摘要目的 研究雄激素联合小剂量全反式维甲酸治疗骨髓增生异常综合征的长期疗效.方法 62例骨髓增生异常综合征(MDS),按FAB诊断分型标准:其中RA48例、RAS2例、RAEB9例、RAEB-t2例、CMML1例.司坦唑醇6 mg/d或炔烃雌烯异(口恶)唑0.6/d,全反式维甲酸10 ms/d.结果 异恶性循环治疗6个月时60例可评价者中36例维持原方案治疗,按照MDS国际工作组疗效判定标准:完全缓解1例,部分缓解6例,血液学改善19例.总有效率43.3%(26/60).RA/RAS有效率50.0%(24/48),RAEB/RAEB-t/CMML有效率16.7%(2/12).有效组和无效组在治疗前后的骨髓原始细胞比例、骨髓增生程度、病态造血以及粒/红差异均无统计学意义.治疗12个月时25例维持原方案治疗:完全缓解1例,部分缓解7例,血液学改善9例.总有效率28.3%(17/60).RA/RAS有效率35.4%(17/48).RAEB/RAEB-t/CMML有效率0%(0/12).长期追访中位时间47个月,39例RA患者有效组、无效组的中位生存期分别为54个月(95%可信区间41,66个月)、23个月(95%可信区间13,32个月),二者差异有统计学意义(P<0.05).不良反应大多数轻微不影响继续治疗.结论 雄激素联合小剂量全反式维甲酸治疗MDS对低危险组患者是一种比较有效、经济、安全的治疗方法,有效者能够改善生活质量,在一定程度上延长生存期.

abstractsObjective To investigate the long-term effects of androgen combined with low dose all-trans-retinoic acid on myelodysplastic syndrome (MDS). Methods Sixty-two MDS patients, 48 with RA, 2 of RAS, 9 with RA with excess of blasts (RAEB), 2 with RAEB-transformation (RAEB-t), and 1 with chronic myelogenous-monocytic leukemia (CMML) according to the FAB subtype standard, received stanazolol (6 mg/d) or danazol (600 mg/d) and low dose all-trans-retinoic acid (ATRA 10 mg/d). Three months later the treatment was discontinued on 22 patients that showed ineffective and 2 more patients withdrew from the treatment due to exacerbation. The remaining 36 patients were treated according to the original protocol, and the doses of these 2 drugs were reduced by half until the condition was exacerbated. Follow-up was conducted for 47 (34 -78) months. Results Mter 6 months of treatment, complete remission (CR) was seen in 1 patient, partial remission (PR) in 6 patients, and hematologic Improvement (HI)in 19 of the 60 patients evaluated with a response rate of 43.3% (26/60) in all patients, 50% (24/48) in RA/RAS group, and 16. 7% (2/12) in RAEB/RAEB-t/CMML group. There were not significant differences in cellularity, dysplastic hematopoiesis, and myeloblast before and after treatment among the RA/RAS patients. After 12 months of treatment, CR was seen in 1 patient, PR in 7, and HI in 9, with a response rate of 28.3 % (17/60) in all patients, 35.4% (17/48) in the RA/RAS group, and 0% (0/12) in the RAEB/RAEB-t/CMML group. Adverse effects were mild and did not require discontinuance of the therapy. The survival time of the 19 patients in the RA group that responded well to treatment was 54 months (41,66), significantly longer than that of the 20 patients without good outcomes [23 months (13,32) , x2= =4.72,P=0.025]. Conclusion Effective, economic, and safe, stanozolol or danazol with low-dose all-trans-retinoic acid improves the life quality and prolongs the survival time of the MDS patients who respond well.