Ocular salvage and vision preservation using a topotecan-based regimen for advanced intraocular retinoblastoma

Rachel C. Brennan, Ibrahim Qaddoumi, Shenghua Mao, Jianrong Wu, Catherine A. Billups, Clinton F. Stewart, Mary Ellen Hoehn, Carlos Rodriguez-Galindo, Matthew Wilson

Research output: Contribution to journalArticle

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Abstract

Purpose: To evaluate topotecan-based therapy for advanced intraocular retinoblastoma. Patients and Methods: Twenty-seven patients with bilateral retinoblastoma (male patients, n = 14; median age, 8.4 months) received chemotherapy: two courses of topotecan plus vincristine followed by three alternating administrations of carboplatin and vincristine for two courses and topotecan plus vincristine for one course, with optional periocular carboplatin. Focal therapy was applied after cycle 2. Event-free survival was defined as avoidance of external-beam radiotherapy (EBRT) and enucleation. Results: Of 54 eyes, 42 were Reese-Ellsworth group IV to V, and 37 were International Classification of Retinoblastoma group C to E. Eleven eyes were enucleated: one at diagnosis, nine with progressive disease including three eyes treated with EBRT, and one that developed neovascular glaucoma after completion of therapy. One patient was removed from protocol with prolonged infection in course 1; 26 patients (51 eyes) were analyzed. At 10 years, cumulative incidence of EBRT was 5.9% (SE ± 3), and event-free survival was 69.2% (SE ± 27.2). Ocular survival for Reese-Ellsworth group IV to V eyes was 77.5% (SE ± 21.2); it was 74.3% (SE ≥ 18.8) for International Classification group C to E eyes. Vision testing (median age, 7 years; range, 5 to 10 years) documented 20/70 vision or better in one eye of 23 patients; 19 (76%) of 25 patients demonstrated 20/40 vision or better in one eye. All patients experienced thrombocytopenia (41 episodes in 275 courses; 15%). There were 29 episodes of febrile neutropenia (10%). Grade 3 diarrhea was present in nine of 27 patients, and one patient had an allergic reaction to carboplatin. All patients are alive at median follow-up 7.4 years (range, 2.7 to 10 years). Conclusion: Topotecan combined with vincristine, carboplatin, and aggressive focal therapies is an effective regimen for the treatment of advanced retinoblastoma and results in globe salvage with vision. Toxicities were anticipated and managed with appropriate supportive care.

Original languageEnglish (US)
Pages (from-to)72-77
Number of pages6
JournalJournal of Clinical Oncology
Volume35
Issue number1
DOIs
StatePublished - Jan 1 2017

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Topotecan
Retinoblastoma
Carboplatin
Vincristine
Radiotherapy
Disease-Free Survival
Neovascular Glaucoma
Therapeutics
Febrile Neutropenia
Thrombocytopenia
Diarrhea
Hypersensitivity

All Science Journal Classification (ASJC) codes

  • Oncology
  • Cancer Research

Cite this

Ocular salvage and vision preservation using a topotecan-based regimen for advanced intraocular retinoblastoma. / Brennan, Rachel C.; Qaddoumi, Ibrahim; Mao, Shenghua; Wu, Jianrong; Billups, Catherine A.; Stewart, Clinton F.; Hoehn, Mary Ellen; Rodriguez-Galindo, Carlos; Wilson, Matthew.

In: Journal of Clinical Oncology, Vol. 35, No. 1, 01.01.2017, p. 72-77.

Research output: Contribution to journalArticle

Brennan, RC, Qaddoumi, I, Mao, S, Wu, J, Billups, CA, Stewart, CF, Hoehn, ME, Rodriguez-Galindo, C & Wilson, M 2017, 'Ocular salvage and vision preservation using a topotecan-based regimen for advanced intraocular retinoblastoma', Journal of Clinical Oncology, vol. 35, no. 1, pp. 72-77. https://doi.org/10.1200/JCO.2016.69.2996
Brennan, Rachel C. ; Qaddoumi, Ibrahim ; Mao, Shenghua ; Wu, Jianrong ; Billups, Catherine A. ; Stewart, Clinton F. ; Hoehn, Mary Ellen ; Rodriguez-Galindo, Carlos ; Wilson, Matthew. / Ocular salvage and vision preservation using a topotecan-based regimen for advanced intraocular retinoblastoma. In: Journal of Clinical Oncology. 2017 ; Vol. 35, No. 1. pp. 72-77.
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AU - Qaddoumi, Ibrahim

AU - Mao, Shenghua

AU - Wu, Jianrong

AU - Billups, Catherine A.

