【摘要】 目的评价甲磺酸伊马替尼(伊马替尼)治疗 Ph 阳性(Ph~+)慢性粒细胞白血病(CML)加速期和急变期的疗效。方法对75例 Ph~+CML 加速期患者和49例急变期患者持续口服伊马替尼400 mg/d 或600 mg/d 进行疗效观察。结果加速期:中位追踪23.0(1.0～64.0)个月,累积获得的血液学总有效率为93.3%,包括完全血液学缓解(CHR)85.3%和回到慢性期(RCP)8.0%,无效6.7%。累积获得的主要细胞遗传学缓解(MCyR)率为33.0%,其中完全细胞遗传学缓解(CCyR)率为28.0%,获得 CCyR 患者中主要分子学缓解(MMoR)率为47.6%。治疗有效者中,预计4年无疾病进展生存(PFS)率和总生存(OS)率分别为48.2%和52.2%。全部患者中,严重白细胞、血红蛋白和血小板减少的发生率分别为37.3%、34.6%和45.3%。急变期:中位追踪4.5(0.3～63.0)个月,累积获得的血液学总有效率为63.3%,包括 CHR 44.9%和 RCP 18.4%,无效36.7%。累积获得的 MCyR 率和 CCyR率均为12.2%,其中 MMoR 率为33.3%。治疗有效者中,预计1年 PFS 率和 OS 率分别为32.8%和46.0%,2年 PFS 率和 OS 率分别为15.8%和21.0%。全部患者中,严再白细胞、血红蛋白和血小板减少的发生率分别为75.5%、71.4%和73.5%。结论①伊马替尼对 Ph~+CML 的疗效随疾病进展的程度递减,严重血液学毒性递增。②多数加速期患者 PFS 期明显延长,特别是获得持久 CCyR 甚至MMoR 者。③绝大部分急变期患者血液学效应短暂,复发率高。更多还原

【Abstract】 Objectives To evaluate the efficacy and safety of imatinib mesylate(imatinib)for pa- tients with Philadelphia chromosome-positive(Ph~+)chronic myeloid leukemia(CML)in accelerated and blastic phase.Methods Seventy-five Ph~+CML patients in accelerated phase and 49 in blastic phase were treated with 400 mg or 600 mg of imatinib once daily.Results For patients in accelerated phase,the cumu- lative hematological response(HR)rate was 93.3%,including cnulplete HR(CHR)rate 85.3%,and re- turning to chronic phase(RCP)rate 8% in a median follow-up of 23.0(1.0-64.0)months.Cumulative major cytogenetic response(MCyR)rate was 33.0%,and complete cytogenetic response(CCyR)rate 28.0%.For patients with CCyR,the major molecular response(MMoR)rate was 47.6%.The estimated 4- year progression-free survival(PFS)rate and overall survival(OS)rate were 48.2% and 52.2% in patients with HR,respectively.Severe leukocytopenia,anemia and thrombncytnpenia occurred in 37.3%,34.6% and 45.3% of all patients,respectively.For patients in blastic phase,the cumulative HR rate was 63.3%, including CHR rate 44.9%,and RCP rate 18.4% in a median follow-up of 4.5(0.3-63.0)months.Cu- mulative MCyR rate and CCyR rate were both 12.2%.For patients with CCyR,the MMoR rate was 33.3%. For patients with HR,the estimated l-year/2-ycar PFS and OS rates were 32.8%/15.8% and 46.0%/ 21.0%,respectively.Severe leukocytopenia,anemia and thrombocytopenia occurred in 75.5%,71.4% and 73.5% of all patients,respectively.Conclusions The efficiency of imatinib was decreasing,and severer hematological toxicities increasing with the disease progressing in patients with Ph~+ CML.hnatinib improves progression-free survival significantly in most patients in accelerated phase,particularly in those with continuous CCyR or MMoR.The response duration in majority of blastie phase patients is short,and the re- lapse rate is high.更多还原