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Case report demonstrates safety and efficacy of intra-arterial melphalan in neonate with advanced retinoblastoma, suggesting potential for broader application.
A bstract This case report describes the administration of intra-arterial chemotherapy (IAC) with melphalan in a 3-week-old neonate with bilateral Group D retinoblastoma (RB), reported from a neurosurgery center in Uzbekistan. IAC is generally avoided in infants under 6 months of age due to technical challenges, and published experience in neonates under 1 month of age remains limited. The infant presented with bilateral leukocoria and was diagnosed through ophthalmologic and ultrasonographic assessments. Three cycles of IAC were administered at 21-day intervals with weight-adjusted melphalan dosing. The treatment was well tolerated, with no procedural complications. Tumor regression and stabilization were achieved across all cycles, supporting the potential of IAC as a globe-salvaging approach in neonates with advanced intraocular RB. IAC is a well-established globe-salvaging approach for advanced intraocular RB. Its use in neonates under 1 month of age is rare and technically demanding. This case contributes to the limited published experience in this age group; highlights the feasibility, safety, and efficacy of early IAC intervention; and offers valuable clinical insights for managing similar cases in resource-limited settings.
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Mahmudov et al. (2026) studied this question.
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