小剂量沙利度胺和泼尼松联合或不联合达那唑治疗原发性骨髓纤维化患者贫血的疗效比较

Comparison of low-dose thalidomide and prednisone combined with or without danazol for the treatment of primary myelofibrosis-associated anemia

目的 观察小剂量沙利度胺和泼尼松联合或不联合达那唑对原发性骨髓纤维化（PMF）患者贫血的治疗效果.方法 以58例HGB＜100 g/L的PMF患者为研究对象.28例患者接受小剂量沙利度胺和泼尼松治疗（沙利度胺＋泼尼松组）,30例接受小剂量沙利度胺和泼尼松联合达那唑治疗（沙利度胺＋泼尼松＋达那唑组）.按照2006年骨髓纤维化研究和治疗国际工作组疗效判断标准评价疗效.治疗后3、12个月检测患者骨髓纤维化程度.结果 58例患者总有效率56.9％（33/58）,其中部分缓解（PR） 1.7％（1/58）,临床改善（CI） 55.2％（32/58）.沙利度胺＋泼尼松组、沙利度胺＋泼尼松＋达那唑组总有效率差异无统计学意义（50.0％对63.3％,P=0.306）,但后者中位有效持续时间和红系临床改善（CI-E）中位有效持续时间优于前者[75（8～206）周对30（8～86）周,P=0.007;61.5（8～194）周对22（8～86）周,P=0.015].两组患者治疗3个月和12个月时骨髓骨髓纤维化程度较治疗前均有显著改善（P值分别为0.000和0.005）.两组不良反应均为1～2级.结论 小剂量沙利度胺和泼尼松为PMF患者贫血的有效治疗方案,联合应用达那唑可显著延长疗效持续时间.

Objective To observe the clinical effects of low-dose thalidomide （THAL） and prednisone （PRED） with or without danazol （DANA） in patients with primary myelofibrosis （PMF） associated anemia.Methods A cohort of 58 PMF patients with anemia （Hb＜100 g/L） were retrospectively studied.Of them,28 patients were treated with THAL and PRED （THAL-PRED group）,and the rest with THAL,PRED and DANA （THAL-PRED-DANA group）.The hematological response was assessed according to the modified criteria of the Intemational Working Group in 2006,and the myelofibrosis degree was evaluated at 3 and 12 month after treatment.Results The total response rate was 56.9％（33/58） including 1.7％ （1/58） partial remission（PR） and 55.2％ （32/58） clinical improvement （CI）.There was no statistical difference in the response rate between THAL-PRED and THAL-PREDDANA groups （50.0％ vs 63.3％,P=0.306）.However,the median response duration of clinical improvement,erythroid response （CI-E） and total response prolonged in THAL-PRED-DANA than THALPRED group （61.5w vs 22w,P=0.015; 75w vs 30w,P=0.007,respectively）.Myelofibrosis degree at 3 and 12 months after treatment decreased significantly than before treatment （P=0.000 and 0.005,respectively）.Side-effects in both groups were only grade 1-2.Conclusions Low-dose THAL together with PRED appeared to be effective in the treatment of PMF-associated anemia,and the response duration would prolong significantly if combined with DANA.