慢性粒细胞白血病异基因造血干细胞移植34例疗效及生活质量随访

Allogeneic hematopoietic stem cell transplantation for 34 patients with chronic myelogenous leukemia: Efficacy and follow-up of quality of life

目的:观察异基因造血干细胞移植治疗慢性粒细胞白血病的疗效并进行生活质量评估。方法:选择2002-03/2007-03在解放军第四军医大学西京医院血液科接受异基因造血干细胞移植18～50岁慢性粒细胞白血病患者34例,其中HLA相合的同胞供者29例,非血缘志愿供者5例。所有患者及其家属对治疗以及研究均知情并签署同意书,医院伦理委员会知情并批准。预处理方案采用改良的马利兰联合环磷酰胺或环磷酰胺联合全身放疗方案。采用标准的环孢素联合短期氨甲喋呤方案预防移植物抗宿主病;无关供者移植加用抗人胸腺细胞球蛋白。所有患者均接受WHOQOL-BREF问卷调查其移植前后的生活质量。结果:所有患者均获得植入。①移植物抗宿主病出现情况:14.7%(5/34)患者出现急性移植物抗宿主病,41.2%(14/34)患者出现慢性移植物抗宿主病。②移植其他并发症:合并重症肝静脉闭塞病1例;并发纯红细胞性再生障碍性贫血3例,甲状腺功能低下2例,女性雌激素水平低下9例,经积极治疗,大部分获得缓解。③长期生存情况:随访3～60个月,现存活25例(73.5%),其中在慢性期移植者,生存率达82.1%(23/28)。26.4%(9/34)患者在移植后1～50个月时死亡,4例死于白血病复发,5例死于移植并发症。④生活质量评分:与患者移植前比较,各项指标均有显著的提高(P<0.05)。结论:异基因造血干细胞移植是治疗慢性粒细胞白血病的有效方法,尤其在慢性期移植效果较好。移植后长期存活患者的生活质量良好,慢性移植物抗宿主病及女性卵巢功能低下是影响患者生活质量的主要因素。

AIM： To observe the curative effect of allogeneic hematopoietic stem cell transplantation （allo-HSCT） on chronic myelogenous leukemia （CML） and evaluate the quality of life. METHODS： Thirty-four 18 to 50-year-old CML patients, who received alIo-HSCT in Department of Hematology, Xijing Hospital of Fourth Military Medical University of Chinese PLA from March 2002 to March 2007, were selected including 29 HLA-identical sibling donors, and 5 HLA-matched unrelated volunteers. The treatment was agreed by the patients and their families, and permitted by Hospital Ethics Committee. Conditioning regimen consisted of cyclophosphamide and total body irradiation or busulphan-cyclophosphamide. Standard methotrexate and cyclosporine were combined for graft versus-host disease （GVHD） prophylaxis. Anti-human thymocyte immunoglobulin （ATG） and CD25 monoclonal antibody were given to unrelated donor HSCT. All the patients alive were evaluated with the questionnaire of.quality of life in WHOQOL-BREF system. RESULTS： All 34 patients got sustained engraftment. ①Acute GVHD and chronic GVHD were found in 5/34 （14.7%） and 14/34 （41.2%） patients, respectively. ②Severe veno-occlusive disease occurred in 1 case, pure red cell aptasia in 3 patients, thyroid hypofunction in 2 cases and decrease in estrogen in 9 cases. Most of the complications were relieved after treatment. GAll subjects were followed up for 3 to 60 months, and 25/34 （73.5%） patients were still alive, of which the survival was 82.1% （23/28） in patients receiving transplantation in chronic stage, and 9/34 （26.4%） died from leukemia relapse （n =4） and transplantation-related complications （n =5）. ③Compared with that before transplantation, the quality of life in all patients was improved significantly （P 〈 0.05）. CONCLUSION： AIIogeneic hematopoietic stem cell transplantation is an effective therapy for CML especially in chronic period. The quality of life of patients alive for a long time after transplantation is good. Chronic GVHD and hypofunction of ovary are main factors for the quality of life.