目的回顾性分析二代酪氨酸激酶抑制剂(second-generation TKI)治疗慢性髓细胞白血病(CML)的临床疗效。方法对近十年来在我院门诊接受二代酪氨酸激酶抑制剂治疗的97例CML患者的临床资料及随访结果进行回顾性分析。其中61例患者为慢性期,26例为加速期,10例为急变期。74例患者于伊马替尼(IM)耐药或不耐受后接受二代TKI治疗,其中57例患者接受尼洛替尼(NIL)治疗,17例患者接受达沙替尼(DAS)治疗;20例患者诊断后即接受二代TKI治疗,其中19例患者接受NIL治疗,1例患者接受DAS治疗;还有3例患者接受二代TKI合并造血干细胞移植(HSCT)及化疗等治疗。治疗期间定期监测患者血液学、细胞遗传学及分子生物学反应,评价治疗反应及疗效,采用Kaplan-Meier曲线进行生存分析。结果随访结束时总完全血液学缓解(CHR)率、主要细胞遗传学缓解(MCyR)率、完全细胞遗传学缓解(CCyR)率和主要分子生物学缓解(MMR)率分别为97.9%、63.9%、60.0%、44.3%,其中除CHR率外,MCyR、CCyR、MMR率慢性期患者均优于进展期(加速期+急变期)患者,差异有统计学意义(P<0.05)。患者的1年、2年、3年和5年总生存(OS)率分别为(90.6±3.0)%、(80.1±4.5)%、(77.5±5.0)%和(64.6±9.3)%;1年、2年、3年和5年无事件生存(EFS)率分别为(81.1±4.0)%、(64.4±5.3)%、(56.4±6.0)%和(46.2±8.2)%;1年、2年、3年和5年无进展生存(PFS)率分别为(87.4±3.4)%、(73.2±4.9)%、(68.9±5.5)%和(57.4±8.7)%,其中慢性期患者总OS、EFS和PFS率均优于进展期患者,差异有统计学意义(P<0.05)。二代TKI一线治疗获得CHR、MCyR、CCyR、MMR率分别是100%、95%、95%、70%,二线治疗获得的则分别是97.3%、56.8%、48.6%、36.5%,其中除CHR率外,MCyR、CCyR、MMR率一线治疗均优于二线治疗,差异有统计学意义(P<0.05)。结论二代TKI治疗CML疗效及预后较好,且一线较二线治疗、慢性期较进展期更能使患者明显获益。 Objective To retrospectively analyze the clinical efficacy of second-generation TKI in the treatment of chronic myelogenous leukemia (CML). Methods The clinical data and follow-up results of 97 CML patients receiving second-generation tyrosine kinase inhibitors in our hospital during the past decade were retrospectively analyzed. Among them, 61 were chronic, 26 were accelerated, and 10 were emergency. Seventy-four patients received second-generation TKI after resistance or intolerance to imatinib (IM), 57 of whom received nilotinib (NIL) and 17 received dasatinib (DAS) Twenty patients were diagnosed with second-generation TKI after treatment, of whom 19 received NIL, 1 received DAS, and 3 received second-generation TKI combined with hematopoietic stem cell transplantation (HSCT) and chemotherapy. Hematology, cytogenetics and molecular biology were regularly monitored during treatment to evaluate the response and efficacy of treatment. Survival analysis was performed using Kaplan-Meier curves. Results The rates of total complete hematological response (CHR), major cytogenetic response (MCyR), complete cytogenetic response (CCyR) and major molecular biological response (MMR) at the end of follow-up were 97.9% and 63.9% , 60.0% and 44.3%, respectively. Except CHR rate, the patients with MCyR, CCyR and MMR rates were better than those with advanced (accelerated + blast crisis), the difference was statistically significant (P <0.05). The overall OS rates at 1 year, 2 years, 3 years and 5 years were (90.6 ± 3.0)%, (80.1 ± 4.5)%, (77.5 ± 5.0)% and (64.6 ± 9.3)%, respectively The event-free survival (EFS) rates were (81.1 ± 4.0)%, (64.4 ± 5.3)%, (56.4 ± 6.0)% and (46.2 ± 8.2)% for 1 year, 2 years, 3 years and 5 years respectively; The PFS rates at 2 years, 3 years and 5 years were (87.4 ± 3.4)%, (73.2 ± 4.9)%, (68.9 ± 5.5)% and (57.4 ± 8.7)%, respectively OS, EFS and PFS rates were better than those in advanced stage, the difference was statistically significant (P <0.05). The second generation of TKI first-line treatment to obtain CHR, MCyR, CCyR, MMR rates were 100%, 95%, 95%, 70%, second-line treatment were 97.3%, 56.8%, 48.6%, 36.5% Rate, MCyR, CCyR, MMR rate of first-line treatment are better than second-line treatment, the difference was statistically significant (P <0.05). Conclusion The second-generation TKI treatment of CML has better curative effect and prognosis, and the first-line treatment is more effective than second-line treatment and chronic phase progression.