重组人血小板生成素治疗原发免疫性血小板减少症的临床观察

Clinical Study on Recombinant Human Thrombopoietin in Treatment of Patients with Primary Immune Thrombocytopenia

目的观察重组人血小板生成素(rhTPO)治疗原发免疫性血小板减少症(ITP)的临床疗效及不良反应,并观察骨髓巨核细胞数与疗效的关系。方法 18例慢性难治性ITP患者皮下注射rhTPO 300 u/kg.d-1,疗程14 d或血小板>100×109/L后停药,动态观察患者血常规,定期监测肝、肾功能及凝血功能,并分析治疗有效患者骨髓巨核细胞数与血小板上升数值之间的相关性。结果 18例患者rhTPO治疗后,完全反应(CR)7例,有效(R)8例,无效(NR)3例。用药前血小板平均值为10±5.4×109/L,治疗后血小板计数平均最高值为116.4±82×109/L,与用药前比较差异有统计学意义(t=4.82,P<0.01)。多数患者对药物耐受良好。rhTPO治疗有效患者骨髓巨核细胞数与血小板上升数值呈正相关(r=0.582,P=0.003)。结论 rhTPO治疗慢性难治性ITP具有良好的疗效,不良反应轻微,骨髓巨核细胞数越多的患者疗效越显著。

Objective To observe the therapeutic effect and toxicity of recombinant human thrombopoietin（rhTPO） on chronic refractory immune thrombocytopenia（ITP） and discuss on the relationship between itstherapeutic effect and the number of bone marrow megakaryocyte.Methods 18 patients with chronic ITP were administered with rhTPO subcut-aneously at 300 u/ kg·d^-1 for 14 days or until the platelet count 〉100×109/L.Blood routine was dynamically examined.Liver function,renal function and blood clotting function were regularly monitored.The correlation between the number of bone marrow megakaryocyte and the increasing platelet count was analyzed.Results Out of 18 patients with ITP,7 cases showed complete response and 8 cases showed good response and 3 cases showed no response to the treatment with rhTPO.The pe ak platelet count after treatment was（116.4±82 ×10^9/L）,signifiantly higher than the mean platelet count（10±5.4）×10^9/L before t reatment（t=4.82,P〈0.01）.The number ofbone marrow megakaryocyte was in a positive correlation with the platelet count in patients with effective treatment（r=0.582,P=0.003）.One mild adverse reaction was observed.Conclusion The rhTPO is safe and efficacy in the treatment of chronic refractory ITP with less adverse reaction.The patients who have more number of bone marrow megakaryocyte have significant herapeutic effect.