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.


METHODS
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.


CONCLUSIONS
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.
Dunkel
Ira J
IJ
Department of Pediatrics, Memorial Sloan-Kettering Cancer Center, New York, New York, USA. dunkeli@mskcc.org
Lee
Thomas C
TC
Shi
Weiji
W
Beaverson
Katherine L
KL
Novetsky
Danielle
D
Lyden
David
D
Finlay
Jonathan L
JL
McCormick
Beryl
B
Abramson
David H
DH
D000970:Antineoplastic Agents; D016190:Carboplatin / Q000008:administration & dosage* / Q000633:toxicity; D003131:Combined Modality Therapy; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D020360:Neoadjuvant Therapy; D012074:Remission Induction; D012175:Retinoblastoma / Q000188:drug therapy* / Q000401:mortality / Q000532:radiotherapy; D016019:Survival Analysis
D000970:Antineoplastic Agents; D016190:Carboplatin