Objective: To investigate the safety and efficacy of eltrombopag for adult patients with chronic immune thrombocytopenia (cITP). Methods: It was a randomised, single-centre, 6 weeks, placebo-controlled study. Beginning in January 29(th), 2013, 35 patients were enrolled, and the trial was completed on May 16(th), 2014. 17 patients were assigned to receive eltrombopag (starting dose 25 mg/d) and 18 were assigned to receive placebo. Results: A total of 35 cases of adult cITP, 6 males and 29 females with a median age of 42(22-66) years were enrolled. One patient withdrew from eltrombopag treatment group for the adverse event (AE) and discontinued treatment. In first two weeks, 27.78% (5/18) of placebo-treated compared with 64.71%(11/17) of eltrombopag-treated patients achieved platelet counts ≥ 30×10(9)/L(P=0.031); Treatment 6 weeks, the proportion of platelet counts reached ≥50×10(9)/L and ≥ 30×10(9)/L in eltrombopag-treated were higher than placebo-treated ones with statistically significant differences in both groups [64.71%(11/17) vs 11.11% (2/18), P=0.001; 76.47% (13/17) vs 38.89% (7/18), P=0.028]; The study also indicated a statistically significant difference in favour of eltrombopag compared with placebo in the odds of achieving the outcome of a platelet count ≥ 50×10(9)/L at least once during 6-week treatment (94.11% vs 33.33%, P<0.001), and 70.59%(12/17) of patients with the platelet count continuously ≥ 50×10(9)/L in 50% of treatment time in eltrombopag-treated group was more than placebo-treated one [11.11%(2/18), P<0.001]. Proportions of patients who required rescue treatment were 44.44% in placebo group and none in eltrombopag-treated one, respectively (P=0.002); The odds of bleeding symptoms with the WHO bleeding scale had no difference in both groups after 6 weeks (P=0.066). Adverse events that occurred more frequently due to eltrombopag than placebo included increased transaminase (3/17) and blood bilirubin (5/17), cerebral infarction(1/17). Conclusions: The thrombopoietin receptor agonist eltrombopag was a suitable therapeutic option for Chinese adults with cITP.
目的： 观察艾曲泊帕治疗成人慢性原发免疫性血小板减少症（ITP）的疗效及安全性。 方法： 2013年1月29日至2014年5月16日，纳入35例慢性ITP患者进行随机、双盲、安慰剂对照临床研究，以25 mg/d为起始剂量给予艾曲泊帕（17例）或安慰剂（18例），疗程为6周。 结果： 35例慢性ITP患者中男6例、女29例，中位年龄42（22~66）岁。艾曲泊帕组退组1例，其余患者均完成治疗。艾曲泊帕组在治疗开始2周内PLT≥30×10(9)/L的患者百分比高于安慰剂组[64.71%（11/17）对27.78%（5/18），P=0.031]。治疗第6周，艾曲泊帕组PLT≥50×10(9)/L、PLT≥30×10(9)/L患者百分比均高于安慰剂组[64.71%（11/17）对11.11%（2/18），P=0.001；76.47%（13/17）对38.89%（7/18），P=0.028]。艾曲泊帕组6周治疗期内至少1次PLT≥50×10(9)/L、50%时间PLT≥50×10(9)/L的患者百分比均高于安慰剂组[94.11%（16/17）对33.33%（6/18），P<0.001；70.59%（12/17）对11.11%（2/18），P<0.001]。安慰剂组8例（44.44%）患者治疗期间增加合并用药，艾曲泊帕组无增加合并用药病例（P=0.002）。治疗第6周WHO出血分级比较：艾曲泊帕组16例均为0级（退组1例未评估），安慰剂组0、1级分别为14、4例，两组差异无统计学意义（P=0.066）。与艾曲泊帕可能相关的不良事件包括转氨酶增高3例、胆红素增高5例、血小板升高相关脑梗死1例。 结论： 艾曲泊帕治疗成人慢性ITP起效时间较快且具有良好的安全性。.
Keywords: Eltrombopag; Outcome; Placebo; Thrombocytopenia.