Objective:Dinutuximab beta (DB) and naxitamab (NAXI) with GM-CSF are used for maintenance treatment of relapsed/refractory neuroblastoma. The objective of this study was to systematically assess comparative efficacy of the two therapies within their designated indications in accordance with established clinical guidelines.Methods:Relevant evidence was identified in systematic literature review. Individual patient data (IPD) from prospective clinical trials of DB were assessed and data on patients with disease in bone or bone marrow, as assessed in MRI, CT, mIBG or biopsy, with incomplete response to previous therapy were included. Patients with complete response, progressive disease and/or soft tissue disease were excluded. DB population was adjusted for sex, MYCN amplification, disease type (relapsed, refractory), and disease site (bone marrow and/or bone) to balance aggregated characteristics of NAXI population. More characteristics were included in sensitivity analyses, including DB treatment without interleukin-2, as currently recommended. Overall response rate (ORR) was assessed as best response.Results:Aggregated data for NAXI from Study 201 (n= 52) and Study 230 (n= 38) and IPD from DB studies (APN311-202, APN311-304,c= 77) met the inclusion criteria. Compared to NAXI, DB significantly extended progression-free survival (PFS): hazard ratio, DB vs. NAXI of 0.47 (95% CI: 0.26 to 0.87,p= 0.015). ORR was 60.1% (95% CI: 48.5% to 71.6%) for DB vs. 43.3% (33.1% to 53.6%) for NAXI (ORR odds ratio, DB vs. NAXI was 1.97, 95% CI: 1.02 to 3.80,p= 0.044). Sensitivity analyses and unadjusted comparisons supported the results.Conclusion:In the indirect comparison, dinutuximab beta significantly extended PFS and increased ORR compared to naxitamab.
目的：地努图希单抗β（DB）与那昔妥单抗（NAXI）联合粒细胞-巨噬细胞集落刺激因子（GM-CSF）被用于复发/难治性神经母细胞瘤的维持治疗。本研究旨在依据现行临床指南，系统评估这两种疗法在其特定适应症范围内的相对疗效。 方法：通过系统性文献检索确定相关证据。对DB前瞻性临床试验中的个体患者数据（IPD）进行评估，纳入经MRI、CT、mIBG或活检证实存在骨或骨髓病灶、且对既往治疗应答不完全的患者。完全缓解、疾病进展和/或软组织病灶患者被排除。对DB人群的性别、MYCN扩增状态、疾病类型（复发/难治）及病灶部位（骨髓和/或骨）进行校正，以平衡NAXI人群的汇总特征。敏感性分析纳入更多特征，包括当前推荐的不联合白细胞介素-2的DB治疗方案。最佳应答被评估为总体缓解率（ORR）。 结果：来自NAXI研究201（n=52）和研究230（n=38）的汇总数据，以及DB研究（APN311-202、APN311-304，n=77）的IPD符合纳入标准。与NAXI相比，DB显著延长无进展生存期（PFS）：风险比（DB vs. NAXI）为0.47（95% CI：0.26-0.87，p=0.015）。DB的ORR为60.1%（95% CI：48.5%-71.6%），NAXI为43.3%（33.1%-53.6%）（ORR比值比DB vs. NAXI为1.97，95% CI：1.02-3.80，p=0.044）。敏感性分析和未校正比较均支持该结果。 结论：在间接比较中，与那昔妥单抗相比，地努图希单抗β能显著延长PFS并提高ORR。
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