AU - Stewart, Clinton F.

AU - Hoehn, Mary Ellen

AU - Rodriguez-Galindo, Carlos

AU - Wilson, Matthew

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N2 - Purpose: To evaluate topotecan-based therapy for advanced intraocular retinoblastoma. Patients and Methods: Twenty-seven patients with bilateral retinoblastoma (male patients, n = 14; median age, 8.4 months) received chemotherapy: two courses of topotecan plus vincristine followed by three alternating administrations of carboplatin and vincristine for two courses and topotecan plus vincristine for one course, with optional periocular carboplatin. Focal therapy was applied after cycle 2. Event-free survival was defined as avoidance of external-beam radiotherapy (EBRT) and enucleation. Results: Of 54 eyes, 42 were Reese-Ellsworth group IV to V, and 37 were International Classification of Retinoblastoma group C to E. Eleven eyes were enucleated: one at diagnosis, nine with progressive disease including three eyes treated with EBRT, and one that developed neovascular glaucoma after completion of therapy. One patient was removed from protocol with prolonged infection in course 1; 26 patients (51 eyes) were analyzed. At 10 years, cumulative incidence of EBRT was 5.9% (SE ± 3), and event-free survival was 69.2% (SE ± 27.2). Ocular survival for Reese-Ellsworth group IV to V eyes was 77.5% (SE ± 21.2); it was 74.3% (SE ≥ 18.8) for International Classification group C to E eyes. Vision testing (median age, 7 years; range, 5 to 10 years) documented 20/70 vision or better in one eye of 23 patients; 19 (76%) of 25 patients demonstrated 20/40 vision or better in one eye. All patients experienced thrombocytopenia (41 episodes in 275 courses; 15%). There were 29 episodes of febrile neutropenia (10%). Grade 3 diarrhea was present in nine of 27 patients, and one patient had an allergic reaction to carboplatin. All patients are alive at median follow-up 7.4 years (range, 2.7 to 10 years). Conclusion: Topotecan combined with vincristine, carboplatin, and aggressive focal therapies is an effective regimen for the treatment of advanced retinoblastoma and results in globe salvage with vision. Toxicities were anticipated and managed with appropriate supportive care.

AB - Purpose: To evaluate topotecan-based therapy for advanced intraocular retinoblastoma. Patients and Methods: Twenty-seven patients with bilateral retinoblastoma (male patients, n = 14; median age, 8.4 months) received chemotherapy: two courses of topotecan plus vincristine followed by three alternating administrations of carboplatin and vincristine for two courses and topotecan plus vincristine for one course, with optional periocular carboplatin. Focal therapy was applied after cycle 2. Event-free survival was defined as avoidance of external-beam radiotherapy (EBRT) and enucleation. Results: Of 54 eyes, 42 were Reese-Ellsworth group IV to V, and 37 were International Classification of Retinoblastoma group C to E. Eleven eyes were enucleated: one at diagnosis, nine with progressive disease including three eyes treated with EBRT, and one that developed neovascular glaucoma after completion of therapy. One patient was removed from protocol with prolonged infection in course 1; 26 patients (51 eyes) were analyzed. At 10 years, cumulative incidence of EBRT was 5.9% (SE ± 3), and event-free survival was 69.2% (SE ± 27.2). Ocular survival for Reese-Ellsworth group IV to V eyes was 77.5% (SE ± 21.2); it was 74.3% (SE ≥ 18.8) for International Classification group C to E eyes. Vision testing (median age, 7 years; range, 5 to 10 years) documented 20/70 vision or better in one eye of 23 patients; 19 (76%) of 25 patients demonstrated 20/40 vision or better in one eye. All patients experienced thrombocytopenia (41 episodes in 275 courses; 15%). There were 29 episodes of febrile neutropenia (10%). Grade 3 diarrhea was present in nine of 27 patients, and one patient had an allergic reaction to carboplatin. All patients are alive at median follow-up 7.4 years (range, 2.7 to 10 years). Conclusion: Topotecan combined with vincristine, carboplatin, and aggressive focal therapies is an effective regimen for the treatment of advanced retinoblastoma and results in globe salvage with vision. Toxicities were anticipated and managed with appropriate supportive care.

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