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1. Antoneli CB, Steinhorst F, de Cássia Braga Ribeiro K, Novaes PE, Chojniak MM, Arias V, de Camargo B: Extraocular retinoblastoma: a 13-year experience. Cancer; 2003 Sep 15;98(6):1292-8
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Extraocular retinoblastoma: a 13-year experience.
  • BACKGROUND: The current study was performed to evaluate two regimens of treatment and to describe clinical and epidemiologic characteristics in patients with extraocular retinoblastoma.
  • METHODS: Eighty-three patients with extraocular retinoblastoma according to Childrens Cancer Group (CCG) classification were admitted to the Pediatric Department of the A. C.
  • The age, gender, race, lag time, first clinical presentation, staging, laterality, and treatment regimen were analyzed.
  • Treatment was comprised of cisplatin, teniposide, vincristine, doxorubicin, and cyclophosphamide during the first treatment period (1987-1991) or cisplatin and teniposide with alternating courses of ifosfamide and etoposide during the second treatment period (1992-2000).
  • The mean lag time was 10.5 months.
  • There was a positive correlation between lag time and age for unilateral tumors (correlation coefficient [r] = 0.35; P = 0.006), whereas the correlation was negative for bilateral tumors (r = -0.12; P = 0.63).
  • The 5-year overall survival was 55.1% in the first treatment period and 59.4% in the second treatment period (P = 0.69).
  • No significant differences with regard to survival rates were noted for unilateral tumors between the two treatment periods (44.6 noted for unilateral tumors vs. 59.1 noted for unilateral tumors).
  • CONCLUSIONS: In the current study, the addition of ifosfamide and etoposide to a treatment regimen comprised of cisplatin, teniposide, vincristine, doxorubicin, and cyclophosphamide did not appear to improve the survival of patients with extraocular retinoblastoma.
  • Patients with dissemination to the central nervous system or metastatic disease remain incurable and die of progressive disease, despite the aggressive treatment.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Retinoblastoma / drug therapy. Retinoblastoma / secondary
  • [MeSH-minor] Child, Preschool. Cisplatin / administration & dosage. Cyclophosphamide / administration & dosage. Doxorubicin / administration & dosage. Etoposide / administration & dosage. Eye Enucleation. Female. Humans. Ifosfamide / administration & dosage. Male. Retinal Neoplasms / pathology. Retinal Neoplasms / surgery. Teniposide / administration & dosage. Time Factors. Vincristine / administration & dosage

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  • [Copyright] Copyright 2003 American Cancer Society.DOI 10.1002/cncr.11647
  • (PMID = 12973854.001).
  • [ISSN] 0008-543X
  • [Journal-full-title] Cancer
  • [ISO-abbreviation] Cancer
  • [Language] eng
  • [Publication-type] Comparative Study; Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 5J49Q6B70F / Vincristine; 6PLQ3CP4P3 / Etoposide; 80168379AG / Doxorubicin; 8N3DW7272P / Cyclophosphamide; 957E6438QA / Teniposide; Q20Q21Q62J / Cisplatin; UM20QQM95Y / Ifosfamide
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2. Chang CY, Hung GY, Hsu WM, Kao SC, Hwang B, Hsieh YL: Retinoblastoma with spinal recurrence presenting as spinal cord compression. J Formos Med Assoc; 2006 Jun;105(6):497-502
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  • [Title] Retinoblastoma with spinal recurrence presenting as spinal cord compression.
  • Central nervous system (CNS) involvement is not rare in extraocular retinoblastoma, and it is not surprising to find it in view of its route of spread.
  • This report describes two patients with unilateral retinoblastoma with spinal recurrence presenting as SCC.
  • The first patient developed erythematous swelling of the right foot and weakness of the bilateral lower limbs at 7 months after left enucleation.
  • The second patient developed weakness of the bilateral lower limbs, and defecative and urinary difficulty for 2 days at 8 months after left enucleation.
  • The first patient refused chemotherapy and survived only 4 months due to disease progression.
  • The second patient received systemic and intrathecal chemotherapy, and survived 19.5 months without disease progression.
  • Spinal recurrence with SCC should be suspected when leg weakness or bowel or bladder disturbance occurs in patients with retinoblastoma.

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  • (PMID = 16801038.001).
  • [ISSN] 0929-6646
  • [Journal-full-title] Journal of the Formosan Medical Association = Taiwan yi zhi
  • [ISO-abbreviation] J. Formos. Med. Assoc.
  • [Language] ENG
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] Singapore
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3. Chantada G, Fandiño A, Dávila MT, Manzitti J, Raslawski E, Casak S, Schvartzman E: Results of a prospective study for the treatment of retinoblastoma. Cancer; 2004 Feb 15;100(4):834-42
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  • [Title] Results of a prospective study for the treatment of retinoblastoma.
  • BACKGROUND: The objectives of this prospective study were to avoid adjuvant treatment for patients with intraocular disease and patients with postlaminar optic nerve invasion (PL-ONI) without full choroidal or scleral invasion.
  • Adjuvant chemotherapy (Regimen 1) was given to patients with scleral invasion, PL-ONI without cut section, and full choroidal and/or scleral invasion.
  • A more intensive regimen of higher dose intravenous chemotherapy (Regimen 2) and local radiotherapy was given to patients with PL-ONI and compromise at the cut end and to patients with overt extraocular disease.
  • METHODS: Six-month intravenous chemotherapy included carboplatin plus etoposide alternating with cyclophosphamide plus vincristine (Regimen 1) and the same drugs at higher dosage plus idarubicin (Regimen 2).
  • None of 22 patients with isolated PL-ONI developed recurrent disease, whereas 2 of 8 patients with concomitant risk factors had tumor recurrences and died.
  • CONCLUSIONS: Adjuvant therapy can be avoided in patients with intraocular and isolated PL-ONI.
  • Patients with PL-ONI who also had other risk factors required intensive adjuvant therapy, such as patients with cut-end and overt extraocular disease.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Neoplasm Invasiveness. Retinoblastoma / drug therapy. Retinoblastoma / radiotherapy
  • [MeSH-minor] Carboplatin / administration & dosage. Chemotherapy, Adjuvant. Child. Child, Preschool. Cyclophosphamide / administration & dosage. Etoposide / administration & dosage. Eye Enucleation. Eye Neoplasms / drug therapy. Eye Neoplasms / pathology. Female. Humans. Idarubicin / administration & dosage. Infant. Male. Neoplasm Recurrence, Local. Optic Nerve Neoplasms / drug therapy. Optic Nerve Neoplasms / pathology. Prospective Studies. Risk Factors. Scleral Diseases / drug therapy. Scleral Diseases / pathology. Survival Analysis. Treatment Outcome. Vincristine / administration & dosage

