艾曲泊帕治疗成人慢性原发免疫性血小板减少症的疗效及安全性

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). CONCLUSION: The thrombopoietin receptor agonist eltrombopag was a suitable therapeutic option for Chinese adults with cITP.