儿童及青少年局部晚期鼻咽癌诱导化疗方案的比较有效性：一项回顾性IPTW分析

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中文摘要

诱导化疗（IC）是局部晚期鼻咽癌（LA-NPC）的一线治疗。然而，关于不同IC方案在儿童患者中的疗效和安全性数据仍然匮乏，因为该群体常被排除在临床试验之外。这项回顾性研究分析了2006年至2022年间接受IC的儿童鼻咽癌患者（年龄≤21岁）。为平衡混杂因素，采用了逆概率治疗加权（IPTW）。在纳入的493例患者中，中位随访时间为69个月。调整前，紫杉烷联合铂类（TP）组的无远处转移生存期（DMFS）显著短于非TP组，后者接受TP联合5-氟尿嘧啶或卡培他滨（TPF/C）、铂类联合5-氟尿嘧啶（PF）或吉西他滨联合铂类（GP）（p=0.032）。IPTW调整后观察到相似结果。TP组与非TP组的总生存期和无进展生存期无显著差异。相反，非TP方案与更高的任何级别毒性发生率相关（92.0% vs. 98.4%；p=0.002）。亚组分析显示，基于TP的IC在年龄≤14岁、EBV-DNA≤4000拷贝/mL以及N3期疾病患者中与更差的DMFS相关。对于儿童LA-NPC，非TP IC方案相比TP方案与更优的DMFS相关，尽管毒性发生率更高。TP在年轻/高风险患者中的潜在劣势具有假设生成性，需要验证。

英文摘要

Induction chemotherapy (IC) is a first-line treatment for locally advanced nasopharyngeal carcinoma (LA-NPC). However, data on the efficacy and safety of different IC regimens in pediatric patients remain scarce, as this group is often excluded from clinical trials. This retrospective study analyzed pediatric NPC patients (age ≤ 21 years) who received IC between 2006 and 2022. To balance confounding factors, inverse probability of treatment weighting (IPTW) was applied. Among 493 included patients, the median follow-up was 69 months. Before adjustment, the taxane-plus-platinum (TP) group showed significantly shorter distant metastasis-free survival (DMFS) than the non-TP group, who received TP plus 5-fluorouracil or capecitabine (TPF/C), platium plus 5-fluorouracil (PF) or gemcitabine plus platium (GP), (p = 0.032). Similar results were observed after IPTW adjustment. And overall survival and progression-free survival did not differ significantly between TP group and non-TP group. Conversely, the non-TP regimen was associated with a higher incidence of any grade toxicities (92.0% vs. 98.4%; p = 0.002). Subgroup analysis revealed that TP-based IC was associated with worse DMFS in patients aged ≤ 14 years, those with EBV-DNA ≤ 4000 copies/mL, and those with N3 stage disease. For pediatric LA-NPC, a non-TP IC regimen was associated with superior DMFS compared to a TP regimen, despite a higher rate of toxicities. The potential disadvantage of TP in younger/high-risk patients is hypothesis-generating and requires validation.