中文摘要：

目的 评价环孢素（CsA）联合沙利度胺治疗骨髓增生异常综合征（MDS）的疗效与不良反应.方法 37例WHO分型（2001）为难治性血细胞减少伴有多系发育异常（RCMD）和难治性贫血伴有原始细胞过多-I（RAEB-I）患者接受CsA联合沙利度胺治疗,CsA起始剂量为3 mg·kg-1·d-1,2周后检测血CsA浓度,根据血药浓度调整用药剂量,使CsA谷浓度维持在100～200μg/L,沙利度胺起始剂量为50 mg/d,1周内增至100 mg/d,无明显不良反应则最大剂量为200 mg/d.依据国际工作组2006疗效标准评价血液学疗效,按美国国立肿瘤研究所常见毒性标准（version 3.0）判定不良反应.同时观察有效持续时间和总体生存率.结果 患者中红系反应率为51.4%（37例中19例） 中性粒细胞反应率为21.2%（33例中7例） 血小板反应率为31.0%（29例中9例） 在依赖输血的32例患者中,15例（46.9%）脱离输血.红系反应中位持续时间为88（4～108）周,血小板反应中位持续时间为78（8～84＋）周,中性粒细胞反应中位持续时间为78（10～84＋）周.中位随访时间为29（4～103）个月,中位生存时间为52个月.不良反应包括1～2级肝肾损害、便秘、嗜睡、头昏、水肿、皮疹、麻木感,经对症治疗后均好转,所有患者未出现3级以上不良反应.结论 CsA联合沙利度胺治疗MDS能有效改善患者贫血,不良反应轻.

英文摘要：

Objective To explore the efficiency and side-effects of the combination of cyclosporine A （ CsA） and thalidomide in patients with myelodysplastic syndromes（ MDS）. Methods A total of thirty-seven patients with MDS-RCMD or-RAEB- I were treated with CsA in combination with thalidomide. The initial CsA dose of 3 mg kg-1 d-1 was administered, all patients had their CsA blood concentration concurrently monitored until it reached and maintained between 100 and 200 μg/L. The initial dose of thalidomide was 50 mg/d, with increasing dose of 50 mg every week until the maximum of 200 mg/d. The hematological response was assessed according to the modified criteria of the International Working Group, and adverse events were graded with the Common Toxicity Criteria （v3.0） of the National Cancer Institute. The response duration and overall survival of the patients were also observed. Results 19/37 cases （51. 4% ） achieved hematologic improvement（HI）-erythroid response（HI-E）, 9/29 cases （31.0%） Hi-platelet response （HI-P） and 7/33 cases （21.2%） HI-neutrophil response （HI-N）. 15 of 32 transfusion-dependent patients （46.9% ） achieved transfusion independence. The median response duration of HI-E, HI-P and HI-N were 88 （4 - 88） weeks, 78（8 -846＋ ） weeks and 78 （10-84＋ ） weeks respectively. The median overall survival was 52 months on a 29（4-103） months median follow-up. Some patients developed grades I - II hepatic or nephritic impairment, constipation, lethargy, dizziness, edema, rashes or numbness, and all were tolerable and reversible. No grade Ⅲ or severer adverse events were observed. Conclusion CsA in combination with thalidomide appears to be effective mainly in inducing HI-E and relatively well-tolerated for the treatment of patients with MDS.