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  • [Copyright] Copyright 2003 American Cancer Society.
  • (PMID = 14770442.001).
  • [ISSN] 0008-543X
  • [Journal-full-title] Cancer
  • [ISO-abbreviation] Cancer
  • [Language] eng
  • [Publication-type] Clinical Trial; Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 5J49Q6B70F / Vincristine; 6PLQ3CP4P3 / Etoposide; 8N3DW7272P / Cyclophosphamide; BG3F62OND5 / Carboplatin; ZRP63D75JW / Idarubicin
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4. Al-Salam S, Algawi K, Alashari M: Malignant non-teratoid medulloepithelioma of ciliary body with retinoblastic differentiation: a case report and review of literature. Neuropathology; 2008 Oct;28(5):551-6
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  • We present a 6-year-old child with intraocular and extraocular mass and high intraocular pressure.
  • A preliminary diagnosis of retinoblastoma with extraocular extension was made.
  • An exenteration of the left globe and orbital tissue was performed.
  • Tumor satellites were seen in the adjacent periocular soft tissue.
  • The treatment involved exenteration of the left globe and orbital tissue with secondary skin graft following chemotherapy.
  • The patient is well and has no recurrence after 1 year of treatment.
  • [MeSH-minor] Biomarkers, Tumor / analysis. Cell Differentiation. Child. Diagnosis, Differential. Glaucoma / etiology. Humans. Immunohistochemistry. Male. Retinoblastoma / pathology

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  • (PMID = 18410270.001).
  • [ISSN] 1440-1789
  • [Journal-full-title] Neuropathology : official journal of the Japanese Society of Neuropathology
  • [ISO-abbreviation] Neuropathology
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] Australia
  • [Chemical-registry-number] 0 / Biomarkers, Tumor
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5. Chantada G, Fandiño A, Casak S, Manzitti J, Raslawski E, Schvartzman E: Treatment of overt extraocular retinoblastoma. Med Pediatr Oncol; 2003 Mar;40(3):158-61
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  • [Title] Treatment of overt extraocular retinoblastoma.
  • BACKGROUND: Overt extraocular retinoblastoma is common in developing countries and little information about its treatment is available.
  • The aim of this study is to report our experience in the treatment of these cases using a uniform approach.
  • PROCEDURE: Patients with overt extraocular retinoblastoma including orbital extension, preauricular lymph node invasion and/or metastatic disease on diagnosis or after extraocular relapse admitted to the Hospital JP Garrahan from August 1987 to December 2000 were retrospectively reviewed.
  • Treatment included: neoadjuvant combination chemotherapy followed by limited surgery in case of orbital extension (enucleation or resection of residual orbital mass) and adjuvant chemotherapy and radiotherapy.
  • Chemotherapy included cyclophosphamide, vincristine, etoposide, doxorubicin (in protocol 87), idarubicin (in protocol 94), cisplatin (in protocol 87), and carboplatin (in protocol 94).
  • CONCLUSIONS: This treatment strategy was highly efficacious for patients with orbital and/or lymph node extension.
  • [MeSH-major] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Retinal Neoplasms / drug therapy. Retinal Neoplasms / radiotherapy. Retinoblastoma / drug therapy. Retinoblastoma / radiotherapy
  • [MeSH-minor] Adolescent. Adult. Aged. Chemotherapy, Adjuvant / methods. Combined Modality Therapy / methods. Developing Countries. Female. Humans. Male. Middle Aged. Neoadjuvant Therapy / methods. Neoplasm Invasiveness. Neoplasm Metastasis. Ophthalmologic Surgical Procedures / methods. Radiotherapy, Adjuvant / methods. Retrospective Studies. Survival Analysis. Time Factors. Treatment Outcome

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  • [Copyright] Copyright 2003 Wiley-Liss, Inc.
  • (PMID = 12518344.001).
  • [ISSN] 0098-1532
  • [Journal-full-title] Medical and pediatric oncology
  • [ISO-abbreviation] Med. Pediatr. Oncol.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
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6. Roth DB, Scott IU, Murray TG, Kaiser PK, Feuer WJ, Hughes JR, Rosa RH Jr: Echography of retinoblastoma: histopathologic correlation and serial evaluation after globe-conserving radiotherapy or chemotherapy. J Pediatr Ophthalmol Strabismus; 2001 May-Jun;38(3):136-43
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  • [Title] Echography of retinoblastoma: histopathologic correlation and serial evaluation after globe-conserving radiotherapy or chemotherapy.
  • PURPOSE: To assess the sensitivity of echography in detecting retinoblastoma, compare tumor features observed by echography with histopathology data, and assess the usefulness of echography in serially following retinoblastoma tumors after globe-conserving treatments.
  • METHODS: The medical and echography records of all patients treated for retinoblastoma at the Bascom Palmer Eye Institute between 1991 and 1997 were reviewed.
  • All eyes underwent pretreatment echographic evaluation, and eyes treated with external beam radiotherapy, brachytherapy, or chemotherapy underwent serial follow-up echography.
  • Echography demonstrated evidence of retinoblastoma in 69 of 69 (100%) eyes and calcification in 63 (91.3%) eyes.
  • Histopathology was superior to echography in detecting optic nerve invasion, extraocular extension, and presence of calcification.
  • CONCLUSION: Echography is a useful adjunct to indirect ophthalmoscopy in establishing the diagnosis of retinoblastoma.
  • While not as specific as histopathology, echographic evaluation before and after treatment of retinoblastoma permits monitoring of treatment response and may aid in detecting recurrent tumor growth or failure to respond to treatment.
  • [MeSH-major] Retinal Neoplasms / ultrasonography. Retinoblastoma / ultrasonography
  • [MeSH-minor] Brachytherapy. Child, Preschool. Drug Therapy. Eye Enucleation. Female. Humans. Infant. Male

