Objective: To explore the clinical characteristics, molecular characteristics, treatment and prognosis of pediatric Philadelphia chromosome-like acute lymphoblastic leukemia (Ph-like ALL) with a therapeutic target.
Methods: A total of 27 patients of Ph-like ALL with targeted drug target were initially diagnosed in Children's Hospital of Soochow University from December 2017 to June 2021. The data of age, gender, white blood cell (WBC) count at initial diagnosis, genetic characteristics, molecular biological changes, chemotherapy regimen, different targeted drugs were given, and minimal residual disease (MRD) on day 19, MRD on day 46, whether hematopoietic stem cell transplantation (HSCT) were retrospective analyed, and the clinical characteristics and treatment effect were summarized. Survival analysis was performed by Kaplan-Meier method.
Results: The intensity of chemotherapy was adjusted according to the MRD level during induced remission therapy in 27 patients, 10 patients were treated with targeted drugs during treatment, and 3 patients were bridged with HSCT, 1 patient died and 2 patients survived. Among the 24 patients who did not receive HSCT, 1 patient developed relapse, and achieved complete remission (CR) after treatment with chimeric antigen receptors T cells (CAR-T). The 3-year overall survival, 3-year relapse-free survival and 3-year event-free survival rate of 27 patients were (95.5±4.4)%, (95.0±4.9)% and (90.7±6.3)% respectively.
Conclusion: Risk stratification chemotherapy based on MRD monitoring can improve the prognosis of Ph-like ALL in children, combined with targeted drugs can achieve complete remission as soon as possible in children whose chemotherapy response is poor, and sequential CAR-T and HSCT can significantly improve the therapeutic effect of Ph-like ALL in children whose MRD is continuously positive during induced remission therapy.
题目: 儿童费城染色体样急性淋巴细胞白血病的临床分析.
目的: 探讨具有治疗靶点的儿童费城染色体样急性淋巴细胞白血病（Ph-like ALL）的临床特点、分子学特征、治疗及预后。.
方法: 2017年12月至2021年6月在苏州大学附属儿童医院初诊且具备靶向药物治疗靶点的儿童Ph-like ALL共27例，回顾性分析患儿年龄、性别、初诊时白细胞计数、遗传学特征、分子生物学改变、化疗方案、给予不同靶向药物、d 19微小残留病（MRD）、d 46 MRD、是否行造血干细胞移植（HSCT）等资料，归纳总结患儿的临床特征及治疗效果。采用Kaplan-Meier方法进行生存分析。.
结果: 27例患儿均根据诱导缓解治疗过程中MRD水平调整化疗强度，10例在治疗过程中加用靶向药，3例患儿桥接HSCT，其中1例死亡，2例存活。24例未行HSCT患儿中，1例患儿出现复发，采用嵌合抗原受体T细胞（CAR-T）治疗后达完全缓解（CR）。27例患儿3年总生存率为（95.5±4.4）%，3年无复发生存率为（95.0±4.9）%，3年无事件生存率为（90.7±6.3）%。.
结论: 基于MRD监测的危险度分层化疗可改善Ph-like ALL患儿预后，对化疗效果欠佳的患儿联合靶向药可尽快完全缓解，诱导缓解治疗过程中MRD持续阳性的Ph-like ALL患儿序贯CAR-T和HSCT能显著提高治疗效果。.
Keywords: CAR-T; targeted drugs; Philadelphia chromosome-like acute lymphoblastic leukemia; children; hematopoietic stem cell transplantation.