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1. Fuchs J, Rydzynski J, Von Schweinitz D, Bode U, Hecker H, Weinel P, Bürger D, Harms D, Erttmann R, Oldhafer K, Mildenberger H, Study Committee of the Cooperative Pediatric Liver Tumor Study Hb 94 for the German Society for Pediatric Oncology and Hematology: Pretreatment prognostic factors and treatment results in children with hepatoblastoma: a report from the German Cooperative Pediatric Liver Tumor Study HB 94. Cancer; 2002 Jul 1;95(1):172-82
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Pretreatment prognostic factors and treatment results in children with hepatoblastoma: a report from the German Cooperative Pediatric Liver Tumor Study HB 94.
  • BACKGROUND: In the past 20 years, a dramatic improvement in the prognosis of patients with hepatoblastoma (HB) has been achieved by combining surgery with chemotherapy in several national and international trials.
  • A worldwide, unsolved problem remains the treatment of patients with advanced or metastatic HB.
  • METHODS: The German Cooperative Pediatric Liver Tumor Study HB 94 was a prospective, multicenter, single-arm study.
  • The protocol assessed the efficiency of chemotherapy consisting of cisplatin, ifosfamide, and doxorubicin (CDDP/IFO/DOXO) and/or etoposide and carboplatin (VP16/CARBO).
  • Long-term DFS was as follows: 26 of 27 patients had Stage I HB, 3 of 3 patients had Stage II HB, 19 of 25 patients had Stage III HB, and 5 of 14 patients had Stage IV.
  • Six children (8%) had no surgical treatment.
  • Twenty-two tumors were resected primarily, and 41 children underwent surgery after initial chemotherapy.
  • Two children underwent liver transplantation.
  • Forty-eight children received primary chemotherapy with CDDP/IFO/DOXO.
  • Eighteen children with advanced or recurrent HB underwent VP16/CARBO chemotherapy, with a response achieved by 12 children.
  • The relevant pretreatment prognostic factors were growth pattern of the liver tumor (P = 0.0135), vascular tumor invasion (P = 0.0039), occurrence of distant metastases (P = 0.0001), initial alpha-fetoprotein level (P = 0.0034), and surgical radicality (P < 0.0001).
  • CONCLUSIONS: The current results underline the necessity of preoperative chemotherapy in all children with HB.
  • [MeSH-major] Hepatoblastoma / therapy. Liver Neoplasms / therapy

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  • [Copyright] Copyright 2002 American Cancer Society.
  • (PMID = 12115331.001).
  • [ISSN] 0008-543X
  • [Journal-full-title] Cancer
  • [ISO-abbreviation] Cancer
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / alpha-Fetoproteins
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2. Furtwaengler R, Reinhard H, Leuschner I, Schenk JP, Goebel U, Claviez A, Kulozik A, Zoubek A, von Schweinitz D, Graf N, Gesellschaft fur Pädiatrische Onkologie und Hämatologie (GPOH) Nephroblastoma Study Group: Mesoblastic nephroma--a report from the Gesellschaft fur Pädiatrische Onkologie und Hämatologie (GPOH). Cancer; 2006 May 15;106(10):2275-83
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  • BACKGROUND: Surgery alone is the appropriate first-line treatment for patients with mesoblastic nephroma (MN).
  • The authors evaluated the outcome of patients with MN who were enrolled in either the International Society of Pediatric Oncology (SIOP) 93-01/GPOH or the SIOP 2001/GPOH Nephroblastoma Study and Trial.
  • The median observation time was 4.2 years.
  • Five patients older than 6 months received preoperative chemotherapy.
  • Nine patients had a Stage III MN, 5 of those patients had tumor ruptures, and 8 had positive surgical margins.
  • After they underwent nephrectomy, 40 patients received no further treatment.
  • For the entire group, event-free survival (EFS) (94%) and overall survival (OS) (95%) were excellent.
  • Patients with a cellular MN, patients with age 3 months or older, and patients with Stage III MN had lower EFS.
  • Three patients developed recurrent disease, and 2 of those patients died.
  • Metastases to the brain, lung, and liver were observed in 1 patient.
  • Nonetheless, a subgroup of patients with MN (Stage III cellular MN in patients age 3 months or older) tends to develop recurrences more often.
  • Further prospective studies will be needed to verify this finding and should help determine whether these patients may benefit from adjuvant therapy.
  • [MeSH-major] Kidney Neoplasms / diagnosis. Kidney Neoplasms / therapy. Nephroma, Mesoblastic / diagnosis. Nephroma, Mesoblastic / therapy
  • [MeSH-minor] Age Factors. Biopsy, Needle. Chemotherapy, Adjuvant. Child. Child, Preschool. Cohort Studies. Female. Germany. Humans. Immunohistochemistry. Infant. Infant, Newborn. Logistic Models. Male. Neoplasm Staging. Nephrectomy / methods. Pediatrics. Prognosis. Proportional Hazards Models. Retrospective Studies. Risk Assessment. Societies, Medical. Statistics, Nonparametric. Survival Analysis. Treatment Outcome

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  • [Copyright] Copyright 2006 American Cancer Society
  • (PMID = 16596620.001).
  • [ISSN] 0008-543X
  • [Journal-full-title] Cancer
  • [ISO-abbreviation] Cancer
  • [Language] eng
  • [Publication-type] Comparative Study; Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
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3. Meyers RL, Rowland JR, Krailo M, Chen Z, Katzenstein HM, Malogolowkin MH: Predictive power of pretreatment prognostic factors in children with hepatoblastoma: a report from the Children's Oncology Group. Pediatr Blood Cancer; 2009 Dec;53(6):1016-22
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  • [Title] Predictive power of pretreatment prognostic factors in children with hepatoblastoma: a report from the Children's Oncology Group.
  • BACKGROUND: PRETEXT is used to stratify risk in children with hepatoblastoma by the Liver Tumor Strategy Group (SIOPEL) of the International Society of Pediatric Oncology (SIOP).
  • A recent analysis excluding patients that did not survive neoadjuvant chemotherapy, concluded that PRETEXT was superior to Children's Oncology Group (COG) stage for predicting survival.
  • Puzzled by this result, we made a similar comparison of PRETEXT and COG stage.
  • This time, however, we include all patients, and we compare predictive value at diagnosis, instead of after neoadjuvant chemotherapy.
  • The 5-year overall survival rates by COG stage were 100%, 97.5%, 100%, 70.2%, and 39.3% for Stage I pure fetal histology (PFH), Stage I unfavorable histology (UH = not PFH), Stage II, Stage III, and Stage IV, respectively.
  • PRETEXT added significant additional prognostic information within the COG Stage III, but not COG Stage IV.
  • Additional prognostic factors statistically significant for an increased risk of death were small-cell-undifferentiated (SCU) histologic subtype and AFP < 100 at diagnosis.
  • CONCLUSIONS: PRETEXT, COG stage, SCU histology, and AFP < 100, as assessed at diagnosis, are important determinants of survival that will allow us to better develop common international criteria for risk stratification.
  • [MeSH-minor] Child. Histology. Humans. Neoplasm Staging / methods. Prognosis. Retrospective Studies. Survival Analysis. Treatment Outcome. alpha-Fetoproteins / analysis

