High-dose dexamethasone plus recombinant human thrombopoietin vs high-dose dexamethasone alone as frontline treatment for newly diagnosed adult primary immune thrombocytopenia: A prospective, multicenter, randomized trial
High-dose dexamethasone plus recombinant human thrombopoietin vs high-dose dexamethasone alone as frontline treatment for newly diagnosed adult primary immune thrombocytopenia: A prospective, multicenter, randomized trial
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高剂量地塞米松联合重组人血小板生成素与单用高剂量地塞米松作为新诊断成人原发性免疫性血小板减少症一线治疗的比较：一项前瞻性、多中心、随机试验
DOI：
10.1002/ajh.25989
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发表时间：
2020-10-19
影响因子：
12.8
通讯作者：
Hou, Ming
中科院分区：
文献类型：
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作者：
Yu, Yafei;Wang, Miaomiao;Hou, Ming
我们进行了一项前瞻性、多中心、随机、对照临床试验，以比较大剂量地塞米松（HD-DXM）联合重组人血小板生成素（rhTPO）与单用HD-DXM治疗新诊断的成人免疫性血小板减少症（ITP）患者的疗效和安全性。入选患者随机分配接受DXM + rhTPO或DXM单药治疗。如果两组在第10天没有达到反应，则重复另一个4天的DXM疗程。HD-DXM + rhTPO组的100例患者和HD-DXM单药治疗组的96例患者被纳入全分析集。HD-DXM联合rhTPO组初治缓解率（89.0%vs66.7%，P <0.001）和完全缓解率（75.0%vs42.7%，P <0.001）均高于HD-DXM单药组。HD-DXM联合rhTPO组6个月的缓解率也高于HD-DXM单药治疗组（51.0%vs 36.5%，P = 0.02;持续CR：46.0%vs 32.3%，P = 0.043）。在整个随访期间，根据Kaplan-Meier分析估计，HD-DXM + rhTPO组的总体缓解持续时间长于HD-DXM单药治疗组（P= .04）。研究药物通常耐受良好。总之，HD-DXM与rhTPO的联合治疗显著改善了初诊ITP患者的初始应答，并产生了良好的SR，因此可以进一步验证为ITP的一线治疗。本研究注册为标识符：NCT 01734044。
We conducted a prospective, multicenter, randomized, controlled clinical trial to compare the efficacy and safety of high-dose dexamethasone (HD-DXM) plus recombinant human thrombopoietin (rhTPO), vs HD-DXM alone in newly diagnosed adult immune thrombocytopenia (ITP) patients. Enrolled patients were randomly assigned to receive DXM plus rhTPO or DXM monotherapy. Another 4-day course of DXM was repeated if response was not achieved by day 10 in both arms. One hundred patients in the HD-DXM plus rhTPO arm and 96 patients in the HD-DXM monotherapy arm were included in the full analysis set. So, HD-DXM plus rhTPO resulted in a higher incidence of initial response (89.0% vs 66.7%,P < .001) and complete response (CR, 75.0% vs 42.7%,P < .001) compared with HD-DXM monotherapy. Response rate at 6 months was also higher in the HD-DXM plus rhTPO arm than that in the HD-DXM monotherapy arm (51.0% vs 36.5%,P= .02; sustained CR: 46.0% vs 32.3%,P= .043). Throughout the follow-up period, the overall duration of response was greater in the HD-DXM plus rhTPO arm compared to the HD-DXM monotherapy arm (P= .04), as estimated by the Kaplan-Meier analysis. The study drugs were generally well tolerated. In conclusion, the combination of HD-DXM with rhTPO significantly improved the initial response and yielded favorable SR in newly diagnosed ITP patients, thus could be further validated as a frontline treatment for ITP. This study is registered as identifier: NCT01734044.