Efficacy and Safety of Anti-GD2 Immunotherapy with Dinutuximab Beta in the Treatment of Relapsed/Refractory High-Risk Neuroblastoma.

IF 4.4 3区医学 Q2 ONCOLOGY

Targeted Oncology Pub Date : 2025-06-03 DOI:10.1007/s11523-025-01155-3

Aleksandra Wieczorek, Katarzyna Śladowska, Holger N Lode

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引用次数: 0

Abstract

Background: High-risk neuroblastoma (HR-NB) is associated with a poor prognosis. Standard first-line maintenance therapy with anti-disialoganglioside 2 (GD2) monoclonal antibodies, such as dinutuximab beta, has improved survival rates; however, approximately 50% of patients experience relapse and ~15% have disease that is refractory to induction therapy.

Objective: This systematic literature review aimed to evaluate response rates, survival outcomes, and safety in patients with relapsed or refractory (R/R) HR-NB receiving dinutuximab beta as maintenance therapy.

Patients and methods: We searched the PubMed, Embase, and Cochrane Library databases and regulatory reports from inception to 1 September 2024, and included studies of patients with R/R HR-NB in which dinutuximab beta (± isotretinoin) was used as maintenance therapy and that reported objective response or survival rates. Studies of dinutuximab beta plus chemotherapy combinations were excluded.

Results: We included nine publications/reports representing seven studies and 442 patients receiving dinutuximab beta. Across studies, the mean age was 5.1-6.4 years, and most patients were male. Reporting of response varied across studies between best response and end-of-treatment response. Best response rates with dinutuximab beta were 28.6-54.8%. All studies reported overall survival (OS), but follow-up times varied. Where reported, 3-year OS rates for patients receiving dinutuximab beta were 54-86% overall, with better OS rates reported for refractory than relapsed patients. Adverse events were frequent but manageable.

Conclusions: Maintenance therapy for patients with R/R HR-NB with dinutuximab beta as monotherapy or in combination with isotretinoin demonstrated efficacy and acceptable safety. Further studies are needed in patients previously treated with anti-GD2 therapies to evaluate efficacy and impact on target antigens.

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抗gd2免疫疗法联合迪努妥昔单抗治疗复发/难治性高危神经母细胞瘤的疗效和安全性

背景：高风险神经母细胞瘤（HR-NB）与不良预后相关。标准的一线维持治疗使用抗双胞苷脂苷2 （GD2）单克隆抗体，如迪努妥昔单抗β，提高了生存率；然而，约50%的患者复发，约15%的患者对诱导治疗难治性。目的：本系统文献综述旨在评估复发或难治性（R/R） HR-NB患者接受迪努妥昔单抗作为维持治疗的反应率、生存结局和安全性。患者和方法：我们检索了PubMed、Embase和Cochrane图书馆数据库以及从成立到2024年9月1日的监管报告，并纳入了使用迪乌妥昔单抗（±异维甲酸）作为维持治疗的R/R HR-NB患者的研究，这些研究报告了客观反应或生存率。排除了迪努妥昔单抗+化疗联合的研究。结果：我们纳入了9篇出版物/报告，代表了7项研究和442名接受迪努妥昔单抗治疗的患者。在所有研究中，平均年龄为5.1-6.4岁，大多数患者为男性。不同研究的应答报告在最佳应答和治疗结束应答之间存在差异。迪努妥昔单抗的最佳有效率为28.6-54.8%。所有研究都报告了总生存期（OS），但随访时间各不相同。据报道，接受迪努妥昔单抗治疗的患者的3年总生存率为54-86%，难治性患者的总生存率高于复发患者。不良事件频繁但可控。结论：替努妥昔单抗或联合异维甲酸对R/R HR-NB患者的维持治疗具有良好的疗效和可接受的安全性。需要进一步研究先前接受过抗gd2治疗的患者，以评估其疗效和对目标抗原的影响。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Targeted Oncology 医学-肿瘤学

CiteScore

8.40

自引率

3.70%

发文量

审稿时长

>12 weeks

期刊介绍： Targeted Oncology addresses physicians and scientists committed to oncology and cancer research by providing a programme of articles on molecularly targeted pharmacotherapy in oncology. The journal includes: Original Research Articles on all aspects of molecularly targeted agents for the treatment of cancer, including immune checkpoint inhibitors and related approaches. Comprehensive narrative Review Articles and shorter Leading Articles discussing relevant clinically established as well as emerging agents and pathways. Current Opinion articles that place interesting areas in perspective. Therapy in Practice articles that provide a guide to the optimum management of a condition and highlight practical, clinically relevant considerations and recommendations. Systematic Reviews that use explicit, systematic methods as outlined by the PRISMA statement. Adis Drug Reviews of the properties and place in therapy of both newer and established targeted drugs in oncology.