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1. Gaspar N, Hartmann O, Munzer C, Bergeron C, Millot F, Cousin-Lafay L, Babin-Boilletot A, Blouin P, Pajot C, Coze C: Neuroblastoma in adolescents. Cancer; 2003 Jul 15;98(2):349-55
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Neuroblastoma in adolescents.
  • Previous studies of patients who were treated with less intensive chemotherapy regimens relative to currently available regimens suggested that adolescents survived longer than younger children, and this finding was related to a lack of myc-N amplification.
  • METHODS: The authors investigated the presentation, treatment, and outcome in 28 adolescent patients who were enrolled in studies of the French Society of Pediatric Oncology during the period from 1987 to 1999 and who were older than age 10 years at the time they were diagnosed with neuroblastoma.
  • RESULTS: None of the six patients with Stage I-II disease either developed recurrent disease or died.
  • At 5 years, disease progression was high (progression-free survival [PFS], 28%) for the 9 adolescents with Stage III disease, but so was survival (overall survival [OS], 86%).
  • The 13 adolescent patients with metastatic neuroblastoma had very poor outcomes (PFS, 18%; OS, 27%).
  • Despite intensive therapy, advanced neuroblastoma appeared to carry a poorer prognosis in adolescent patients compared with children, although patients with Stage III disease had a more indolent course.
  • No difference was found between adolescent patients and children regarding the clinical presentation, treatment schedule, or doses and tolerance of chemotherapy.
  • CONCLUSIONS: Adolescent patients with advanced neuroblastoma had less favorable outcomes compared with children, even if survival in adolescents with Stage III disease seemed longer.
  • [MeSH-major] Abdominal Neoplasms / diagnosis. Neuroblastoma / diagnosis
  • [MeSH-minor] Adolescent. Case-Control Studies. Child. Disease-Free Survival. Female. Humans. Male. Neoplasm Recurrence, Local. Neoplasm Staging. Prognosis. Retrospective Studies. Treatment Outcome

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  • [Copyright] Copyright 2003 American Cancer Society.
  • (PMID = 12872356.001).
  • [ISSN] 0008-543X
  • [Journal-full-title] Cancer
  • [ISO-abbreviation] Cancer
  • [Language] eng
  • [Publication-type] Evaluation Studies; Journal Article
  • [Publication-country] United States
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2. Laprie A, Michon J, Hartmann O, Munzer C, Leclair MD, Coze C, Valteau-Couanet D, Plantaz D, Carrie C, Habrand JL, Bergeron C, Chastagner P, Défachelles AS, Delattre O, Combaret V, Bénard J, Pérel Y, Gandemer V, Rubie H, Neuroblastoma Study Group of the French Society of Pediatric Oncology: High-dose chemotherapy followed by locoregional irradiation improves the outcome of patients with international neuroblastoma staging system Stage II and III neuroblastoma with MYCN amplification. Cancer; 2004 Sep 1;101(5):1081-9
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  • [Title] High-dose chemotherapy followed by locoregional irradiation improves the outcome of patients with international neuroblastoma staging system Stage II and III neuroblastoma with MYCN amplification.
  • BACKGROUND: The objective of this study was to determine whether systemic and regional, intensified treatment can improve the outcome of children who present with a localized neuroblastoma (NB) with MYCN amplification (MNA).
  • METHODS: Between 1990 and 2000, 610 children with localized NB were included in the Localized Neuroblastoma 90 (NBL 90) and NBL 94 study from the French Society of Pediatric Oncology.
  • Among them, 32 children had MNA with Stage II or III NB.
  • During the first period of the study, 20 children (Group A) received postoperative conventional chemotherapy (CT) and/or radiotherapy (RT), depending on each patient's postoperative status.
  • RESULTS: The two groups were comparable with regard to prognostic factors (age, location of the primary lesion, International Neuroblastoma Staging System stage, lymph node invasion) and response to preoperative CT.
  • CONCLUSIONS: Postoperative intensification treatment with HDC, SCR, and locoregional RT resulted in higher survival rates when compared with standard treatment alone and should be considered in the treatment plan for children who are diagnosed with Stage II or III NB and MYCN amplification.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Gene Amplification. Genes, myc. Neuroblastoma / genetics. Neuroblastoma / therapy
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Combined Modality Therapy. Gamma Rays. Humans. Infant. Infant, Newborn. Lymph Nodes / pathology. Neoplasm Invasiveness. Neoplasm Recurrence, Local. Neoplasm Staging. Postoperative Care. Prognosis. Survival Rate. Treatment Outcome

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  • [Copyright] Copyright 2004 American Cancer Society.
  • (PMID = 15329919.001).
  • [ISSN] 0008-543X
  • [Journal-full-title] Cancer
  • [ISO-abbreviation] Cancer
  • [Language] eng
  • [Publication-type] Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
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3. Stefanowicz J, Izycka-Swieszewska E, Drozyńska E, Pienczk J, Połczyńska K, Czauderna P, Sierota D, Bień E, Stachowicz-Stencel T, Kosiak W, Balcerska A: Neuroblastoma and opsoclonus-myoclonus-ataxia syndrome--clinical and pathological characteristics. Folia Neuropathol; 2008;46(3):176-85
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  • [Title] Neuroblastoma and opsoclonus-myoclonus-ataxia syndrome--clinical and pathological characteristics.
  • All cases were in stage II or III of the disease, with no metastases or MYCN amplification.
  • The group included two ganglioneuroblastomas, one ganglioneuroma and one differentiating neuroblastoma.
  • Immunohistochemical analysis of inflammatory infiltrations revealed mixed type populations of lymphocytes with prevalence of the cytotoxic type (CD8 and CD56-positive cells).
  • All patients were treated by surgery alone or with adjuvant chemotherapy with a positive outcome.
  • Children with OMA and neuroblastoma despite a good oncological prognosis often present permanent neurological and developmental deficits.


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4. Feng C, Tang SQ, Wang JW, Liu Y, Yang G: [Curative effects of the protocol of CDV combined with CiE as pre-operative chemotherapy in high-risk childhood neuroblastoma]. Zhongguo Dang Dai Er Ke Za Zhi; 2009 Nov;11(11):885-7
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  • [Title] [Curative effects of the protocol of CDV combined with CiE as pre-operative chemotherapy in high-risk childhood neuroblastoma].
  • OBJECTIVE: To evaluate the effects and the toxicity of the protocol of CDV combined with CiE as pre-operative chemotherapy in childhood stage IV neuroblastoma.
  • METHODS: The clinical data of 27 children aged from 1.2 to 8 years with neuroblastoma in stage IV was retrospectively studied.
  • Neuroblastoma therapeutic response evaluation criterion and common terminology criteria for adverse events of National Cancer Institute were used to evaluate effects and chemotherapy related toxicity.
  • RESULTS: All patients received the pre-operative chemotherapy.
  • After chemotherapy, 24 patients received operations.
  • The most common chemotherapy related toxicity was bone marrow suppression: grade IV suppression of neutrophils (n = 27), reduction in hemoglobin (III grade, n = 7; IV grade, n = 20) and reduction in platelet (III grade, n = 2; IV grade, n = 25).
  • I or II grade lesions of digestive, liver and kidney were found and could be recovered after therapy.
  • CONCLUSIONS: The protocol of CDV combined with CiE as pre-operative chemotherapy might be effective in children with stage IV neuroblastoma.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Neuroblastoma / drug therapy

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  • (PMID = 20113653.001).
  • [ISSN] 1008-8830
  • [Journal-full-title] Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics
  • [ISO-abbreviation] Zhongguo Dang Dai Er Ke Za Zhi
  • [Language] chi
  • [Publication-type] English Abstract; Journal Article
  • [Publication-country] China
  • [Chemical-registry-number] 5J49Q6B70F / Vincristine; 6PLQ3CP4P3 / Etoposide; 7GR28W0FJI / Dacarbazine; 8N3DW7272P / Cyclophosphamide; Q20Q21Q62J / Cisplatin; CVD protocol
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5. Valteau-Couanet D, Michon J, Boneu A, Rodary C, Perel Y, Bergeron C, Rubie H, Coze C, Plantaz D, Bernard F, Chastagner P, Bouzy J, Hartmann O: Results of induction chemotherapy in children older than 1 year with a stage 4 neuroblastoma treated with the NB 97 French Society of Pediatric Oncology (SFOP) protocol. J Clin Oncol; 2005 Jan 20;23(3):532-40
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  • [Title] Results of induction chemotherapy in children older than 1 year with a stage 4 neuroblastoma treated with the NB 97 French Society of Pediatric Oncology (SFOP) protocol.
  • PURPOSE: To test the metastatic response rate in stage 4 neuroblastoma, using dose-intensive induction chemotherapy in a multi-institutional setting.
  • The International Neuroblastoma Staging system was used with an emphasis on skeletal evaluation by 123-iodine-metaiodobenzylguanidine (MIBG) scintigraphy.
  • A phase II study evaluating the metastasis complete response rate after induction chemotherapy was conducted in patients who had positive metastatic sites on MIBG scans at diagnosis.
  • CONCLUSION: The N7 induction chemotherapy protocol was feasible in a multicentric setting.
  • In our hands, escalating doses of cyclophosphamide and prolonging conventional chemotherapy with the same drugs failed to improve the metastasis complete response rate.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Neoplasm Metastasis. Neuroblastoma / drug therapy. Neuroblastoma / pathology
  • [MeSH-minor] 3-Iodobenzylguanidine. Child. Child, Preschool. Cisplatin / administration & dosage. Cyclophosphamide / administration & dosage. Dose-Response Relationship, Drug. Doxorubicin / administration & dosage. Etoposide / administration & dosage. Female. Humans. Infant. Male. Neutropenia / chemically induced. Radiopharmaceuticals. Thrombocytopenia / chemically induced. Treatment Outcome. Vincristine / administration & dosage

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  • (PMID = 15659499.001).
  • [ISSN] 0732-183X
  • [Journal-full-title] Journal of clinical oncology : official journal of the American Society of Clinical Oncology
  • [ISO-abbreviation] J. Clin. Oncol.
  • [Language] eng
  • [Publication-type] Clinical Trial; Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Radiopharmaceuticals; 35MRW7B4AD / 3-Iodobenzylguanidine; 5J49Q6B70F / Vincristine; 6PLQ3CP4P3 / Etoposide; 80168379AG / Doxorubicin; 8N3DW7272P / Cyclophosphamide; Q20Q21Q62J / Cisplatin; CAV protocol; VP-P protocol
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6. Meir HM, Balawi I, Nayel H, Yousef MK, Badawood S, Al-Mobarak M: Neuroblastoma. Saudi Med J; 2001 Aug;22(8):674-80
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  • [Title] Neuroblastoma.
  • OBJECTIVE: Due to the poor results achieved on combination chemotherapy and the unproven cost-effectiveness value of myeloablative therapy, the question has been raised; should patients with stage IV neuroblastoma be actively treated?
  • The aim of the current study is to analyze retrospectively treatment results of 43 children with neuroblastoma with special stress on the rate and duration of remission in children with disseminated neuroblastoma.
  • METHODS: Treatment of children with neuroblastoma consisted of surgical removal of the tumor, if possible, followed by chemotherapy for unresectable residual tumor including metastases.
  • Second look surgery was performed to resect residual masses rendered resectable on chemotherapy in the absence of distal metastases.
  • The chemotherapy protocol used in the current study consisted of alternating combination chemotherapy regimens containing, Cyclophosphamide, Vincristine and Doxorubicin, alternating with Cis-platinum and Etoposide.
  • According to the childrens cancer study group (CCSG) staging system, only 6 patients (14%) had localized tumors (stages I and II).
  • Two patients (5%) were found to have stage IV.
  • Stage III was documented in 5 patients (12%).
  • The bone marrow was the most common site of metastatic deposit, encountered in 23 patients out of the 30 with stage IV disease (77%).
  • Out of the 12 evaluable non-stage IV patients, only one patient (8%) showed treatment failure.
  • Assessment of response by the end of the 6th month from the date of diagnosis revealed that out of the 27 evaluable patients with stage IV, 4 patients achieved complete remission, 7 patients achieved very good partial remission, 8 patients achieved partial remission and 4 patients achieved mixed response.
  • Three patients showed progressive disease on chemotherapy.
  • Assessment of response to treatment by the end of the 12th month from diagnosis revealed that 6 patients (2 complete remissions, 1 very good partial response, 3 partial responses) were maintaining their remission.
  • CONCLUSION: Results of treatment by multiagent chemotherapy regimens used in the current study show that children with neuroblastoma, even those with advanced stages, should receive the benefit of intensive multimodal therapy, even those with partial response to initial therapy.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Neuroblastoma / drug therapy. Neuroblastoma / surgery
  • [MeSH-minor] Child. Child, Preschool. Cisplatin / administration & dosage. Combined Modality Therapy. Cyclophosphamide / administration & dosage. Disease Progression. Doxorubicin / administration & dosage. Etoposide / administration & dosage. Female. Humans. Infant. Male. Neoplasm Staging. Retrospective Studies. Second-Look Surgery. Survival Rate. Treatment Outcome. Vincristine / administration & dosage

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  • (PMID = 11573111.001).
  • [ISSN] 0379-5284
  • [Journal-full-title] Saudi medical journal
  • [ISO-abbreviation] Saudi Med J
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] Saudi Arabia
  • [Chemical-registry-number] 5J49Q6B70F / Vincristine; 6PLQ3CP4P3 / Etoposide; 80168379AG / Doxorubicin; 8N3DW7272P / Cyclophosphamide; Q20Q21Q62J / Cisplatin
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7. López-Aguilar E, Cerecedo-Díaz F, Rivera-Márquez H, Valdéz-Sánchez M, Sepúlveda-Vildósola AC, Delgado Huerta S, Vera-Hermosillo H, Vázquez-Langle JR, Wanzke del Angel V: [Neuroblastoma: prognostic factors and survival. Experience in Hospital de Pediatria del Centro Medico Nacional del Siglo XXI and review of the literature]. Gac Med Mex; 2003 May-Jun;139(3):209-14
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  • [Title] [Neuroblastoma: prognostic factors and survival. Experience in Hospital de Pediatria del Centro Medico Nacional del Siglo XXI and review of the literature].
  • [Transliterated title] Neuroblastoma: factores pronósticos y sobrevida. Experiencia en el Hospital de Pediatría del Centro Médico Nacional Siglo XXI y revisión de la literatura.
  • Neuroblastoma (NB) is the most frequent extracranial solid tumor in children according to the literature.
  • We included all patients admitted to our hospital during the previous five years and who had not received any treatment.
  • Patients with stages I, II, and IV received cyclophosphamide and epirrubicin.
  • Patients with stages were III and IV received the same chemotherapy alternating with cisplatinum., ifosfamide and etoposide during 12 months as well as massive doses of 131-MIBG and surgical ablation of the remaining tumor when possible.
  • The most important prognostic factors are still considered to be age, stage and histology.
  • [MeSH-major] Neuroblastoma / diagnosis
  • [MeSH-minor] Adolescent. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Child. Child, Preschool. Female. Humans. Infant. Infant, Newborn. Male. Mexico. Neoplasm Staging. Prognosis. Prospective Studies. Survival Analysis