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  • (PMID = 11386645.001).
  • [ISSN] 0191-3913
  • [Journal-full-title] Journal of pediatric ophthalmology and strabismus
  • [ISO-abbreviation] J Pediatr Ophthalmol Strabismus
  • [Language] eng
  • [Publication-type] Comparative Study; Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
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7. Bakhshi S, Meel R, Mohanti BK, Hasan Naqvi SG: Treatment and outcome of nonmetastatic extraocular retinoblastoma with a uniform chemotherapy protocol. J Pediatr Hematol Oncol; 2010 Mar;32(2):e42-5
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  • [Title] Treatment and outcome of nonmetastatic extraocular retinoblastoma with a uniform chemotherapy protocol.
  • Nonmetastatic extraocular retinoblastoma is a common entity in South-East Asia.
  • We did a retrospective study of patients treated for isolated extraocular retinoblastoma, that is, International retinoblastoma staging system stages II and III, using a uniform chemotherapy protocol at our oncology center, between June 2003 and June 2008.
  • Out of the 25 patients having nonmetastatic extraocular retinoblastoma, 6 were in stage II, and 19 in stage III.
  • This is the largest case series of nonmetastatic extraocular retinoblastoma from South-East Asia.
  • [MeSH-major] Orbital Neoplasms / drug therapy. Retinoblastoma / drug therapy
  • [MeSH-minor] Adolescent. Child. Child, Preschool. Disease-Free Survival. Female. Humans. Infant. Male. Neoplasm Staging. Retrospective Studies. Treatment Outcome

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  • (PMID = 20168241.001).
  • [ISSN] 1536-3678
  • [Journal-full-title] Journal of pediatric hematology/oncology
  • [ISO-abbreviation] J. Pediatr. Hematol. Oncol.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
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8. Murthy R, Honavar SG, Vemuganti GK, Naik MN, Reddy VP: Systemic metastasis following hyphema drainage in an unsuspected retinoblastoma. J Pediatr Ophthalmol Strabismus; 2007 Mar-Apr;44(2):120-3
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  • [Title] Systemic metastasis following hyphema drainage in an unsuspected retinoblastoma.
  • Biopsy revealed extraocular retinoblastoma and lymph node metastasis.
  • Computed tomography showed an intraocular mass with intracranial extension.
  • She died of metastatic disease despite intensive chemotherapy.
  • Retinoblastoma should be suspected in a child with hyphema following trivial trauma.
  • [MeSH-major] Brain Neoplasms / secondary. Conjunctival Neoplasms / secondary. Drainage. Hyphema / surgery. Retinal Neoplasms / pathology. Retinoblastoma / secondary
  • [MeSH-minor] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Calcinosis / etiology. Calcinosis / radiography. Calcinosis / ultrasonography. Child. Fatal Outcome. Female. Humans. Intraocular Pressure. Lymphatic Metastasis. Tomography, X-Ray Computed

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  • (PMID = 17410964.001).
  • [ISSN] 0191-3913
  • [Journal-full-title] Journal of pediatric ophthalmology and strabismus
  • [ISO-abbreviation] J Pediatr Ophthalmol Strabismus
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] United States
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9. Makimoto A: Results of treatment of retinoblastoma that has infiltrated the optic nerve, is recurrent, or has metastasized outside the eyeball. Int J Clin Oncol; 2004 Feb;9(1):7-12
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Results of treatment of retinoblastoma that has infiltrated the optic nerve, is recurrent, or has metastasized outside the eyeball.
  • Since the development of chemotherapy regimens for patients with retinoblastoma started in the 1950s, various agents and regimens have been employed for various kinds of patients.
  • Chemotherapy has been employed for:.
  • With the addition of appropriate chemotherapies to the conventional treatment modalities such as enucleation and radiotherapy, patients with advanced retinoblastoma are expected to obtain a survival benefit.
  • Moreover, a new modality combined with autologous stem cell support allowed us to use high-dose alkylating agents such as thiotepa, melphalan, and cyclophosphamide, which resulted in better prognosis for patients with metastatic retinoblastoma.
  • Because of the small number of patients with retinoblastoma and the diversity of the disease characteristics in individual patients, there have been no clinical trials to determine whether to recommend a particular regimen, or to identify specific criteria in patients who would benefit from chemotherapy.
  • Well-designed prospective controlled trials are warranted to establish a standard treatment strategy for patients with extraocular retinoblastoma.
  • [MeSH-major] Neoplasm Recurrence, Local / secondary. Neoplasm Recurrence, Local / therapy. Optic Nerve / pathology. Retinal Neoplasms / pathology. Retinal Neoplasms / therapy. Retinoblastoma / pathology. Retinoblastoma / therapy
  • [MeSH-minor] Drug Therapy / trends. Eye / pathology. Humans. Orbital Neoplasms / pathology. Orbital Neoplasms / therapy. Stem Cell Transplantation / trends. Transplantation, Autologous

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  • (PMID = 15162820.001).
  • [ISSN] 1341-9625
  • [Journal-full-title] International journal of clinical oncology
  • [ISO-abbreviation] Int. J. Clin. Oncol.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't; Review
  • [Publication-country] Japan
  • [Number-of-references] 30
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10. Taçyildiz N, Yavuz G, Unal E, Gündüz K, Günalp I, Ekinci C: Encouraging result of tamoxifen in a retinoblastoma patient with central nervous system metastasis. Pediatr Hematol Oncol; 2003 Sep;20(6):473-6
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  • [Title] Encouraging result of tamoxifen in a retinoblastoma patient with central nervous system metastasis.
  • Extraocular retinoblastoma occurs more frequently in developing countries as a delayed diagnosis and prognosis of patients with conventional therapy is very poor.
  • Metastatic retinoblastoma, especially in the central nervous system (CNS), is a highly lethal disease.
  • Tamoxifen has been used in some previous studies with variety of responses to therapy in patients with unresectable recurrent brain tumors.
  • A 7-year-old girl with recurrent metastatic retinoblastoma received 60 mg/m2 tamoxifen in addition to chemotherapy and CNS radiotherapy.
  • The authors think that tamoxifen can be added to treatment protocols of metastatic retinoblastoma to provide longer and at least higher quality of life for these patients.
  • [MeSH-major] Angiogenesis Inhibitors / therapeutic use. Eye Neoplasms / surgery. Meningeal Neoplasms / secondary. Retinoblastoma / secondary. Skull Neoplasms / secondary. Tamoxifen / therapeutic use
  • [MeSH-minor] Antineoplastic Combined Chemotherapy Protocols / administration & dosage. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Carboplatin / administration & dosage. Child, Preschool. Combined Modality Therapy. Cranial Irradiation. Cyclophosphamide / administration & dosage. Cytarabine / administration & dosage. Doxorubicin / administration & dosage. Etoposide / administration & dosage. Eye Enucleation. Female. Glucocorticoids / administration & dosage. Humans. Methotrexate / administration & dosage. Vincristine / administration & dosage

