Efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia: stage 2 results from a multicenter phase III study
Efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia: stage 2 results from a multicenter phase III study
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DOI：
10.1080/09537104.2020.1847267
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发表时间：
2020-11-29
期刊：
影响因子：
3.3
通讯作者：
Yang, Renchi
中科院分区：
文献类型：
--
作者：
Liu, Xiaofan;Hou, Ming;Yang, Renchi
这项III期、随机化、安慰剂对照研究分为3个阶段（6周、随机化、安慰剂对照阶段1; 24周、开放标签阶段2;和连续扩展阶段3），评估了艾曲泊帕在中国慢性免疫性血小板减少症（ITP）患者中的长期疗效和安全性。本文介绍了第二阶段的结果。总体而言，150例患者（安慰剂-艾曲泊帕[P-E]，50例;艾曲泊帕-艾曲泊帕[E-E]，100例）接受开放标签艾曲泊帕治疗。中位血小板计数维持在41 × 10（9）/L至80 × 10（9）/L之间。两组中大多数患者（P-E，90.0%; E-E，81.8%）在艾曲泊帕治疗后至少一次达到血小板计数≥ 30 x 10（9）/L和≥ 2倍基线血小板计数。总体而言，32%的患者在≥ 75%的血小板计数评估中达到血小板计数≥ 50 × 10（9）/L。与基线相比，两组在第2阶段期间罕见出血和临床显著出血事件的趋势降低。在基线时接受≥ 1种ITP药物治疗的患者中，P-E组70.4%和E-E组40.8%减少或永久停用≥ 1种ITP药物。第2阶段结果进一步证明了艾曲泊帕在中国慢性ITP患者中的可持续长期疗效和良好耐受性，以及有利的获益-风险比。
This phase III, randomized, placebo-controlled study conducted in three stages (6-week, randomized, placebo-controlled stage 1; 24-week, open-label stage 2; and continuous extension stage 3) assessed the long-term efficacy and safety of eltrombopag use in Chinese patients with chronic immune thrombocytopenia (ITP). This article presents the results from stage 2. Overall, 150 patients (placebo-eltrombopag [P-E], 50; eltrombopag-eltrombopag [E-E], 100) received open-label eltrombopag. The median platelet count was maintained between 41 x 10(9)/L and 80 x 10(9)/L. Most patients in both groups (P-E, 90.0%; E-E, 81.8%) achieved platelet counts >= 30 x 10(9)/L and >= 2 times the baseline platelet count at least once with eltrombopag treatment. Overall, 32% of patients achieved platelet counts >= 50 x 10(9)/L in >= 75% of platelet count assessments. Both groups showed a decreased tendency to infrequent bleeding and clinically significant bleeding events during stage 2 compared with baseline. Among patients who received >= 1 ITP medication at baseline, 70.4% in the P-E group and 40.8% in the E-E group reduced or permanently stopped >= 1 of their ITP medications. The stage 2 results further demonstrated a sustainable long-term efficacy and good tolerability of eltrombopag with a favorable benefit-risk ratio in Chinese chronic ITP patients.