环孢菌素A为主方案治疗骨髓增生异常综合征  

Cyclosporine A Based Therapy for Myelodysplastic Syndrome

作　　者：李振玲[1] 龚明[1] 徐韶华[1] 黄泛舟[1] 陈艳荣[1] 马一盖[1] 

机构地区：[1]中日友好医院血液科,北京100029

出　　处：《中国实验血液学杂志》2005年第5期867-870,共4页Journal of Experimental Hematology

摘　　要：为了研究CsA为主方案治疗骨髓增生异常综合征(MDS)的疗效,给16例MDS患者(RA10例,RAEB1型6例)口服CsA2.5-5.5mg/(kg·d),其中2例单用CsA,14例合用CsA、重组人促红细胞生成素和(或)雄激素、维生素D3,疗程2周-2年(平均8个月)。疗效判定按照MDS国际工作组标准,分为完全缓解(CR)、部分缓解(PR)、血液学改善(HI)和无效(NR),前三者均视为有效。结果表明:12例获HI,2例获PR,2例NR,总有效率87.5%。中性粒细胞、红系细胞及血小板升高反应率分别为83.3%、66.7%和60%,最早起效时间分别为2周、1个月及1个月。治疗前13例(81.25%)依赖输血,治疗后仅5例(31.25%)依赖输血。10例RA中9例有效,1例无效;6例RAEB中1例无效并转为RAEBt死亡,5例近期有效。副作用主要为齿龈增生、多毛和轻度肝肾功能异常。结论:以CsA为主的治疗方案能改善MDS的血象,减少输血,近期疗效好。患者口服CsA3-5mg/(kg·d)能很好耐受。To determine the efficacy and tolerance to cyclosporine A （CsA） based therapy in patients with myelodysplastic syndrome （MDS）, 16 patients with MDS consisting of 10 refractory anemia （RA） and 6 refractory anemia with accessary blasts less than 10% （RAEB-1） were analyzed. Five patients had hypocellular bone marrows and 11 patients had normocellular or hypercellular marrows, The dose of CsA was 2.5 -5.5 mg/（ kg·d） for 2 weeks to 2 years （mean 8 months）. Two out of 16 patients were treated with CsA alone, 14 patients were treated with CsA, recombinant human erythropoietin, androgens, 1,25 dihydroxy vitamin D3 or two or three of them combination with CsA. Treatment responses were classified according to the International Working Group （IWG） criteria as complete remission （CR）, partial remission （PR）, hematological improvement （HI） and no response （NR）. Patients who obtained CR, PR or HI were defined as responders. The results showed that HI was observed in 12 patients, PR in 2 patients and NR in 2 patients. Total response rate was 87.5%. Response rates shown in neutrophil lineage, platelet and erythroid lineage were 83.3%, 66.7% and 60%, respectively; their shortest time requied to obtain some hematologic improvement after initiation of CsA therapy was 2 weeks, 1 month and 1 month, respectively. Of 13 patients being transfusion-dependent before treatment, 3 patients did not need transfusion any more and 5 showed the reduced transfusion requirements after CsA therapy. In 10 patients with RA, 9 responded to CsA. Of 6 patients with RAEB, 1 patient had no response and died of RAEB-t and 5 patients had transient responses. One of the latter transformed to CMML and two relapsed. The total response rate decreased to 50% in the patients with CsA therapy lasting more than 3 months at the end of following-up. The adverse effects included hirsutism, hyperplastic gingiva, reversible hepatic and renal dysfunction. In conclusion, the usefulness of CsA based therapy for MDS-RA and RAEB-1

关 键 词：骨髓增生异常综合征 免疫抑制治疗 环孢菌素A EP0 雄激素 

分 类 号：R733.7[医药卫生—肿瘤] R979.5[医药卫生—临床医学]