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  • (PMID = 19588519.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / U10 CA098413; United States / NCI NIH HHS / CA / U10 CA098413-08; United States / NCI NIH HHS / CA / U10 CA098543; United States / NCI NIH HHS / CA / U10 CA098543-08
  • [Publication-type] Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / alpha-Fetoproteins
  • [Other-IDs] NLM/ NIHMS183909; NLM/ PMC4408767
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4. Meany HJ, Seibel NL, Sun J, Finklestein JZ, Sato J, Kelleher J, Sondel P, Reaman G: Phase 2 trial of recombinant tumor necrosis factor-alpha in combination with dactinomycin in children with recurrent Wilms tumor. J Immunother; 2008 Sep;31(7):679-83
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  • Tumor necrosis factor (TNF), a peptide produced by macrophages with cytostatic and cytolytic effects, demonstrated single agent antitumor activity and synergistic effect when administered with dactinomycin in in vitro tumor cell lines, in vivo xenograft models, and adult and pediatric phase 1 clinical trials.
  • This phase 2 pediatric trial evaluated the efficacy and further defined the toxicity profile of recombinant TNF (rTNF) and dactinomycin in patients with recurrent or refractory Wilms tumor.
  • On this 2 stage Children's Cancer Group trial, dactinomycin (15 microg/kg/d, IV) immediately followed by rTNF (200 microg/m/d, IV), once daily for 5 consecutive days, was administered to patients with recurrent or refractory Wilms tumor.
  • Nineteen of 21 consecutive patients, ranging 0.9 to 16.5 years of age at the time of initial diagnosis, were evaluable for response and toxicity.
  • Following 59 patient treatment cycles, the most commonly observed grade 3/4 toxicities were thrombocytopenia (40.7%), elevated liver transaminases (23.7%), neutropenia (20.3%), leucopenia (13.6%), anemia (11.9%), and myalgias (10.2%).
  • Before completion of stage 2, the study closed due to unavailability of rTNF.

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  • (PMID = 18600176.001).
  • [ISSN] 1537-4513
  • [Journal-full-title] Journal of immunotherapy (Hagerstown, Md. : 1997)
  • [ISO-abbreviation] J. Immunother.
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CA / U10 CA013539; United States / NCI NIH HHS / CA / CA013539-29; United States / NCI NIH HHS / CA / U10 CA013539-29; United States / NCI NIH HHS / CA / U10 CA098543; United States / NCI NIH HHS / CA / U10 CA098543-01
  • [Publication-type] Clinical Trial, Phase II; Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Recombinant Proteins; 0 / Tumor Necrosis Factor-alpha; 1CC1JFE158 / Dactinomycin
  • [Other-IDs] NLM/ NIHMS107700; NLM/ PMC2677078
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5. Navid F, Furman WL, Fleming M, Rao BN, Kovach S, Billups CA, Cain AM, Amonette R, Jenkins JJ, Pappo AS: The feasibility of adjuvant interferon alpha-2b in children with high-risk melanoma. Cancer; 2005 Feb 15;103(4):780-7
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • BACKGROUND: It has been shown that induction high-dose interferon alpha-2b (IFN-alpha-2b) followed by maintenance therapy improves recurrence-free survival in adults with high-risk, resected melanoma.
  • In this study, the feasibility and toxicity of this regimen were evaluated in newly diagnosed pediatric patients with Stage III melanoma involving regional lymph nodes.
  • METHODS: Fifteen patients age <or=18 years with newly diagnosed Stage III melanoma were enrolled on an institutional protocol.
  • Patients were treated with wide local excision, sentinel lymph node biopsy, lymph node dissection, and adjuvant biotherapy, consisting of induction therapy with 20 million IU/m2 per day IFN-alpha-2b intravenously 5 times per week for 4 weeks followed by maintenance therapy with IFN-alpha-2b 10 million IU/m2 per day subcutaneously 3 times per week for 48 weeks.
  • RESULTS: All patients completed induction therapy, and nine patients completed maintenance therapy.
  • Three patients currently are receiving maintenance, 2 patients developed recurrent disease on maintenance therapy, and 1 patient stopped maintenance therapy 5 weeks early.
  • During induction therapy, Grade 3-4 toxicities included 14 episodes of neutropenia in 11 patients, 3 episodes of leukopenia in 2 patients, and 6 episodes of liver transaminase elevations in 5 patients.
  • During maintenance therapy, Grade 3-4 toxicities included 23 episodes of neutropenia in 10 patients and 2 episodes of liver transaminase elevations in 2 patients.
  • All toxicities were reversible with interruption or dose modification of therapy, and no patients were taken off study due to toxicity.
  • CONCLUSIONS: High dose IFN-alpha-2b for 4 weeks followed by a lower dose maintenance phase for 48 weeks was feasible in children with Stage III melanoma and was associated with tolerable toxicity.
  • [MeSH-major] Antineoplastic Agents / adverse effects. Interferon-alpha / adverse effects. Melanoma / drug therapy. Skin Neoplasms / drug therapy
  • [MeSH-minor] Adolescent. Chemotherapy, Adjuvant. Child. Child, Preschool. Feasibility Studies. Humans. Infant. Lymph Node Excision. Recombinant Proteins. Sentinel Lymph Node Biopsy. Survival

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  • [Copyright] Copyright (c) 2005 American Cancer Society.
  • (PMID = 15660397.001).
  • [ISSN] 0008-543X
  • [Journal-full-title] Cancer
  • [ISO-abbreviation] Cancer
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / CA-21765
  • [Publication-type] Clinical Trial; Clinical Trial, Phase I; Journal Article; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, P.H.S.
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 0 / Interferon-alpha; 0 / Recombinant Proteins; 99210-65-8 / interferon alfa-2b
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6. Katzenstein HM, Krailo MD, Malogolowkin MH, Ortega JA, Qu W, Douglass EC, Feusner JH, Reynolds M, Quinn JJ, Newman K, Finegold MJ, Haas JE, Sensel MG, Castleberry RP, Bowman LC: Fibrolamellar hepatocellular carcinoma in children and adolescents. Cancer; 2003 Apr 15;97(8):2006-12
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  • [Title] Fibrolamellar hepatocellular carcinoma in children and adolescents.
  • BACKGROUND: Children with hepatocellular carcinoma (HCC) were treated on a prospective, randomized trial and were then analyzed to determine whether children with the fibrolamellar (FL) histologic variant of HCC have a more favorable presentation, increased surgical resectability, greater response to therapy, and improved outcome compared with children who have typical HCC.
  • METHODS: Forty-six patients were enrolled on Pediatric Intergroup Hepatoma Protocol INT-0098 (Pediatric Oncology Group Study 8945/Children's Cancer Group Study 8881) between August 1989 and December 1992.
  • After undergoing initial surgery or biopsy, children with Stage I HCC (n = 8 patients), Stage III HCC (n = 25 patients), and Stage IV HCC (n = 13 patients) were assigned randomly, regardless of histology, to receive treatment either with cisplatin, vincristine, and fluorouracil (n = 20 patients) or with cisplatin and continuous-infusion doxorubicin (n = 26 patients).
  • There was no difference in the number of patients with advanced-stage disease, the incidence of surgical resectability at diagnosis, or the response to treatment between patients with FL-HCC and patients with typical HCC.
  • CONCLUSIONS: Children with FL-HCC do not have a favorable prognosis and do not respond any differently to current therapeutic regimens than patients with typical HCC.
  • Children with initially resectable HCC have a good prognosis irrespective of histologic subtype, whereas outcomes are poor uniformly for children with advanced-stage disease.
  • The use of novel chemotherapeutic agents and the incorporation of other treatment modalities are indicated to improve the dismal survival of pediatric patients with all histologic variants of advanced-stage HCC.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Carcinoma, Hepatocellular / drug therapy. Carcinoma, Hepatocellular / surgery. Liver Neoplasms / drug therapy. Liver Neoplasms / surgery
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Cisplatin / administration & dosage. Cohort Studies. Disease-Free Survival. Doxorubicin / administration & dosage. Female. Fluorouracil / administration & dosage. Hepatoblastoma. Humans. Infant. Infusions, Intravenous. Male. Neoplasm Staging. Prognosis. Prospective Studies. Risk Factors. Time Factors. Treatment Outcome. Vincristine / administration & dosage. alpha-Fetoproteins / analysis

