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目的多中心前瞻性研究环孢素 A(CsA)治疗骨髓增生异常综合征(MDS)的疗效。方法分析 CsA 治疗33例 MDS,其中难治性贫血(RA)24例,环形铁粒幼细胞增多性难治性贫血(RAS)2例,难治性贫血伴原始细胞增多(RAEB)7例。给药方法:新山地明3～5 mg·kg~(-1)·d~(-1),分2次服用。疗程中根据 CsA 血浓度和疗效调整剂量,疗程3个月以上。结果治疗3个月时,按 MDS国际工作组评价标准,可评价的32例患者总血液学改善率18/32(56.3%)。随访结束时,中数随访期14个月,自然病程改变4例(4/32):1例完全缓解,3例部分缓解。血液学改善16例,总有效率为20/32(62.5%)。结论本文证实了 CsA 治疗 RA、RAEB 型患者有效,对于增生低下和增生活跃骨髓增生异常综合征均有效,治疗有效患者生存期较无效患者明显延长。
Objective To investigate the efficacy of cyclosporine A (CsA) in the treatment of myelodysplastic syndrome (MDS). Methods Thirty-three patients with MDS were enrolled in this study. Among them, 24 were refractory anemia (RA), 2 were RKA (rimmed sideroblasts), and 7 were refractory anemia with rheumatoid arthritis (RAEB). Method of administration: nerolimi 3 ~ 5 mg · kg -1 (-1) · d -1, taking 2 times. Course of treatment according to CsA blood concentration and efficacy of dose adjustment, treatment of more than 3 months. Results At 3 months of treatment, according to MDS International Working Group evaluation criteria, the total hematological improvement rate of 32 patients evaluated was 18/32 (56.3%). At the end of follow-up, the median follow-up period was 14 months. Four patients (4/32) had changes in their natural course of disease: one had complete remission and three had partial remission. Hematology improved in 16 cases, the total effective rate was 20/32 (62.5%). Conclusions This study demonstrates that CsA is effective in patients with RA and RAEB and is effective in patients with hyperplasia and hyperplasia of active myelodysplastic syndromes. Patients in the treatment-effective patients have significantly longer survival than those in patients with ineffective treatment.