Original StudyTOXICITY AND EFFICACY OF INTRAVITREAL MELPHALAN FOR RETINOBLASTOMA 25 µg Versus 30 µgLiao, Albert MD, MS*,†; Hsieh, Terry MD, PhD*,‡; Francis, Jasmine H. MD*,§; Lavery, Jessica A. MS¶; Mauguen, Audrey PhD¶; Brodie, Scott E. MD, PhD**; Abramson, David H. MD, FACS*,§Author Information *Ophthalmic Oncology Service, Memorial Sloan-Kettering Cancer Center, New York, New York; †Emory Eye Center, Emory University School of Medicine, Atlanta, Georgia; ‡Stein Eye Institute, David Geffen School of Medicine at UCLA, Los Angeles, California; §Department of Ophthalmology, Weill-Cornell Medical Center, New York, New York; ¶Department of Epidemiology and Biostatistics, Memorial Sloan-Kettering Cancer Center, New York, New York; and **Department of Ophthalmology, NYU Langone Health, New York, New York. Reprint requests: David H. Abramson, MD, FACS, Ophthalmic Oncology Service, Memorial Sloan Kettering Cancer Center, 1275 York Ave, New York, NY 10065; e-mail: [email protected] Supported in part by grants from the Fund for Ophthalmic Knowledge, Inc, New York, NY and Cancer Center Support Grant (P30 CA008748). The sponsor or funding organization had no role in the design or conduct of this research. Alkeran and Evomela, the products used in this study, are not labeled for the use under discussion. None of the authors has any conflicting interests to disclose. Supplemental digital content is available for this article. Direct URL citations appear in the printed text and are provided in the HTML and PDF versions of this article on the journal's Web site (www.retinajournal.com). Retina: January 2021 - Volume 41 - Issue 1 - p 208-212 doi: 10.1097/IAE.0000000000002782 Buy SDC Metrics AbstractIn Brief Purpose: To compare retinal toxicity as measured by electroretinogram, ocular, and patient survival in retinoblastoma treated with intravitreal melphalan at two concentrations (25 vs. 30 µg). Methods: Single-center, retrospective analysis of retinoblastoma eyes receiving 25-µg or 30-µg intravitreal melphalan from September 2012 to January 2019. Ocular toxicity was measured by electroretinogram of evaluable injections in 449 injections in 136 eyes. A repeated-measures linear mixed model with a random intercept and slope was applied to account for repeated measures for each eye. Results: Average decline in electroretinogram after each additional injection was −4.9 µV (95% confidence interval −6.3 to −3.4); electroretinogram declined by −4.6 µV (95% confidence interval −7.0 to −2.2) after 25-µg injections and −5.2 µV (95% confidence interval −6.6 to −3.8) after 30-µg injections (P = 0.66). Injection at a new clock site hour was associated with a −3.91-µV lower average (95% confidence interval −7.8 to −0.04). Conclusion: Electroretinogram-measured toxicity in retinoblastoma eyes treated with intravitreal injections was not found to be different across 25-µg and 30-µg injections. There were no cases of extraocular extension or metastatic deaths in our patient population. We did not detect a difference in toxicity as measured by electroretinogram for retinoblastoma eyes treated with 25 µg versus 30 µg of intravitreal melphalan. Intravitreal melphalan injections at new sites on the eye were associated with increased electroretinogram-measured toxicity.