Background: The incorporation of anti-GD2 antibodies such as ch14.18/SP2/0 into the multimodal treatment of high-risk neuroblastoma (HR-NB) patients has improved their outcomes. As studies assessing the long-term outcomes, long-term sequelae, and health-related quality of life (HRQoL) of this treatment are limited, this retrospective analysis aimed to explore these. Patients and Methods: Between 1991 and 2002, 65 children received a multimodal treatment, including ch14.18/SP2/0, for primary HR-NB. All received chemotherapy according to the NB90/NB97 trial, 51 received high-dose chemotherapy, and all received ch14.18/SP2/0 treatment. We analyzed the long-term sequelae and HRQoL (EORTC QLQ-C30), and evaluated overall and event-free survival (OS/EFS). Results: Twenty-five survivors were evaluated for HRQoL and long-term effects. All reported long-term sequelae, including ototoxicity in 16/25 (64%), cardiac toxicity in 6/25 (24%), and endocrine toxicity in 19/25 (76%) patients. Chronic diarrhea was reported in 20% of female patients. Seven patients developed autoimmune diseases. HRQoL scores were better across multiple scales than those of the matched German general population. Twenty-five-year OS and EFS were 50.8% (95% confidence interval: 31–55) and 43% (30.1–55.3), with 33 (50.8%) long-term survivors. Thirty-two patients died: 28 (43.1%) because of progression/relapse and 4 (6.2%) because of secondary neoplasms. Conclusions: Multimodal treatment, including ch14.18/SP2/0, can achieve long-term survival in HR-NB patients, with a substantial proportion of survivors reporting better HRQoL compared to the general population. All patients reported long-term side effects mostly attributable to chemotherapy and radiotherapy. The relatively high prevalence of autoimmune diseases and persistent diarrhea warrants additional longitudinal research on individuals treated with anti-GD2 antibodies.
背景：在高危神经母细胞瘤（HR-NB）患者的多模式治疗方案中加入ch14.18/SP2/0等抗GD2抗体已改善其预后。由于评估该治疗方案长期结局、远期后遗症及健康相关生活质量（HRQoL）的研究有限，本回顾性分析旨在探讨这些方面。患者与方法：1991年至2002年间，65例原发性HR-NB患儿接受了包含ch14.18/SP2/0的多模式治疗。所有患者均按NB90/NB97试验方案接受化疗，51例接受高剂量化疗，且均接受ch14.18/SP2/0治疗。我们分析了远期后遗症和HRQoL（采用EORTC QLQ-C30量表），并评估总生存期和无事件生存期（OS/EFS）。结果：对25例存活者进行了HRQoL和远期影响评估。所有患者均报告存在远期后遗症，其中16/25例（64%）出现耳毒性，6/25例（24%）出现心脏毒性，19/25例（76%）出现内分泌毒性。20%的女性患者报告慢性腹泻。7例患者出现自身免疫性疾病。在多个维度上，HRQoL评分均优于匹配的德国普通人群。25年OS和EFS分别为50.8%（95%置信区间：31-55）和43%（30.1-55.3），其中33例（50.8%）为长期存活者。32例患者死亡：28例（43.1%）因疾病进展/复发，4例（6.2%）因继发性肿瘤。结论：包含ch14.18/SP2/0的多模式治疗可使HR-NB患者获得长期生存，且相当比例存活者的HRQoL优于普通人群。所有患者均报告远期副作用，主要归因于化疗和放疗。自身免疫性疾病和持续性腹泻的较高发生率提示，需对接受抗GD2抗体治疗的个体开展更多纵向研究。
肿瘤(癌症)患者之家