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  • [Copyright] Copyright 2003 American Cancer Society.
  • (PMID = 12673731.001).
  • [ISSN] 0008-543X
  • [Journal-full-title] Cancer
  • [ISO-abbreviation] Cancer
  • [Language] eng
  • [Publication-type] Clinical Trial; Comparative Study; Journal Article; Randomized Controlled Trial; Research Support, U.S. Gov't, P.H.S.; Review
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / alpha-Fetoproteins; 5J49Q6B70F / Vincristine; 80168379AG / Doxorubicin; Q20Q21Q62J / Cisplatin; U3P01618RT / Fluorouracil
  • [Number-of-references] 36
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7. 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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  • 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.
  • Dose-limiting toxicity was observed in only 1 patient (Grade 4 liver toxicity).
  • [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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8. Gokhale CD, Udipi SA, Ambaye RY, Pai SK, Advani SH: Post-therapy profile of serum total cholesterol, retinol and zinc in pediatric acute lymphoblastic leukemia and non-Hodgkin's lymphoma. J Am Coll Nutr; 2007 Feb;26(1):49-56
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  • [Title] Post-therapy profile of serum total cholesterol, retinol and zinc in pediatric acute lymphoblastic leukemia and non-Hodgkin's lymphoma.
  • OBJECTIVE: To assess serum albumin, total cholesterol, retinol, zinc and hemoglobin in children who had completed treatment for acute lymphoblastic leukemia (ALL) and Non-Hodgkin's lymphoma (NHL).
  • Comparisons were made to stage of treatment (maintenance 6 with post-therapy), type of treatment (chemotherapy and radiation with only chemotherapy) and type of malignancy (ALL with NHL).
  • RESULTS: Only serum albumin in patients included at Maintenance(6) was significantly higher (t = 2.31, p = 0.05) than post-therapy patients.
  • No significant difference in serum values was observed by type of treatment.
  • CONCLUSION: The results of the present study indicate that cancer and its treatment did not have any long-lasting effect on serum albumin, total cholesterol, retinol, zinc and hemoglobin.
  • Majority of subjects had low serum retinol suggestive of depleted liver reserves.
  • A higher percentage of patients with low serum retinol levels may also be attributed to the possibility of urinary losses of retinol that occur during episodes of infection while on immunosuppressive anti-cancer drug therapy.
  • [MeSH-major] Lymphoma, Non-Hodgkin / blood. Nutritional Status. Precursor Cell Lymphoblastic Leukemia-Lymphoma / blood. Trace Elements / blood. Vitamin A / blood. Vitamins / blood


9. Katzenstein HM, Krailo MD, Malogolowkin MH, Ortega JA, Liu-Mares W, Douglass EC, Feusner JH, Reynolds M, Quinn JJ, Newman K, Finegold MJ, Haas JE, Sensel MG, Castleberry RP, Bowman LC: Hepatocellular carcinoma in children and adolescents: results from the Pediatric Oncology Group and the Children's Cancer Group intergroup study. J Clin Oncol; 2002 Jun 15;20(12):2789-97
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  • [Title] Hepatocellular carcinoma in children and adolescents: results from the Pediatric Oncology Group and the Children's Cancer Group intergroup study.
  • PURPOSE: To determine surgical resectability, event-free survival (EFS), and toxicity in children with hepatocellular carcinoma (HCC) randomized to treatment with either cisplatin (CDDP), vincristine, and fluorouracil (regimen A) or CDDP and continuous-infusion doxorubicin (regimen B).
  • PATIENTS AND METHODS: Forty-six patients were enrolled onto Pediatric Intergroup Hepatoma Protocol INT-0098 (Pediatric Oncology Group (POG) 8945/Children's Cancer Group (CCG) 8881).
  • After initial surgery or biopsy, children with stage I (n = 8), stage III (n = 25), and stage IV (n = 13) HCC were randomly assigned to receive regimen A (n = 20) or regimen B (n = 26).
  • Patients with stage I, III, and IV had 5-year EFS estimates of 88% (SD = 12%), 8% (SD = 5%), and 0%, respectively.
  • Events occurred before postinduction surgery I in 18 (47%) of 38 patients with stage III or IV disease, and tumor resection was possible in two (10%) of the remaining 20 children with advanced-stage disease after chemotherapy.
  • CONCLUSION: Children with initially resectable HCC have a good prognosis and may benefit from the use of adjuvant chemotherapy.
  • Outcome was uniformly poor for children with advanced-stage disease treated with either regimen.
  • New therapeutic strategies are needed for the treatment of advanced-stage pediatric HCC.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Carcinoma, Hepatocellular / drug therapy. Carcinoma, Hepatocellular / surgery. Liver Neoplasms / drug therapy. Liver Neoplasms / surgery
  • [MeSH-minor] Adolescent. Chemotherapy, Adjuvant. Child. Child, Preschool. Cisplatin / administration & dosage. Disease-Free Survival. Doxorubicin / administration & dosage. Female. Fluorouracil / administration & dosage. Humans. Infant. Infant, Newborn. Infusions, Intravenous. Male. Neoplasm Staging. Prognosis. Treatment Outcome. Vincristine / administration & dosage

