ArticleFrontiers in oncology2026
Efficacy of high-dose chemotherapy combined with hematopoietic stem cell transplantation in advanced neuroblastoma.
Article in Frontiers in oncology, 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
Background: High-dose chemotherapy plus hematopoietic stem cell transplantation (HDC+HSCT) is widely used as consolidation for advanced-stage neuroblastoma, but real-world comparative evidence versus high-dose chemotherapy (HDC) alone remains limited. This study aimed to compare the efficacy and safety of HDC+HSCT versus HDC alone in pediatric patients with advanced-stage neuroblastoma. Methods: In this retrospective cohort study, pediatric patients with advanced-stage neuroblastoma treated between October 2017 and October 2022 were identified from institutional medical records. Patients were classified according to the consolidation strategy actually delivered: HDC alone (n=108) or HDC+HSCT (n=103). The primary outcomes were overall survival (OS) and event-free survival (EFS), both calculated from the start of consolidation therapy. Secondary outcomes included objective response rate (ORR) at the first post-consolidation assessment and treatment-related toxicities. Survival was evaluated using Kaplan-Meier analysis and Cox proportional hazards models. Results: The HDC+HSCT group showed a higher ORR than the HDC group (78.6% vs 64.8%, P = 0.026). OS estimates were also higher in the HDC+HSCT group, with a median OS of 34.7 versus 26.1 months (log-rank P = 0.016); the adjusted hazard ratio (HR) for OS was 0.62 (95% confidence interval [CI] 0.41-0.94, P = 0.024). EFS did not differ significantly between groups (median, 21.9 vs 19.9 months; log-rank P = 0.937; adjusted HR 0.98, 95% CI 0.70-1.36, P = 0.901). Grade ≥3 bacterial infection (35.0% vs 22.2%, P = 0.040), sepsis (11.7% vs 1.9%, P = 0.004), mucositis (31.1% vs 17.6%, P = 0.022), and renal toxicity (9.7% vs 1.9%, P = 0.014) were more frequent in the HDC+HSCT group. Conclusion: HDC+HSCT was associated with higher post-consolidation ORR and more favorable OS estimates than HDC alone, while EFS was similar between groups. It was also associated with more selected severe toxicities, underscoring the need for intensive supportive care. Prospective studies are needed to validate these findings.
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