Retinoblastoma patient outcomes in the contemporary era: The RIVERBOAT Consortium.

Abstract

10062 Background: Retinoblastoma (RB) is the most common childhood eye tumor. Intraocular RB cure rates approach 100%. Therefore, treatment now focuses on globe salvage and preserving functional vision. The Research into Visual Endpoints and RB Health Outcomes After Treatment (RIVERBOAT) consortium was established to examine patient outcomes in the transitional era from systemic to intraocular therapy. Methods: Patients with RB treated at 13 North American centers from 2008-2022 were identified. Medical record abstraction was performed for disease presentation, visual acuity, treatment, globe salvage and functional outcome. Enrollment on the study included submission of retrospective data and prospective data on newly diagnosed patients as well as retrospective data from chart reviews. Results: 830 patients (68% white race, 77% non-Hispanic ethnicity) were enrolled. In 420 of these, data was limited to chart review. Median age at diagnosis of the 830 patients (1165 eyes) was 1 year (0 - 16.3) and at enrollment was 7.8 years (0 - 28.6). 60% had unilateral disease and the eye group distribution (International Intraocular Retinoblastoma Classification) was 10% A, 16% B, 12% C, 31% D, 27% E, and 4% unknown (UK). Of the 1165 affected eyes, major treatment modalities included primary enucleation (15%), systemic chemotherapy (SC) (36%), intraarterial chemotherapy (IAC) (10%), SC followed by IAC in 9%, and intravitreal chemotherapy in combination in 6%. SC only was used in 55-59% of those with A-C eyes compared to 29% of D eyes. IAC only was used in 20% of D eyes and 16% of C eyes. Secondary enucleation occurred in 151 eyes (14%): 63/416 (15%) of SC; 34/120 (28%) of IAC; 25/106 (24%) of SC followed by IAC; 29/523 (6%) other treatments. The overall globe salvage rate was 86%. Second malignancies occurred in 5, metastatic disease in 8 and pineoblastoma in 3 patients. Eleven patients died of RB (1%). Visual acuity after treatment was reported in 229 eyes: 106 (eye group 24 A, 37 B, 22 C, 19 D, 3 E, 1 UK) had normal vision (20/20-20/40). 38 eyes, (6 A, 11 B, 4 C, 10 D, 5 E, 2 UK) had moderate vision loss ( > 20/40 – 20/70). Twenty-five eyes (5 B, 2 C, 16 D, 2 E) had low vision ( > 20/70 < 20/200), and 60 eyes (12 B, 7 C, 32 D, 8 E, 1 UK) were legally blind (>20/200). In those treated with IAC only, normal vision was found in 30% of eyes, moderate vision loss in 22%, low vision in 13%, and legal blindness in 35%. In those treated with SC only, normal vision was noted in 53% of eyes, moderate vision loss in 11%, low vision in 11%, and legal blindness in 25%. In those treated with SC followed by IAC, normal vision was reported in 32%, moderate vision in 26%, low vision in 5% and legal blindness in 37%. Conclusions: We demonstrate the significant benefits witnessed by the evolution of RB therapy. Cure rates remain high, with a very low incidence of second malignancies, metastatic disease or trilateral RB. Eye salvage rate was excellent, avoiding low vision or legal blindness in two-thirds of the patients.