A single-arm study of systemic and sub-Tenon chemotherapy for Groups C and D intraocular retinoblastoma: A Children's Oncology Group study (ARET 0231).

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.

• Authors: Rima F Jubran, Judith G Villablanca, Mark Krailo, Jin Piao, Li Huang, A Linn Murphree, Joan O'Brien, Dan Gombos, Carol L Shields, Anna Meadows, Murali Chintagumpala
• Journal: Pediatric blood & cancer (Pediatr Blood Cancer) Vol. 67 Issue 9 Pg. e28502 (09 2020) ISSN: 1545-5017 [Electronic] United States
• PMID: 32589362 (Publication Type: Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)
• Copyright: © 2020 Wiley Periodicals, Inc.

Chemical References

• Vincristine
• Etoposide
• Carboplatin

Topics

• Adolescent
• Adult
• Antineoplastic Combined Chemotherapy Protocols (therapeutic use)
• Carboplatin (administration & dosage)
• Child
• Child, Preschool
• Etoposide (administration & dosage)
• Female
• Follow-Up Studies
• Humans
• Infant
• Male
• Prognosis
• Retinal Neoplasms (drug therapy, pathology)
• Retinoblastoma (drug therapy, pathology)
• Tenon Capsule
• Vincristine (administration & dosage)
• Young Adult