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  • (PMID = 12065555.001).
  • [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
  • [Grant] United States / NCI NIH HHS / CA / R25 CA092049
  • [Publication-type] Clinical Trial; Journal Article; Multicenter Study; Randomized Controlled Trial; Research Support, U.S. Gov't, P.H.S.
  • [Publication-country] United States
  • [Chemical-registry-number] 5J49Q6B70F / Vincristine; 80168379AG / Doxorubicin; Q20Q21Q62J / Cisplatin; U3P01618RT / Fluorouracil
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10. Dunkel IJ, Khakoo Y, Kernan NA, Gershon T, Gilheeney S, Lyden DC, Wolden SL, Orjuela M, Gardner SL, Abramson DH: Intensive multimodality therapy for patients with stage 4a metastatic retinoblastoma. Pediatr Blood Cancer; 2010 Jul 15;55(1):55-9
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Intensive multimodality therapy for patients with stage 4a metastatic retinoblastoma.
  • BACKGROUND: We previously reported promising pilot results treating patients with stage 4a metastatic retinoblastoma with combined intensive conventional chemotherapy, high-dose chemotherapy with autologous hematopoietic stem cell rescue, and radiation therapy and now present an expanded and updated series.
  • PROCEDURE: Fifteen patients with bone marrow (n = 14), bone (n = 10), orbit (n = 9), and/or liver (n = 4) disease were treated.
  • Induction chemotherapy usually consisted of vincristine, cyclophosphamide, cisplatin, and etoposide.
  • The high-dose chemotherapy regimen included carboplatin and thiotepa alone (n = 1) or with etoposide (n = 5) or topotecan (n = 7).
  • RESULTS: Bone marrow cleared at first post-initiation of chemotherapy examination in all patients and stem cells were harvested after a median of 3.5 cycles of chemotherapy (range 3-6 cycles).
  • Two patients progressed prior to high-dose chemotherapy and died.
  • Thirteen received high-dose chemotherapy at a median of 6 months post-diagnosis of metastases (range 4-8 months).
  • Six of the 10 survivors received radiation therapy.
  • Three patients developed secondary osteosarcoma 14, 4, and 9 years after diagnosis of metastatic disease.
  • CONCLUSIONS: Intensive multimodality therapy including high-dose chemotherapy with autologous hematopoietic stem cell rescue was curative for the majority of patients with stage 4a metastatic retinoblastoma treated.
  • The contribution of external beam radiation therapy is unclear.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Retinal Neoplasms / secondary. Retinal Neoplasms / therapy. Retinoblastoma / secondary. Retinoblastoma / therapy
  • [MeSH-minor] Child, Preschool. Cisplatin / adverse effects. Cisplatin / therapeutic use. Combined Modality Therapy. Cyclophosphamide / adverse effects. Cyclophosphamide / therapeutic use. Etoposide / adverse effects. Etoposide / therapeutic use. Follow-Up Studies. Hematopoietic Stem Cell Transplantation. Humans. Infant. Neoplasm Staging. Recurrence. Retrospective Studies. Survival Analysis. Transplantation, Autologous. Vincristine / adverse effects. Vincristine / therapeutic use

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  • (PMID = 20486171.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 5J49Q6B70F / Vincristine; 6PLQ3CP4P3 / Etoposide; 8N3DW7272P / Cyclophosphamide; Q20Q21Q62J / Cisplatin
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11. Pham TH, Iqbal CW, Grams JM, Zarroug AE, Wall JC, Ishitani MB, Nagorney DM, Moir C: Outcomes of primary liver cancer in children: an appraisal of experience. J Pediatr Surg; 2007 May;42(5):834-9
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Outcomes of primary liver cancer in children: an appraisal of experience.
  • INTRODUCTION: Hepatoblastoma (HB) and hepatocellular carcinoma (HCC) are the most common primary liver cancers in children.
  • Recent advances in management of pediatric liver cancer have improved disease-specific survival (DSS).
  • This is a review of our experience with childhood liver malignancy over the past 3 decades.
  • MATERIALS AND METHODS: A retrospective chart review from 1975 to 2005 identified patients who were 18 years old or younger with a histologically confirmed diagnosis of primary liver cancer.
  • Patients were staged according to the Children's Cancer Group and Pediatric Oncology Group (CCG/POG) system.
  • RESULTS: Fifty-two patients were confirmed to have primary liver cancers, where 24 (46%) patients had HB, 22 (42%) had HCC, 3 (6%) had sarcomas, and 3 (6%) had other histologies.
  • Most patients underwent major liver resection (n = 45, 87%), including: lobectomy (n = 25, 48%), and trisegmentectomy (n = 11, 21%).
  • Three patients underwent liver transplantation (n = 3, 6%) for advanced local disease.
  • Forty-five (87%) received primary or neoadjuvant and/or adjuvant chemotherapy.
  • Complete gross resection (stage I and II) was achieved in 37 (71%) patients.
  • CONCLUSION: Complete resection of the pediatric primary liver tumors remains the cornerstone of treatment to achieve cure.
  • Major liver resection can be performed with minimal perioperative mortality and morbidity.
  • Liver transplantation in conjunction with chemotherapy may have an increasing role in the management of locally advanced primary liver cancers.
  • [MeSH-major] Carcinoma, Hepatocellular / therapy. Hepatoblastoma / therapy. Liver Neoplasms / therapy
  • [MeSH-minor] Adolescent. Analysis of Variance. Child. Child, Preschool. Combined Modality Therapy. Hepatectomy. Humans. Liver Transplantation. Registries. Retrospective Studies. Survival Rate. Treatment Outcome

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  • (PMID = 17502194.001).
  • [ISSN] 1531-5037
  • [Journal-full-title] Journal of pediatric surgery
  • [ISO-abbreviation] J. Pediatr. Surg.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
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12. Smith MA, Morton CL, Phelps DA, Kolb EA, Lock R, Carol H, Reynolds CP, Maris JM, Keir ST, Wu J, Houghton PJ: Stage 1 testing and pharmacodynamic evaluation of the HSP90 inhibitor alvespimycin (17-DMAG, KOS-1022) by the pediatric preclinical testing program. Pediatr Blood Cancer; 2008 Jul;51(1):34-41
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  • [Title] Stage 1 testing and pharmacodynamic evaluation of the HSP90 inhibitor alvespimycin (17-DMAG, KOS-1022) by the pediatric preclinical testing program.
  • BACKGROUND: Alvespimycin (17-DMAG, KOS-1022), a potent small-molecule inhibitor of the protein chaperone Hsp90, is being developed as an anticancer agent because of the multiple Hsp90 client proteins involved in cancer cell growth and survival.
  • PROCEDURES: Alvespimycin was tested against the in vitro panel of the Pediatric Preclinical Testing Program (PPTP) at concentrations from 1 nM to 10 microM and was tested against the PPTP's in vivo tumor panels by intraperitoneal administration using a 50 mg/kg BID twice weekly x 6 weeks dose and schedule.
  • Hsp70 induction in tumor and liver tissue was used as a pharmacodynamic measure of Hsp90 inhibition and stress response induction.
  • Using the time to event activity measure, alvespimycin had intermediate or high activity against 4 of 28 evaluable solid tumor xenografts, including 3 of 4 alveolar rhabdomyosarcoma xenografts (one with a partial response).
  • Hsp70 induction was observed in tumor tissue from both responding and non-responding xenografts.
  • The greatest drug effect was observed for the alveolar rhabdomyosarcoma xenografts in the rhabdomyosarcoma panel.

