慢性粒细胞白血病治疗进展研究

Research Progress in the Treatment of Chronic Myeloid Leukemia

摘要: 目的：系统梳理慢性粒细胞白血病(Chronic Myeloid Leukemia, CML)治疗的最新进展，总结各类治疗方案的疗效、局限性与发展趋势。方法：通过文献综述，整合近年来CML在化学治疗、免疫调节、靶向治疗、细胞治疗及基因与表观治疗等领域的研究成果，重点分析酪氨酸激酶抑制剂(Tyrosine Kinase Inhibitor, TKI)的迭代与联合治疗策略。结果：当前CML治疗已从传统治疗方式转向以TKI为核心的精准化、个体化模式。一代TKI伊马替尼可提升患者长期生存，二代TKI能进一步提高分子缓解率，而三代TKI可克服T315I耐药突变，新型变构抑制剂阿西米尼展现出较高的选择性。联合治疗成为目前提高CML疗效、降低耐药的重要方向。对于造血干细胞移植仍用于耐药或进展期患者，CAR-T与基因编辑等新兴疗法尚处探索阶段。结论：当前CML治疗已从传统治疗方式转向以TKI为核心的精准化、个体化模式。一代TKI伊马替尼提升患者长期生存率，二代TKI能进一步提高分子缓解率，而三代TKI可克服T315I耐药突变，新型变构抑制剂阿西米尼展现出较高的选择性。联合治疗成为目前提升CML疗效、降低耐药的重要方向。对于造血干细胞移植仍用于耐药或进展期患者，CAR-T与基因编辑等新兴疗法尚处探索阶段。

Abstract: Objective: To systematically review the latest advances in the treatment of Chronic Myeloid Leukemia (CML), and to summarize the efficacy, limitations, and development trends of various therapeutic regimens. Methods: Through a literature review, this study integrates recent research achievements in CML treatment areas including chemotherapy, immunomodulation, targeted therapy, cell therapy, and gene and epigenetic therapy, with a focus on the iteration of Tyrosine Kinase Inhibitors (TKIs) and combination therapy strategies. Results: Current CML treatment has shifted from traditional approaches to a precision and individualized model centered on TKIs. The first-generation TKI imatinib improves long-term patient survival, second-generation TKIs can further increase molecular response rates, and third-generation TKIs can overcome the T315I resistance mutation, while the novel allosteric inhibitor asciminib demonstrates high selectivity. Combination therapy has become an important direction to enhance efficacy and reduce drug resistance. Hematopoietic stem cell transplantation remains an option for drug-resistant or advanced-stage patients, while emerging therapies such as CAR-T and gene editing are still in the exploratory stage. Conclusion: Current CML treatment has shifted from traditional approaches to a precision and individualized model centered on TKIs. The first-generation TKI imatinib improves long-term patient survival, second-generation TKIs can further increase molecular response rates, and third-generation TKIs can overcome the T315I resistance mutation, while the novel allosteric inhibitor asciminib demonstrates high selectivity. Combination therapy has become an important direction to enhance efficacy and reduce drug resistance. Hematopoietic stem cell transplantation remains an option for drug-resistant or advanced-stage patients, while emerging therapies such as CAR-T and gene editing are still in the exploratory stage.