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  • (PMID = 14631622.001).
  • [ISSN] 0888-0018
  • [Journal-full-title] Pediatric hematology and oncology
  • [ISO-abbreviation] Pediatr Hematol Oncol
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] England
  • [Chemical-registry-number] 0 / Angiogenesis Inhibitors; 0 / Glucocorticoids; 04079A1RDZ / Cytarabine; 094ZI81Y45 / Tamoxifen; 5J49Q6B70F / Vincristine; 6PLQ3CP4P3 / Etoposide; 80168379AG / Doxorubicin; 8N3DW7272P / Cyclophosphamide; BG3F62OND5 / Carboplatin; YL5FZ2Y5U1 / Methotrexate; CEV regimen
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11. Armenian SH, Panigrahy A, Murphree AL, Jubran RF: Management of retinoblastoma with proximal optic nerve enhancement on MRI at diagnosis. Pediatr Blood Cancer; 2008 Oct;51(4):479-84
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Management of retinoblastoma with proximal optic nerve enhancement on MRI at diagnosis.
  • BACKGROUND: In North America, retinoblastoma rarely presents with gross clinical evidence of tumor involving the optic nerve.
  • Due to poor outcomes in patients with metastatic disease, historical treatment for patients with clinical evidence of extraocular optic nerve involvement has included upfront enucleation followed by aggressive adjuvant chemotherapy.
  • Little is known about the role of neoadjuvant therapy in the setting of orbital optic nerve enhancement on magnetic resonance imaging (MRI) at diagnosis.
  • METHODS: A retrospective review of consecutive retinoblastoma cases at Childrens Hospital Los Angeles over a 3-year period (2004-2006) found to have gadolinium contrast enhancement in the proximal portion of optic nerve on MRI at diagnosis.
  • All patients received neoadjuvant chemotherapy prior to enucleation.
  • CONCLUSIONS: Neoadjuvant chemotherapy is well tolerated prior to enucleation of retinoblastoma-containing eyes associated with contrast enhancement of the proximal optic nerve on MRI at diagnosis.
  • Such an approach may be used to decrease intensity or duration of chemotherapy and need for external beam radiation.
  • [MeSH-major] Optic Nerve Neoplasms / diagnosis. Optic Nerve Neoplasms / drug therapy. Retinal Neoplasms / diagnosis. Retinal Neoplasms / drug therapy. Retinoblastoma / diagnosis. Retinoblastoma / drug therapy
  • [MeSH-minor] Antineoplastic Agents / adverse effects. Antineoplastic Agents / therapeutic use. Child, Preschool. Female. Humans. Infant. Magnetic Resonance Imaging. Male. Treatment Outcome

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  • [Copyright] (c) 2008 Wiley-Liss, Inc.
  • (PMID = 18478574.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents
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12. Leal-Leal CA, Rivera-Luna R, Flores-Rojo ME, Amador-Zarco J, Juarez-Echenique C: Treatment of metastatic retinoblastoma with paclitaxel. Preliminary results of a pilot study with seven patients. J Clin Oncol; 2004 Jul 15;22(14_suppl):8569

  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Treatment of metastatic retinoblastoma with paclitaxel. Preliminary results of a pilot study with seven patients.
  • : 8569 Background: Metastatic retinoblastoma has a very high mortality rate in most of the cases in spite that international efforts have been made with different chemotherapy regimens.
  • Only high doses of chemotherapy followed by steam cells rescue have proved to provide long lasting survival.
  • METHODS: In a prospective study patients with pathological diagnosis of metastatic retinoblastoma were included, regardless of having recurrent disease or metastasis at time of diagnosis.
  • Once the staging procedure with imaging was performed, the patients were treated with paclitaxel as a single agent at a dose of 150 mg/m<sup>2</sup> days 1, 4 and 7 days every 21 days from 2-6 courses. RESULTS: .
  • At the present time 7 patients were registered into the study.
  • The time prior to develop metastasis was 11 months and a SD of 13 months.
  • All patients presented complete remission as per CAT scan and MRI studies by the second chemotherapy cycle, even intraocular dieses went to ptisis bublbi.
  • By the 20<sup>th</sup> month of follow up all children have died with tumor activity.
  • CONCLUSIONS: In this pilot study, placlitaxel showed to be an effective drug against intraocular and metastatic Retinoblastoma.
  • However, it should be tested in combination with other drugs in order to obtain long lasting survival for affected patients.

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  • (PMID = 28013865.001).
  • [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. Dunkel IJ, Lee TC, Shi W, Beaverson KL, Novetsky D, Lyden D, Finlay JL, McCormick B, Abramson DH: A phase II trial of carboplatin for intraocular retinoblastoma. Pediatr Blood Cancer; 2007 Oct 15;49(5):643-8
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  • [Title] A phase II trial of carboplatin for intraocular retinoblastoma.
  • BACKGROUND: Retinoblastoma patients with RB1 germline mutations are at risk of developing second malignancies and external beam radiation therapy increases the risk.
  • Carboplatin-containing chemotherapy regimens in conjunction with local therapies have been investigated for intraocular retinoblastoma, but the lack of data regarding the efficacy of single agent intravenous carboplatin prompted this phase II study.
  • PROCEDURE: Twenty-five patients (43 eyes) were treated with intravenous carboplatin (18.7 mg/kg for patients < 12 kg, 560 mg/m(2) for patients >/= 12 kg).
  • RESULTS: All patients were extraocular disease free during the follow-up period (median 76.3 months).
  • The 5-year overall ocular and ocular event-free survivals were 93.3% (95% CI, 84.4-100%) and 43.5% (95% CI, 25.8-61.3%) for eyes treated for Reese-Ellsworth (RE) group 1-3 disease and 25.0% (95% CI, 1.0-50.0%) and 8.3% (95% CI, 0-24.0%) for RE group 4-5 disease, respectively.
  • No non-hematopoietic serious or permanent toxicities related to the chemotherapy were observed.
  • CONCLUSION: When used as a neoadjuvant agent, carboplatin usually leads to objective responses of intraocular retinoblastoma.
  • The 5-year ocular event-free survival appears inferior to other protocols using more extensive chemotherapy, but with greater radiation therapy usage, overall ocular survival rate for RE group 1-3 eyes was excellent.
  • [MeSH-major] Carboplatin / administration & dosage. Retinoblastoma / drug therapy
  • [MeSH-minor] Antineoplastic Agents. Combined Modality Therapy. Disease-Free Survival. Female. Humans. Infant. Infant, Newborn. Male. Neoadjuvant Therapy. Remission Induction. Survival Analysis

