Outcomes of neonatal retinoblastoma in pre-chemotherapy and chemotherapy eras

PURPOSE: To quantify outcomes for neonatal retinoblastoma patients treated during the pre-chemotherapy (1980–1994) and chemotherapy (1995–2018) eras. METHODS: Retrospective review of retinoblastoma patients diagnosed within the first 28 days of life between 1/1/1980 and 11/30/2018. Student's t-test, Chi-square, and Fisher's exact test were performed to compare treatments and outcomes by era. RESULTS: There were 68 patients with neonatal retinoblastoma (12% unilateral and 88% bilateral). According to era (pre-chemotherapy vs. chemotherapy), the number of treated patients was 26 (38%) vs. 42 (62%). Primary treatment was external beam radiotherapy (50% vs. 1%, P < 0.001), plaque radiotherapy (17% vs. 0%, P < 0.001), focal treatment (transpupillary thermotherapy or cryotherapy) only (21% vs. 14%, P = 0.33), intravenous chemotherapy (0% vs. 81%, P < 0.001), enucleation (10% vs. 4%, P = 0.26), or exenteration (2% vs. 0%, P = 0.37). Outcomes included tumor control (79% vs. 94%, P = 0.02), globe salvage (75% vs. 91%, P = 0.02), final gross visual acuity for salvaged eyes 20/200 or better (66% vs. 89%, P < 0.01), and death (19% vs. 0%, P < 0.01). CONCLUSION: Chemotherapy advancements for neonatal retinoblastoma have improved tumor control, globe salvage, visual acuity, and patient survival.