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  • (PMID = 12872413.001).
  • [ISSN] 0016-3813
  • [Journal-full-title] Gaceta médica de México
  • [ISO-abbreviation] Gac Med Mex
  • [Language] spa
  • [Publication-type] English Abstract; Journal Article
  • [Publication-country] Mexico
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8. Opel D, Poremba C, Simon T, Debatin KM, Fulda S: Activation of Akt predicts poor outcome in neuroblastoma. Cancer Res; 2007 Jan 15;67(2):735-45
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  • [Title] Activation of Akt predicts poor outcome in neuroblastoma.
  • Whereas aberrant activation of the phosphatidylinositol 3'-kinase (PI3K)/Akt pathway, a key survival cascade, has previously been linked to poor prognosis in several human malignancies, its prognostic effect in neuroblastoma has not yet been explored.
  • We therefore investigated the phosphorylation status of Akt, S6 ribosomal protein as target of mammalian target of rapamycin, and extracellular signal-regulated kinase (ERK) in 116 primary neuroblastoma samples by tissue microarray and its correlation with established prognostic markers and survival outcome.
  • Here, we provide for the first time evidence that phosphorylation of Akt at serine 473 (S473) and/or threonine 308 (T308), S6 ribosomal protein, and ERK frequently occurs in primary neuroblastoma.
  • Importantly, we identified Akt activation as a novel prognostic indicator of decreased event-free or overall survival in neuroblastoma, whereas phosphorylation of S6 ribosomal protein or ERK had no prognostic effect.
  • In addition, Akt activation correlated with variables of aggressive disease, including MYCN amplification, 1p36 aberrations, advanced disease stage, age at diagnosis, and unfavorable histology.
  • Monitoring Akt at T308 or both phosphorylation sites improved the prognostic significance of Akt activation in neuroblastoma specimens compared with S473 phosphorylation.
  • Parallel experiments in neuroblastoma cell lines revealed that activation of Akt by insulin-like growth factor (IGF)-I significantly inhibited tumor necrosis factor-related apoptosis-inducing ligand- or chemotherapy-induced apoptosis in a PI3K-dependent manner because the PI3K inhibitor LY294002 completely reversed the IGF-I-mediated protection of neuroblastoma cells from apoptosis.
  • By showing that activation of Akt correlates with poor prognosis in primary neuroblastoma in vivo and with apoptosis resistance in vitro, our findings indicate that Akt presents a clinically relevant target in neuroblastoma that warrants further investigation.
  • [MeSH-major] Neuroblastoma / enzymology. Proto-Oncogene Proteins c-akt / metabolism
  • [MeSH-minor] Apoptosis / drug effects. Cell Line, Tumor. Child, Preschool. Cohort Studies. Doxorubicin / pharmacology. Enzyme Activation. Humans. Infant. Insulin-Like Growth Factor I / physiology. Insulin-Like Growth Factor II / pharmacology. Phosphatidylinositol 3-Kinases / antagonists & inhibitors. Phosphatidylinositol 3-Kinases / metabolism. Phosphorylation. Prognosis. Signal Transduction. TNF-Related Apoptosis-Inducing Ligand / pharmacology


9. Granata C, Fagnani AM, Gambini C, Boglino C, Bagnulo S, Cecchetto G, Federici S, Inserra A, Michelazzi A, Riccipetitoni G, Rizzo A, Tamaro P, Jasonni V, De Bernardi B: Features and outcome of neuroblastoma detected before birth. J Pediatr Surg; 2000 Jan;35(1):88-91
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  • [Title] Features and outcome of neuroblastoma detected before birth.
  • Optimal strategy has not yet been defined for these patients, because knowledge on this particular neuroblastoma (NB) population is still limited.
  • However, definite guidelines are needed to avoid inadequate treatment.
  • The authors analyzed the cases of antenatally detected NB (ADNB) reported in the Italian Neuroblastoma Registry during the past 6 years to elucidate the features of this subset of NB.
  • METHODS: The Italian Neuroblastoma Registry was reviewed for the period January 1993 to December 1998 to collect clinical, radiographic, surgical, and histopathological data on ADNB cases.
  • NB stage was evaluated according to INSS criteria.
  • All patients had undergone imaging (computed tomography or magnetic resonance imaging) of the primary tumor and bone marrow biopsy before surgical resection.
  • Stage distribution was stage I, 13 cases; stage II-A, 1 case; stage II-B, 1 case; stage IV-S, 2 cases.
  • N-myc amplification was detected only in a newborn with stage II-A NB, who died of massive bleeding 2 days after tumor resection.
  • Both diploidy and deletion of 1p were observed in a newborn who subsequently died of disease progression despite surgery, chemotherapy, and radiation therapy.
  • Aggressive treatment may not always be necessary.
  • [MeSH-major] Adrenal Gland Neoplasms / diagnosis. Neuroblastoma / diagnosis. Prenatal Diagnosis


10. Paulino AC: Palliative radiotherapy in children with neuroblastoma. Pediatr Hematol Oncol; 2003 Mar;20(2):111-7
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  • [Title] Palliative radiotherapy in children with neuroblastoma.
  • Limited information is available regarding the efficacy of external beam radiation therapy in the palliation of metastatic disease from neuroblastoma.
  • There were 26 soft tissue (group I), 19 bone (group II), 5 brain (group III), and 3 hepatic (group IV) treated sites.
  • Median radiotherapy doses for groups I, II, III, and IV sites were 2000, 2000, 2400, and 450 cGy, respectively.
  • For group II sites, complete response was complete pain relief without medication, partial response was > or =50% pain relief with or without medication, no response was <50% change in pain with medication, and progressive disease was increase in pain and/or medication.
  • Duration of response was until death in 18 responders (90%); 2 patients relapsed with an increasing soft tissue mass at 5 months and 1 year after palliative radiotherapy.
  • For group II sites, complete response was seen in 8 (42%) while partial response was documented in 7 (37%).
  • The only patient who survived had a stage IV-S neuroblastoma with liver metastases and is alive 13 years after hepatic irradiation.
  • Radiotherapy is an effective treatment for palliation of symptomatic metastatic disease in children with neuroblastoma.
  • [MeSH-major] Neuroblastoma / radiotherapy. Palliative Care
  • [MeSH-minor] Adolescent. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Bone Neoplasms / radiotherapy. Bone Neoplasms / secondary. Brain Neoplasms / radiotherapy. Brain Neoplasms / secondary. Child. Child, Preschool. Combined Modality Therapy. Cranial Irradiation. Cyclophosphamide / administration & dosage. Disease Progression. Doxorubicin / administration & dosage. Female. Humans. Infant. Liver Neoplasms / drug therapy. Liver Neoplasms / radiotherapy. Liver Neoplasms / secondary. Male. Pain / radiotherapy. Radioisotope Teletherapy. Remission Induction. Retrospective Studies. Soft Tissue Neoplasms / radiotherapy. Soft Tissue Neoplasms / secondary. Survival Analysis. Treatment Outcome

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  • (PMID = 12554522.001).
  • [ISSN] 0888-0018
  • [Journal-full-title] Pediatric hematology and oncology
  • [ISO-abbreviation] Pediatr Hematol Oncol
  • [Language] eng
  • [Publication-type] Evaluation Studies; Journal Article
  • [Publication-country] England
  • [Chemical-registry-number] 80168379AG / Doxorubicin; 8N3DW7272P / Cyclophosphamide
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11. Frantz CN, London WB, Diller L, Seeger R, Sawyer K: Recurrent neuroblastoma: Randomized treatment with topotecan + cyclophosphamide (T+C) vs. topotecan alone(T). A POG/CCG Intergroup Study. J Clin Oncol; 2004 Jul 15;22(14_suppl):8512

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  • [Title] Recurrent neuroblastoma: Randomized treatment with topotecan + cyclophosphamide (T+C) vs. topotecan alone(T). A POG/CCG Intergroup Study.
  • : 8512 Background: In Phase I/II studies, T and the combination of T+C each demonstrated promising activity against recurrent neuroblastoma, so a randomized study was performed.
  • METHODS: Eligible patients were <21 yrs with evaluable disease at first recurrence after a first regimen of aggressive chemotherapy.
  • 13 patients received "therapeutic window" deferoxamine for 3-6 weeks as part of the study.
  • T received T 2.4mg/m2/d×5d q21d.
  • Best response according to the International Neuroblastoma Response Criteria was analyzed in a two-stage sequential design.
  • In the calculation of time to progressive disease, patients taken off study for stem cell transplant, refusal, or unrelated death were censored at that time.
  • At least eight more responders in T+C than in T were required by the two-stage design in order to conclude superiority of T+C; with only seven, there is insufficient evidence to reach such a conclusion.
  • For both EFS and S treatment comparisons, the curves were not significantly different.
  • However, the time to disease progression was significantly longer for T+C.
  • CONCLUSIONS: There is a trend toward better results with T+C than T.
  • Incorporation of the combination into front line treatment plans deserves further exploration.

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  • (PMID = 28013780.001).
  • [ISSN] 1527-7755
  • [Journal-full-title] Journal of clinical oncology : official journal of the American Society of Clinical Oncology
  • [ISO-abbreviation] J. Clin. Oncol.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
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12. Arafat W, Abdelghany A, Awad N: A clinical trial to study the effect of adding cis-retinoic acid during and after conventional treatment for pediatric neuroblastoma patient. J Clin Oncol; 2009 May 20;27(15_suppl):10057

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  • [Title] A clinical trial to study the effect of adding cis-retinoic acid during and after conventional treatment for pediatric neuroblastoma patient.
  • : 10057 Background: Neuroblastoma is predominantly a tumor of early childhood, with two thirds of the cases presenting in children younger than 5 years.
  • Cis-retinoic acid has been used as maintenance therapy for treatment of advanced neuroblastoma after BMT.
  • Patients who received cis-retinoic acid had significantly better 3-year event-free survival than patients receiving no maintenance therapy.
  • However, there is no data available about usingcis-retinoic acid during induction phase of chemotherapy.
  • The aim of this study is to evaluate the efficacy of cis-retinoic acid when used in combination with conventional chemotherapy in pediatric patient who is newly diagnosed with locally advanced neuroblastoma.
  • METHODS: Seventeen newly diagnosed children with locally advanced neuroblastoma who is candidate to receive chemotherapy were also received cis-retinoic acid starting at a dose of 160 /m<sup>2</sup> day (day 2-15 of chemotherapy) first the 3 patients, 20% dose reduction were allowed if toxicity occurred in first cohort of patient. . Patients were giving the drugs for at least 4 cycles.
  • RT-PCR analysis of several related genes was done from tumor, sample after treatment.
  • Patients were stage III, IV (70%), II (30%).
  • CONCLUSIONS: Cis-retinoic acid at dose 130mg/m<sup>2</sup> is a well tolerated drug with chemotherapy.
  • Response to treatment is better than historical control.

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  • (PMID = 27962453.001).
  • [ISSN] 1527-7755
  • [Journal-full-title] Journal of clinical oncology : official journal of the American Society of Clinical Oncology
  • [ISO-abbreviation] J. Clin. Oncol.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
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13. Bansal D, Marwaha RK, Trehan A, Rao KL, Gupta V: Profile and outcome of neuroblastoma with convertional chemotherapy in children older than one year: a 15-years experience. Indian Pediatr; 2008 Feb;45(2):135-9
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  • [Title] Profile and outcome of neuroblastoma with convertional chemotherapy in children older than one year: a 15-years experience.
  • The clinical profile and outcome of neuroblastoma in 103 children, older than one-year is presented.
  • 74 had Stage IV, 27 Stage III and one patient each had Stage I or II disease.
  • Treatment included chemotherapy followed by surgical resection/debulking.
  • Chemotherapy consisted of OPEC (vincristine, cyclophosphamide, cisplatin and etoposide).
  • The caretakers of 54 (52.4%) children either did not opt for or defaulted therapy, whilst 3 patients died before chemotherapy could be initiated.
  • Of the remaining 46 patients, the tumor progressed during therapy in 19 (41.3%).
  • Majority of children presented with advanced disease and the outcome was dismal with conventional non-myloablative chemotherapy.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Brain Neoplasms / drug therapy. Brain Neoplasms / pathology. Neuroblastoma / drug therapy. Neuroblastoma / pathology


14. Frappaz D, Perol D, Michon J, Berger C, Coze C, Bernard JL, Zucker JM, Philip T: The LMCE5 unselected cohort of 25 children consecutively diagnosed with untreated stage 4 neuroblastoma over 1 year at diagnosis. Br J Cancer; 2002 Nov 18;87(11):1197-203
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  • [Title] The LMCE5 unselected cohort of 25 children consecutively diagnosed with untreated stage 4 neuroblastoma over 1 year at diagnosis.
  • The Lyon-Marseille-Curie-Est (LMCE) of France cooperative group has previously reported successive series of unselected stage four children older than 1 year at diagnosis with metastatic neuroblastoma (LMCE 1 and 3).
  • Based on improvements reported with post induction chemotherapy, the LMCE 5 used post induction for all children, but omitted total body irradiation and immunomagnetic purging in megatherapy regimen for all children.
  • After surgery treatment was stratified based on response and eligible children received etoposide carboplatin (LMCE 5A : n=10)+/-doxorubicin (LMCE 5B-C n=13) followed by megatherapy (melphalan without total body irradiation and unpurged peripheral blood stem cell rescue).
  • The increase in drug doses during induction did not improve remission rate.
  • Modified strategies for induction, non toxic alternative to total body irradiation, and post megatherapy regimen should be developed.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Neuroblastoma / drug therapy
  • [MeSH-minor] Adolescent. Carboplatin / administration & dosage. Child. Child, Preschool. Cisplatin / administration & dosage. Cohort Studies. Cyclophosphamide / administration & dosage. Disease-Free Survival. Doxorubicin / administration & dosage. Etoposide / administration & dosage. Female. Humans. Infant. Male. Melphalan / administration & dosage. Peripheral Blood Stem Cell Transplantation. Treatment Outcome

