Intra-Arterial Chemotherapy (Ophthalmic Artery Chemosurgery) for Group D Retinoblastoma

• Published in: PloS one 2016-01, Vol.11 (1), p.e0146582-e0146582
• Main Authors: Abramson, David H, Daniels, Anthony B, Marr, Brian P, Francis, Jasmine H, Brodie, Scott E, Dunkel, Ira J, Gobin, Y Pierre
• Format: Article
• Language: English
• Subjects: Analysis; Antineoplastic Agents - pharmacology; Antineoplastic Agents - therapeutic use; Archives & records; Bridge failure; Cancer; Cancer therapies; Carboplatin; Care and treatment; Chemotherapy; Classification; Demographics; Demography; Drug dosages; Enucleation; Eye; Fatalities; Humans; Infant; Infants; Infusions, Intra-Arterial; Intra-arterial injections; Intravenous administration; Melphalan; Metastases; Neutropenia; Oncology; Ophthalmic Artery - drug effects; Ophthalmic Artery - pathology; Patients; Pediatrics; Quality; Radiation therapy; Retina; Retinoblastoma; Retinoblastoma - drug therapy; Risk factors; Salvage; Success; Survival; Topotecan; Treatment Outcome; Tumors; Veins & arteries
• ISSN: 1932-6203; 1932-6203
• DOI: 10.1371/journal.pone.0146582

To report globe salvage rates, patient survival and adverse events of ophthalmic artery chemosurgery (OAC) for International Classification of Retinoblastoma (ICRB) group D retinoblastoma (naive and after prior failures). Single institution retrospective review of all Group D eyes treated with OAC from 5/2006-12/2012. Patients were treated according to our previously-published techniques. Primary outcome was globe retention without need for external beam radiotherapy (EBRT). Demographics, prior treatments, OAC agents used, and adverse events were also recorded. 112 group D eyes (103 patients) that underwent OAC were included (average follow-up was 34 months, range: 2-110 months). 47 eyes were treatment-naïve, 58 eyes received prior treatments elsewhere, and 7 young infants (7 eyes) underwent our published "bridge therapy" (single agent intravenous carboplatin) until old enough to undergo OAC. Median number of OAC sessions/eye was 3 (range 1-9). 110/112 eyes received intra-arterial melphalan, but only 31 eyes received melphalan alone. 43 eyes received carboplatin, and 78 eyes received topotecan (never as a single agent). 80/112 eyes received >1 drug over their treatment course, and 39 eyes received all three agents. 24 eyes (16 pretreated, 7 treatment-naïve, 1 bridge) failed treatment and required enucleation during the study period. Enucleation and EBRT were avoided in 88/112 eyes (78.6%; including 40/47 [85.1%] treatment-naïve eyes, 42/58 [72.4%] previously-treated eyes, and 6/7 eyes [85.7%] among bridge patients). By Kaplan-Meier survival analysis, globe salvage rate was 74% at 110 months among all patients, and 85% at 110 months in the treatment-naïve subgroup. Transient grade 3/4 neutropenia was more common in patients receiving OAC bilaterally. No child died of metastatic disease. OAC is effective for curing group D retinoblastoma, achieving rates of globe salvage many times higher than systemic chemotherapy (10-47%), even in eyes that previously failed other treatments. OAC can be performed multiple times, using multiple agents, on one or both eyes of patients.