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  • [Copyright] (c) 2008 Wiley-Liss, Inc.
  • (PMID = 18260120.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] ENG
  • [Grant] United States / NCI NIH HHS / CM / N01 CM042216; United States / NCI NIH HHS / CA / CA108786; United States / NCI NIH HHS / CA / CA21765; United States / NCI NIH HHS / CM / N01-CM-42216
  • [Publication-type] Journal Article; Research Support, N.I.H., Extramural
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 0 / Benzoquinones; 0 / HSP90 Heat-Shock Proteins; 0 / Lactams, Macrocyclic; 001L2FE0M3 / 17-(dimethylaminoethylamino)-17-demethoxygeldanamycin
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13. Kushner BH, Kramer K, LaQuaglia MP, Modak S, Cheung NK: Liver involvement in neuroblastoma: the Memorial Sloan-Kettering Experience supports treatment reduction in young patients. Pediatr Blood Cancer; 2006 Mar;46(3):278-84
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  • [Title] Liver involvement in neuroblastoma: the Memorial Sloan-Kettering Experience supports treatment reduction in young patients.
  • BACKGROUND: We reviewed clinical and biologic findings in a series of infants with neuroblastoma (NB) in liver.
  • The aim was to gain insights into improving therapy.
  • PATIENTS AND METHODS: Among 19 newly or recently diagnosed infants with NB in liver, 1987-2002, those with stage 4 involving bone received chemotherapy, while those without bone or extensive bone marrow (BM) involvement were observed or received limited treatment if NB caused life-threatening symptoms.
  • We assessed results in the context of NB treatment risk stratification, which is based on age, stage, and selected biologic features (MYCN, ploidy, histology).
  • RESULTS: Six of eight infants with bone involvement became long-term event-free survivors including 1/2 with MYCN amplification and four who received only 4-6 cycles of chemotherapy; at the end of treatment, four infants had abnormalities in liver +/- the primary site, but these resolved.
  • All 11 infants without bone lesions became long-term survivors with either no cytotoxic therapy or only one cycle of chemotherapy (+/- radiotherapy to liver), including four who had stage 4 and one stage 4S patient who still had NB in BM at age 15 months.
  • CONCLUSIONS: Treatment reduction should be considered for subsets of infants with non-MYCN-amplified widespread NB: stage 4 without bone or extensive BM involvement may not require cytotoxic therapy, stage 4S with symptomatic hepatomegaly may not require multiple cycles of chemotherapy, and classic stage 4 may do well with limited chemotherapy.
  • Persistent liver abnormalities post-treatment may not require continued therapy to achieve a radiologic complete remission.
  • [MeSH-major] Liver Neoplasms / therapy. Neuroblastoma / therapy
  • [MeSH-minor] Bone Marrow Neoplasms / mortality. Bone Marrow Neoplasms / pathology. Bone Marrow Neoplasms / secondary. Bone Marrow Neoplasms / therapy. Combined Modality Therapy. Disease-Free Survival. Female. Humans. Infant. Infant, Newborn. Liver / pathology. Male. Remission Induction. Retrospective Studies

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  • [CommentIn] Pediatr Blood Cancer. 2006 Mar;46(3):269-70 [16261577.001]
  • (PMID = 16124002.001).
  • [ISSN] 1545-5009
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / CA61017; United States / NCI NIH HHS / CA / CA72868
  • [Publication-type] Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
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14. Malek MM, Shah SR, Atri P, Paredes JL, DiCicco LA, Sindhi R, Soltys KA, Mazariegos GV, Kane TD: Review of outcomes of primary liver cancers in children: our institutional experience with resection and transplantation. Surgery; 2010 Oct;148(4):778-82; discussion 782-4
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  • [Title] Review of outcomes of primary liver cancers in children: our institutional experience with resection and transplantation.
  • BACKGROUND: Operative intervention plays an important role in the management of primary liver cancers in children.
  • Recent improvements in diagnostic modalities, pre- and postoperative chemotherapy, and operative technique have all led to improved survival in these patients.
  • Both hepatic resection and orthotopic liver transplantation are effective operations for pediatric liver tumors; which intervention is pursued is based on preoperative extent of disease.
  • This is a review of our institution's experience with operative management of pediatric liver cancer over an 18-year period.
  • METHODS: A retrospective chart review from 1990 to 2007 identified patients who were ≤18 years old who underwent operative intervention for primary liver cancer.
  • Demographics, type of operation, intraoperative details, pre- and postoperative management, as well as outcomes were recorded for all patients.
  • RESULTS: Fifty-four patients underwent 57 operations for primary liver cancer, 30 of whom underwent resection; the remaining 27 underwent orthotopic liver transplantation.
  • Twenty patients had stage 1 or 2 disease and 34 patients had stage 3 or 4 disease.
  • Forty-eight (89%) patients received preoperative chemotherapy.
  • Postoperative chemotherapy was given to 92% of patients.
  • Only 6 patients had a recurrence of their cancer, 5 after liver resection, 3 of whom later received a transplant.
  • There was only 1 recurrence after liver transplantation.
  • CONCLUSION: Operative intervention plays a critical role in the management of primary liver cancer in the pediatric population.
  • Neoadjuvant chemotherapy can be given if the tumor seems unresectable at diagnosis.
  • If chemotherapy is unable to sufficiently downstage the tumor, orthotopic liver transplantation becomes the patient's best option.
  • Our institution has had considerable experience with both resection and liver transplantation in the treatment of pediatric primary liver cancer, with good long-term outcomes.
  • [MeSH-major] Carcinoma, Hepatocellular / surgery. Hepatectomy. Hepatoblastoma / surgery. Liver Neoplasms / surgery. Liver Transplantation
  • [MeSH-minor] Antineoplastic Agents / therapeutic use. Chemotherapy, Adjuvant. Child. Child, Preschool. Humans. Infant. Neoadjuvant Therapy. Retrospective Studies. Survival Analysis. Treatment Outcome

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  • [Copyright] Copyright © 2010 Mosby, Inc. All rights reserved.
  • (PMID = 20728194.001).
  • [ISSN] 1532-7361
  • [Journal-full-title] Surgery
  • [ISO-abbreviation] Surgery
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents
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15. Randall RJ: Hepatitis C virus infection and long-term survivors of childhood cancer: issues for the pediatric oncology nurse. J Pediatr Oncol Nurs; 2001 Jan-Feb;18(1):4-15
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  • [Title] Hepatitis C virus infection and long-term survivors of childhood cancer: issues for the pediatric oncology nurse.
  • Patients with HCV have a broad spectrum of symptoms, which vary from elevated liver function test results to cirrhosis, liver cancer and end stage liver disease.
  • Past treatment therapies have not been highly effective; however, a new treatment is currently available.
  • Therefore, cancer survivors who received blood products to combat chemotherapy induced anemia and thrombocytopenia before 1980 represent a population at risk.
  • [MeSH-major] Blood Transfusion / adverse effects. Hepatitis C / etiology. Neoplasms / therapy. Survivors
  • [MeSH-minor] Algorithms. Child. Humans. Interferon-alpha / therapeutic use. United States / epidemiology


