大剂量化疗联合造血干细胞移植治疗晚期神经母细胞瘤的疗效
Efficacy of high-dose chemotherapy combined with hematopoietic stem cell transplantation in advanced neuroblastoma.
摘要(中文译文)
大剂量化疗联合造血干细胞移植（HDC+HSCT）被广泛用作晚期神经母细胞瘤的巩固治疗，但其与单纯大剂量化疗（HDC）相比的真实世界对比证据仍然有限。本研究旨在比较HDC+HSCT与单纯HDC在晚期神经母细胞瘤患儿中的疗效与安全性。在这项回顾性队列研究中，通过机构病历资料筛选出2017年10月至2022年10月期间接受治疗的晚期神经母细胞瘤患儿。患者根据实际接受的巩固治疗策略分为两组：单纯HDC组（n=108）和HDC+HSCT组（n=103）。主要结局为总生存期（OS）和无事件生存期（EFS），均从巩固治疗开始计算。次要结局包括巩固治疗后首次评估时的客观缓解率（ORR）以及治疗相关毒性。生存分析采用Kaplan-Meier法和Cox比例风险模型。HDC+HSCT组的ORR高于HDC组（78.6% vs 64.8%，P = 0.026）。HDC+HSCT组的OS估计值也更高，中位OS分别为34.7个月和26.1个月（log-rank P = 0.016）；OS的校正风险比（HR）为0.62（95%置信区间[CI] 0.41-0.94，P = 0.024）。两组间EFS无显著差异（中位EFS分别为21.9个月和19.9个月；log-rank P = 0.937；校正HR 0.98，95% CI 0.70-1.36，P = 0.901）。HDC+HSCT组中≥3级细菌感染（35.0% vs 22.2%，P = 0.040）、脓毒症（11.7% vs 1.9%，P = 0.004）、黏膜炎（31.1% vs 17.6%，P = 0.022）和肾毒性（9.7% vs 1.9%，P = 0.014）的发生率更高。与单纯HDC相比，HDC+HSCT与更高的巩固治疗后ORR及更有利的OS估计值相关，而两组间EFS相似。HDC+HSCT还与更多特定严重毒性相关，凸显了加强支持治疗的必要性。尚需前瞻性研究来验证这些发现。
Abstract
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. 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. 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. 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.
如何引用
AMA
Lin Wang, Jian Gao, Boshen Shu, Shufeng Zhang, Yinghao Sun, Xunqian Wang, et al. Efficacy of high-dose chemotherapy combined with hematopoietic stem cell transplantation in advanced neuroblastoma.. Frontiers in oncology. 2026; doi:10.3389/fonc.2026.1819260.
APA
Lin Wang, Jian Gao, Boshen Shu, Shufeng Zhang, Yinghao Sun, Xunqian Wang, et al (2026). Efficacy of high-dose chemotherapy combined with hematopoietic stem cell transplantation in advanced neuroblastoma.. Frontiers in oncology. https://doi.org/10.3389/fonc.2026.1819260
GB/T 7714
Lin Wang, Jian Gao, Boshen Shu, Shufeng Zhang, Yinghao Sun, Xunqian Wang, et al. Efficacy of high-dose chemotherapy combined with hematopoietic stem cell transplantation in advanced neuroblastoma.[J]. Frontiers in oncology, 2026 doi:10.3389/fonc.2026.1819260.
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