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  • (PMID = 12439705.001).
  • [ISSN] 0007-0920
  • [Journal-full-title] British journal of cancer
  • [ISO-abbreviation] Br. J. Cancer
  • [Language] eng
  • [Publication-type] Clinical Trial; Clinical Trial, Phase II; Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't
  • [Publication-country] Scotland
  • [Chemical-registry-number] 6PLQ3CP4P3 / Etoposide; 80168379AG / Doxorubicin; 8N3DW7272P / Cyclophosphamide; BG3F62OND5 / Carboplatin; Q20Q21Q62J / Cisplatin; Q41OR9510P / Melphalan
  • [Other-IDs] NLM/ PMC2408899
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15. London WB, Chang MN: One- and two-stage designs for stratified phase II clinical trials. Stat Med; 2005 Sep 15;24(17):2597-611
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  • [Title] One- and two-stage designs for stratified phase II clinical trials.
  • A global one-sample test for response rates for stratified phase II clinical trials is proposed.
  • Such a test is analogous to that of a stratified log-rank test for time-to-event data.
  • Both one- and two-stage tests are developed, and conditional and unconditional approaches are introduced in each case, where the conditional approach involves conditioning on the observed samples sizes within the strata.
  • The methodology generates samples sizes and stopping boundaries that provide designs with the desired power and type I error probability.
  • These methods are useful for designing stratified phase II clinical trials.
  • An application to a Children's Oncology Group phase II clinical trial in relapsed neuroblastoma patients is presented.
  • [MeSH-major] Clinical Trials, Phase II as Topic / methods. Data Interpretation, Statistical
  • [MeSH-minor] Antineoplastic Agents / therapeutic use. Antineoplastic Combined Chemotherapy Protocols. Child. Child, Preschool. Cyclophosphamide / therapeutic use. Humans. Infant. Neoplasm Recurrence, Local / prevention & control. Neuroblastoma / drug therapy. Research Design. Topotecan / therapeutic use

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  • [Copyright] Copyright (c) 2005 John Wiley & Sons, Ltd.
  • [CommentIn] Stat Med. 2006 Sep 30;25(18):3220-2; author reply 3222-3 [16906490.001]
  • (PMID = 16118809.001).
  • [ISSN] 0277-6715
  • [Journal-full-title] Statistics in medicine
  • [ISO-abbreviation] Stat Med
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / CA29139; United States / NCI NIH HHS / CA / CA98413-01
  • [Publication-type] Journal Article; Research Support, N.I.H., Extramural; Research Support, U.S. Gov't, P.H.S.
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 7M7YKX2N15 / Topotecan; 8N3DW7272P / Cyclophosphamide
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16. Tsuchida Y, Shitara T, Kuroiwa M, Ikeda H: Current treatment and future directions in neuroblastoma. Indian J Pediatr; 2003 Oct;70(10):809-12
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  • [Title] Current treatment and future directions in neuroblastoma.
  • OBJECTIVE: The International Neuroblastoma Staging System (INSS) and Pathology Classification (INPC) were applied to analyze the results of treatment of 644 patients with neuroblastoma treated in Japan during the years from 1995 to 1999, and it was found that the pathology classification (INPC) showed the strongest relevance to prognosis compared to other factors such as stage, MYNC amplification, DNA ploidy and 1p-deletion.
  • Current results of treatment for advanced neuroblastoma are still not satisfactory, so innovative therapeutic methods have been sought during the past 10 years.
  • RESULTS: Irinotecan is a water-soluble derivative of camptothecin, which is isolated from a Chinese tree, Camptotheca acuminata; Its effectiveness against neuroblastoma was confirmed by in vivo preclinical studies, and phase I clinical trials in Japan concluded the maximum tolerated dose of this agent is 160-180 mg/m2/day for 3 consecutive days, repeated after 25 days off.
  • Phase II trials with this dose began, and we could obtain some encouraging results with the clinical use of irinotecan. rhEndostatin has been studied in in vivo experimental models.
  • CONCLUSION: Irinotecan appears to be promising when it is given to the patients neuroblastoma, whereas rhEndostatin needs to have more preclinical studies before it is used in patients.
  • [MeSH-major] Angiogenesis Inhibitors / therapeutic use. Antineoplastic Agents, Phytogenic / therapeutic use. Brain Neoplasms / drug therapy. Camptothecin / analogs & derivatives. Camptothecin / therapeutic use. Endostatins / therapeutic use. Neuroblastoma / drug therapy
  • [MeSH-minor] Child. Child, Preschool. Drug Administration Schedule. Humans. Infant. Neoplasm Staging. Prospective Studies. Survival Rate

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  • (PMID = 14649477.001).
  • [ISSN] 0019-5456
  • [Journal-full-title] Indian journal of pediatrics
  • [ISO-abbreviation] Indian J Pediatr
  • [Language] eng
  • [Publication-type] Clinical Trial; Journal Article
  • [Publication-country] India
  • [Chemical-registry-number] 0 / Angiogenesis Inhibitors; 0 / Antineoplastic Agents, Phytogenic; 0 / Endostatins; 0H43101T0J / irinotecan; XT3Z54Z28A / Camptothecin
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17. Meir HM, Balawi I, Nayel H, Yousef MK, Badawood S, Al-Mobarak M: Neuroblastoma: A clinical review. Neurosciences (Riyadh); 2001 Oct;6(4):213-9

  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Neuroblastoma: A clinical review.
  • OBJECTIVE: Due to the poor results achieved on combination chemotherapy and the unproven cost-effectiveness value of myeloablative therapy, the question has been raised; should patients with stage IV neuroblastoma be actively treated?
  • The aim of the current study is to analyze retrospectively treatment results of 43 children with neuroblastoma with special stress on the rate and duration of remission in children with disseminated neuroblastoma.
  • METHODS: Treatment of children with neuroblastoma consisted of surgical removal of the tumor, if possible, followed by chemotherapy for unresectable residual tumor including metastases.
  • Second look surgery was performed to resect residual masses rendered resectable on chemotherapy in the absence of distal metastases.
  • The chemotherapy protocol used in the current study consisted of alternating combination chemotherapy regimens containing, Cyclophosphamide, Vincristine and Doxorubicin, alternating with Cis-platinum and Etoposide.
  • According to the childrens cancer study group (CCSG) staging system, only 6 patients (14%) had localized tumors (stages I and II).
  • Two patients (5%) were found to have stage IV.
  • Stage III was documented in 5 patients (12%).
  • The bone marrow was the most common site of metastatic deposit, encountered in 23 patients out of the 30 with stage IV disease (77%).
  • Out of the 12 evaluable non-stage IV patients, only one patient (8%) showed treatment failure.
  • Assessment of response by the end of the 6th month from the date of diagnosis revealed that out of the 27 evaluable patients with stage IV, 4 patients achieved complete remission, 7 patients achieved very good partial remission, 8 patients achieved partial remission and 4 patients achieved mixed response.
  • Three patients showed progressive disease on chemotherapy.
  • Assessment of response to treatment by the end of the 12th month from diagnosis revealed that 6 patients (2 complete remissions, 1 very good partial response, 3 partial responses) were maintaining their remission.
  • CONCLUSION: Results of treatment by multiagent chemotherapy regimens used in the current study show that children with neuroblastoma, even those with advanced stages, should receive the benefit of intensive multimodal therapy, even those with partial response to initial therapy.

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  • (PMID = 24185182.001).
  • [ISSN] 1319-6138
  • [Journal-full-title] Neurosciences (Riyadh, Saudi Arabia)
  • [ISO-abbreviation] Neurosciences (Riyadh)
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] Saudi Arabia
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18. Martínez Ibáñez V, Abad P, Gallego S, Sánchez de Toledo J, Marqués A, Boix Ochoa J: [Neuroblastoma: biological markers, surgery, and clinical course]. Cir Pediatr; 2000 Apr;13(2):47-53
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  • [Title] [Neuroblastoma: biological markers, surgery, and clinical course].
  • INTRODUCTION: The treatment of neuroblastoma is basically chemotherapy, and surgery, in spite of advances, this kind of tumor is nowadays a surgical challenge.
  • PURPOSE: The aim of this study was to evaluate the impact of our therapy in this kind of pediatric tumors.
  • MATERIAL AND METHODS: 32 consecutive patients with abdominal neuroblastoma, aged between 1 month and 10 years old, median age 3 years old, observed from 1993 through 1997 have been studied.
  • Several parameters: age, ferritin, deletion of the chromosome 1p36, chromosomic ploidy, LDH, N-myc gene amplification and enolase neuron specific were studied and were related with the histology by Joshi and the International Neuroblastoma Staging System (INSS) in order to know the prognosis.
  • All the patients were treated by means of chemotherapy and surgery, and some cases with radiotherapy and bone marrow transplantation.
  • RESULTS: Two patients presented the tumor in stage I (INSS) and three in stage II.
  • In the stage IV: five died, two live with disease (1 local recurrence and one metastasis), five live free of disease, and one did not maintain the follow-up.
  • In the stage IV-S, the three patients live free of disease.
  • Of all the parameters studied, we consider the ones with biggest prognostic efficacy are, the age, the stage INSS, the histology Shimada and the N-myc amplification.
  • [MeSH-major] Abdominal Neoplasms / surgery. Neuroblastoma / surgery

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  • (PMID = 12602001.001).
  • [ISSN] 0214-1221
  • [Journal-full-title] Cirugía pediátrica : organo oficial de la Sociedad Española de Cirugía Pediátrica
  • [ISO-abbreviation] Cir Pediatr
  • [Language] spa
  • [Publication-type] English Abstract; Journal Article
  • [Publication-country] Spain
  • [Chemical-registry-number] 0 / Biomarkers, Tumor
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19. Garaventa A, Luksch R, Biasotti S, Severi G, Pizzitola MR, Viscardi E, Prete A, Mastrangelo S, Podda M, Haupt R, De Bernardi B: A phase II study of topotecan with vincristine and doxorubicin in children with recurrent/refractory neuroblastoma. Cancer; 2003 Dec 1;98(11):2488-94
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  • [Title] A phase II study of topotecan with vincristine and doxorubicin in children with recurrent/refractory neuroblastoma.
  • BACKGROUND: A Phase II trial in children with advanced neuroblastoma was carried out in five Italian institutions to evaluate the antitumor activity and tolerability of topotecan followed by vincristine and doxorubicin.
  • METHODS: Children older than age 1 year with Stage III or Stage IV neuroblastoma, all of whom had been treated previously with chemotherapy and were diagnosed with either refractory or recurrent disease, were treated with topotecan at an intravenous dose of 1.5 mg/m(2) (the dose was 0.75 mg/m(2) for patients who were treated within 1 year of previous megatherapy) per day for 5 days followed by 48-hour intravenous infusions of 2 mg/m(2) vincristine and 45 mg/m(2) doxorubicin.
  • Cycles of therapy were repeated every 3 weeks.
  • RESULTS: Twenty-five patients (2 with Stage III disease and 23 with Stage IV disease; 19 with refractory disease and 6 with recurrent disease) were treated with a total of 115 cycles.
  • Fifteen patients were alive at the time of the current report and were progression free at 4-16 months (median, 9 months) after the first course of this treatment.
  • CONCLUSIONS: The topotecan-vincristine-doxorubicin combination was active and well tolerated in previously treated patients with advanced neuroblastoma.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Neuroblastoma / drug therapy
  • [MeSH-minor] Child. Child, Preschool. Disease Progression. Doxorubicin / administration & dosage. Female. Humans. Infant. Male. Survival Analysis. Topotecan / administration & dosage. Treatment Outcome. Vincristine / administration & dosage

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  • [Copyright] Copyright 2003 American Cancer Society.
  • (PMID = 14635085.001).
  • [ISSN] 0008-543X
  • [Journal-full-title] Cancer
  • [ISO-abbreviation] Cancer
  • [Language] eng
  • [Publication-type] Clinical Trial; Clinical Trial, Phase II; Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 5J49Q6B70F / Vincristine; 7M7YKX2N15 / Topotecan; 80168379AG / Doxorubicin
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20. Mitchell WG, Davalos-Gonzalez Y, Brumm VL, Aller SK, Burger E, Turkel SB, Borchert MS, Hollar S, Padilla S: Opsoclonus-ataxia caused by childhood neuroblastoma: developmental and neurologic sequelae. Pediatrics; 2002 Jan;109(1):86-98
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  • [Title] Opsoclonus-ataxia caused by childhood neuroblastoma: developmental and neurologic sequelae.
  • OBJECTIVE: Opsoclonus-ataxia, also called "dancing eye syndrome," is a serious neurologic condition that is often a paraneoplastic manifestation of occult neuroblastoma in early childhood.
  • Despite resection of tumor and immunosuppressive therapy, outcome generally includes significant developmental and behavioral sequelae.
  • There is controversy about how treatment alters outcome.
  • The goals of this study were to understand the ongoing neurologic and developmental deficits of children who are treated for opsoclonus-ataxia with associated neuroblastoma; to relate treatment history to outcome; and to quantify objectively the acute changes in motor function, speech, mood, and behavior related to intravenous immunoglobulin (IVIg) treatment.
  • METHODS: Patients were children with opsoclonus-ataxia caused by neuroblastoma, regardless of interval since diagnosis.
  • Additional examinations were performed immediately before and 2 to 3 days after treatment with IVIg in 5 children.
  • All had a stage I or II neuroblastoma resected 3 months to 11 years previously.
  • None received any other treatment for the tumor.
  • Three had received other immunosuppressive treatment, including cyclophosphamide.
  • Immediate versus delayed treatment was not associated with better outcome.
  • CONCLUSIONS: Opsoclonus-ataxia caused by neuroblastoma causes substantial developmental sequelae that are not adequately prevented by current treatment.
  • The increased deficits in older children raise concern that this represents a progressive encephalopathy rather than a time-limited single insult.
  • [MeSH-major] Abdominal Neoplasms / complications. Ataxia / etiology. Neuroblastoma / complications. Paraneoplastic Syndromes, Nervous System / etiology. Thoracic Neoplasms / complications
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Cross-Sectional Studies. Female. Head and Neck Neoplasms / complications. Head and Neck Neoplasms / diagnosis. Head and Neck Neoplasms / drug therapy. Humans. Immunoglobulins, Intravenous / therapeutic use. Infant. Male

