重组人血小板生成素治疗特发性和血液肿瘤化疗后血小板减少症的Ⅲ期临床研究

Treatment of thrombocytopenia with recombinant human thrombopoietin in patients with idiopathic thrombocytopenic purpura and hematological malignancies following chemotherapy: Phase Ⅲ clinical cohort study

目的 :评估重组人血小板生成素 (rhTPO)治疗特发性和化疗后血小板减少的临床安全性和有效性 .方法 :①病例分组 :4例非霍奇金淋巴瘤 (NHL)患者入选随机交叉对照组 ,6例巩固治疗期急性白血病患者入选非随机自身对照组 ;难治性血小板减少性紫癜 (ITP)组入选 9例患者 ;②用药方法 :rhTPO 1 μg/kg ,皮下注射 ,每日 1次 ,疗程 1 4d ;③统计分析 :病例资料采用SPSS软件统计分析 .结果 :①血液肿瘤组 :试验组rhTPO治疗后血小板数均值、最高值皆大于对照 ,两周期血小板数用药前后差值相差显著 (P <0 .0 5 ) ,试验组血小板数低于 5 0× 1 0 9/L的持续日数、恢复至 75× 1 0 9/L和 1 0 0× 1 0 9/L以上需要日数均有不同程度缩短 ;②难治性ITP组 :rhTPO治疗后患者血小板数均值渐升高 ,至 1 7d达 1 0 1× 1 0 9/L ;用药前后血小板差值为 85 .6 7× 1 0 9/L ,其中血小板数达 30× 1 0 9/L ,5 0× 1 0 9/L及 1 0 0× 1 0 9/L所需日数分别为 9.89,1 3.5 6及 1 9.78d .结论 :rhTPO对部分难治性ITP及血液肿瘤化疗所致的血小板减少有一定疗效 。

AIM: To assess the clinical safety and efficiency of recombinant human thrombopoietin (rhTPO) for treatment of idiopathic and chemotherapy induced thrombocytopenia. METHODS: Four patients with non Hodgkin's lymphoma (NHL) entered randomized, cross controlled group, and 6 patients with acute leukemia for consolidation treatment entered non randomized, self controlled group. Also, 9 patients with refractory idiopathic thrombocytopenic purpura (ITP) were enrolled in the trial. rhTPO was daily administered subcutaneously at a dose of 1 μg/kg for a duration of 14 days. All the trial data were analyzed with statistical software SPSS 10.0. RESULTS: After therapy with rhTPO in patients with hematological malignancies, both median platelet count and median peak platelet count were increased; there was significant difference in median differential platelet count between trial cycle and control cycle ( P < 0.05); the days with platelet count <50×10 9/L and days for platelet recovering ≥75×10 9/L or ≥100×10 9/L were shorter to a different degree. After treatment of refractory ITP with rhTPO, the median platelet count gradually rose to maximum of 101×10 9/L; the median differential platelet count between before and after administration was 85.67×10 9/L; the days for platelet count ≥30×10 9/L, ≥50×10 9/L and ≥100×10 9/L were 9.89, 13.56 and 19.78, respectively. CONCLUSION: rhTPO seems to be safe and may attenuate idiopathic or chemotherapy induced thrombocytopenia without obviously adverse action.