Neuroblastoma presents with two patterns of disease: locoregional or systemic. The poor prognostic risk factors of locoregional neuroblastoma (LR-NB) include age,MYCNorMDM2-CDK4amplification, 11q, histology, diploidy withALKorTERTmutations, andATRXaberrations. Anti-GD2 immunotherapy has significantly improved the outcome of high-risk (HR) NB and is mostly effective against osteomedullary minimal residual disease (MRD), but less so against soft tissue disease. The question is whether adding anti-GD2 monoclonal antibodies (mAbs) benefits patients with HR-NB compounded by only soft tissue. We reviewed 31 patients treated at SJD for HR-NB with no osteomedullary involvement at diagnosis. All tumors had molecular genetic features of HR-NB. The outcome after first-line treatment showed 25 (80.6%) patients achieving CR. Thirteen patients remain in continued CR, median follow-up 3.9 years. We analyzed whether adding anti-GD2 immunotherapy to first-line treatment had any prognostic significance. The EFS analysis using Cox models showed a HR of 0.20,p= 0.0054, and an 80% decrease in the risk of relapse in patients treated with anti-GD2 immunotherapy in the first line. Neither EFS nor OS were significantly different by CR status after first-line treatment. In conclusion, adding treatment with anti-GD2 mAbs at the stage of MRD helps prevent relapse that unequivocally portends poor survival.
神经母细胞瘤呈现两种疾病模式：局部区域性或全身性。局部区域神经母细胞瘤（LR-NB）的不良预后风险因素包括年龄、MYCN或MDM2-CDK4扩增、11q异常、组织学特征、伴有ALK或TERT突变的二倍体以及ATRX畸变。抗GD2免疫疗法显著改善了高危（HR）神经母细胞瘤的预后，主要对骨髓微小残留病（MRD）有效，但对软组织病灶效果较弱。问题在于，对于仅伴有软组织病灶的高危神经母细胞瘤患者，加用抗GD2单克隆抗体（mAbs）是否具有临床获益。我们回顾了圣胡安·德·迪奥斯医院收治的31例确诊时无骨髓侵犯的高危神经母细胞瘤患者。所有肿瘤均具有高危神经母细胞瘤的分子遗传学特征。一线治疗后结果显示，25例（80.6%）患者达到完全缓解。13例患者持续保持完全缓解状态，中位随访时间3.9年。我们分析了在一线治疗基础上加用抗GD2免疫疗法是否具有预后意义。采用Cox模型进行的无事件生存期分析显示，一线接受抗GD2免疫治疗的患者复发风险降低80%（风险比0.20，p=0.0054）。无论一线治疗后是否达到完全缓解状态，无事件生存期和总生存期均无显著差异。综上所述，在微小残留病阶段加用抗GD2单克隆抗体治疗有助于预防复发，而复发明确预示着不良生存结局。
肿瘤(癌症)患者之家