ArticleFrontiers in pediatrics2026
Efficacy and safety analysis of autologous hematopoietic stem cell transplantation for extraocular retinoblastoma.
Article in Frontiers in pediatrics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
This study aimed to assess the efficacy and safety of autologous hematopoietic stem cell transplantation (Auto-HSCT) in pediatric patients with extraocular retinoblastoma. We performed a retrospective analysis of clinical data from 12 children diagnosed with extraocular retinoblastoma at our institution between September 2018 and February 2023. A conditioning regimen consisting of Etoposide, Thiotepa, and Carboplatin was administered to all patients, with individualized adjustments based on patient-specific characteristics. Notably, one patient with trilateral retinoblastoma who underwent salvage transplantation received a Melphalan/Busulfan-based conditioning regimen. All 12 patients successfully underwent stem cell mobilization, apheresis, and auto-HSCT, with no transplant-related mortality reported. The median follow-up duration after high-dose chemotherapy and auto-HSCT was 27 months (range, 2-59 months). During the follow-up period, 8 patients (66.7%) achieved sustained disease-free survival (DFS), whereas 4 patients (33.3%) died. Among the deceased, one case was attributed to disease progression in a patient with trilateral retinoblastoma. Stratified analysis demonstrated that the DFS rate was 50% (2/4) in patients with central nervous system (CNS) metastasis, in contrast to a significantly higher DFS rate of 85.7% (6/7) in those without CNS involvement. In conclusion, high-dose chemotherapy combined with auto-HSCT is an effective therapeutic strategy for extraocular retinoblastoma in children, characterized by reversible adverse events and favorable tolerability. Patients with retinoblastoma complicated by CNS metastasis exhibit a poor prognosis. For children with extraocular retinoblastoma without CNS involvement, auto-HSCT serves as a viable treatment option with potential curative efficacy.
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