An Wen-Bin, Yang Wen-Yu
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin 300020, China.
Zhongguo Dang Dai Er Ke Za Zhi. 2025 Jul 15;27(7):792-801. doi: 10.7499/j.issn.1008-8830.2503021.
Pediatric chronic myeloid leukemia (CML) is more aggressive than adult CML, with unique molecular characteristics and a higher propensity for lymphoid blast crisis. The application of tyrosine kinase inhibitors (TKIs) has significantly improved the prognosis of pediatric CML. Based on international consensus and clinical experience, this article proposes standardized diagnosis and treatment recommendations for pediatric CML, covering initial therapy selection, efficacy evaluation, drug switching, and management of adverse effects. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is recommended only for patients with disease progression or failure of multiple lines of TKI therapy. For children newly diagnosed with CML in accelerated phase, high-dose imatinib or second-generation TKIs are recommended as first-line therapy. Those achieving optimal responses should continue maintenance therapy, while non-responders require switching to alternative TKIs and consider allo-HSCT. For blast-phase CML, induction therapy requires a combination of TKIs and chemotherapy, with allo-HSCT serving as the core curative intervention. This article highlights common but challenging problems (poor response, drug intolerance, and disease progression) in pediatric CML treatment using three typical cases, aiming to optimize treatment strategies. Furthermore, the goal of achieving treatment-free remission needs to be further addressed through multi-center clinical studies.
儿童慢性髓系白血病(CML)比成人CML更具侵袭性,具有独特的分子特征,且发生淋巴细胞急变的倾向更高。酪氨酸激酶抑制剂(TKIs)的应用显著改善了儿童CML的预后。基于国际共识和临床经验,本文提出了儿童CML的标准化诊断和治疗建议,涵盖初始治疗选择、疗效评估、换药以及不良反应管理。仅建议对疾病进展或多线TKI治疗失败的患者进行异基因造血干细胞移植(allo-HSCT)。对于新诊断为加速期CML的儿童,推荐高剂量伊马替尼或第二代TKIs作为一线治疗。达到最佳反应的患者应继续维持治疗,而无反应者则需要换用其他TKIs并考虑allo-HSCT。对于急变期CML,诱导治疗需要联合使用TKIs和化疗,allo-HSCT作为核心的治愈性干预措施。本文通过三个典型病例突出了儿童CML治疗中常见但具有挑战性的问题(反应不佳、药物不耐受和疾病进展),旨在优化治疗策略。此外,需要通过多中心临床研究进一步探讨实现无治疗缓解的目标。