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  • [ErratumIn] Pediatrics 2002 Oct;110(4):853-4
  • (PMID = 11773546.001).
  • [ISSN] 1098-4275
  • [Journal-full-title] Pediatrics
  • [ISO-abbreviation] Pediatrics
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Immunoglobulins, Intravenous
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21. Sun XF, Liu DG, Su YS, Lin TY, Chen XQ, He YJ: [Evaluation of efficacy of treatment for 30 children with neuroblastoma]. Ai Zheng; 2003 Dec;22(12):1343-5
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  • [Title] [Evaluation of efficacy of treatment for 30 children with neuroblastoma].
  • BACKGROUND & OBJECTIVE: Neuroblastoma is one of common solid tumors in children.
  • The major treatment modality for neuroblastoma (NB) is chemotherapy combined with operation or irradiation.
  • Further study is needed for improving cure rate of neuroblastoma.
  • This study was designed to evaluate the efficacy of children with neuroblastoma treated in Cancer Center, Sun Yat-sen University, and to explore reasonable therapy strategy.
  • These patients were treated with chemotherapy plus operation or radiation.
  • The stages were as follow: II, n=2; III, n=12; IV, n=15; IVS, n=1.
  • Chemotherapy regimen was CAV (cyclophosphamide 750 mg/m(2) d1, vincristine 1.5 mg/m(2), d1, Adriamycin 50 mg/m(2) d1) alternated with EP (teniposide or etoposide 60 mg/m(2) d1-d5, cisplatin 20 mg/m(2) d1-d5).
  • The resection would be done after 4 to 6 cycles of chemotherapy if possible.
  • The chemotherapy or radiation would be done after resection.
  • If operation was not available, the patients continued to receive the chemotherapy.
  • The patients with stage IVS only received cyclophosphamide plus vincristine.
  • RESULTS: Among 30 patients, 2 cases achieved complete remission (CR 6.7%) by chemotherapy alone; 21 cases achieved partial remission (PR 70%); 6 cases showed no change (NC 20%); 1 cases showed progressive diseases (3.3%).
  • Overall response rate (CR+PR) were 76.7% by chemotherapy alone.
  • The 2-year overall survival rate was 47.8% for all patients; 100% for Stage II/IVS, 34% for stage III, 22% for stage IV, respectively.
  • CONCLUSION: Chemotherapy plus operation or radiation is the major treatment for neuroblastoma.
  • CAV/EP alternative chemotherapy is the active regimen for NB.
  • Advance stage NB needs further study for improving the prognosis.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Cyclophosphamide / therapeutic use. Doxorubicin / therapeutic use. Neuroblastoma / therapy. Vincristine / therapeutic use
  • [MeSH-minor] Child. Child, Preschool. Combined Modality Therapy. Etoposide / administration & dosage. Female. Humans. Infant. Male. Neoplasm Staging. Retrospective Studies. Teniposide / administration & dosage. Treatment Outcome

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  • (PMID = 14693065.001).
  • [Journal-full-title] Ai zheng = Aizheng = Chinese journal of cancer
  • [ISO-abbreviation] Ai Zheng
  • [Language] chi
  • [Publication-type] English Abstract; Journal Article
  • [Publication-country] China
  • [Chemical-registry-number] 5J49Q6B70F / Vincristine; 6PLQ3CP4P3 / Etoposide; 80168379AG / Doxorubicin; 8N3DW7272P / Cyclophosphamide; 957E6438QA / Teniposide; CAV protocol
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22. London WB, Frantz CN, Campbell LA, Seeger RC, Brumback BA, Cohn SL, Matthay KK, Castleberry RP, Diller L: Phase II randomized comparison of topotecan plus cyclophosphamide versus topotecan alone in children with recurrent or refractory neuroblastoma: a Children's Oncology Group study. J Clin Oncol; 2010 Aug 20;28(24):3808-15
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  • [Title] Phase II randomized comparison of topotecan plus cyclophosphamide versus topotecan alone in children with recurrent or refractory neuroblastoma: a Children's Oncology Group study.
  • PURPOSE: Single-agent topotecan (TOPO) and combination topotecan and cyclophosphamide (TOPO/CTX) were compared in a phase II randomized trial in relapsed/refractory neuroblastoma.
  • Because responders often underwent further therapies, novel statistical methods were required to compare the long-term outcome of the two treatments.
  • PATIENTS AND METHODS: Children with refractory/recurrent neuroblastoma (only one prior aggressive chemotherapy regimen) were randomly assigned to daily 5-day topotecan (2 mg/m(2)) or combination topotecan (0.75 mg/m(2)) and cyclophosphamide (250 mg/m(2)).
  • A randomized two-stage group sequential design enrolled 119 eligible patients.
  • Long-term outcome of protocol therapy was assessed using novel methods-causal inference-which allowed adjustment for the confounding effect of off-study therapies.
  • Adjusting for randomized treatment effect and subsequent autologous stem-cell transplantation, there was no difference between TOPO and TOPO/CTX in terms of the proportion alive at 2 years.
  • After adjustment for subsequent therapies, no difference was detected in the proportion alive at 2 years.
  • Causal inference methods for assessing long-term outcomes of phase II therapies after subsequent treatment can elucidate effects of initial therapies.

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  • [Cites] J Clin Oncol. 2001 Jan 1;19(1):213-9 [11134215.001]
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  • (PMID = 20660830.001).
  • [ISSN] 1527-7755
  • [Journal-full-title] Journal of clinical oncology : official journal of the American Society of Clinical Oncology
  • [ISO-abbreviation] J. Clin. Oncol.
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / U10 CA29139; United States / NCI NIH HHS / CA / U10 CA98413; United States / NCI NIH HHS / CA / U10 CA098413; United States / NCI NIH HHS / CA / U10 CA13539; United States / NCI NIH HHS / CA / U10 CA029139
  • [Publication-type] Clinical Trial, Phase II; Comparative Study; Journal Article; Randomized Controlled Trial; Research Support, N.I.H., Extramural
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 7M7YKX2N15 / Topotecan; 8N3DW7272P / Cyclophosphamide
  • [Other-IDs] NLM/ PMC2940398
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23. Garaventa A, Boni L, Lo Piccolo MS, Tonini GP, Gambini C, Mancini A, Tonegatti L, Carli M, di Montezemolo LC, Di Cataldo A, Casale F, Mazzocco K, Cecchetto G, Rizzo A, Bernardi B, Italian Cooperative Group for Neuroblastoma: Localized unresectable neuroblastoma: results of treatment based on clinical prognostic factors. Ann Oncol; 2002 Jun;13(6):956-64
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  • [Title] Localized unresectable neuroblastoma: results of treatment based on clinical prognostic factors.
  • BACKGROUND: We previously reported that stage 3 neuroblastoma comprises (i) a low-risk group including all infants (age 0-11 months) as well as older children with non-abdominal primaries, and (ii) a high-risk group made up of children >1 year of age with abdominal primaries.
  • Aggressive chemotherapy was effective only in the latter group.
  • PATIENTS AND TREATMENT: On this basis, in 1990 we designed a new protocol by which all low-risk patients received standard-dose chemotherapy, while the high-risk ones received very aggressive chemotherapy.
  • CONCLUSIONS: In low-risk stage 3 neuroblastoma, standard-dose chemotherapy is associated with an excellent chance of being cured.
  • Aggressive chemotherapy is effective for high-risk patients, but results are still unsatisfactory.
  • MYCN gene amplification is a prognostic indicator for most, but not all, treatment failures.

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  • (PMID = 12123342.001).
  • [ISSN] 0923-7534
  • [Journal-full-title] Annals of oncology : official journal of the European Society for Medical Oncology
  • [ISO-abbreviation] Ann. Oncol.
  • [Language] ENG
  • [Publication-type] Comparative Study; Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] England
  • [Chemical-registry-number] 5J49Q6B70F / Vincristine; 80168379AG / Doxorubicin; 8N3DW7272P / Cyclophosphamide
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24. Okazaki T, Kohno S, Mimaya J, Hasegawa S, Urushihara N, Yoshida A, Kawano S, Kusafuka J, Horikoshi Y, Takashima Y, Aoki K, Hamazaki M: Neuroblastoma detected by mass screening: the Tumor Board's role in its treatment. Pediatr Surg Int; 2004 Jan;20(1):27-32
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  • [Title] Neuroblastoma detected by mass screening: the Tumor Board's role in its treatment.
  • Japan has a nationwide mass-screening program for neuroblastoma in 6-month-old infants.
  • Neuroblastoma can regress spontaneously, and some institutions observe selected cases.
  • We evaluated the management of screened neuroblastoma at our hospital since 1997 when an observation program was introduced.
  • Criteria for the observation program were stage-I, stage-II, or stage-IVs tumors, urinary vanillylmandelic acid (VMA) and homovanillic acid (HVA) levels <40 microg/mg creatinine, tumor <5 cm in diameter, no invasion to the intraspinal canal or great vessels, and parental consent to participate.
  • Patients who did not meet observation criteria underwent surgery or mild chemotherapy according to the location of the tumor.
  • If patients met observation criteria after chemotherapy, surgical intervention was no longer performed.
  • Thirty-six patients attended our hospital for screened neuroblastoma from 1997 to 2002.
  • Four patients underwent primary surgery (group C), and 19 patients had chemotherapy initially.
  • Fourteen patients met observation criteria after chemotherapy and two are still having chemotherapy (group D).
  • There was no evidence of unfavorable prognosis in any of the 33 subjects, although 1 patient who underwent primary surgery had a vanishing kidney 1 year later and 1 patient had multiple bony metastases after complete resection of tumor, which was treated by chemotherapy.
  • Until the real significance of mass screening for neuroblastoma as a public health measure is confirmed, observation with careful follow-up should be adopted more extensively because it has a favorable outcome in many cases, and is associated with minimal therapeutic complications.
  • [MeSH-major] Mass Screening. Neuroblastoma / prevention & control
  • [MeSH-minor] Adrenal Gland Neoplasms / pathology. Adrenal Gland Neoplasms / prevention & control. Adrenal Gland Neoplasms / surgery. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Creatinine / urine. Follow-Up Studies. Homovanillic Acid / urine. Hospitals, Pediatric. Humans. Infant. Japan. Neoplasm Regression, Spontaneous. Neoplasm Staging. Parental Consent. Prognosis. Proto-Oncogene Proteins c-myc / analysis. Retroperitoneal Neoplasms / pathology. Retroperitoneal Neoplasms / prevention & control. Retroperitoneal Neoplasms / urine. Treatment Outcome. Vanilmandelic Acid / urine

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  • (PMID = 14689211.001).
  • [ISSN] 0179-0358
  • [Journal-full-title] Pediatric surgery international
  • [ISO-abbreviation] Pediatr. Surg. Int.
  • [Language] eng
  • [Publication-type] Comparative Study; Journal Article
  • [Publication-country] Germany
  • [Chemical-registry-number] 0 / Proto-Oncogene Proteins c-myc; 55-10-7 / Vanilmandelic Acid; AYI8EX34EU / Creatinine; X77S6GMS36 / Homovanillic Acid
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25. de Buys Roessingh AS, Rougemont AL, Wiesenauer C, Barrette S, Bouron-Dal Soglio D, Lallier M: Implication of unfavorable histology, MYCN amplification and diploidy for stage I and II neuroblastomas. Eur J Pediatr Surg; 2008 Dec;18(6):410-4
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  • [Title] Implication of unfavorable histology, MYCN amplification and diploidy for stage I and II neuroblastomas.
  • BACKGROUND: Surgery is the first line treatment for low-grade neuroblastomas.
  • In stage I tumors, the presence of MYCN amplification is rarely detected and the Shimada histology is not always taken into consideration when deciding on the treatment.
  • METHODS: We analyzed the assessment and follow-up of children with low-grade neuroblastomas (stages I and II) with or without MYCN amplification, with either a favorable or unfavorable histology and with or without tumor cell diploidy.
  • RESULTS: From 1995 to 2006, out of 114 neuroblastomas, nine (7.9 %) were stage I and 21 (18.4 %) stage II.
  • Of these 30 patients, 27 underwent surgery alone and three received chemotherapy after surgery.
  • The combination of MYCN amplification, unfavorable histology and diploidy was noted in one patient who developed metastases within two months.
  • Unfavorable histology alone was noted in four patients, of whom one suffered a recurrence of the tumor (previously stage I) and three are tumor-free after six years.
  • CONCLUSION: Because MYCN amplification and unfavorable histology are rare in early stage neuroblastomas, these tumors may be misclassified if they are not investigated further.
  • It seems that no single clinical or biological feature can be considered a significant factor in establishing a prognosis or determining whether additional treatment is required.
  • [MeSH-major] DNA, Neoplasm / metabolism. Diploidy. Gene Amplification. Genetic Markers. Neuroblastoma / pathology. Nuclear Proteins / genetics. Oncogene Proteins / genetics

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  • (PMID = 19012235.001).
  • [ISSN] 0939-7248
  • [Journal-full-title] European journal of pediatric surgery : official journal of Austrian Association of Pediatric Surgery ... [et al] = Zeitschrift für Kinderchirurgie
  • [ISO-abbreviation] Eur J Pediatr Surg
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] Germany
  • [Chemical-registry-number] 0 / DNA, Neoplasm; 0 / Genetic Markers; 0 / MYCN protein, human; 0 / Nuclear Proteins; 0 / Oncogene Proteins
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26. Ashton LJ, Murray JE, Haber M, Marshall GM, Ashley DM, Norris MD: Polymorphisms in genes encoding drug metabolizing enzymes and their influence on the outcome of children with neuroblastoma. Pharmacogenet Genomics; 2007 Sep;17(9):709-17
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  • [Title] Polymorphisms in genes encoding drug metabolizing enzymes and their influence on the outcome of children with neuroblastoma.
  • BACKGROUND: Although several studies have shown that drug metabolizing enzyme gene polymorphisms may influence the impact of therapy in childhood leukemia, no comprehensive investigations have been carried out in children with neuroblastoma.
  • The aim of this study was to identify polymorphisms in the genes encoding phase I and II drug metabolizing enzymes associated with the risk of relapse or death in a cohort of 209 children with neuroblastoma.
  • METHODS: Real-time PCR allelic discrimination was used to characterize the presence of polymorphisms in DNA from children with neuroblastoma.
  • RESULTS: As previously reported, amplification of MYCN (hazards ratio=4.25, 95% confidence interval=2.76-6.56, P<0.001), unfavorable stage (hazard ratio=4.14, 95% confidence interval=2.3-7.47, P<0.001) or age more than 1 year at diagnosis (hazard ratio=1.86, 95% confidence interval=1.19-2.92, P=0.007) were all associated with an increased risk of relapse or death.
  • In multivariate analysis, children who were GSTM1 null were more likely to relapse or die during follow-up after adjusting for MYCN amplification, stage and age at diagnosis (hazard ratio=1.6, 95% confidence interval=1.02-2.9, P=0.04).
  • CONCLUSIONS: These observations suggest that the NAT1*11 variant and the GSTM1 wild-type genotype contribute to a more favorable outcome in patients treated for neuroblastoma and are the first to demonstrate a relationship between NAT1 and GSTM1 genotypes in childhood neuroblastoma.
  • [MeSH-major] Neuroblastoma / enzymology. Neuroblastoma / genetics. Pharmaceutical Preparations / metabolism

