目的 研究高危神经母细胞瘤（neuroblastoma, NB）患儿诱导治疗反应与预后的相关性，分析其影响因素。 方法 回顾性分析2019年1月—2023年12月期间上海市儿童医院诊治的55例高危NB患儿的资料，根据国际NB反应标准评估诱导反应，分为诱导治疗反应良好组（完全缓解或非常好的部分缓解）及诱导治疗反应不良组（部分缓解+疾病进展+混合性反应+无反应），分析两组患儿的临床生物学特点、治疗情况及影响预后的相关因素。 结果 55例高危NB患儿中，男性29例，女性26例，中位发病年龄为39个月。随访至2024年12月31日，3年总生存（overall survival rate, OS）率及无事件生存（event-free survival, EFS）率分别为（83.8±5.3）%、（47.0±10.3）%。初诊时神经元特异性烯醇化酶水平、诱导治疗反应、放疗、复发是影响患儿EFS率及OS率的预后因素（P<0.05）。诱导治疗反应良好组及不良组的3年OS率分别为（83.5±7.4）%、（66.7±13.6）%（P=0.012），3年EFS率分别为（62.8±10.4）%、（27.8±14.8）%（P<0.001）。疾病初诊时存在颅内转移与诱导治疗反应不良相关（P=0.033）；血小板计数≥400×109/L与更好的诱导治疗反应相关（P=0.002）。 结论 诱导治疗反应显著影响高危NB的预后。疾病初诊时无颅内转移及血小板计数≥400×109/L与诱导治疗反应良好相关。
Objective To investigate the correlation between induction therapy response and prognosis in children with high-risk neuroblastoma, and to analyze factors associated with the induction therapy response. Methods Data of 55 children with high-risk neuroblastoma diagnosed and treated at Shanghai Children's Hospital from January 2019 to December 2023 were retrospectively reviewed. Induction response was assessed according to the International Neuroblastoma Response Criteria and patients were categorized into a good-response group (complete response or very good partial response) and a poor-response group (partial response, progressive disease, mixed response, or no response). Clinical and biological characteristics, treatments, and prognostic factors were analyzed. Results Among the 55 children, 29 were male and 26 were female; the median age at onset was 39 months. Follow-up was performed until December 31, 2024. The 3-year overall survival (OS) and event-free survival (EFS) rates were (83.8±5.3)% and (47.0±10.3)%, respectively. Neuron-specific enolase level at initial diagnosis, induction therapy response, radiotherapy, and recurrence were prognostic factors for EFS and OS (P<0.05). The 3-year OS was (83.5±7.4)% in the good-response group and (66.7±13.6)% in the poor-response group (P=0.012), while the 3-year EFS was (62.8±10.4)% and (27.8±14.8)%, respectively (P<0.001). Intracranial metastasis at initial diagnosis was associated with a poor induction response (P=0.033). A platelet count ≥400×109/L was associated with a better induction response (P=0.002). Conclusions Induction therapy response is a significant prognostic factor in high-risk neuroblastoma. Absence of intracranial metastasis and a platelet count ≥400×109/L at initial diagnosis are associated with a favorable induction therapy response.
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