16. Ortega JA, Douglass EC, Feusner JH, Reynolds M, Quinn JJ, Finegold MJ, Haas JE, King DR, Liu-Mares W, Sensel MG, Krailo MD: Randomized comparison of cisplatin/vincristine/fluorouracil and cisplatin/continuous infusion doxorubicin for treatment of pediatric hepatoblastoma: A report from the Children's Cancer Group and the Pediatric Oncology Group. J Clin Oncol; 2000 Jul;18(14):2665-75
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  • [Title] Randomized comparison of cisplatin/vincristine/fluorouracil and cisplatin/continuous infusion doxorubicin for treatment of pediatric hepatoblastoma: A report from the Children's Cancer Group and the Pediatric Oncology Group.
  • PURPOSE: Previous studies demonstrated that chemotherapy with either cisplatin, vincristine, and fluorouracil (regimen A) or cisplatin and continuous infusion doxorubicin (regimen B) improved survival in children with hepatoblastoma.
  • After initial surgery, patients with stage I-unfavorable histology (UH; n = 43), stage II (n = 7), stage III (n = 83), and stage IV (n = 40) hepatoblastoma were randomized to receive regimen A (n = 92) or regimen B (n = 81).
  • Patients with stage I-favorable histology (FH; n = 9) were treated with four cycles of doxorubicin alone.
  • RESULTS: There were no events among patients with stage I-FH disease.
  • Patients with stage I-UH, stage II, stage III, or stage IV disease had 5-year EFS estimates of 91% (SD = 4%), 100%, 64% (SD = 5%), and 25% (SD = 7%), respectively.
  • At postinduction surgery I, patients with stage III or IV disease who were found to be tumor-free had no events; those who had complete resections achieved a 5-year EFS of 83% (SD = 6%); other patients with stage III or IV disease had worse outcome.
  • CONCLUSION: Treatment outcome was not significantly different between regimen A and regimen B.
  • Excellent outcome was achieved for patients with stage I-UH and stage II hepatoblastoma and for subsets of patients with stage III disease.
  • New treatment strategies are needed for the majority of patients with advanced-stage hepatoblastoma.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Hepatoblastoma / drug therapy. Liver Neoplasms / drug therapy
  • [MeSH-minor] Antibiotics, Antineoplastic / therapeutic use. Child. Child, Preschool. Cisplatin / administration & dosage. Combined Modality Therapy. Doxorubicin / administration & dosage. Doxorubicin / therapeutic use. Female. Fluorouracil / administration & dosage. Humans. Infant. Male. Neoplasm Staging. Proportional Hazards Models. Survival Analysis. Treatment Outcome. Vincristine / administration & dosage

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  • (PMID = 10894865.001).
  • [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
  • [Grant] United States / NCI NIH HHS / CA / R25 CA092049; United States / NCI NIH HHS / CA / CA 13539; United States / NCI NIH HHS / CA / CA 30969
  • [Publication-type] Clinical Trial; Comparative Study; Journal Article; Multicenter Study; Randomized Controlled Trial; Research Support, U.S. Gov't, P.H.S.
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antibiotics, Antineoplastic; 5J49Q6B70F / Vincristine; 80168379AG / Doxorubicin; Q20Q21Q62J / Cisplatin; U3P01618RT / Fluorouracil; AP protocol 1
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17. Pass AK, McLin VA, Rushton JR, Kearney DL, Hastings CA, Margolin JF: Vanishing bile duct syndrome and Hodgkin disease: a case series and review of the literature. J Pediatr Hematol Oncol; 2008 Dec;30(12):976-80
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  • This is the first description in which the diagnosis of vanishing bile duct syndrome (VBDS) preceded the diagnosis of Hodgkin disease (HD) by several months, and for which patients received modifications to modern MOPP-ABV chemotherapy with successful clinical remission.
  • VBDS is an uncommon form of liver disease manifested by severe cholestasis and progressive liver failure.
  • We report 2 cases of stage IIIB pediatric HD and VBDS.
  • Because VBDS is progressive and the only curative treatment is liver transplant, it is imperative to recognize that children with VBDS may also have concurrent HD.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Cholestasis / diagnosis. Hodgkin Disease / diagnosis
  • [MeSH-minor] Bleomycin / therapeutic use. Child. Combined Modality Therapy. Doxorubicin / therapeutic use. Humans. Male. Mechlorethamine / therapeutic use. Prednisone / therapeutic use. Procarbazine / therapeutic use. Radiotherapy Dosage. Remission Induction. Syndrome. Treatment Outcome. Vinblastine / therapeutic use. Vincristine / therapeutic use

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  • (PMID = 19131796.001).
  • [ISSN] 1536-3678
  • [Journal-full-title] Journal of pediatric hematology/oncology
  • [ISO-abbreviation] J. Pediatr. Hematol. Oncol.
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 11056-06-7 / Bleomycin; 35S93Y190K / Procarbazine; 50D9XSG0VR / Mechlorethamine; 5J49Q6B70F / Vincristine; 5V9KLZ54CY / Vinblastine; 80168379AG / Doxorubicin; VB0R961HZT / Prednisone; MOPP-ABV protocol
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18. Reingruber B, Boettcher MI, Klein P, Hohenberger W, Pelz JO: Hyperthermic intraperitoneal chemoperfusion is an option for treatment of peritoneal carcinomatosis in children. J Pediatr Surg; 2007 Sep;42(9):E17-21
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  • [Title] Hyperthermic intraperitoneal chemoperfusion is an option for treatment of peritoneal carcinomatosis in children.
  • BACKGROUND: Gastrointestinal carcinomas in childhood are rare and frequently present at an advanced stage.
  • In addition to surgery and intravenous chemotherapy, hyperthermic intraperitoneal chemoperfusion (HIPEC) may be an option for selected patients.
  • METHODS: After treating a series of adult patients, HIPEC for peritoneal carcinomatosis from a signet cell carcinoma of the colon was performed intraoperatively in a 12-year-old boy.
  • We performed intraoperative drug level monitoring and daily postoperative liver and kidney function tests and differential blood counts.
  • Perfusate and venous drug levels were similar to those in an adult case.
  • [MeSH-major] Antibiotics, Antineoplastic / administration & dosage. Carcinoma, Signet Ring Cell / drug therapy. Carcinoma, Signet Ring Cell / secondary. Chemotherapy, Cancer, Regional Perfusion. Hyperthermia, Induced. Mitomycin / administration & dosage. Peritoneal Neoplasms / drug therapy. Peritoneal Neoplasms / secondary