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  • (PMID = 17700360.001).
  • [ISSN] 1744-6872
  • [Journal-full-title] Pharmacogenetics and genomics
  • [ISO-abbreviation] Pharmacogenet. Genomics
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / DNA Primers; 0 / Isoenzymes; 0 / MYCN protein, human; 0 / Nuclear Proteins; 0 / Oncogene Proteins; 0 / Pharmaceutical Preparations; 9035-51-2 / Cytochrome P-450 Enzyme System; EC 2.3.1.5 / Arylamine N-Acetyltransferase; EC 2.3.1.5 / N-acetyltransferase 1; EC 2.5.1.18 / Glutathione Transferase; EC 2.5.1.18 / glutathione S-transferase M1
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27. Tang JY, Pan C, Chen J, Xu M, Chen J, Xue HL, Gu LJ, Dong R, Ye H, Zhou M, Wang YP: [Comprehensive protocol for diagnosis and treatment of childhood neuroblastoma--results of 45 cases]. Zhonghua Er Ke Za Zhi; 2006 Oct;44(10):770-3
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  • [Title] [Comprehensive protocol for diagnosis and treatment of childhood neuroblastoma--results of 45 cases].
  • OBJECTIVE: The aim of the paper was to improve the prognosis of neuroblastoma (NB) stage III and IV in children through the comprehensive therapy including chemotherapy, delayed tumor resection, autologous stem cell transplantation (ASCT) and inducing differentiation and to analyze the factors affecting the prognosis.
  • METHODS: Newly diagnosed neuroblastoma patients seen from Oct.1998 to Dec.2003 were divided into high, medium and low risk groups depending on clinical stage and age.
  • Comprehensive protocol included accurate staging, delayed and/or second tumor resection for stage III and IV patients, chemotherapy of different intensity mainly composed of cell cycle nonspecific drugs and 13-cis-retinoid for inducing cell differentiation.
  • ASCT was given at the end of therapy for high risk group.
  • Nine cases had stage I, 1 case had stage II, 8 cases had III, 26 cases had stage IV and 1 case had stage IVs of the tumor.
  • Depending on the age and stage of the tumor, 26 cases were aligned into high risk protocol, 10 into medium risk and 9 into low risk groups.
  • Of the thirty-nine patients, 31 achieved complete remission (CR) and 8 partial remission (PR) after surgery and/or chemotherapy.
  • No death occurred from treatment complication.
  • Statistical analysis showed that the age older than 18 months, and stage III and IV of the tumor were the factors predicting poor prognosis (P = 0.04 and 0.003, respectively).
  • Age older than 18 months, and stage III and IV were the factors suggesting poor prognosis.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Hematopoietic Stem Cell Transplantation. Neuroblastoma / diagnosis. Neuroblastoma / therapy. Transplantation, Autologous
  • [MeSH-minor] Age Factors. Chemotherapy, Adjuvant. Child. Child, Preschool. Female. Follow-Up Studies. Humans. Infant. Male. Neoplasm Recurrence, Local / prevention & control. Neoplasm Recurrence, Local / surgery. Neoplasm Recurrence, Local / therapy. Neoplasm Staging. Prognosis. Remission Induction / methods. Retrospective Studies. Severity of Illness Index. Survival Rate. Time Factors. Treatment Outcome

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  • (PMID = 17229383.001).
  • [ISSN] 0578-1310
  • [Journal-full-title] Zhonghua er ke za zhi = Chinese journal of pediatrics
  • [ISO-abbreviation] Zhonghua Er Ke Za Zhi
  • [Language] chi
  • [Publication-type] English Abstract; Journal Article
  • [Publication-country] China
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28. Raguénez G, Douc-Rasy S, Blanc E, Goldschneider D, Barrois M, Valteau-Couanet D, Bénard J: [A functional gene map is required to adapt therapy of metastatic neuroblastoma]. Bull Cancer; 2001 Mar;88(3):295-304
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  • [Title] [A functional gene map is required to adapt therapy of metastatic neuroblastoma].
  • [Transliterated title] Vers une carte génétique fonctionnelle des neuroblastomes métastiques pour une thérapeutique adaptée.
  • Neuroblastoma is a very common solid tumor which arises in childhood and shows an extreme heterogeneity at the clinical, histological and genetic levels.
  • Besides age and stage, N-myc amplification and 1p deletion are prognostic factors of the disease: in Europe, these genetic markers are used to conduct therapy.
  • In France, N-myc amplification is a factor of bad prognosis which leads, in all forms of the disease including localised forms and metastatic forms of children aged of less than 1 year, to a myeloablative treatment with autologous hematopoietic stem cells transplantation.
  • Moreover, experimental models of human metastatic neuroblastoma have been obtained in which variations of genes transcript levels involved in these pathways, are observed.
  • The current break-through of cDNA microarrays allows to develop a dynamic transcriptomic scanning of these models as well as of tumors and bone marrows from patients upon conventional chemotherapy.
  • ii) to apply adapted treatment;.
  • iii) to develop new therapeutic strategies.
  • [MeSH-major] Genes, myc / genetics. Neuroblastoma / genetics

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  • (PMID = 11313207.001).
  • [ISSN] 0007-4551
  • [Journal-full-title] Bulletin du cancer
  • [ISO-abbreviation] Bull Cancer
  • [Language] fre
  • [Publication-type] English Abstract; Journal Article
  • [Publication-country] France
  • [Chemical-registry-number] 0 / Neoplasm Proteins; 0 / Nerve Growth Factors
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29. Don S, Verrills NM, Liaw TY, Liu ML, Norris MD, Haber M, Kavallaris M: Neuronal-associated microtubule proteins class III beta-tubulin and MAP2c in neuroblastoma: role in resistance to microtubule-targeted drugs. Mol Cancer Ther; 2004 Sep;3(9):1137-46
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  • [Title] Neuronal-associated microtubule proteins class III beta-tubulin and MAP2c in neuroblastoma: role in resistance to microtubule-targeted drugs.
  • Advanced stage neuroblastoma has a poor clinical outcome and microtubule-destabilizing agents, such as the Vinca alkaloids, are an important component in the treatment of this childhood cancer.
  • To date, studies examining the contribution of microtubules and associated proteins to the efficacy of microtubule-destabilizing agents in neuroblastoma have been limited.
  • In this study, BE2-C neuroblastoma cells previously selected for resistance to either vincristine (BE/VCR10) or colchicine (BE/CHCb0.2) were found to display significant decreases in neuronal-specific class III beta-tubulin.
  • Interestingly, vincristine-selected cells exhibited increased levels of polymerized tubulin that were not due to alpha-tubulin and class I, II, or III beta-tubulin mutations.
  • This is the first report describing specific microtubule alterations in neuroblastoma cells resistant to tubulin-targeted agents.
  • The results indicate a need to identify the factors responsible for resistance to tubulin-targeted agents in neuroblastoma so that improved and novel treatment strategies can be developed for this drug refractory disease.
  • [MeSH-major] Drug Resistance, Neoplasm. Microtubule-Associated Proteins / metabolism. Microtubules / drug effects. Neuroblastoma / drug therapy. Neuroblastoma / metabolism. Tubulin / metabolism

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  • (PMID = 15367708.001).
  • [ISSN] 1535-7163
  • [Journal-full-title] Molecular cancer therapeutics
  • [ISO-abbreviation] Mol. Cancer Ther.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / MAP2 protein, human; 0 / MAP4; 0 / Microtubule Proteins; 0 / Microtubule-Associated Proteins; 0 / Phosphoproteins; 0 / Protein Isoforms; 0 / STMN1 protein, human; 0 / Stathmin; 0 / Tubulin; P88XT4IS4D / Paclitaxel
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30. Kretschmar CS, Kletzel M, Murray K, Thorner P, Joshi V, Marcus R, Smith EI, London WB, Castleberry R: Response to paclitaxel, topotecan, and topotecan-cyclophosphamide in children with untreated disseminated neuroblastoma treated in an upfront phase II investigational window: a pediatric oncology group study. J Clin Oncol; 2004 Oct 15;22(20):4119-26
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  • [Title] Response to paclitaxel, topotecan, and topotecan-cyclophosphamide in children with untreated disseminated neuroblastoma treated in an upfront phase II investigational window: a pediatric oncology group study.
  • PURPOSE: Most children older than 1 year of age with metastatic neuroblastoma (NB) die despite intensive chemotherapy and bone marrow transplantation.
  • The Pediatric Oncology Group conducted a study of paclitaxel, topotecan, and topotecan-cyclophosphamide (topo-cyclo) in newly diagnosed children with stage IV NB.
  • Patients were re-evaluated after two courses and then treated with intensive induction therapy and bone marrow transplantation.
  • Four patients had grade 3 to 4 allergic reactions to paclitaxel; most patients developed grade 3 to 4 marrow suppression after topotecan or topo-cyclo.
  • Neither disease-free survival nor overall survival differed significantly between children who received a phase II agent and those who did not.
  • [MeSH-major] Antineoplastic Agents / therapeutic use. Cyclophosphamide / therapeutic use. Neuroblastoma / drug therapy. Paclitaxel / therapeutic use. Topotecan / therapeutic use
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Cohort Studies. Disease-Free Survival. Drug Administration Schedule. Female. Humans. Infant. Male. Survival Rate

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  • (PMID = 15483021.001).
  • [ISSN] 0732-183X
  • [Journal-full-title] Journal of clinical oncology : official journal of the American Society of Clinical Oncology
  • [ISO-abbreviation] J. Clin. Oncol.
  • [Language] eng
  • [Publication-type] Clinical Trial; Clinical Trial, Phase II; Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 7M7YKX2N15 / Topotecan; 8N3DW7272P / Cyclophosphamide; P88XT4IS4D / Paclitaxel
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31. Bégaud-Grimaud G, Battu S, Lazcoz P, Castresana JS, Jauberteau MO, Cardot PJ: Study of the phenotypic relationship in the IMR-32 human neuroblastoma cell line by sedimentation field flow fractionation. Int J Oncol; 2007 Oct;31(4):883-92
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  • [Title] Study of the phenotypic relationship in the IMR-32 human neuroblastoma cell line by sedimentation field flow fractionation.
  • Neuroblastoma (NB) is the most common childhood solid tumor.
  • Cell type heterogeneity is observed either in the morphological appearance of NB tumors or in cell lines isolated from tumor specimens.
  • NB consists of two principal neoplastic cell types: i) neuroblastic or N-type (undifferentiated cells); and ii) stromal or S-type (differentiated cells).
  • As NB cells seem to have the capacity to differentiate spontaneously in vivo and in vitro, their heterogeneity could affect treatment outcome, in particular the response to apoptosis induced by chemotherapy.
  • Therefore, it is important to understand the underlying process governing changes in differentiation in order to improve treatment response and NB patient outcome and the neoplastic population in IMR-32 represented a good model for such a study.
  • Results showed that this cell line was extremely heterogeneous and highly variable in its stage of differentiation and we demonstrated that sedimentation field flow fractionation (SdFFF) permitted the isolation of 2 N-phenotypes and contributed to the understanding of the IMR-32 cell population dynamics.
  • The first N-phenotype forms a pool of quiescent undifferentiated cells while the second one was able to proliferate (incorporation of BrdU) and also give rise to adherent S-type cells (PSA-N-CAM+ and N-CAM+).
  • [MeSH-major] Cell Differentiation. Cell Fractionation. Fractionation, Field Flow. Neuroblastoma / metabolism. Neuroblastoma / pathology

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  • (PMID = 17786321.001).
  • [ISSN] 1019-6439
  • [Journal-full-title] International journal of oncology
  • [ISO-abbreviation] Int. J. Oncol.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] Greece
  • [Chemical-registry-number] 0 / Neural Cell Adhesion Molecule L1; 0 / Sialic Acids; 0 / polysialyl neural cell adhesion molecule; G34N38R2N1 / Bromodeoxyuridine
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32. Hase T, Ohta S, Tani T, Mizukuro T, Mekata E, Naitoh H, Shimadera S, Fujino S, Taga T: Outcome of infants with neuroblastoma detected by mass screening and surgically treated in Shiga Prefecture, Japan: what is the role of surgery? Pediatr Surg Int; 2002 Sep;18(5-6):289-94
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  • [Title] Outcome of infants with neuroblastoma detected by mass screening and surgically treated in Shiga Prefecture, Japan: what is the role of surgery?
  • To investigate retrospectively the clinical and biological features that influence the outcome of infants with neuroblastoma (NB) detected by mass screening (NBMS), and to construct surgical strategies to deal with NBMS, 20 infants diagnosed as having either NB or ganglioneuroblastoma (GNB) between 1986 and 1998 were enrolled in a study.
  • The following factors were analyzed by multivariate analysis: age, stage according to the Japanese staging system at the time of diagnosis, site of the primary tumor, histologic findings, preoperative urinary vanillylmandelic acid (VMA) and homovanillic acid (HVA) levels, VMA/HVA ratio, lactate dehydrogenase, neuron-specific enolase, Shimada's histologic classification, amplification of the N- myc oncogene by Southern blot analysis, nuclear content, and chromosomal abnormality.
  • Four infants had stage I, 6 stage II, 3 stage III, 3 stage IVB, and 4 stage IV disease.
  • The infant with N- myc-positive NB (stage II) died 23 months after surgery in spite of aggressive postoperative chemotherapy.
  • The N- myc-positive NB case implies that even in locoregional NB detected by NBMS, surgical excision should play a central role in the diagnosis of its oncogenic characteristics and indicate any subsequent therapy.
  • [MeSH-major] Adrenal Gland Neoplasms / surgery. Neuroblastoma / surgery. Retroperitoneal Neoplasms / surgery
  • [MeSH-minor] Biomarkers, Tumor / urine. Female. Homovanillic Acid / urine. Humans. Infant. Japan. Male. Mass Screening. Neoplasm Staging. Prognosis. Retrospective Studies. Treatment Outcome. Vanilmandelic Acid / urine

