Concizumab in hemophilia with inhibitors: longer-term efficacy and safety results from the phase 3 explorer7 study.

Results from the phase 3 explorer7 study confirmed superiority of concizumab prophylaxis over no prophylaxis in reducing the annualized bleeding rate (ABR) in people with hemophilia A or B with inhibitors (HAwI/HBwI).

第三阶段explorer7研究的结果证实，在减少有抑制物的血友病A或B患者（HAwI/HBwI）的年化出血率（ABR）方面，Concizumab预防治疗优于无预防治疗。

Male patients aged ≥12 years were randomized 1:2 to no prophylaxis (group 1) or concizumab prophylaxis (group 2), or nonrandomly allocated to concizumab prophylaxis (groups 3 and 4).

年龄≥12岁的男性患者按1:2的比例随机分为无预防组（第1组）或concizumab预防组（第2组），或非随机分配至concizumab预防组（第3组和第4组）。

After ≥24 weeks of treatment, patients in group 1 could switch to concizumab prophylaxis.

治疗≥24周后，第1组的患者可以转换为concizumab预防治疗。

At the 56-week cutoff (defined as when all patients in groups 2-4 had completed the visit at 56 weeks or permanently discontinued treatment), bleed-related efficacy, pharmacokinetics and pharmacodynamics, and safety were assessed.

在56周的截止日期（定义为所有第2-4组患者已完成56周的访问或永久停止治疗时），评估了与出血相关的疗效、药代动力学和药效学以及安全性。

Of the 133 patients enrolled (HAwI, n = 80; HBwI, n = 53), 114 received concizumab prophylaxis (groups 2-4) and 19 were randomized to no prophylaxis (group 1).

在入组的133名患者中（HAwI，n = 80；HBwI，n = 53），有114人接受了concizumab预防治疗（第2-4组），另外19人被随机分配到无预防治疗组（第1组）。

After ≥24 weeks, 13 patients from group 1 switched to concizumab.

在≥24周后，组1中的13名患者改用concizumab治疗。

Median ABR for treated spontaneous and traumatic bleeding episodes in patients receiving concizumab was 0.8 (interquartile range [IQR], 0.0-3.2) at the 56-week cutoff, consistent with the low bleeding rates (median ABR, 0.0; IQR, 0.0-3.3) at the 32-week cutoff.

在接受concizumab治疗的患者中，自发性和创伤性出血事件的中位年出血率（ABR）在56周时为0.8（四分位数范围[IQR]，0.0-3.2），与32周时的低出血率（中位ABR，0.0；IQR，0.0-3.3）一致。

Concizumab and free-TFPI concentration remained stable over time.

Concizumab 和游离 TFPI 浓度随时间保持稳定。

No new safety concerns were reported.

未报告新的安全问题。

Longer-term (≥1 year) efficacy and safety results of concizumab prophylaxis for HAwI/HBwI were consistent with the 32-week cutoff results in explorer7.

在对HAwI/HBwI进行concizumab预防的长期（≥1年）疗效和安全性结果与explorer7试验中32周截止点的结果一致。

This trial was registered at www.clinicaltrials.gov as #NCT04083781.

该试验已在www.clinicaltrials.gov上注册，注册号为#NCT04083781。