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  • [Copyright] (c) 2007 Wiley-Liss, Inc.
  • (PMID = 17301956.001).
  • [ISSN] 1545-5009
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Clinical Trial, Phase II; Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents; BG3F62OND5 / Carboplatin
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14. Stannard C, Sealy R, Hering E, Hough J, Knowles R, Lecuona K, Reddi VB: Postenucleation orbits in retinoblastoma: treatment with 125I brachytherapy. Int J Radiat Oncol Biol Phys; 2002 Dec 1;54(5):1446-54
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Postenucleation orbits in retinoblastoma: treatment with 125I brachytherapy.
  • PURPOSE: Children with retinoblastoma that extends into or through the choroid, sclera, or optic nerve are at risk of developing orbital disease, as well as metastases.
  • Previously, these enucleated orbits were treated with external beam radiotherapy in addition to chemotherapy.
  • In 1983, he developed a technique to irradiate the contents of the orbit while limiting the dose to the bony orbit and eyelids.
  • Between 1983 and 2000, 57 orbits were treated in 56 children with retinoblastoma.
  • They received a median dose of 34 Gy in 70 h; 30 also received chemotherapy.
  • Children with tumor at the resection line of the nerve also received treatment to the craniospinal axis.
  • Eight of the 13 patients with microscopic extraocular tumor survived a median of 29 months (range 5-156).
  • No orbital recurrences developed in any of the patients.
  • CONCLUSION: Orbital brachytherapy is an effective method of irradiating the orbit to prevent recurrent tumor, the treatment time is short, and the cosmesis is much more acceptable than with other forms of irradiation.
  • [MeSH-major] Brachytherapy / instrumentation. Brachytherapy / methods. Eye Neoplasms / radiotherapy. Iodine Radioisotopes / therapeutic use. Retinoblastoma / radiotherapy
  • [MeSH-minor] Antineoplastic Agents / therapeutic use. Child. Child, Preschool. Combined Modality Therapy. Dose-Response Relationship, Radiation. Follow-Up Studies. Hot Temperature. Humans. Infant. Neoplasm Metastasis. Radiometry. Time Factors. Treatment Outcome

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  • (PMID = 12459368.001).
  • [ISSN] 0360-3016
  • [Journal-full-title] International journal of radiation oncology, biology, physics
  • [ISO-abbreviation] Int. J. Radiat. Oncol. Biol. Phys.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 0 / Iodine Radioisotopes
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15. Menon BS, Alagaratnam J, Juraida E, Mohamed M, Ibrahim H, Naing NN: Late presentation of retinoblastoma in Malaysia. Pediatr Blood Cancer; 2009 Feb;52(2):215-7
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Late presentation of retinoblastoma in Malaysia.
  • AIMS: The aims of this study were to review the presenting features, treatment and outcome for Malaysian children with retinoblastoma currently.
  • RESULTS: One hundred five children were diagnosed to have retinoblastoma.
  • Thirty-three children (31%) deferred treatment for 6 months or more.
  • Overall, 56 children had extraocular disease (55%), 52 at presentation, 4 later.
  • Seventy-one children (68%) underwent primary enucleation, 76 received chemotherapy (72%), and 23 radiotherapy (22%).
  • CONCLUSION: Retinoblastoma in Malaysia is still characterized by predominantly extraocular disease due to late presentation and high rates of abandonment.
  • [MeSH-major] Retinoblastoma / pathology
  • [MeSH-minor] Adolescent. Antineoplastic Agents / therapeutic use. Child. Child, Preschool. Ethnic Groups. Exophthalmos / etiology. Eye Enucleation. Female. Follow-Up Studies. Humans. Infant. Malaysia / epidemiology. Male. Orbital Cellulitis / etiology. Prospective Studies. Radiotherapy. Strabismus / etiology. Survival Rate

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  • [Copyright] (c) 2008 Wiley-Liss, Inc.
  • (PMID = 18855905.001).
  • [ISSN] 1545-5017
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents
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16. Chantada GL, Dunkel IJ, Antoneli CB, de Dávila MT, Arias V, Beaverson K, Fandiño AC, Chojniak M, Abramson DH: Risk factors for extraocular relapse following enucleation after failure of chemoreduction in retinoblastoma. Pediatr Blood Cancer; 2007 Sep;49(3):256-60
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Risk factors for extraocular relapse following enucleation after failure of chemoreduction in retinoblastoma.
  • OBJECTIVE: To assess the outcome and determine risk factors for extraocular relapse in patients with retinoblastoma who had been enucleated after failure of chemoreduction.
  • Extraocular relapse was defined as an event.
  • Adjuvant chemotherapy was given to eight patients (6.5%) because of scleral invasion.
  • Four patients had an extraocular relapse after enucleation, two of whom survive after intensive treatment including stem cell rescue.
  • Only scleral invasion and bilateral enucleation were significantly associated with extraocular relapse.
  • CONCLUSIONS: The risk of extraocular relapse is low after enucleation following failure of chemoreduction.
  • Patients who underwent bilateral enucleation and those with scleral invasion are at higher risk of extraocular relapse.
  • [MeSH-major] Eye Enucleation. Retinal Neoplasms / surgery. Retinoblastoma / secondary. Retinoblastoma / surgery
  • [MeSH-minor] Antineoplastic Agents / therapeutic use. Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Carboplatin / therapeutic use. Chemotherapy, Adjuvant. Child. Child, Preschool. Female. Humans. Infant. Male. Neoplasm Invasiveness. Retrospective Studies. Risk Factors. Survival Analysis