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  • [CommentIn] Pediatr Surg Int. 2002 Sep;18(5-6):288 [12415340.001]
  • (PMID = 12415341.001).
  • [ISSN] 0179-0358
  • [Journal-full-title] Pediatric surgery international
  • [ISO-abbreviation] Pediatr. Surg. Int.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] Germany
  • [Chemical-registry-number] 0 / Biomarkers, Tumor; 55-10-7 / Vanilmandelic Acid; X77S6GMS36 / Homovanillic Acid
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33. Donfrancesco A, Jenkner A, Castellano A, Ilari I, Milano GM, De Sio L, Cozza R, Fidani P, Deb G, De Laurentis C, Inserra A, Dominici C: Ifosfamide/carboplatin/etoposide (ICE) as front-line, topotecan/cyclophosphamide as second-line and oral temozolomide as third-line treatment for advanced neuroblastoma over one year of age. Acta Paediatr Suppl; 2004 May;93(445):6-11
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  • [Title] Ifosfamide/carboplatin/etoposide (ICE) as front-line, topotecan/cyclophosphamide as second-line and oral temozolomide as third-line treatment for advanced neuroblastoma over one year of age.
  • Children affected by advanced neuroblastoma have a discouraging prognosis, but intensive induction chemotherapy may increase the complete response rate.
  • The combination of ifosfamide, carboplatin and etoposide (ICE) was used for the first time as front-line regimen in patients with stage 4 neuroblastoma over the age of 1 y.
  • Similarly, second-line treatment for children with relapsed neuroblastoma, particularly after high-dose chemotherapy, has been unsatisfactory.
  • Furthermore, there is a need for effective palliative treatment in patients failing therapy.
  • Temozolomide, a new dacarbazine analog with optimal oral bioavailability, is being used in an ongoing phase II study as an alternative to oral etoposide.
  • Seventeen patients with stage 4 neuroblastoma have entered the ICE study; 15/16 (94%) major responses after induction were observed and 6/16 (37%) evaluable patients are disease free after a median of 51 mo.
  • Second-line treatment was effective in obtaining remissions, some of them long lasting.
  • Third-line treatment did not elicit measurable responses in neuroblastoma, but achieved prolonged freedom from disease progression and excellent palliation in several patients.
  • [MeSH-major] Antineoplastic Agents, Alkylating / therapeutic use. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Dacarbazine / analogs & derivatives. Dacarbazine / therapeutic use. Neuroblastoma / drug therapy
  • [MeSH-minor] Adolescent. Adult. Carboplatin / administration & dosage. Child. Child, Preschool. Cyclophosphamide / administration & dosage. Drug Resistance, Neoplasm. Etoposide / administration & dosage. Female. Humans. Ifosfamide / administration & dosage. Infant. Male. Neoplasm Recurrence, Local / drug therapy. Palliative Care. Topotecan / administration & dosage. Treatment Outcome

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  • (PMID = 15176712.001).
  • [ISSN] 0803-5326
  • [Journal-full-title] Acta paediatrica (Oslo, Norway : 1992). Supplement
  • [ISO-abbreviation] Acta Paediatr Suppl
  • [Language] eng
  • [Publication-type] Clinical Trial; Clinical Trial, Phase II; Journal Article
  • [Publication-country] Norway
  • [Chemical-registry-number] 0 / Antineoplastic Agents, Alkylating; 6PLQ3CP4P3 / Etoposide; 7GR28W0FJI / Dacarbazine; 7M7YKX2N15 / Topotecan; 85622-93-1 / temozolomide; 8N3DW7272P / Cyclophosphamide; BG3F62OND5 / Carboplatin; UM20QQM95Y / Ifosfamide
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34. Pradhan KR, Johnson CS, Vik TA, Sender LS, Kreissman SG: A novel intensive induction therapy for high-risk neuroblastoma utilizing sequential peripheral blood stem cell collection and infusion as hematopoietic support. Pediatr Blood Cancer; 2006 Jun;46(7):793-802
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  • [Title] A novel intensive induction therapy for high-risk neuroblastoma utilizing sequential peripheral blood stem cell collection and infusion as hematopoietic support.
  • OBJECTIVE: To determine the feasibility, toxicities, and the response rate (RR) of a dose intensive, submyeloablative, induction chemotherapy protocol termed EPiC (etoposide, carboplatin, and intensive cyclophosphamide) utilizing sequential peripheral blood stem cell (PBSC) collection and infusion as hematopoietic support in children with newly diagnosed Stage 4 neuroblastoma.
  • PATIENTS AND METHODS: Twenty-five children (age >1 year) with Stage 4 neuroblastoma were enrolled.
  • Following EPiC and surgical resection of the primary tumor, patients proceeded to various consolidation therapies.
  • RR was scored using the International Neuroblastoma Response Criteria.
  • RESULTS: Using PBSC infusion following EPiC chemotherapy resulted in a dose intensity averaging 85% of intended dose intensity; and in early neutrophil but not platelet recovery.
  • CONCLUSION: EPiC is a feasible, well-tolerated, sub-myeloablative, induction chemotherapy protocol for children with high-risk neuroblastoma.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / administration & dosage. Neuroblastoma / therapy. Peripheral Blood Stem Cell Transplantation / methods
  • [MeSH-minor] Carboplatin / administration & dosage. Carboplatin / adverse effects. Child. Child, Preschool. Combined Modality Therapy. Etoposide / administration & dosage. Etoposide / adverse effects. Female. Hematopoietic Stem Cell Mobilization. Humans. Infant. Linear Models. Male. Pilot Projects. Remission Induction. Survival Analysis

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  • [Copyright] (c) 2005 Wiley-Liss, Inc.
  • (PMID = 16206215.001).
  • [ISSN] 1545-5009
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Clinical Trial, Phase II; Journal Article; Multicenter Study
  • [Publication-country] United States
  • [Chemical-registry-number] 6PLQ3CP4P3 / Etoposide; BG3F62OND5 / Carboplatin
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35. Castel V, Cañete A, Navarro S, García-Miguel P, Melero C, Acha T, Navajas A, Badal MD: Outcome of high-risk neuroblastoma using a dose intensity approach: improvement in initial but not in long-term results. Med Pediatr Oncol; 2001 Dec;37(6):537-42
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  • [Title] Outcome of high-risk neuroblastoma using a dose intensity approach: improvement in initial but not in long-term results.
  • BACKGROUND: Stage 4 and MYCN amplified (MNA) neuroblastoma in children have a poor prognosis.
  • PROCEDURE: High-risk children were studied according to the International Neuroblastoma Staging System, then treated with high-dose cyclophosphamide and high-dose carboplatin, followed by surgery and autologous stem cell transplant or maintenance chemotherapy.
  • RESULTS: From June 1992 to December 1998, 83 children were admitted in the study (72 stage 4> 1 year, 5 stage 4 MNA infants, and 6 MNA stage 3 children); tumor tissue was obtained from 73, MYCN was performed in 65, being amplified in 21 (32%).
  • Induction chemotherapy was administered in the expected time in 35% of patients.
  • Its toxicity was mainly hematologic followed by infections, and there were 3 chemotherapy-related deaths.
  • Chemotherapy plus surgery produced some response in 90% of patients, 53% were in CR/VGPR; 49 children received autologous SCT, and 16 received maintenance chemotherapy for 9 months.
  • CONCLUSIONS: High-dose cyclophosphamide and high-dose carboplatin are effective in the initial treatment of neuroblastoma; combined with surgery they produce some response in most patients.
  • Survival time has also been prolonged but most patients relapse with metastases.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Hematopoietic Stem Cell Transplantation. Neuroblastoma / drug therapy. Neuroblastoma / mortality
  • [MeSH-minor] Adolescent. Carboplatin / administration & dosage. Child. Child, Preschool. Cyclophosphamide / administration & dosage. Disease-Free Survival. Drug Administration Schedule. Female. Humans. Infant. Male. Neoplasm Staging. Spain. Survival Analysis. Survivors. Transplantation Conditioning. Treatment Outcome

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  • [Copyright] Copyright 2001 Wiley-Liss, Inc.
  • (PMID = 11745893.001).
  • [ISSN] 0098-1532
  • [Journal-full-title] Medical and pediatric oncology
  • [ISO-abbreviation] Med. Pediatr. Oncol.
  • [Language] eng
  • [Publication-type] Clinical Trial; Clinical Trial, Phase II; Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 8N3DW7272P / Cyclophosphamide; BG3F62OND5 / Carboplatin
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36. Yoneda A, Oue T, Imura K, Inoue M, Yagi K, Kawa K, Nishikawa M, Morimoto S, Nakayama M: Observation of untreated patients with neuroblastoma detected by mass screening: a "wait and see" pilot study. Med Pediatr Oncol; 2001 Jan;36(1):160-2
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Observation of untreated patients with neuroblastoma detected by mass screening: a "wait and see" pilot study.
  • BACKGROUND: Recent studies have indicated that mass screening for neuroblastoma detects tumors that otherwise would have regressed spontaneously without recognition.
  • PROCEDURE: Eighteen patients were detected by mass screening between June 1994 and December 1996.
  • Eight of these cases matched the following criteria and entered the observation program: Stage I or II, less than 5 cm in diameter; no involvement of large vessels or organs; not difficult to resect; informed consent.
  • CONCLUSIONS: At least 60% of neuroblastoma cases who entered our observation program regressed spontaneously.
  • [MeSH-major] Adrenal Gland Neoplasms / epidemiology. Mass Screening. Neoplasm Regression, Spontaneous. Neuroblastoma / epidemiology. Retroperitoneal Neoplasms / epidemiology
  • [MeSH-minor] Biomarkers, Tumor. Chemotherapy, Adjuvant. Disease Progression. Follow-Up Studies. Ganglioneuroblastoma / drug therapy. Ganglioneuroblastoma / epidemiology. Ganglioneuroblastoma / pathology. Ganglioneuroblastoma / surgery. Ganglioneuroblastoma / urine. Genes, myc. Homovanillic Acid / urine. Humans. Infant. Japan / epidemiology. Neoplasm Proteins / blood. Phosphopyruvate Hydratase / blood. Pilot Projects. Prognosis. Prospective Studies. Registries. Treatment Outcome. Vanilmandelic Acid / urine

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  • (PMID = 11464874.001).
  • [ISSN] 0098-1532
  • [Journal-full-title] Medical and pediatric oncology
  • [ISO-abbreviation] Med. Pediatr. Oncol.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Biomarkers, Tumor; 0 / Neoplasm Proteins; 55-10-7 / Vanilmandelic Acid; EC 4.2.1.11 / Phosphopyruvate Hydratase; X77S6GMS36 / Homovanillic Acid
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37. Adamson PC, Matthay KK, O'Brien M, Reaman GH, Sato JK, Balis FM: A phase 2 trial of all-trans-retinoic acid in combination with interferon-alpha2a in children with recurrent neuroblastoma or Wilms tumor: A Pediatric Oncology Branch, NCI and Children's Oncology Group Study. Pediatr Blood Cancer; 2007 Oct 15;49(5):661-5
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  • [Title] A phase 2 trial of all-trans-retinoic acid in combination with interferon-alpha2a in children with recurrent neuroblastoma or Wilms tumor: A Pediatric Oncology Branch, NCI and Children's Oncology Group Study.
  • BACKGROUND: The combination of the antiproliferative and differentiation-inducing effects of retinoids together with the antiproliferative, immunostimulatory, and differentiation-potentiating effects of interferon-alpha (IFN-alpha) were the basis for the development of this combination in pediatric patients with refractory neuroblastoma or Wilms tumor.
  • PROCEDURE: A phase 2 trial of all-trans-retinoic acid (ATRA), administered orally at a dose of 90 mg/m(2)/day in three divided doses for 3 consecutive days per week, and IFN-alpha2a, administered subcutaneously daily at a dose of 3 x 10(6) U/m(2)/day for 5 consecutive days per week, in 4 week cycles was performed.
  • A two-stage design was used for each disease stratum.
  • RESULTS: Seventeen patients (16 evaluable) with neuroblastoma, median age 9 years, and 15 patients (14 evaluable) with Wilms tumor, median age 6 years, were enrolled.
  • Four patients with neuroblastoma had stable disease for 12 or more weeks.
  • CONCLUSIONS: The combination of ATRA and IFN-alpha2a was inactive in children with relapsed or refractory neuroblastoma and Wilms tumor.
  • The lack of activity with this combination in children with refractory neuroblastoma is similar to the disappointing phase 2 results of single agent 13-cis-retinoic-acid (13cRA) and does not support further development of ATRA for children with relapsed neuroblastoma.
  • [MeSH-major] Interferon-alpha / administration & dosage. Neuroblastoma / drug therapy. Tretinoin / administration & dosage. Wilms Tumor / drug therapy
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Drug Therapy, Combination. Humans. Infant. Recombinant Proteins. Remission Induction. Salvage Therapy / methods. Treatment Outcome


38. Geoerger B, Brasme JF, Daudigeos-Dubus E, Opolon P, Venot C, Debussche L, Vrignaud P, Vassal G: Anti-insulin-like growth factor 1 receptor antibody EM164 (murine AVE1642) exhibits anti-tumour activity alone and in combination with temozolomide against neuroblastoma. Eur J Cancer; 2010 Dec;46(18):3251-62
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  • [Title] Anti-insulin-like growth factor 1 receptor antibody EM164 (murine AVE1642) exhibits anti-tumour activity alone and in combination with temozolomide against neuroblastoma.
  • Insulin-like growth factor 1 receptor (IGF-1R) is overexpressed in many tumours and contributes to tumourigenicity, cell proliferation, metastasis and resistance, thus representing a promising therapeutic target.
  • We investigated the EM164 for its therapeutic potential against childhood neuroblastoma.
  • Sensitivity was independent from culture serum conditions, intensity of IGF-1R expression and IGF-II secretion, although associated with inhibition of AKT activation.
  • In vivo, EM164 administered intravenously at 40 mg/kg twice weekly for 4 weeks yielded significant tumour growth delays (TGD) of 13.4d in advanced stage IGR-N91 and 12.9 d in SK-N-AS tumours compared to controls (p = 0.02 and p = 0.0059, respectively).
  • Simultaneous treatment of EM164 0.7 μg/mL and temozolomide resulted in enhanced activity in vitro.
  • In vivo, treatment with temozolomide at the maximum tolerated dose (100mg/kg/d for 5 consecutive days) and EM164 yielded a significantly greater TGD of 29.1d (p<0.01) and two complete tumour regressions (CR) compared to 18.1d (p = ns) and one CR for EM164 alone and 16.1d (p = ns) for temozolomide alone.
  • Our results demonstrate the potential of the anti-IGF-1R antibody alone and in combination with alkylating agents and support the therapeutic development of the AVE1642 for aggressive neuroblastoma.
  • [MeSH-major] Antibodies, Monoclonal / therapeutic use. Antineoplastic Agents, Alkylating / therapeutic use. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Neuroblastoma / drug therapy. Receptor, IGF Type 1 / antagonists & inhibitors
  • [MeSH-minor] Animals. Antibodies, Monoclonal, Humanized. Cell Line, Tumor. Cell Proliferation / drug effects. Dacarbazine / administration & dosage. Dacarbazine / analogs & derivatives. Female. Humans. Mice. Mice, Nude. Mitogen-Activated Protein Kinases / drug effects. Proto-Oncogene Proteins c-akt / drug effects. Xenograft Model Antitumor Assays / methods

