低剂量反应停治疗原发性骨髓纤维化的临床研究

Clinical efficacy of low dose thalidomide in the treatment of patients with idiopathic myelofibrosis

目的：探讨低剂量反应停治疗原发性骨髓纤维化的临床疗效。方法：原发性骨髓纤维化患者21例，反应停剂量为≤100mg／d，根据治疗前后外周血白细胞、血红蛋白、血小板、脾脏大小的变化及临床症状的改善来评定疗效，并观察骨髓病理检查及药物的不良反应。结果：21例患者中有18例完成了6个月的疗程，2／4患者白细胞从〉15×10^9／L[（15．4～31．2）×10^9／L]降至〈15×10^9／L；对于贫血的改善，CR2例（2／18，11．1％），PR5例（5／18，27．8％），MR4例（4／18，22．2％）；6／10例患者血小板从〈100×10^9／L[（30～90）×10^9／L]升至正常；18例脾大的患者中，有1例CR，2例PR，2例脾脏缩小≥25％，3例脾脏缩小〈25％，有效率为44．4％。5例治疗有效者复查骨髓活检较治疗前无明显变化。反应停的不良反应主要表现为疲乏、便秘及淡漠。结论：反应停是一种安全、经济、有效的治疗原发性骨髓纤维化的新方法。

Objective：To explore the clinical efficacy of low dose thalidomide in the treatment of patients with idiopathic myelofibrosis. Method;21 patients received less than 100 mg/d thalidomide, the hematological indicators such as white blood cell counts, hemoglobin level, platelet counts, the size of spleen and the clinical symptoms were observed to assess the clinical efficacy. The changes of marrow biopsy and side effects were observed syn chronously. Result; 18/21 （85.7 %） patients had finished the 6-month treatment, the white blood cell counts of 2/4 patients（50%） had declined from〉15×10^9/L[-（15.4～31.2）×10^9/L] to〈15× 10^9/L;11/18（61. 1%） patients had achieved increase in their hemoglobin, including 2 CR（2/18,11.1%）, 5 PR（5/18,27.8%）, 4 MR（4/18, 22.2%）. The platelet counts of 6/10 patients（60%） had increased from〈100×10^9/L[-（30～90）×10^9/L] to normal value. 8/18（44.4%） patients has a decrease in their spleen size, including 1 CR, 2 PR, 2 had a decrease by 25%, 3 had a decrease〈25%. There was no significant changes in the biopsy results of the 5 responders. The main side effects were mainly fatigue, constipation and indifference. Conclusion：Thalidomide is a safety, economical, effective new method to treat IMF.