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  • [Copyright] Copyright (c) 2006 Wiley-Liss, Inc.
  • (PMID = 17029248.001).
  • [ISSN] 1545-5009
  • [Journal-full-title] Pediatric blood & cancer
  • [ISO-abbreviation] Pediatr Blood Cancer
  • [Language] eng
  • [Publication-type] Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents; BG3F62OND5 / Carboplatin
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17. Chantada GL, Casco F, Fandiño AC, Galli S, Manzitti J, Scopinaro M, Schvartzman E, de Dávila MT: Outcome of patients with retinoblastoma and postlaminar optic nerve invasion. Ophthalmology; 2007 Nov;114(11):2083-9
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Outcome of patients with retinoblastoma and postlaminar optic nerve invasion.
  • PURPOSE: To evaluate the outcome of patients with retinoblastoma and postlaminar optic nerve invasion (PLONI).
  • Patients were stratified into 2 risk groups: the high-risk group included those with concomitant full choroidal and/or scleral invasion and were given adjuvant chemotherapy.
  • Those without these features were considered low risk and chemotherapy was withheld after 1994.
  • MAIN OUTCOME MEASURES: Extraocular relapse and survival according to stratification.
  • Three had extraocular relapse (involving the central nervous system; all died of disease).
  • Microscopic scleral invasion was associated to extraocular relapse (P = 0.05).
  • Eighteen received postenucleation chemotherapy and none relapsed.
  • Twenty-one received no adjuvant therapy and 2 had a systemic relapse but were successfully retrieved.
  • CONCLUSIONS: Patients with PLONI have an excellent outcome with current therapy.
  • Risk stratification according to the presence of concomitant choroidal and/or scleral invasion may help in the decision of giving adjuvant therapy.
  • [MeSH-major] Optic Nerve Neoplasms / pathology. Retinal Neoplasms / pathology. Retinoblastoma / pathology
  • [MeSH-minor] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Child. Child, Preschool. Disease-Free Survival. Female. Humans. Infant. Male. Neoplasm Invasiveness. Neoplasm Recurrence, Local. Retrospective Studies. Survival Rate. Treatment Outcome

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  • (PMID = 17459482.001).
  • [ISSN] 1549-4713
  • [Journal-full-title] Ophthalmology
  • [ISO-abbreviation] Ophthalmology
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
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18. Parulekar MV: Retinoblastoma - current treatment and future direction. Early Hum Dev; 2010 Oct;86(10):619-25
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  • [Title] Retinoblastoma - current treatment and future direction.
  • Retinoblastoma is the commonest primary ocular malignancy of childhood.
  • Heritable retinoblastoma is a cancer susceptibility syndrome.
  • Early detection and prompt treatment can give cure rates up to 95% for intraocular tumours, but extraocular disease carries a very high mortality.
  • The diagnosis is essentially clinical and biopsy is contraindicated due to the risk of extraocular spread.
  • Treatment requires significant multidisciplinary input, with local ophthalmic treatment, systemic chemotherapy and external beam or plaque radiotherapy, or surgery to remove the affected eye.
  • Screening of family members is essential for early detection.
  • Newer treatment modalities including intra-arterial chemotherapy have been added to the therapeutic armamentarium in recent years.
  • [MeSH-major] Retinal Neoplasms / diagnosis. Retinoblastoma / diagnosis

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  • [Copyright] Copyright © 2010 Elsevier Ireland Ltd. All rights reserved.
  • (PMID = 20889272.001).
  • [ISSN] 1872-6232
  • [Journal-full-title] Early human development
  • [ISO-abbreviation] Early Hum. Dev.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] Ireland
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19. Cuenca A, Giron F, Castro D, Fandiño A, Guitter M, de Dávila MT, Chantada G: Microscopic scleral invasion in retinoblastoma: clinicopathological features and outcome. Arch Ophthalmol; 2009 Aug;127(8):1006-10
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  • [Title] Microscopic scleral invasion in retinoblastoma: clinicopathological features and outcome.
  • OBJECTIVE: To describe the clinical and pathological features of patients with retinoblastoma and microscopic scleral invasion.
  • METHODS: We reviewed all pathology slides of patients with microscopic scleral invasion who were included in 3 prospective treatment protocols (1988-2007).
  • All patients received adjuvant chemotherapy (moderately intensive chemotherapy in the first 2 protocols or a more intensive combination in the third one).
  • Sixteen were treated with moderately intensive chemotherapy and 16 received a higher-intensity regimen.
  • Seven patients had an extraocular relapse (central nervous system metastasis, n = 4; systemic metastasis, n = 2; and involving the orbit, n = 3, isolated in 1 and combined with central nervous system disease in 2).
  • CONCLUSIONS: Microscopic scleral invasion might be a risk factor for extraocular relapse, and more intensive chemotherapy results in improved survival for these patients.
  • [MeSH-major] Eye Neoplasms / pathology. Neoplasm Recurrence, Local / pathology. Retinal Neoplasms / pathology. Retinoblastoma / pathology. Scleral Diseases / pathology
  • [MeSH-minor] Chemotherapy, Adjuvant. Child. Child, Preschool. Eye Enucleation. Female. Follow-Up Studies. Humans. Infant. Male. Neoplasm Invasiveness. Prospective Studies. Survival Rate

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  • (PMID = 19667337.001).
  • [ISSN] 1538-3601
  • [Journal-full-title] Archives of ophthalmology (Chicago, Ill. : 1960)
  • [ISO-abbreviation] Arch. Ophthalmol.
  • [Language] eng
  • [Publication-type] Journal Article; Research Support, Non-U.S. Gov't
  • [Publication-country] United States
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20. Kim JW, Yau JW, Moshfeghi D, Fishman M: Orbital fibrosis and intraocular recurrence of retinoblastoma following periocular carboplatin. J Pediatr Ophthalmol Strabismus; 2010;47 Online:e1-4
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  • [Title] Orbital fibrosis and intraocular recurrence of retinoblastoma following periocular carboplatin.
  • A 5-month-old infant with bilateral advanced retinoblastoma underwent six cycles of systemic chemotherapy.
  • Following the third injection, the patient developed periocular ecchymosis and magnetic resonance imaging demonstrated abnormal signal characteristics at the site of injection.
  • An orbital biopsy did not demonstrate extraocular tumor extension, but histopathologic examination revealed severe orbital fibrosis and fat necrosis.
  • Following the biopsy, the patient developed an intraocular tumor recurrence at the same location where the carboplatin injections had been given and enucleation was performed to prevent tumor spread.
  • In this case, a child developed orbital scarring and intraocular tumor recurrence at the site of injection following treatment with periocular carboplatin.
  • [MeSH-major] Antineoplastic Agents / adverse effects. Carboplatin / adverse effects. Neoplasm Recurrence, Local / chemically induced. Orbit / pathology. Retinal Neoplasms / pathology. Retinoblastoma / pathology
  • [MeSH-minor] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Etoposide / therapeutic use. Eye Enucleation. Female. Fibrosis. Humans. Infant. Injections, Intraocular. Magnetic Resonance Imaging. Neoplasm Seeding. Tomography, X-Ray Computed. Vincristine / therapeutic use

