Eye-preserving therapies for advanced retinoblastoma: a multicenter cohort of 1678 patients in China.

Abstract Purpose Multiple eye-preserving treatments have been widely used in China for the last 15 years, however, controversy remains on using eye-preserving therapies for advanced retinoblastoma (The International Intraocular Retinoblastoma Classification: groups D and E). This study attempts to estimate the impact of eye-preserving therapies for the long-term prognosis of advanced retinoblastoma with regard to overall survival and ocular salvage. Design A retrospective cohort study covering all 31 provinces (38 retinoblastoma treating centers) of Chinese mainland. Participants A total of 1678 patients diagnosed with groups D or E retinoblastoma from January 2006 to May 2016. Methods Medical charts review was performed. The patients were divided into primary enucleation and eye-preserving groups, and they were followed up for survival status. The impact of initial treatment on survival was evaluated by Cox analyses. Main Outcome Measures Overall survival and final eye-preservation rate. Results After a median follow-up period of 43.9 months, 196 (12%) patients died, and the 5-year overall survival was 86%. In total, the eyeball preservation rate was 48%. In this cohort, 1172 (70%) patients had unilateral retinoblastoma, whereas 506 (30%) were bilateral. For unilateral patients, 570 (49%) eyes had primary enucleation, and 602 (51%) patients had eye-preserving therapies initially. During the follow-up (median: 45.6 months), 59 (10%) patients from primary enucleation group and 56 (9.3%) patients from eye-preserving group died. Multivariate Cox analyses indicated no significant difference in overall survival between the two groups (HR=1.25; 95%CI:0.85-1.84; p=0.250). For bilateral patients, only 95 (19%) eyes had primary enucleation, and 411 (81%) patients had eye-preserving therapies initially. During the follow-up (median: 40.1 months), 12 (13%) patients from primary enucleation group and 69 (17%) patients from eye-preserving group died. For bilateral retinoblastoma with the worse eye of group E, patients had primary enucleation exhibited better overall survival (HR=2.35; 95%CI:1.10-5.01; p=0.027), however, this survival advantage was not evident until passing 22.6 months after initial diagnosis. Conclusion Eye-preserving therapies have been widely used for advanced retinoblastoma in China. Bilateral patients with the worse eye of group E initially underwent eye-preserving therapies exhibited a worse overall survival. The choice of primary treatment for advanced retinoblastoma should be carefully weighed.