重组人血小板生成素治疗儿童重症免疫性血小板减少症25例临床观察

Clinical analysis of Recombinant Humanized Thrombopietin for Treating 25 Children with Severe Immune Thrombocytopenia

本研究旨在通过对国产重组人血小板生成素(rhTPO)治疗儿童重症免疫性血小板减少症(ITP)的临床资料分析,探讨儿童患者使用rhTPO的疗效及安全性。采用回顾分析方法,收集北京儿童医院2009年12月-2012年11月收入院的25例予rhTPO治疗(连续14 d)的重症免疫性血小板减少症患儿临床资料。分析rhTPO治疗儿童重症免疫性血小板减少症的疗效及安全性。结果表明:25例病例用药前血小板中位数为4×109/L(0×109/L-10×109/L),用药后最高值中位数为71×109/L(14×109/L-439×109/L),升至最高值中位时间为11 d(范围3-15d)。停用TPO后,血小板计数逐渐下降。疗效分析显示,完全反应11/25例(44%),有效8/25例(32%),无效6/25例(24%),总有效率19/25(76%)。对于治疗有效的患者,用药后血小板最高值中位数为112×109/L(43×109/L-439×109/L),升至最高值中位时间为12 d(范围7-15 d);达到有效疗效的平均中位时间为4 d(范围1-11d)。治疗有效者停药2周时平均血小板计数仍显著高于治疗前(P<0.05)。12例予rhTPO治疗前用足量丙种球蛋白治疗无效的病例,在rhTPO治疗结束后予丙种球蛋白治疗有效。应用rhTPO治疗期间,2例患者出现轻微不良反应,用药后及随访过程中未发现其他相关副作用。结论:儿童重症ITP应用rhTPO治疗安全有效,可以帮助患儿更平稳度过严重出血的危险期,但rhTPO作用不持久,停用后血小板计数逐渐回落,需维持治疗以巩固疗效。

This study was aimed to evaluate the efficacy and safety of recombinant humanized thrombopietin （rhTPO） for treating children with severe immune thrombocytopenia （ITP）. A total of 25 patients with severe ITP who accepted rhTPO treatment for 14 days between December,2009 and November,2012 in Beijing Children＇s Hospital was retrospectively analyzed. The results showed that the median platelet counts of all 25 patients increased from the lowest level 4.0 × 109/L（0 × 109/L - 10 ×109/L） to the highest level 71×109/L（ 14 ×109/L - 439 ×10a/L） on median 11 days（range from 3 days to 15 days）. After rhTPO discontinuation, the platelet counts of patients gradually decreased. Complete response rate was 44% （ 11/25 ）, response rate was 32% （8/25）, non-response rate was 24% （6/25） and total response rate was 76% （ 19/25 ）. The platele count in the patients who showed complete response to rhTPO therapy reached the highest 112 × 109/L（43× 109/L - 439 ×109/L） on median 12 days（ range from 7 days to 15 days）. The patients showed response to rhTPO treatment on median 4 days （range from 1 days to 11 days）. The platelet count decreased gradually after the discontinuation of rhTPO administration but still significantly higher on 28 days than the level before the treatment （P 〈 0.05 ）. 12 patients who did not respond to γ-globulin before rhTPO treatment showed response to γ-globulin after the discontinuation of rhTPO therapy. 2 patients showed mild clinical adverse reaction. It is concluded that rhTPO is an effective and safe treatment method for children with severe ITP. It will help the patient smoothly through the dangerous period of severe bleeding, but the platelet count decreases gradually after rhTPO disco-ntinuation. Maintenance treatment is needed to consolidate the curative efficacy.