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  • [Copyright] Copyright 2010, SLACK Incorporated.
  • (PMID = 21214147.001).
  • [ISSN] 1938-2405
  • [Journal-full-title] Journal of pediatric ophthalmology and strabismus
  • [ISO-abbreviation] J Pediatr Ophthalmol Strabismus
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 5J49Q6B70F / Vincristine; 6PLQ3CP4P3 / Etoposide; BG3F62OND5 / Carboplatin; CEV regimen
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21. Goto H, Kousaka A, Takano S, Usui M: Recurrence of retinoblastoma 12 years after brachytherapy. Am J Ophthalmol; 2002 Nov;134(5):773-5
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Recurrence of retinoblastoma 12 years after brachytherapy.
  • PURPOSE: To report a case of retinoblastoma that recurred 12 years after brachytherapy.
  • METHODS: A 2-month-old boy presented in December 1983 with bilateral retinoblastoma and was treated with bilateral 198Au plaque radiotherapy, photocoagulation, and cryotherapy.
  • RESULTS: In January 1996, cytopathologic examination of large keratic precipitates in the right eye demonstrated cells consistant with retinoblastoma.
  • The right eye was enucleated and diffuse retinoblastoma was noted histopathologically.
  • Systemic chemotherapy was given, and there has been no local recurrence or extraocular metastasis for 5 years.
  • CONCLUSIONS: This case emphasizes that long-term follow-up is essential for managing retinoblastoma after eye-preserving conservative therapy.
  • [MeSH-major] Brachytherapy. Gold Radioisotopes / therapeutic use. Neoplasm Recurrence, Local. Retinal Neoplasms / radiotherapy. Retinoblastoma / radiotherapy
  • [MeSH-minor] Child. Combined Modality Therapy. Cryosurgery. Eye Enucleation. Follow-Up Studies. Humans. Laser Coagulation. Male. Visual Acuity

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  • (PMID = 12429261.001).
  • [ISSN] 0002-9394
  • [Journal-full-title] American journal of ophthalmology
  • [ISO-abbreviation] Am. J. Ophthalmol.
  • [Language] eng
  • [Publication-type] Case Reports; Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Gold Radioisotopes
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22. Gündüz K, Müftüoglu O, Günalp I, Unal E, Taçyildiz N: Metastatic retinoblastoma clinical features, treatment, and prognosis. Ophthalmology; 2006 Sep;113(9):1558-66
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Metastatic retinoblastoma clinical features, treatment, and prognosis.
  • PURPOSE: To evaluate the clinical features, treatment, and prognosis in patients with metastatic retinoblastoma.
  • PARTICIPANTS: Eighteen consecutive patients with metastatic retinoblastoma who were diagnosed and managed at the Oncology Service of Ankara University, Turkey, between January 1999 and January 2005.
  • Histopathologic confirmation of retinoblastoma via enucleation, exenteration, or orbital biopsy was obtained in each patient.
  • The status of extraocular spread (optic nerve, extrascleral extension, or both) was assessed based on histopathologic or MRI results.
  • Systemic treatment for metastatic retinoblastoma consisted of chemotherapy and radiotherapy (craniospinal, orbital, or both), if necessary.
  • MAIN OUTCOME MEASURES: Status of extraocular spread, site of metastasis, and survival from metastatic retinoblastoma.
  • Ten patients had unilateral retinoblastoma, 7 had bilateral retinoblastoma, and 1 had trilateral retinoblastoma.
  • All patients with metastatic retinoblastoma had histopathologic or MRI evidence of unilateral extraocular disease characterized by optic nerve involvement, extrascleral extension, or both.
  • CONCLUSIONS: The prognosis for metastatic retinoblastoma is dismal and the presence of CNS involvement may portend an even worse outcome.
  • [MeSH-major] Retinal Neoplasms / pathology. Retinal Neoplasms / therapy. Retinoblastoma / secondary. Retinoblastoma / therapy
  • [MeSH-minor] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Bone Neoplasms / mortality. Bone Neoplasms / secondary. Bone Neoplasms / therapy. Brain Neoplasms / mortality. Brain Neoplasms / secondary. Brain Neoplasms / therapy. Child. Child, Preschool. Eye Enucleation. Eye Evisceration. Female. Humans. Infant. Magnetic Resonance Imaging. Male. Neoplasm Invasiveness. Optic Nerve Neoplasms / mortality. Optic Nerve Neoplasms / secondary. Optic Nerve Neoplasms / therapy. Orbital Neoplasms / mortality. Orbital Neoplasms / secondary. Orbital Neoplasms / therapy. Prognosis. Radiotherapy, High-Energy. Retrospective Studies. Survival Rate

