分子靶向和免疫治疗在儿童恶性实体瘤中的应用进展  

Advance of molecular targeted agents and immunotherapy applied to children with malignant solid tumors

作　　者：蒋莎义[1] 吕志宝[1] JIANG Shayi;LYU Zhibao(Department of Hematology and Oncology,Children's Hospital of Shanghai,Shanghai 200062,China)

机构地区：[1]上海市儿童医院血液肿瘤科,上海200062

出　　处：《上海医药》2022年第S02期214-223,共10页Shanghai Medical & Pharmaceutical Journal

摘　　要：目的:深入了解小分子靶向药物和免疫治疗在儿童恶性实体瘤中的应用进展。方法:对获批用于儿童恶性实体瘤的分子靶向和免疫治疗药物的儿科临床研究文献进行综述。结果:分别有近10种小分子靶向药物和免疫治疗药物用于儿童恶性实体瘤临床,主要治疗复发难治或/和手术难以切除的病例。Ⅰ期临床均显示出良好的耐受性。某些肿瘤完全或部分客观反应,如伴ALK异常的炎性肌成纤维细胞瘤和间变大淋巴瘤对克唑替尼,TRK融合肿瘤对拉罗替尼,都有可观反应。在免疫治疗药物中疗效显著的是GD2单克隆抗体,提高了高危神经母细胞瘤患儿生存率20%左右。结论:小分子靶向和免疫治疗药物是目前儿童复发难治恶性实体瘤的治疗策略,进一步提高了这类患儿的生存率,也是探索新疗法的研究方向。Objective:To further understand the advance of small molecule targeted drugs and immunotherapy applying in children with malignant solid tumors.Methods:To review the research literature on the application of molecular targeted and immunotherapeutic drugs approved for children with malignant solid tumors.Results:There are nearly 10 small molecule targeted drugs and immunotherapeutic drugs used in the clinical treatment of children’s malignant solid tumors.At present,it is mainly used for patients with recurrence and refractory tumors and/or difficult to resect by surgery.PhaseⅠclinical trials show good tolerance.Some tumors have complete or partial objective response.For example,inflammatory myofibroblastoma and anaplastic lymphoma with abnormal ALK have objective response to crizotinib and TRK fusion tumor to larotrectinib.Among immunotherapeutic drugs,GD2 monoclonal antibody is an outstanding one,improving the survival rate of children with high-risk neuroblastoma by about 20%.Conclusion:Small molecule targeted drugs and immunotherapies are currently the treatment strategies for children with relapsed and refractory malignant solid tumors,which further improve the survival rate of such children and are also the exploration direction of new therapies.

关 键 词：分子靶向药物 免疫治疗 恶性实体肿瘤 儿童 

分 类 号：R733.7[医药卫生—肿瘤]