Safety and efficacy of rilzabrutinib vs placebo in adults with immune thrombocytopenia: the phase 3 LUNA3 study.

The phase 3 LUNA3 study in previously treated adults with persistent/chronic ITP evaluated oral rilzabrutinib 400 mg twice daily (n = 133) vs placebo (n = 69) for 24 weeks.

在一项针对先前接受治疗的成人持续/慢性ITP患者的第三阶段LUNA3研究中，评估了每日两次口服400毫克的rilzabrutinib（n = 133）与安慰剂（n = 69）进行24周的治疗效果。

At baseline overall, median age was 47 years, 63% female, 7.7 year median ITP duration, and 28% prior splenectomy.

基线时总体中位年龄为47岁，女性占63%，中位免疫性血小板减少症(ITP)病程为7.7年，28%的患者曾接受过脾切除术。

Overall (N = 202), 85 (64%) rilzabrutinib and 22 (32%) placebo patients achieved platelet response (≥50 × 109/L or 30 × 109/L to <50 × 109/L and doubled from baseline) during the first 12 weeks and were eligible to continue.

在总体202名患者中，85名（64%）接受rilzabrutinib治疗和22名（32%）接受安慰剂治疗的患者在前12周内达到了血小板反应（≥50 × 10^9/L或30 × 10^9/L至<50 × 10^9/L，并且是从基线时的两倍），这些患者有资格继续治疗。

The primary end point, durable platelet response (platelet count ≥50 × 109/L for ≥two-thirds of ≥8 of the last 12 of 24 weeks without rescue therapy), was observed in 31 (23%) rilzabrutinib vs 0 placebo patients (P < .0001).

主要终点，持久的血小板反应（血小板计数≥50 × 10^9/L，持续时间≥三分之二的≥8周，共24周，无需救援治疗），在31名（23%）rilzabrutinib患者中观察到，而安慰剂组患者中为0（P < .0001）。

All secondary efficacy end points were significantly superior for rilzabrutinib (P < .05).

所有次要疗效终点对于rilzabrutinib均显著优越（P < .05）。

Median time to first platelet response was 15 days in rilzabrutinib responders.

rilzabrutinib反应者的首次血小板反应中位时间为15天。

Rilzabrutinib significantly reduced rescue therapy use by 52% (P = .0007) and improved week 25 bleeding scores (P = .0006).

rilzabrutinib显著减少了52%的救援治疗使用（P = .0007），并改善了第25周的出血评分（P = .0006）。

Improved physical fatigue was sustained from week 13 (P = .01) through 25 (P = .0003).

从第13周（P = .01）到第25周（P = .0003），身体疲劳的改善得到了持续。

Treatment-related adverse events were mainly grade 1/2.

治疗相关的不良事件主要是1级/2级。

One rilzabrutinib patient with multiple risk factors had serious treatment-related grade 3 peripheral embolism (lower left leg), and another died from unrelated pneumonia.

一位存在多种风险因素的rilzabrutinib患者出现了严重的与治疗相关的3级外周栓塞（左下肢），另一位患者因非相关肺炎死亡。

Rilzabrutinib in patients who failed multiple previous ITP therapies showed rapid and durable platelet response, reduced rescue medication and bleeding, improved physical fatigue, and favorable safety.

在之前接受过多次ITP治疗失败的患者中，rilzabrutinib显示出快速且持久的血小板反应，减少了急救药物的使用和出血事件，改善了身体疲劳，并具有良好的安全性。

Trial registration: www.clinicaltrials.gov (#NCT04562766) and www.clinicaltrialsregister.eu (#2020-002063-60).

试验注册信息：www.clinicaltrials.gov (#NCT04562766) 和 www.clinicaltrialsregister.eu (#2020-002063-60).