Efficacy and safety of marstacimab prophylaxis in hemophilia A/B with inhibitors: results from the phase 3 BASIS trial.

We present efficacy and safety for individuals with inhibitors.

我们展示了有抑制物的个体的疗效和安全性。

The open-label, single-arm, phase 3 study evaluated once-weekly subcutaneous flat-dose marstacimab in males aged 12 to <75 years with severe hemophilia A or moderately severe to severe hemophilia B.

这项开放标签、单臂、III期研究评估了每周一次皮下注射固定剂量的marstacimab在12至<75岁患有严重血友病A或中度至严重血友病B的男性中的效果。

Participants with inhibitors received bypassing agents (on-demand or routine prophylaxis) during a 6-month observational phase (OP) before entering a 12-month active treatment phase (ATP) with marstacimab.

有抑制物的参与者在进入为期12个月的活性治疗阶段（ATP）之前，会在为期6个月的观察阶段（OP）接受旁路药物（按需或常规预防）治疗。

Primary end points were annualized bleeding rate (ABR) of treated bleeds and safety.

主要终点是处理出血的年化出血率(ABR)和安全性。

Of 60 participants with inhibitors in the OP, 51 entered the ATP and received marstacimab.

在OP中有60名携带抑制物的参与者，其中51名进入了ATP并接受了marstacimab治疗。

In the on-demand group (n = 48), mean estimated ABR declined from 19.78 (95% confidence interval[CI], 16.12-24.27) in the OP to 1.39 (95% CI, 0.85-2.29) during the ATP (ABR ratio, 0.07 [95% CI, 0.042-0.118]; 2-sided P< .0001).

在按需治疗组（n = 48）中，平均估算的年出血率（ABR）从开放标签研究（OP）期间的19.78（95%置信区间[CI]，16.12-24.27）下降到活性治疗期（ATP）期间的1.39（95% CI，0.85-2.29）（ABR比率，0.07 [95% CI，0.042-0.118]；双侧P< .0001）。

Results were consistent by hemophilia type (ABR ratio, 0.05 [hemophilia A, n = 40]; 0.13 [hemophilia B, n = 8]).

按血友病类型分层的结果是一致的（ABR比率，0.05 [血友病A，n = 40]；0.13 [血友病B，n = 8]）。

Participants reported significant improvements in health-related quality of life.

参与者报告了健康相关生活质量的显著改善。

Adverse events were common but mostly mild; 1 treatment-related grade 3 skin rash led to discontinuation.

不良事件很常见，但大多数是轻度的；1例与治疗相关的3级皮疹导致了治疗的中断。

Antidrug antibodies were detected in 19.6% of participants, with no apparent effect on efficacy or safety.

在参与者中检测到19.6%的抗药物抗体，但对疗效或安全性似乎没有明显影响。

In participants with inhibitors, marstacimab was associated with reduced bleeding rates and an acceptable safety profile, with no thromboembolic events.

在有抑制物的参与者中，marstacimab与降低出血率和可接受的安全性剖面相关，未发生血栓栓塞事件。

Marstacimab may be a viable treatment option for people with hemophilia A or B with inhibitors.

Marstacimab 可能是血友病A或B患者伴有抑制物的一个可行治疗选择。

This trial was registered at www.clinicaltrials.gov as #NCT03938792.

这项试验已在 www.clinicaltrials.gov 注册，注册号为 #NCT03938792。

ClinicalTrials.gov identifier: NCT03938792.

ClinicalTrials.gov 注册号：NCT03938792。