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  • (PMID = 16828510.001).
  • [ISSN] 1549-4713
  • [Journal-full-title] Ophthalmology
  • [ISO-abbreviation] Ophthalmology
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
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23. Stannard C, Sealy R, Hering E, Korrubel J, Hill J, Barron A, Knowles R: Localized whole eye radiotherapy for retinoblastoma using a (125)I applicator, "claws". Int J Radiat Oncol Biol Phys; 2001 Oct 1;51(2):399-409
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  • [Source] The source of this record is MEDLINE®, a database of the U.S. National Library of Medicine.
  • [Title] Localized whole eye radiotherapy for retinoblastoma using a (125)I applicator, "claws".
  • PURPOSE: To treat children with retinoblastoma, who require whole eye radiotherapy, with a specially designed (125)I applicator that irradiates the eye while sparing the surrounding tissues.
  • METHODS AND MATERIALS: Under general anesthesia, a pericorneal ring is attached to the 4 extraocular muscles, and 4 appendages, each loaded with (125)I seeds, are inserted beneath the conjunctiva in-between each pair of muscles and attached anteriorly to the ring.
  • Eighteen received a median dose of 28 Gy during 91 hours and 11 received 40 Gy during 122 hours, when the relative biologic effectiveness was taken as 1 instead of 1.5.
  • Six had received prior chemotherapy.
  • Although 22 eyes responded, local control was achieved in 13 patients, 3 of whom required additional treatment for new tumors; a further 3 required additional treatment for tumor recurrence as well as new tumors.
  • Three developed cataracts 7, 8, and 12 years later, 1 of which has been removed.
  • CONCLUSIONS: This is a new way of irradiating the whole eye with a minimal dose to the surrounding tissues.
  • The treatment time is only 5 days.
  • [MeSH-major] Brachytherapy / instrumentation. Iodine Radioisotopes / therapeutic use. Retinoblastoma / radiotherapy
  • [MeSH-minor] Antineoplastic Combined Chemotherapy Protocols / therapeutic use. Child. Child, Preschool. Equipment Design. Eye Enucleation. Female. Humans. Infant. Male. Radiotherapy Dosage

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  • (PMID = 11567814.001).
  • [ISSN] 0360-3016
  • [Journal-full-title] International journal of radiation oncology, biology, physics
  • [ISO-abbreviation] Int. J. Radiat. Oncol. Biol. Phys.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Iodine Radioisotopes
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24. Chantada GL, Fandiño AC, Casak SJ, Mato G, Manzitti J, Schvartzman E: Activity of topotecan in retinoblastoma. Ophthalmic Genet; 2004 Mar;25(1):37-43
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  • [Title] Activity of topotecan in retinoblastoma.
  • PURPOSE: To report our experience with topotecan in children with relapsed/refractory metastatic and intraocular retinoblastoma.
  • If obvious progression was detected by physical examination in patients with overt extraocular disease or if progressive disease was noted after fundoscopic examination in patients with intraocular disease, a second cycle was not administered.
  • RESULTS: Nine patients (6 extraocular, 3 intraocular) were treated from November 1998 to March 2002.
  • In patients with extraocular disease, there were three partial responses, two cases of stable disease, and one case of progressive disease.
  • Two patients with relapsed/resistant intraocular disease had partial response. allowing local therapy to be performed, and the third patient had progressive disease.
  • The drug was well-tolerated.
  • No patient developed fever or documented infections.
  • CONCLUSION: Topotecan is active in extraocular and relapsed/resistant intraocular retinoblastoma.
  • The role of this drug in the treatment of retinoblastoma should be explored in further studies.
  • [MeSH-major] Antineoplastic Agents / therapeutic use. Enzyme Inhibitors / therapeutic use. Retinal Neoplasms / drug therapy. Retinoblastoma / drug therapy. Topotecan / therapeutic use
  • [MeSH-minor] Adult. Aged. Aged, 80 and over. Female. Humans. Infusions, Intravenous. Male. Middle Aged. Tomography, X-Ray Computed. Topoisomerase I Inhibitors

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  • (PMID = 15255113.001).
  • [ISSN] 1381-6810
  • [Journal-full-title] Ophthalmic genetics
  • [ISO-abbreviation] Ophthalmic Genet.
  • [Language] eng
  • [Publication-type] Journal Article
  • [Publication-country] Netherlands
  • [Chemical-registry-number] 0 / Antineoplastic Agents; 0 / Enzyme Inhibitors; 0 / Topoisomerase I Inhibitors; 7M7YKX2N15 / Topotecan
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25. Chévez-Barrios P, Chintagumpala M, Mieler W, Paysse E, Boniuk M, Kozinetz C, Hurwitz MY, Hurwitz RL: Response of retinoblastoma with vitreous tumor seeding to adenovirus-mediated delivery of thymidine kinase followed by ganciclovir. J Clin Oncol; 2005 Nov 1;23(31):7927-35
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  • [Title] Response of retinoblastoma with vitreous tumor seeding to adenovirus-mediated delivery of thymidine kinase followed by ganciclovir.
  • PURPOSE: To evaluate the feasibility and safety of adenovirus-mediated gene therapy as a treatment for tumor seeds in the vitreous of children with retinoblastoma.
  • PATIENTS AND METHODS: An Institutional Biosafety Committee-, Institutional Review Board-, Recombinant DNA Advisory Committee-, and US Food and Drug Administration-approved phase I study used intrapatient dose escalation of adenoviral vector containing a herpes simplex thymidine kinase gene (AdV-TK) followed by systemic administration of ganciclovir to treat bilateral retinoblastoma with vitreous tumor seeding refractory to standard therapies.
  • One patient was free of active vitreous tumor seeds 38 months after therapy.
  • There has been no evidence of extraocular spread of tumor along the needle tract in any patient.
  • CONCLUSION: AdV-TK followed by ganciclovir can be administered safely to children with retinoblastoma.
  • Suicide gene therapy may contribute to the treatment of children with retinoblastoma tumor seeds in the vitreous, a resistant complication of retinoblastoma.
  • [MeSH-major] Adenoviruses, Human / genetics. Antiviral Agents / therapeutic use. Ganciclovir / therapeutic use. Neoplasm Seeding. Retinal Neoplasms / therapy. Retinoblastoma / therapy. Thymidine Kinase / therapeutic use. Vitreous Body / pathology
  • [MeSH-minor] Child. Combined Modality Therapy. Feasibility Studies. Gene Transfer Techniques. Genetic Therapy. Genetic Vectors. Humans. Neoplasm Recurrence, Local / enzymology. Neoplasm Recurrence, Local / genetics. Neoplasm Recurrence, Local / therapy

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  • (PMID = 16258092.001).
  • [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 / CA97762
  • [Publication-type] Clinical Trial, Phase I; Journal Article; Research Support, N.I.H., Extramural; Research Support, U.S. Gov't, P.H.S.
  • [Publication-country] United States
  • [Chemical-registry-number] 0 / Antiviral Agents; EC 2.7.1.21 / Thymidine Kinase; P9G3CKZ4P5 / Ganciclovir
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