中文摘要：

目的比较甲磺酸伊马替尼和异基因造血干细胞移植治疗慢性髓细胞白血病（CML）慢性期的疗效。方法选取南方医科大学南方医院血液科2002年2月至2012年12月198例CML慢性期接受甲磺酸伊马替尼或异基因造血干细胞移植治疗的患者，评价两种治疗方法的疗效、生存及疾病进展和不良反应情况。其中甲磺酸伊马替尼组115例，初始剂量400mg／d，此后依据血象及疗效调整剂量，所有患者每1～3个月复查骨髓细胞学及遗传学1次，以此判断血液学反应和细胞遗传学反应。移植组共83例，移植采用清髓性预处理方案，常规采用甲氨蝶呤加环孢素A进行移植物抗宿主病（GVHD）的预防，部分联合应用麦考酚酸酯和抗胸腺细胞球蛋白，移植后常规检测植活证据、细胞及分子遗传学缓解情况。结果伊马替尼组86例患者中59例（68．6％）在12个月获得完全细胞遗传学缓解（CCyR），而移植组移植后12个月70例患者中67例（95．7％）获得CCyR。伊马替尼组与移植组累计复发率分别为14．8％（17／115）、12．3％（10／81）。移植组GVHD发生率及感染率较高，而伊马替尼组患者不良反应大多可耐受。患者伊马替尼组10年累计生存率高于移植组（93．9％比77．1％，P=0．015）；10年无疾病进展生存率两组分别为86．1％、88．0％（P=0．508），差异无统计学意义。对于Sokal评分中、高危患者的累计生存率，伊马替尼组均高于移植组（96．4％比68．0％，P=0．049；92．6％比57．1％，P=0．017），而无疾病进展生存率两组差异均无统计学意义（89．3％比88．0％，P=0．942；70．4％比85．7％，P=0．405）；低危患者总生存率与无疾病进展生存率两组差异均无统计学意义。对年龄≥30岁的患者，伊马替尼组累计生存率高于移植组（94．7％比73．5％，P=0．009），而两组无疾病进展生存率分别为84．2％、94?

英文摘要：

Objective To compare the treatment efficacy of imatinib mesylate versus allogeneic hematopoietic stem cell transplantation （allo-HSCT） for patients with chronic myeloid leukemia in chronic phase. Methods The efficacy, overall survival, progression-free survival and adverse events were evaluated in 198 patients on these two therapies from February 2002 to December 2012 at our hospital. One hundred and fifteen cases in imatinib group （n = 115） received imatinib at an initial daily dose of 400 mg and then dose was adjusted according to blood routine test and therapy response. All patients were evaluated for hematologic, cytogenetic and molecular responses every 1 - 3 months. The allo-HSCT group （ n = 83 ） received myeloablative preconditioning regimen and methotrexate （MTX） and cyclosporine A （ CsA ） were used for graft-versus-host disease （GVHD） partially plus mycophenolate mofetil （MMF） and antihuman thymocyte globulin （ATG）. The engraftment evidence and evolution of cytogenetic and molecular responsewas conventionally detected after allo-HSCT. Results In imatinib group, 59 of 86 （68.6%） cases achieved complete cytogenetic response （CCyR） in the 12 months after therapy, while 67 of 70 （95.7%） cases achieved CCyR in allo-HSCT group. The relapse rates of two groups were 14. 8% （17/115） ,12. 3% （10/81） respectively. The adverse reaction of imatinib in imatinib group was obviously much more tolerable for patients compared with frequently occurred GVHD and infection in allo-HSCT group. The 10-year cumulative overall survival （OS） rate was 93.9% in imatinib group and 77.1% in allo-HSCT group （P = 0. 015）. And the lO-year cumulative progression-free survival （PFS） rate was 86. 1% in imatinib group versus 88. 0% in allo-HSCT group（P =0. 508）. For Sokal rating stratified analysis, the cumulative OS rates of two groups were 96.4% and 68.0% （P = 0. 049） for intermediate-risk patients, 92. 6% and 57.1% （P = 0. 017） for high-risk patients while the cum