First-line chemotherapy with local treatment can prevent external-beam irradiation and enucleation in low-stage intraocular retinoblastoma.
OBJECTIVE
To evaluate the efficacy of first-line chemotherapy (CT) in preventing external-beam radiotherapy (EBR) and/or enucleation in patients with retinoblastoma (Rbl).


METHODS
Twenty-four patients with newly diagnosed unilateral or bilateral Rbl received CT associated with local treatment (LT). Two to five courses of etoposide and carboplatin were administered at 3- to 4-week intervals, depending on tumor response, and were completed each time by LT.


RESULTS
Tumor response was observed in all eyes. Twenty-one of 24 patients showed a complete response (CR) that persisted at a median follow-up (FU) of 31 months (range, 4 to 41 months). Among the three patients who relapsed, two were lost to FU and one died of progressive disease. CR was achieved by CT and LT alone in 15 (71.4%) of 21 patients with less advanced disease (groups I to III). Six other patients with advanced disease (groups IV and V) experienced treatment failure and needed salvage treatment by EBR and/or enucleation. The difference between the two patient groups with regard to disease stage was statistically significant (P <.0001). EBR could be avoided in 13 (68.4%) of 19 patients, who presented with groups I to III (15 eyes) and group V (one eye) disease, whereas enucleation could be avoided in only two (40%) of five.


CONCLUSIONS
CT combined with intensive LT is effective in patients with groups I to III Rbl, permitting the avoidance of EBR in the majority of these young children and, thus, reducing the risk of long-term sequelae. This is in contrast with the disappointing results for patients with groups IV and V Rbl, in whom EBR and/or enucleation was needed.
Pediatric Department, Hematology-Oncology Unit, and Department of Radiooncology, Centre Hospitalier Universitaire Vaudois, and Jules Gonin Eye Hospital, Lausanne, Switzerland. mnenadov@hola.hospvd.ch
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Beck
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Balmer
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Dessing
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Pica
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Munier
D000293:Adolescent; D000328:Adult; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D016190:Carboplatin / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D018450:Disease Progression; D005047:Etoposide / Q000008:administration & dosage; D015353:Eye Enucleation; D005260:Female; D006801:Humans; D008297:Male; D019572:Retinal Neoplasms / Q000188:drug therapy* / Q000532:radiotherapy / Q000601:surgery; D012175:Retinoblastoma / Q000188:drug therapy* / Q000532:radiotherapy / Q000601:surgery; D016896:Treatment Outcome
D005047:Etoposide; D016190:Carboplatin