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  • (PMID = 17848227.001).
  • [ISSN] 1531-5037
  • [Journal-full-title] Journal of pediatric surgery
  • [ISO-abbreviation] J. Pediatr. Surg.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antibiotics, Antineoplastic; 50SG953SK6 / Mitomycin
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19. Malogolowkin M, Cotton CA, Green DM, Breslow NE, Perlman E, Miser J, Ritchey ML, Thomas PR, Grundy PE, D'Angio GJ, Beckwith JB, Shamberger RC, Haase GM, Donaldson M, Weetman R, Coppes MJ, Shearer P, Coccia P, Kletzel M, Macklis R, Tomlinson G, Huff V, Newbury R, Weeks D, National Wilms Tumor Study Group: Treatment of Wilms tumor relapsing after initial treatment with vincristine, actinomycin D, and doxorubicin. A report from the National Wilms Tumor Study Group. Pediatr Blood Cancer; 2008 Feb;50(2):236-41
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  • [Title] Treatment of Wilms tumor relapsing after initial treatment with vincristine, actinomycin D, and doxorubicin. A report from the National Wilms Tumor Study Group.
  • OBJECTIVE: We evaluated the use of alternating cycles of cyclophosphamide/etoposide and carboplatin/etoposide in children entered on National Wilms Tumor Study (NWTS)-5 who were diagnosed between August 1, 1995 and May 31, 2002 and who relapsed after chemotherapy with vincristine, actinomycin D, and doxorubicin (VAD) and radiation therapy (DD-4A).
  • PATIENTS AND METHODS: One hundred three patients who relapsed or had progressive disease after initial VAD chemotherapy and radiation therapy were registered on stratum C of the NWTS-5 Relapse protocol.
  • Twelve patients were not evaluable: five due to insufficient data, six due to major protocol violations, and one for refusal of therapy.
  • Among the 91 remaining patients, 14 with stage V Wilms tumor (WT), 1 with contralateral relapse, and 16 who did not achieve a complete response (CR) to the initial three-drug chemotherapy were not included in this analysis.
  • Relapse treatment included alternating courses of the drug pairs cyclophosphamide/etoposide and carboplatin/etoposide, surgery, and radiation therapy.
  • The lung was the only site of relapse for 33 patients; other sites of relapse included the operative bed, the abdomen, and liver.
  • CONCLUSION: These results demonstrate that approximately one-half of children with unilateral WT who relapse after initial treatment with VAD and radiation therapy can be successfully retreated.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / administration & dosage. Kidney Neoplasms / drug therapy. Wilms Tumor / drug therapy
  • [MeSH-minor] Carboplatin / administration & dosage. Child, Preschool. Combined Modality Therapy. Dactinomycin / administration & dosage. Doxorubicin / administration & dosage. Drug Administration Schedule. Etoposide / administration & dosage. Female. Humans. Infant. Infant, Newborn. Male. Neoplasm Staging. Recurrence

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  • [Copyright] (c) 2007 Wiley-Liss, Inc.
  • (PMID = 17539021.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / CA-42326
  • [Publication-type] Clinical Trial; Journal Article; Multicenter Study; Research Support, N.I.H., Extramural
  • [Publication-country] United States
  • [Chemical-registry-number] 1CC1JFE158 / Dactinomycin; 6PLQ3CP4P3 / Etoposide; 80168379AG / Doxorubicin; BG3F62OND5 / Carboplatin
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20. Pillon M, Piglione M, Garaventa A, Conter V, Giuliano M, Arcamone G, Mura R, Cellini M, D'Amore ES, Varotto S, Mussolin L, Rosolen A, AIEOP-NHL Committee: Long-term results of AIEOP LNH-92 protocol for the treatment of pediatric lymphoblastic lymphoma: a report of the Italian Association of Pediatric Hematology and Oncology. Pediatr Blood Cancer; 2009 Dec;53(6):953-9
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  • [Title] Long-term results of AIEOP LNH-92 protocol for the treatment of pediatric lymphoblastic lymphoma: a report of the Italian Association of Pediatric Hematology and Oncology.
  • BACKGROUND: Lymphoblastic lymphoma (LBL) is the second most frequent lymphoma subtype in childhood.
  • It is commonly treated according to therapy strategies for lymphoblastic leukemia.
  • METHODS: The AIEOP LNH-92 protocol was a modified LSA2-L2 therapy used for both T- and B-cell precursor LBL and included Induction, Consolidation, and Maintenance treatment with a total duration of 11 and 24 months for stages I and II, stages III and IV disease, respectively.
  • Outcome was comparable to most concomitant international protocols for LBL, but inferior to recent trials that included reinduction treatment or a higher intensity therapy for high stage disease.
  • Nevertheless, an intensified treatment is warranted for high stage disease.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Precursor Cell Lymphoblastic Leukemia-Lymphoma / therapy
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Drug-Induced Liver Injury. Female. Hematologic Diseases / chemically induced. Humans. Infant. Male. Remission Induction. Survival Analysis

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  • [CommentIn] Pediatr Blood Cancer. 2009 Dec;53(6):917-9 [19672977.001]
  • (PMID = 19621432.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Clinical Trial; Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
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21. Aretz S, Koch A, Uhlhaas S, Friedl W, Propping P, von Schweinitz D, Pietsch T: Should children at risk for familial adenomatous polyposis be screened for hepatoblastoma and children with apparently sporadic hepatoblastoma be screened for APC germline mutations? Pediatr Blood Cancer; 2006 Nov;47(6):811-8
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  • BACKGROUND: Hepatoblastoma (HB) is the most frequent liver tumor in childhood, occurring in the first few years of life.
  • Surgery combined with chemotherapy has resulted in dramatic improvements in prognosis.
  • Compared to the general population, the risk of HB is 750-7,500 times higher in children predisposed to familial adenomatous polyposis (FAP), an autosomal-dominant cancer predispostion syndrome caused by germline mutations in the tumor suppressor gene APC.
  • PROCEDURE: In our sample of 1,166 German FAP families, all known cases of HB were registered.
  • RESULTS: In the FAP families, seven unrelated cases of HB are documented; three had been detected at an advanced stage.
  • In Beckwith-Wiedemann syndrome (BWS), recent studies suggest an earlier detection of both Wilms tumor and HB by frequent screening.
  • We discuss the rationale and implications of a screening program; besides the examination procedure itself, screening for HB in children of FAP patients would have important consequences for the policy of predictive testing in FAP.
  • [MeSH-major] Adenomatous Polyposis Coli / genetics. Genes, APC. Germ-Line Mutation. Hepatoblastoma / genetics. Liver Neoplasms / genetics


22. Aydin GB, Ciftçi AO, Yalçin B, Akçören Z, Cağlar M, Senocak ME, Büyükpamukçu M: Paratesticular metastasis from Wilms tumor associated with a hydrocele. Pediatr Blood Cancer; 2006 Jul;47(1):97-9
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  • Metastatic sites other than the lungs, lymph nodes, and liver are unusual for Wilms tumor (WT).
  • We report a 3-year-old boy with stage IIA WT, who experienced paratesticular metastasis 2 months after surgery for an abdominal recurrence.
  • One month after the end of 12 months of salvage chemotherapy and abdominal radiotherapy, the patient has no evidence of disease.
  • [MeSH-minor] Child, Preschool. Combined Modality Therapy. Humans. Male. Orchiectomy. Testicular Neoplasms / secondary

