Autologous hematopoietic stem cell transplantation improves disease-free survival in pediatric extraocular retinoblastoma patients

Photo by Nick Design / Unsplash

Frontiers in Medicine Published April 29, 2026 Medically reviewed April 29, 2026 DOI ↗ By Dr. Sofia Müller, MD · Lifespan & Whole-Person Care

Key Takeaway

Consider autologous hematopoietic stem cell transplantation for pediatric extraocular retinoblastoma without CNS involvement.

This retrospective analysis evaluated 12 pediatric patients with extraocular retinoblastoma or trilateral retinoblastoma at a single institution. The intervention involved autologous hematopoietic stem cell transplantation using a conditioning regimen of Etoposide, Thiotepa, and Carboplatin. One patient received Melphalan and Busulfan instead. The median follow-up duration was 27 months, with a range from 2 to 59 months.

Primary analysis showed that 8 patients (66.7%) achieved sustained disease-free survival, whereas 4 patients (33.3%) died. Among patients with central nervous system metastasis, disease-free survival was 50% (2 of 4). In patients without central nervous system involvement, disease-free survival was 85.7% (6 of 7).

Safety data indicated favorable tolerability with reversible adverse events. One serious adverse event was attributed to disease progression in a patient with trilateral retinoblastoma. The study did not report discontinuations or p-values. Practice relevance notes that this approach serves as a viable treatment option for children with extraocular retinoblastoma without central nervous system involvement.

Study Details

Study typeCohort

EvidenceLevel 3

PublishedApr 2026

View Original 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.