PDF(Pubmed)
Abstract：
Neuroblastoma presents with two patterns of disease: locoregional or systemic. The poor prognostic risk factors of locoregional neuroblastoma (LR-NB) include age, MYCN or MDM2-CDK4 amplification, 11q, histology, diploidy with ALK or TERT mutations, and ATRX aberrations. 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.
摘要：
神经母细胞瘤表现为两种类型的疾病：局部或全身性。局部神经母细胞瘤(LR-NB)预后不良的危险因素包括年龄、MYCN或MDM2-CDK4扩增，11q,组织学,具有ALK或TERT突变的二倍体，和ATRX像差。抗GD2免疫疗法显著改善了高危（HR）NB的预后，对骨髓质微小残留病（MRD）大多有效。但对软组织疾病就不那么重要了。问题是添加抗GD2单克隆抗体（mAb）是否有益于仅由软组织复合的HR-NB患者。我们回顾了在SJD接受HR-NB治疗的31例患者，诊断时无骨髓质受累。所有肿瘤均具有HR-NB的分子遗传学特征。一线治疗后的结果显示25例（80.6%）患者达到CR。13名患者仍处于持续CR状态，中位随访3.9年。我们分析了在一线治疗中加入抗GD2免疫疗法是否具有任何预后意义。使用Cox模型的EFS分析显示HR为0.20，p=0.0054，一线用抗GD2免疫疗法治疗的患者复发风险降低80％。一线治疗后，EFS和OS的CR状态均无明显差异。总之,在MRD阶段加用抗GD2单克隆抗体治疗有助于预防复发,而复发无疑预示着患者的生存率较差.