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  • [Copyright] Copyright 2006 Wiley-Liss, Inc.
  • (PMID = 16049972.001).
  • [ISSN] 1545-5009
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article; Review
  • [Publication-country] United States
  • [Number-of-references] 16
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23. De Backer A, Madern GC, Oosterhuis JW, Hakvoort-Cammel FG, Hazebroek FW: Ovarian germ cell tumors in children: a clinical study of 66 patients. Pediatr Blood Cancer; 2006 Apr;46(4):459-64
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  • [Title] Ovarian germ cell tumors in children: a clinical study of 66 patients.
  • BACKGROUND: Ovarian germ cell tumors are rare in childhood.
  • The aim of this study is to review clinical presentation, management, and outcome in a two-center series of girls with ovarian germ cell tumor.
  • PROCEDURE: The records of 66 patients (median age 9 years) with histologically proven ovarian germ cell tumor (either benign or malignant), treated over a 44-year-span, were reviewed.
  • Most patients (52) were stage I, 4 were stage II, 6 stage III, and 1, with liver metastases, stage IV.
  • Unilateral salpingo-oophorectomy was the most frequently performed procedure (n = 46), and ovarian-sparing tumorectomy was performed in 9 patients (one bilaterally).
  • Histologically, teratomas were found most frequently (mature: 45, immature: 9), followed by mixed tumors (n = 7), yolk sac tumors (n = 3), dysgerminoma (n = 2), gonadoblastoma (n = 2), and embryonal carcinoma (n = 1).
  • Surgical removal of the tumor with or without the ovary and/or adnex was the sole treatment in 55 patients, chemotherapy was administered in 10 and radiotherapy + chemotherapy in one.
  • The 64 survivors are now between 8 months and 44 years after treatment.
  • CONCLUSIONS: With a recurrence rate of 4.5% and a mortality rate of 3%, this series confirms the excellent prognosis for girls with ovarian germ cell tumor (GCT).
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Neoplasms, Germ Cell and Embryonal / drug therapy. Ovarian Neoplasms / drug therapy
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Female. Humans. Infant. Infant, Newborn. Magnetic Resonance Imaging. Neoplasm Staging. Tomography, X-Ray Computed. Treatment Outcome

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  • (PMID = 16206211.001).
  • [ISSN] 1545-5009
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Journal Article; Multicenter Study
  • [Publication-country] United States
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24. Radford IR: Gd-Tex Pharmacyclics Inc. Curr Opin Investig Drugs; 2000 Dec;1(4):524-8
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  • Pharmacyclics is developing Gd-Tex (gadolinium texaphyrin) as a radiosensitizer for the potential treatment of various cancers including brain metastases and primary brain tumors, pancreatic tumors, lung tumors and pediatric cancers [196711], [348919].
  • Phase I clinical trials for the treatment of primary brain tumors and pancreatic cancer have been initiated while several trials in other cancer types are in the planning stages [367716].
  • In September 1998, Pharmacyclics announced the initiation of a pivotal phase III trial for the treatment of patients with brain metastases.
  • In September 2000, Pharmacyclics and the National Cancer Institute (NCI) initiated two phase I trials of Gd-Tex.
  • The first was to determine the safety of two different dosing regimens of the drug during preoperative radiotherapy after induction chemotherapy in patients with stage IIA non-small cell lung cancer (NSCLC).
  • Full results were announced in October 1998 at the American Society of Therapeutic Radiology and Oncology.
  • Gd-Tex was well tolerated, and liver enzyme elevation was the dose-limiting effect, which was reversible.
  • Death due to tumor progression was seen in 15% of the Gd-Tex group as opposed to 35% in the control group [302872].
  • In March 1997 the Decision Network of the NCI voted to sponsor additional clinical indications including adult and pediatric brain tumors, as well as cancers involving the lung, head & neck, pancreas and prostrate.
  • Two phase I trials of Gd-Tex for the treatment of primary brain tumors commenced in August 1998 under a CRADA with the NCI [237538], [295592], [348919].
  • [MeSH-major] Drugs, Investigational / therapeutic use. Neoplasms / therapy. Organometallic Compounds / therapeutic use. Radiation-Sensitizing Agents / therapeutic use

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  • (PMID = 11249709.001).
  • [ISSN] 1472-4472
  • [Journal-full-title] Current opinion in investigational drugs (London, England : 2000)
  • [ISO-abbreviation] Curr Opin Investig Drugs
  • [Language] eng
  • [Publication-type] Journal Article; Review
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Drugs, Investigational; 0 / Gd-Tex complex; 0 / Organometallic Compounds; 0 / Radiation-Sensitizing Agents
  • [Number-of-references] 33
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25. 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.
  • Secondary objectives included further evaluation of the toxicity and pharmacokinetic profile of Rebeccamycin analogue in children with relapsed and refractory cancer.
  • 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.
  • With a global response rate of 3% observed in children with relapsed CNS and non-CNS solid tumors, further development of Rebeccamycin analogue in pediatric solid tumors is not recommended.
  • [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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26. Modak S, Gerald W, Cheung NK: Disialoganglioside GD2 and a novel tumor antigen: potential targets for immunotherapy of desmoplastic small round cell tumor. Med Pediatr Oncol; 2002 Dec;39(6):547-51
The Lens. Cited by Patents in .

  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Disialoganglioside GD2 and a novel tumor antigen: potential targets for immunotherapy of desmoplastic small round cell tumor.
  • BACKGROUND: Desmoplastic small round cell tumor (DSRCT) is an aggressive and often misdiagnosed neoplasm of children and young adults.
  • It is chemotherapy-sensitive, yet patients often relapse off therapy because of residual microscopic disease at distant sites: peritoneum, liver, lymph node, and lung.
  • Monoclonal antibodies selective for cell surface tumor-associated antigens may have utility for diagnosis and therapy of MRD, as recently demonstrated in advanced-stage neuroblastoma (JCO 16: 3053, 1998).
  • 8H9 recognizes a 58 kDa surface antigen expressed among neuroectodermal, mesenchymal, and epithelial tumors with restricted expression on normal tissues.
  • Both G(D2) and the 58 kDa antigen were localized to tumor cell membrane and stroma.
  • CONCLUSIONS: G(D2) and the novel tumor antigen recognized by 8H9 are potential targets for immunodiagnosis and antibody-based therapy of DSRCT.
  • [MeSH-minor] Adolescent. Adult. Antigens, Surface / analysis. Female. Humans. Immunoglobulin G / therapeutic use. Immunohistochemistry. Immunologic Tests. Male. Middle Aged

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  • [Copyright] Copyright 2002 Wiley-Liss, Inc.
  • (PMID = 12376975.001).
  • [ISSN] 0098-1532
  • [Journal-full-title] Medical and pediatric oncology
  • [ISO-abbreviation] Med. Pediatr. Oncol.
  • [Language] eng
  • [Grant] United States / NCI NIH HHS / CA / CA 61017
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, Non-P.H.S.; Research Support, U.S. Gov't, P.H.S.
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / 3F8 antibody; 0 / Antibodies, Monoclonal; 0 / Antigens, Neoplasm; 0 / Antigens, Surface; 0 / Gangliosides; 0 / Immunoglobulin G; 65988-71-8 / ganglioside, GD2
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