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  • [Copyright] Copyright © 2010 Elsevier Ltd. All rights reserved.
  • (PMID = 20591650.001).
  • [ISSN] 1879-0852
  • [Journal-full-title] European journal of cancer (Oxford, England : 1990)
  • [ISO-abbreviation] Eur. J. Cancer
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] England
  • [Chemical-registry-number] 0 / AVE1642; 0 / Antibodies, Monoclonal; 0 / Antibodies, Monoclonal, Humanized; 0 / Antineoplastic Agents, Alkylating; 7GR28W0FJI / Dacarbazine; 85622-93-1 / temozolomide; EC 2.7.10.1 / Receptor, IGF Type 1; EC 2.7.11.1 / Proto-Oncogene Proteins c-akt; EC 2.7.11.24 / Mitogen-Activated Protein Kinases
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39. Drozynska E, Stefanowicz J, Polczynska K, Balcerska A, Czauderna P: [Application of Ethyol during chemotherapy in patients with impaired kidney function. Description of three cases]. Med Wieku Rozwoj; 2001 Jul-Sep;5(3 Suppl 1):7-13
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  • [Title] [Application of Ethyol during chemotherapy in patients with impaired kidney function. Description of three cases].
  • Nephrotoxicity is observed in 30% of patient's treated with chemotherapy schedules based on cisplatin analogues.
  • One of them, a 5 month old boy presented with agenesis of the right kidney and stage II tumour in the left kidney.
  • The third patient was a girl aged 5 who had isolated relapse of neuroblastoma in the central nervous system.
  • Primary treatment caused nephrotoxic complications such as Fanconi syndrome and diminished glomerular filtration (66 ml/min).
  • [MeSH-major] Amifostine / therapeutic use. Antineoplastic Combined Chemotherapy Protocols / adverse effects. Glomerular Filtration Rate / drug effects. Kidney Diseases / chemically induced. Radiation-Protective Agents / therapeutic use
  • [MeSH-minor] Central Nervous System Neoplasms / drug therapy. Child. Child, Preschool. Female. Humans. Infant. Kidney Neoplasms / drug therapy. Male. Neuroblastoma / drug therapy. Treatment Outcome. Wilms Tumor / drug therapy

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  • (PMID = 12004147.001).
  • [Journal-full-title] Medycyna wieku rozwojowego
  • [ISO-abbreviation] Med Wieku Rozwoj
  • [Language] pol
  • [Publication-type] Case Reports; English Abstract; Journal Article
  • [Publication-country] Poland
  • [Chemical-registry-number] 0 / Radiation-Protective Agents; M487QF2F4V / Amifostine
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40. Shi X, Zhu XD, Wang HM: [Effect of supporting the healthy energy and strengthening Pi principle of TCM combined with chemotherapy in treating children with solid tumors]. Zhongguo Zhong Xi Yi Jie He Za Zhi; 2007 Jun;27(6):542-5
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  • [Title] [Effect of supporting the healthy energy and strengthening Pi principle of TCM combined with chemotherapy in treating children with solid tumors].
  • OBJECTIVE: To study the clinical validity in improving quality of life (QOL) of patients and alleviating adverse reaction of chemotherapy in treating children with solid tumors by Chinese herbal medicine for supporting healthy energy and strengthening Pi.
  • METHODS: A prospective cohort study was conducted in 60 children with solid tumor in stage II-IV, who were assigned to two groups, 30 in each group.
  • All children received chemotherapy and those in the observed group were given Chinese herbs according to syndrome differentiation additionally.
  • The conditions of the two groups were compared after 6-month treatment.
  • RESULTS: Compared with before treatment, the white blood cells (WBC), hemoglobin (Hb), and platelet (PLT) all increased in the observed group after treatment (P < 0.01, P < 0.01, P < 0.05), while in the control group, the WBC and Hb have no significant difference (P > 0.05) and PLT decreased (P < 0.05) after treatment.
  • Comparison between the two groups, the clinical symptom score in the observed group has significant difference after treatment (P < 0.05).
  • CONCLUSION: Chinese herbal medicine for supporting healthy energy, strengthening Pi and supplementing qi-blood is good for alleviating the adverse reaction and improving patients' peripheral blood picture in children with solid tumor undergoing chemotherapy.

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  • (PMID = 17633370.001).
  • [ISSN] 1003-5370
  • [Journal-full-title] Zhongguo Zhong xi yi jie he za zhi Zhongguo Zhongxiyi jiehe zazhi = Chinese journal of integrated traditional and Western medicine
  • [ISO-abbreviation] Zhongguo Zhong Xi Yi Jie He Za Zhi
  • [Language] CHI
  • [Publication-type] English Abstract; Journal Article; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
  • [Publication-country] China
  • [Chemical-registry-number] 0 / Drugs, Chinese Herbal
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41. Berg SL, Blaney SM, Sullivan J, Bernstein M, Dubowy R, Harris MB, Pediatric Oncology Group: Phase II trial of pyrazoloacridine in children with solid tumors: a Pediatric Oncology Group phase II study. J Pediatr Hematol Oncol; 2000 Nov-Dec;22(6):506-9
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  • [Title] Phase II trial of pyrazoloacridine in children with solid tumors: a Pediatric Oncology Group phase II study.
  • The Pediatric Oncology Group conducted a phase II study to determine the activity of PZA administered as a 3-hour infusion.
  • PATIENTS AND METHODS: The activity of PZA was evaluated in patients with a variety of childhood solid tumors including rhabdomyosarcoma, Ewing sarcoma/peripheral neuroectodermal tumor, neuroblastoma, osteogenic sarcoma, Wilms tumor, or other solid tumors (excluding brain tumors).
  • In addition to a standard three-stage design to test the drug's activity in each tumor type, a global stopping rule was used such that if no complete or partial responses (CR or PR) occurred in the first 35 patients (pooled across all strata except "other"), the study would be closed.

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  • (PMID = 11132217.001).
  • [ISSN] 1077-4114
  • [Journal-full-title] Journal of pediatric hematology/oncology
  • [ISO-abbreviation] J. Pediatr. Hematol. Oncol.
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / CA-15525; United States / NCI NIH HHS / CA / U10 CA098413; United States / NCI NIH HHS / CA / CA-03161; United States / NCI NIH HHS / CA / U10 CA098543; United States / NCI NIH HHS / CA / CA-41573
  • [Publication-type] Clinical Trial; Clinical Trial, Phase II; Journal Article; Multicenter Study; Research Support, U.S. Gov't, P.H.S.
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Acridines; 0 / Antineoplastic Agents; 0 / Pyrazoles; 99009-20-8 / NSC 366140
  • [Other-IDs] NLM/ NIHMS522396; NLM/ PMC4008246
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42. Langevin AM, Bernstein M, Kuhn JG, Blaney SM, Ivy P, Sun J, Chen Z, Adamson PC, Children's Oncology Group: A phase II trial of rebeccamycin analogue (NSC #655649) in children with solid tumors: a Children's Oncology Group study. Pediatr Blood Cancer; 2008 Mar;50(3):577-80
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  • [Title] A phase II trial of rebeccamycin analogue (NSC #655649) in children with solid tumors: a Children's Oncology Group study.
  • BACKGROUND: Rebeccamycin Analogue (NSC #655649), a chemically synthesized glycosyl-dichloro-indolocarbazole derivative of rebeccamycin with topoisomerase inhibiting activity, has in vitro activity against pediatric tumor cell lines and tumor specimens including rhabdomyosarcoma, neuroblastoma, Ewing's sarcoma and medulloblastoma.
  • PROCEDURE: The primary objective of this trial was to determine the response rate to Rebeccamycin analogue NSC #655649 in children with refractory solid and CNS tumors.
  • A two-stage design was used for this Phase II trial.
  • Rebeccamycin analogue, 650 mg/m(2), was administered every 21 days, and could be escalated to 780 mg/m(2) in subsequent cycles to achieve a maximum plasma drug concentration >5 microg/ml.
  • Of 126 evaluable patients for response, only 4 patients had an objective response: 3 patients with rhabdomyosarcoma (1 CR and 2 PR) and 1 patient with neuroblastoma (1 PR).
  • [MeSH-major] Aminoglycosides / therapeutic use. Antibiotics, Antineoplastic / therapeutic use. Neoplasms / drug therapy
  • [MeSH-minor] Adolescent. Adult. Bone Marrow Diseases / chemically induced. Carbazoles. Central Nervous System Neoplasms / drug therapy. Child. Child, Preschool. Drug-Induced Liver Injury / etiology. Female. Glucosides. Humans. Infant. Infant, Newborn. Infusions, Intravenous. Male. Neoplasm Proteins / antagonists & inhibitors. Pancreatitis / chemically induced. Rhabdomyosarcoma / drug therapy. Salvage Therapy. Topoisomerase II Inhibitors

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  • [Copyright] (c) 2007 Wiley-Liss, Inc.
  • (PMID = 17610262.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / CA98543; United States / NCI NIH HHS / CA / P30CA-54174
  • [Publication-type] Clinical Trial, Phase II; Journal Article; Multicenter Study; Research Support, N.I.H., Extramural
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Aminoglycosides; 0 / Antibiotics, Antineoplastic; 0 / Carbazoles; 0 / Glucosides; 0 / Neoplasm Proteins; 0 / Topoisomerase II Inhibitors; A60X6MBU6G / becatecarin
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43. Jacobs S, Fox E, Krailo M, Hartley G, Navid F, Wexler L, Blaney SM, Goodwin A, Goodspeed W, Balis FM, Adamson PC, Widemann BC: Phase II trial of ixabepilone administered daily for five days in children and young adults with refractory solid tumors: a report from the children's oncology group. Clin Cancer Res; 2010 Jan 15;16(2):750-4
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  • [Title] Phase II trial of ixabepilone administered daily for five days in children and young adults with refractory solid tumors: a report from the children's oncology group.
  • EXPERIMENTAL DESIGN: We conducted a phase II trial of ixabepilone (8 mg/m(2)/dose for 5 days every 21 days) using a two-stage design in taxane-naïve children and young adults with treatment-refractory, measurable rhabdomyosarcoma, Ewing sarcoma family tumors, osteosarcoma, synovial sarcoma, or malignant peripheral nerve sheath tumor, neuroblastoma, and Wilms tumor.

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  • (PMID = 20068084.001).
  • [ISSN] 1078-0432
  • [Journal-full-title] Clinical cancer research : an official journal of the American Association for Cancer Research
  • [ISO-abbreviation] Clin. Cancer Res.
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / U10 CA098413; United States / NCI NIH HHS / CA / U10 CA 98543; United States / Intramural NIH HHS / / ; United States / NCI NIH HHS / CA / U10 CA098543-08; None / None / / U10 CA098543-08; United States / NCI NIH HHS / CA / U10 CA098543; United States / NCI NIH HHS / CA / U10 CA098413-08; None / None / / U10 CA098413-08
  • [Publication-type] Clinical Trial, Phase II; Journal Article; Research Support, N.I.H., Extramural; Research Support, N.I.H., Intramural
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 0 / Epothilones; K27005NP0A / ixabepilone
  • [Other-IDs] NLM/ NIHMS160304; NLM/ PMC3086796
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44. Beaty O 3rd, Berg S, Blaney S, Malogolowkin M, Krailo M, Knight R, Schaiquevich P, Stewart C, Chen Z, Nelson M, Voss S, Ivy SP, Adamson PC: A phase II trial and pharmacokinetic study of oxaliplatin in children with refractory solid tumors: a Children's Oncology Group study. Pediatr Blood Cancer; 2010 Sep;55(3):440-5
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  • [Title] A phase II trial and pharmacokinetic study of oxaliplatin in children with refractory solid tumors: a Children's Oncology Group study.
  • BACKGROUND: Platinating agents are used in the treatment of a spectrum of childhood cancers.
  • A phase 2 study was performed to estimate the response rate to single agent oxaliplatin in patients with refractory pediatric solid tumors, and to further describe the toxicities and pharmacokinetics of the drug in this population.
  • Histologies included: Ewing sarcoma/peripheral PNET, osteosarcoma, rhabdomyosarcoma, neuroblastoma, high and low grade astrocytoma, brain stem glioma, ependymoma, hepatoblastoma and selected rare tumors.
  • A two-stage design, enrolling 10 + 10 subjects, was used for each disease stratum.
  • Five subjects completed 17 treatment cycles.

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  • [Copyright] 2010 Wiley-Liss, Inc.
  • (PMID = 20658614.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / U10CA98413; United States / NCI NIH HHS / CA / U10CA98543; United States / NCI NIH HHS / CA / U10 CA098413; United States / NCI NIH HHS / CA / U10 CA098543-08; United States / NCI NIH HHS / CA / U10 CA098543; United States / NCI NIH HHS / CA / U10 CA098413-08
  • [Publication-type] Clinical Trial, Phase II; Journal Article; Multicenter Study; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 0 / Organoplatinum Compounds; 04ZR38536J / oxaliplatin
  • [Other-IDs] NLM/ NIHMS218622; NLM/ PMC4665115
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45. Canete A, Gerrard M, Rubie H, Castel V, Di Cataldo A, Munzer C, Ladenstein R, Brichard B, Bermúdez JD, Couturier J, de Bernardi B, Pearson AJ, Michon J: Poor survival for infants with MYCN-amplified metastatic neuroblastoma despite intensified treatment: the International Society of Paediatric Oncology European Neuroblastoma Experience. J Clin Oncol; 2009 Mar 1;27(7):1014-9
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  • [Title] Poor survival for infants with MYCN-amplified metastatic neuroblastoma despite intensified treatment: the International Society of Paediatric Oncology European Neuroblastoma Experience.
  • PURPOSE: To report the results of a prospective, nonrandomized European study on infants with neuroblastoma and MYCN gene amplification.
  • PATIENTS AND METHODS: Infants with neuroblastoma (stage 2, 3, 4, and 4s) and MYCN gene amplification who were diagnosed between 1999 and 2004 were eligible for enrollment onto the study.
  • After diagnosis, staging, and mandatory biologic studies, induction chemotherapy (IC) with conventional drugs was administered, followed by delayed surgery, megatherapy (busulfan-melphalan as a conditioning regimen), and local radiotherapy.
  • RESULTS: Of the 46 infants enrolled onto the study, 35 infants were eligible; of these 35 infants, 97% had metastatic spread (24 infants had stage 4, and 10 infants had stage 4s).
  • Two-year overall survival (OS) was 30% (SE, 0.08), with median survival time of 12 months, and 23 deaths due to disease.
  • Two-year, event-free survival (EFS) was 29% (SE, 0.07).
  • The treatment was well tolerated with no deaths as a result of toxicity or severe toxicity.
  • Stage and high lactate dehydrogenase reached significance in the univariate analysis (P = .028 and .039, respectively for OS; and P = .05 and .031 respectively, for EFS).
  • CONCLUSION: Although treatment was well tolerated, survival was poor and our IC failed to achieve a satisfactory response in 30% of our patients.
  • New therapeutic approaches and more intense world-wide collaboration are needed to achieve a cure in this population.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Gene Amplification. Genes, myc. Neuroblastoma / drug therapy
  • [MeSH-minor] Combined Modality Therapy. Cyclophosphamide / administration & dosage. Doxorubicin / administration & dosage. Drug-Related Side Effects and Adverse Reactions. Female. Humans. Infant. Infant, Newborn. Leukapheresis. Male. Peripheral Blood Stem Cell Transplantation. Prospective Studies. Survival Analysis. Transplantation Conditioning. Vincristine / administration & dosage

