Intravitreal Melphalan versus Topotecan for Vitreous Seeds in Retinoblastoma: A Comparative Study of 64 Asian Indian Eyes.
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PURPOSE: To compare the outcomes of intravitreal melphalan (IVit-M) versus intravitreal topotecan (IVit-T) for vitreous seeds (VS) in retinoblastoma (RB). DESIGN: Retrospective interventional study. PARTICIPANTS: Patients of RB with VS receiving intravitreal chemotherapy (IVit-C) between December 2012 and December 2022, at a single quaternary ocular oncology referral center. INTERVENTION: Intravitreal melphalan injection of 25 μg/0.1 mL in 25 eyes and IVit-T of 30 μg/0.15 mL in 39 eyes. MAIN OUTCOME MEASURES: Resolution of VS, globe salvage, IVit-C-related complications. RESULTS: The mean age at presentation was 28 months (median, 24 months; range, 4-144 months) for the IVit-M group and 25 months (median, 24 months; range, 2-60 months) for the IVit-T group. At the time of initiation of intravitreal injection, the VS belonged to type 1 (1 [4%] vs. 1 [3%]), type 2 (9 [36%] vs. 14 [36%]), type 3 (8 [32%] vs. 18 [46%]), or a combination of these (7 [28%] vs. 6 [15%]) in IVit-M and IVit-T groups, respectively. Complete resolution of VS after IVit-C was seen in 22 (92%) and 28 (72%) eyes (P = 0.069) in IVit-M and IVit-T groups, respectively. At a mean follow-up period of 44 months after the first IVit-C injection in the IVit-M group and 19 months in the IVit-T group, globe salvage was higher in the IVit-T group (77%) than in the IVit-M group (60%, P = 0.148), respectively. Posterior segment complications were significantly higher in the IVit-M group than in the IVit-T group (P < 0.001). These included retinal pigment epithelial atrophy (P < 0.001), optic atrophy (P < 0.001), vascular attenuation (P < 0.001), and retinal/subretinal hemorrhages (P = 0.004). CONCLUSIONS: Intravitreal topotecan is as efficacious as IVit-M for controlling VS in RB, with a better safety profile in pigmented Asian Indian eyes. FINANCIAL DISCLOSURE(S): Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.