To compare the efficacy and safety of high-dose chemotherapy plus hematopoietic stem cell transplantation (HDC+HSCT) versus high-dose chemotherapy (HDC) alone in pediatric patients with advanced-stage neuroblastoma, with a focus on both outcomes and safety.
Key Findings:
The HDC+HSCT group had a higher ORR (78.6% vs 64.8%, P = 0.026).
Median OS was longer in the HDC+HSCT group (34.7 months vs 26.1 months, log-rank P = 0.016; adjusted HR for OS was 0.62, 95% CI 0.41–0.94, P = 0.024).
EFS did not significantly differ 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).
Higher rates of grade ≥3 bacterial infection, sepsis, mucositis, and renal toxicity were observed in the HDC+HSCT group.
Interpretation:
HDC+HSCT is associated with improved post-consolidation ORR and OS compared to HDC alone, although EFS is similar. The increased severe toxicities highlight the need for intensive supportive care and careful patient monitoring.
Limitations:
Retrospective design may introduce selection bias, potentially affecting the validity of the findings.
Single-center study limits generalizability to broader populations.
Potential confounding factors not fully controlled may influence outcomes.
Conclusion:
HDC+HSCT offers better outcomes in terms of ORR and OS but comes with increased severe toxicities, necessitating further prospective studies to validate these findings and explore strategies to mitigate risks.
