Abstract
Background: Eyes with Group D intraocular retinoblastoma have low salvage rates. A pilot study showed safety and efficacy of sub-Tenon's fascia carboplatin with systemic chemotherapy supporting further study. Methods: Children with newly diagnosed bilateral intraocular retinoblastoma with at least one remaining Group C or D eye were treated with six courses of carboplatin/etoposide/vincristine (CEV) with sub-Tenon's fascia carboplatin for Group C/D eyes during courses 2-4. Local ophthalmic therapy started at course 3. The primary study objective was to determine the 1-year failure rate of Group D eyes. Results: The study closed prematurely due to poor accrual and 22 of 30 patients were evaluable for failure rate, contributing 25 Group D and four Group C eyes. Among the 25 Group D eyes, there were 13 failures within the first year of study enrollment including eight needing external beam radiotherapy (EBR) and five needing enucleation, resulting in 1-year failure rate of 52%. The failure rate was significantly lower than the historical rate of 70% (P =.039). The 1-year eye preservation rate for Group D eyes was 80% (20/25). One-year failure rate for Group C eyes was 25% (1/4); 1-year preservation rate was 100% without need for EBR. Systemic toxicity included Grade 3 hearing loss in two subjects, infections, neutropenia, and thrombocytopenia. Ocular toxicities included periorbital fat atrophy (13/29 = 45% eyes), optic nerve atrophy (1/29 = 3% eyes), and restrictive fibrosis (1/29 = 3% eyes). Conclusions: Sub-Tenon's fascia carboplatin plus CEV was partially effective in Group D intraocular retinoblastoma but had unacceptable ocular toxicities.
Original language | English (US) |
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Article number | e28502 |
Pages (from-to) | e28502 |
Journal | Pediatric Blood and Cancer |
Volume | 67 |
Issue number | 9 |
DOIs | |
State | Published - Sep 1 2020 |
Keywords
- clinical trial
- retinoblastoma
- sub-Tenon chemotherapy
- Retinal Neoplasms/drug therapy
- Prognosis
- Follow-Up Studies
- Humans
- Child, Preschool
- Infant
- Male
- Vincristine/administration & dosage
- Tenon Capsule
- Antineoplastic Combined Chemotherapy Protocols/therapeutic use
- Etoposide/administration & dosage
- Young Adult
- Adolescent
- Adult
- Carboplatin/administration & dosage
- Female
- Child
- Retinoblastoma/drug therapy
ASJC Scopus subject areas
- Hematology
- Oncology
- Pediatrics, Perinatology, and Child Health