采用包括抗GD2免疫疗法在内的多模式治疗的高危神经母细胞瘤患者的长期结局和生活质量：一项回顾性队列研究
Long-Term Outcomes and Quality of Life of High-Risk Neuroblastoma Patients Treated with a Multimodal Treatment Including Anti-GD2 Immunotherapy: A Retrospective Cohort Study.
作者信息
Flaadt Tim, Rehm Jonas, Simon Thorsten, Hero Barbara, Ladenstein Ruth L, Lode Holger N, Grabow Desiree, Nolte Sandra, Crazzolara Roman, Greil Johann, Ebinger Martin, Abele Michael, Holzer Ursula, Döring Michaela, Schulte Johannes H, Bader Peter, Schlegel Paul-Gerhardt, Eyrich Matthias, Lang Peter, Klingebiel Thomas, Handgretinger Rupert
机构信息
Department of Haematology and Oncology, University Children's Hospital, Eberhard Karls University Tuebingen, 72076 Tuebingen, Germany.
Department of Paediatric Oncology and Haematology, University Hospital, University of Cologne, 50937 Cologne, Germany.
出版信息
Cancers (Basel). 2025 Jan 5;17(1):149. doi: 10.3390/cancers17010149.
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
将抗GD2抗体（如ch14.18/SP2/0）纳入高危神经母细胞瘤（HR-NB）患者的多模式治疗中，已改善了他们的治疗结果。由于评估这种治疗的长期结果、长期后遗症以及健康相关生活质量（HRQoL）的研究有限，这项回顾性分析旨在探索这些方面。
患者与方法
1991年至2002年间，65名儿童因原发性HR-NB接受了包括ch14.18/SP2/0的多模式治疗。所有患者均根据NB90/NB97试验接受化疗，51名接受了大剂量化疗，且所有患者均接受了ch14.18/SP2/0治疗。我们分析了长期后遗症和HRQoL（欧洲癌症研究与治疗组织核心生活质量问卷QLQ-C30），并评估了总生存率和无事件生存率（OS/EFS）。
结果
对25名幸存者进行了HRQoL和长期影响评估。所有患者均报告有长期后遗症，包括16/25（64%）的患者出现耳毒性，6/25（24%）的患者出现心脏毒性，19/25（76%）的患者出现内分泌毒性。20%的女性患者报告有慢性腹泻。7名患者患上自身免疫性疾病。多个量表的HRQoL评分均优于匹配的德国普通人群。25年总生存率和无事件生存率分别为50.8%（95%置信区间：31-55）和43%（30.1-55.3），有33名（50.8%）长期幸存者。32名患者死亡：28名（43.1%）死于疾病进展/复发，4名（6.2%）死于继发性肿瘤。
结论
包括ch14.18/SP2/0的多模式治疗可使HR-NB患者实现长期生存，相当一部分幸存者报告的HRQoL优于普通人群。所有患者均报告有主要归因于化疗和放疗的长期副作用。自身免疫性疾病和持续性腹泻的相对高患病率需要对接受抗GD2抗体治疗的个体进行更多纵向研究。
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