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A phase II trial of carboplatin for intraocular retinoblastoma
Author(s) -
Dunkel Ira J.,
Lee Thomas C.,
Shi Weiji,
Beaverson Katherine L.,
Novetsky Danielle,
Lyden David,
Finlay Jonathan L.,
McCormick Beryl,
Abramson David H.
Publication year - 2007
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.21163
Subject(s) - medicine , carboplatin , retinoblastoma , ophthalmology , chemotherapy , cisplatin , genetics , biology , gene
Abstract 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). Patients received a median of two cycles of carboplatin (range one to five cycles) beginning at a median age of 5 months (range 14 days to 22 months). Results All patients were extraocular disease free during the follow‐up period (median 76.3 months). Responses were noted in 33 of 36 evaluable eyes (92%). 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. Pediatr Blood Cancer 2007;49:643–648. © 2007 Wiley‐Liss, Inc.