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  • (PMID = 19171715.001).
  • [ISSN] 1527-7755
  • [Journal-full-title] Journal of clinical oncology : official journal of the American Society of Clinical Oncology
  • [ISO-abbreviation] J. Clin. Oncol.
  • [Language] eng
  • [Publication-type] Clinical Trial; Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 5J49Q6B70F / Vincristine; 80168379AG / Doxorubicin; 8N3DW7272P / Cyclophosphamide
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46. Morovic A, Damjanov I: Neuroectodermal ovarian tumors: a brief overview. Histol Histopathol; 2008 06;23(6):765-71
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  • Primary neuroectodermal tumors of the ovary are rare monophasic teratomas composed exclusively or almost exclusively of neuroectodemal tissue.
  • These tumors were classified as ependymoma, astrocytoma, glioblastoma multiforme, ependymoblastoma or as primitive neuroepithelial tumors such as medullo-blastoma, medulloepithelioma and neuroblastoma.
  • The review of the literature shows that most patients with clinical stage I and II were treated surgically, whereas those with stage III or IV tumors received additional radiation or chemotherapy, or both.
  • The clinical stage at the time of diagnosis is the most important prognostic parameter of these tumors.
  • [MeSH-minor] Biomarkers, Tumor / analysis. Combined Modality Therapy. Female. Humans. Immunohistochemistry. Neoplasm Staging. Prognosis

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  • (PMID = 18366014.001).
  • [ISSN] 1699-5848
  • [Journal-full-title] Histology and histopathology
  • [ISO-abbreviation] Histol. Histopathol.
  • [Language] eng
  • [Publication-type] Journal Article; Review
  • [Publication-country] Spain
  • [Chemical-registry-number] 0 / Biomarkers, Tumor
  • [Number-of-references] 30
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47. Vaidya SJ, Payne GS, Leach MO, Pinkerton CR: Potential role of magnetic resonance spectroscopy in assessment of tumour response in childhood cancer. Eur J Cancer; 2003 Apr;39(6):728-35
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  • An early non-invasive indicator of tumour response to therapy would provide useful information regarding the effectiveness of therapy.
  • This might be a relevant prognostic factor in new patients and in phase II studies could facilitate recommendations at an early stage as to whether to continue treatment.
  • [MeSH-minor] Child. Humans. Lymphoma, Non-Hodgkin / drug therapy. Lymphoma, Non-Hodgkin / metabolism. Neuroblastoma / drug therapy. Neuroblastoma / metabolism. Phosphorus. Protons. Sarcoma / drug therapy. Sarcoma / metabolism

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  • (PMID = 12651196.001).
  • [ISSN] 0959-8049
  • [Journal-full-title] European journal of cancer (Oxford, England : 1990)
  • [ISO-abbreviation] Eur. J. Cancer
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't; Review
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Protons; 27YLU75U4W / Phosphorus
  • [Number-of-references] 49
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48. Terenziani M, D'Angelo P, Bisogno G, Boldrini R, Cecchetto G, Collini P, Conte M, De Laurentis T, Ilari I, Indolfi P, Inserra A, Piva L, Siracusa F, Spreafico F, Tamaro P, Lo Curto M: Teratoma with a malignant somatic component in pediatric patients: the Associazione Italiana Ematologia Oncologia Pediatrica (AIEOP) experience. Pediatr Blood Cancer; 2010 Apr;54(4):532-7
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  • PROCEDURE: The Associazione Italiana Ematologia Oncologia Pediatrica identified 14 cases of TMSC.
  • RESULTS: The series (9 female, 5 male) showed the following disease: testis (2), sacrococcygeal (3), ovary (3), retroperitoneum (3), mediastinum (2), and foot soft tissue (1).
  • Distribution of the somatic component was: carcinoma (4), pancreatic neuroendocrine tumor (1), neuroblastoma (3), rhabdomyosarcoma (3), rhabdomyosarcoma plus liposarcoma, chondrosarcoma, neurogenic sarcoma (1), chondrosarcoma plus neuroectodermal sarcoma (1), malignant peripheral nerve sheath tumor (1).
  • Three patients were in stage I, four in stage II, three in stage III, and four in stage IV.
  • Chemotherapy optimized for histology should include reagents directed to the somatic malignancy, if chemosensitive.
  • Malignant GCT warrants GCT-directed chemotherapy.
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Female. Humans. Infant. Infant, Newborn. Italy. Male. Neoplasm Staging. Prognosis. Retrospective Studies. Treatment Outcome

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  • (PMID = 20049928.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
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49. Hu WH, Xie FY, Fang SH, Jiao JJ, Yan C, Peng WJ, Fu XY, Zhang F: [Cancer of the nasal cavity]. Zhonghua Zhong Liu Za Zhi; 2005 Feb;27(2):117-21
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  • The 5-year survival rate was 55.8% in squamous-cell carcinoma, 44.0% in adenocarcinoma, 59.7% in undifferentiated carcinoma, 76.3% in adenoid cystic carcinoma, 71.4% in mucoepidermoid carcinoma, 25.0% in rhabdomyosarcoma, 26.7% in malignant melanoma, 50.0% in neuroblastoma (P > 0.05).
  • The 5-year survival rate was 73.8% in patients whose cancer completely disappeared after treatment.
  • The 5-year survival was 78.3% in stage I disease, 56.4% in stage II disease, 54.2% in stage III and 35.9% in stage IV (P < 0.05).
  • That with chemotherapy only was 25.0% whereas that of patients treated with combination treatment was 61.8% (P > 0.05).
  • CONCLUSION: Clinical stage, immediate therapeutic response and involvement of sphenoidal or maxillary sinus; but not the pathologic type, the presence of cervical metastasis nor the method of treatment, are the factors affecting the prognosis of patients with nasal carcinoma.
  • [MeSH-major] Nasal Cavity. Nose Neoplasms / mortality. Nose Neoplasms / therapy
  • [MeSH-minor] Adolescent. Adult. Aged. Aged, 80 and over. Child. Combined Modality Therapy. Female. Humans. Male. Middle Aged. Multivariate Analysis. Prognosis. Survival Rate

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  • (PMID = 15946555.001).
  • [ISSN] 0253-3766
  • [Journal-full-title] Zhonghua zhong liu za zhi [Chinese journal of oncology]
  • [ISO-abbreviation] Zhonghua Zhong Liu Za Zhi
  • [Language] chi
  • [Publication-type] English Abstract; Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] China
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50. Tröbs RB, Hänsel M, Friedrich T, Bennek J: A 23-year experience with malignant renal tumors in infancy and childhood. Eur J Pediatr Surg; 2001 Apr;11(2):92-8
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  • A retrospective analysis of 77 children treated between 1974 and 1996 was undertaken to evaluate morbidity and the evolution of therapy.
  • We observed 3 children of school age with renal carcinoma and one patient with an intrarenal neuroblastoma.
  • Comparing relapse-free survival of stages I, II and III, respectively, there was a reduced survival rate for stage III (p=0.019).
  • According to the SIOP/GPOH protocol in 1989, the regimen was switched from primary surgery to preoperative chemotherapy without biopsy in 1989 (11 pats.).
  • During preoperative chemotherapy a venous occlusive disease of the liver occurred in 2 patients.
  • Preoperative chemotherapy led to an impressive tumor shrinkage in the majority of patients.
  • In our experience, reduction of tumor volume due to preoperative chemotherapy facilitates tumor removal by surgery and may prevent tumor spillage and the deleterious effects of radiation in young children.
  • Surgery without delay is necessary if the diagnosis is unclear or the tumor fails to respond to preoperative chemotherapy.
  • [MeSH-minor] Antineoplastic Agents / therapeutic use. Chemotherapy, Adjuvant. Child. Child, Preschool. Disease-Free Survival. Female. Humans. Infant. Male. Neoplasm Recurrence, Local / epidemiology. Neoplasm Staging. Prognosis. Retrospective Studies

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  • (PMID = 11371043.001).
  • [ISSN] 0939-7248
  • [Journal-full-title] European journal of pediatric surgery : official journal of Austrian Association of Pediatric Surgery ... [et al] = Zeitschrift für Kinderchirurgie
  • [ISO-abbreviation] Eur J Pediatr Surg
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] Germany
  • [Chemical-registry-number] 0 / Antineoplastic Agents
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51. Jakobisiak M, Golab J: Potential antitumor effects of statins (Review). Int J Oncol; 2003 Oct;23(4):1055-69
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • Statins, which have been introduced to the clinic for the treatment of hypercholesterolemia, are competitive inhibitors of 3-hydroxy-3-methylglutaryl-coenzyme A (HMG-CoA) reductase, the major rate-limiting enzyme that controls the conversion of HMG-CoA to mevalonic acid (MA).
  • They have also been found to display antitumor effects against melanoma, mammary carcinoma, pancreatic adenocarcinoma, fibrosarcoma, glioma, neuroblastoma, and lymphoma in animal tumor models resulting in retardation of tumor growth, and/or inhibition of the metastatic process.
  • The results of several clinical studies of statins in cancer patients including phase I, phase I/II, and phase II trials have been published.
  • Although evaluation of the therapeutic efficacy is not the purpose of early clinical trials and all conclusions might be premature at this stage, some preliminary conclusions have already been drawn.
  • The results of these studies do not show any significant therapeutic effects of statins in cancer patients.
  • However, the results of one of these studies suggest that statins could effectively strengthen the therapeutic activity of some chemotherapeutics.
  • However, as toxic side effects of statins have been particularly evident in their combination with some other drugs great caution should be advised while planning clinical trials based on combination therapy including statins in cancer patients.
  • [MeSH-major] Antineoplastic Agents / therapeutic use. Hydroxymethylglutaryl-CoA Reductase Inhibitors / therapeutic use. Neoplasms / drug therapy

  • MedlinePlus Health Information. consumer health - Cancer Chemotherapy.
  • MedlinePlus Health Information. consumer health - Statins.
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  • (PMID = 12963986.001).
  • [ISSN] 1019-6439
  • [Journal-full-title] International journal of oncology
  • [ISO-abbreviation] Int. J. Oncol.
  • [Language] eng
  • [Publication-type] Journal Article; Review
  • [Publication-country] Greece
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 0 / Hydroxymethylglutaryl-CoA Reductase Inhibitors
  • [Number-of-references] 230
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52. Perek D, Brozyna A, Dembowska-Baginska B, Stypinska M, Sojka M, Bacewicz L, Polnik D, Kalicinski P: [Tumours in newborns and infants up to three months of life. One institution experience]. Med Wieku Rozwoj; 2006 Jul-Sep;10(3 Pt 1):711-23
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • INTRODUCTION: Newborns and infants up to three months of life are a specific group of population in paediatric oncology due to immaturity of tissues and organs and rarity of neoplastic diseases in this group of patients (pts).
  • There are no strict therapeutic procedures established for these children.
  • THE AIM of our study was to examine distribution of tumours in newborns and infants up to 3 months of age treated in our institution and to present our own experience in the treatment of these patients.
  • Distribution of tumour types in newborns and babies from 1 to 3 months of age was analyzed separately.
  • Due to similar growth pattern, response to treatment and it's side effects in newborns and small infants, treatment results were evaluated for the whole group.
  • The second most common was neuroblastoma (NBL) 22%.
  • There were also 3 cases of soft tissue sarcomas (STS), 2 central nervous system tumours (CNS), 2 retinoblastoma (RB), 2 hepatoblastoma (HB).
  • It concerned pts with MT--28, IT--3 pts, yolk sac tumour (YST)--1 pt and malignant tumours (stage I and II): 8-NBL, 2-CNS tumours, 2 STS, 3-HB, 1-WT.
  • Eleven pts underwent combined treatment of chemotherapy and surgery: 5 with stage III and IV NBL, 6 with other tumours.
  • Chemotherapy alone was administered to 7 pts in whom local advancement of disease enabled surgery and to pts with RBL.
  • Four pts with NBL (2 stage IV and 2 stage IVS) were treated with irradiation to the liver only.
  • One pt, critically ill, died before any treatment.
  • GCT and neuroblastoma are the most common tumours in newborns and infants up to 3 months of age.
  • 2. Newborns and small infants with advanced neoplastic disease, similarly to older children can be cured with chemotherapy.
  • 3. Individual approach is warranted in newborns and small infants and treatment should be carried out in specialized centres.
  • 4. All patients who completed treatment of any tumour type should be followed up by a pediatric oncologist.
  • [MeSH-major] Infant Welfare / statistics & numerical data. Neoplasms / epidemiology. Neoplasms / therapy

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  • (PMID = 17317902.001).
  • [Journal-full-title] Medycyna wieku rozwojowego
  • [ISO-abbreviation] Med Wieku Rozwoj
  • [Language] pol
  • [Publication-type] English Abstract; Journal Article
  • [Publication-country] Poland
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53. Surico G, Muggeo P, De Leonardis F, Rigillo N: New paclitaxel-cisplatin based chemotherapy regimen for advanced stage, recurrent, or refractory neuroblastoma-preliminary report. Med Pediatr Oncol; 2003 Feb;40(2):130-2
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] New paclitaxel-cisplatin based chemotherapy regimen for advanced stage, recurrent, or refractory neuroblastoma-preliminary report.
  • [MeSH-major] Adrenal Gland Neoplasms / drug therapy. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Bone Marrow Neoplasms / drug therapy. Bone Neoplasms / drug therapy. Cisplatin / therapeutic use. Neoplasm Recurrence, Local / drug therapy. Neuroblastoma / drug therapy. Paclitaxel / therapeutic use. Retroperitoneal Neoplasms / drug therapy
  • [MeSH-minor] Child. Child, Preschool. Fatal Outcome. Female. Humans. Male. Neoplasm Staging. Salvage Therapy. Taxoids






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