Jiang Chiyi, Zhao Wen, Qin Maoquan, Jin Mei, Chang Lungji, Ma Xiaoli
Beijing Key Laboratory of Pediatric Hematology Oncology, National Discipline of Pediatrics, Ministry of Education, MOE Key Laboratory of Major Diseases in Children, Hematology Oncology Center, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing.
Geno-immune Medical Institute, Shenzhen, China.
Medicine (Baltimore). 2019 Oct;98(43):e17572. doi: 10.1097/MD.0000000000017572.
Rhabdomyosarcoma (RMS) is a common soft tissue sarcoma in children with high malignancy. The prognosis of refractory recurrent RMS is extremely poor, and the 5-year survival rate is less than 20%.
We reported a 2-year-old male patient with RMS who underwent 3 operations and 2 recurrences while being treated with regular multidisciplinary therapy.
A diagnosis of embryonal rhabdomyosarcoma with primary bladder (IIIa, TNM stage 2, and medium risk group) was made.
After repeated recurrence, the patient was treated with chimeric antigen receptor T (CAR-T) cells, which had a safety mechanism and specifically bound the CD56 antigen in the fourth generation.
The process of CAR-T cell transfusion was smooth, and there were no significant cytokine release syndrome manifestations after reinfusion. The patient was in complete remission at last follow-up visit after 3.5 years.
CD56-CAR-T cell therapy is a safe and effective approach and may be an option for children with solid tumors who are nonresponsive to conventional radiotherapy and chemotherapy, or are unsuitable for hematopoietic stem cell transplantation.
横纹肌肉瘤（RMS）是儿童常见的高度恶性软组织肉瘤。难治性复发性RMS的预后极差，5年生存率低于20%。
我们报告了一名2岁男性RMS患者，在接受常规多学科治疗期间接受了3次手术和2次复发。
诊断为原发性膀胱胚胎性横纹肌肉瘤（IIIa，TNM分期2期，中度风险组）。
反复复发后，患者接受了嵌合抗原受体T（CAR-T）细胞治疗，该细胞具有安全机制且在第四代中特异性结合CD56抗原。
CAR-T细胞输注过程顺利，再输注后无明显细胞因子释放综合征表现。在最后一次随访时，即3.5年后，患者完全缓解。
CD56-CAR-T细胞疗法是一种安全有效的方法，对于对传统放疗和化疗无反应或不适合造血干细胞移植的实体瘤儿童可能是一种选择。
