无抑制剂的血友病A或B患者的Concizumab预防：来自3期explorer8研究的患者报告的结果

IF 3.4 3区医学 Q2 HEMATOLOGY

Research and Practice in Thrombosis and Haemostasis Pub Date : 2025-02-01 DOI:10.1016/j.rpth.2025.102705

Pantep Angchaisuksiri , Sylvia von Mackensen , Shashikant Apte , Gary Benson , Hermann Eichler , Amy Findley , Tadashi Matsushita , Camila M. Mazini Tavares , Morten Puggaard Ravn , Jameela Sathar , Laura Villarreal Martinez , Guy Young

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引用次数: 0

摘要

患者报告的结果（PROs）可以为患者对concizumab的看法提供有用的见解，concizumab是一种抗组织因子途径抑制剂单克隆抗体，用于血友病A （HA）或血友病B （HB）的每日一次皮下预防，有或没有抑制剂。目的评价3期explorer8研究（NCT04082429）的PROs。方法招募年龄≥12岁的HA/HB无抑制剂患者，按1:2随机分为无预防/按需治疗组（第1组）或包珠单抗预防组（第2组）或包珠单抗预防组（第3组和第4组）。PRO问卷包括36项简明健康调查版本2、成人血友病生活质量问卷、血友病治疗体验量表和血友病患者偏好问卷。结果1组和2组患者从基线到第24周的36项简短健康调查版本2“身体疼痛”和“身体功能”变化的估计治疗差异分别为9.5点（95% CI， 2.4至16.7）和0.3点（95% CI, - 5.1至5.6）。第1组和第2组患者在第24周的治疗差异估计为血友病成人生活质量问卷“总分”为- 18.0分（95% CI, - 26.4至- 9.5），“身体健康”为- 16.8分（95% CI, - 32.2至- 1.4）。血友病治疗经验测量和血友病患者偏好问卷调查结果倾向于使用concizumab预防而不是没有预防或以前的治疗。结论：来自3期explorer8研究的pro数据为HA/HB患者提供了额外的支持，与不进行预防相比，使用concizumab预防是一种治疗选择。

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Concizumab prophylaxis in people with hemophilia A or B without inhibitors: patient-reported outcome results from the phase 3 explorer8 study

Background

Patient-reported outcomes (PROs) can provide useful insights into patient perception of concizumab, an anti–tissue factor pathway inhibitor monoclonal antibody intended for once-daily, subcutaneous prophylaxis for hemophilia A (HA) or hemophilia B (HB), with and without inhibitors.

Objectives

To evaluate PROs from the phase 3 explorer8 study (NCT04082429).

Methods

Male patients aged ≥12 years with HA/HB without inhibitors were enrolled and randomized 1:2 to no prophylaxis/on-demand treatment (arm 1) or concizumab prophylaxis (arm 2) or allocated to concizumab prophylaxis (arms 3 and 4). PRO questionnaires included the 36-item short-form health survey version 2, Haemophilia Quality of Life Questionnaire for Adults, Hemophilia Treatment Experience Measure, and Haemophilia Patient Preference Questionnaire.

Results

Estimated treatment difference for change in 36-item short-form health survey version 2 “bodily pain” and “physical functioning” from baseline to week 24 between patients in arms 1 and 2 was 9.5 points (95% CI, 2.4 to 16.7) and 0.3 points (95% CI, −5.1 to 5.6), respectively. Estimated treatment difference at week 24 between patients in arms 1 and 2 was −18.0 points (95% CI, −26.4 to −9.5) for Haemophilia Quality of Life Questionnaire for Adults “total score” and −16.8 points (95% CI, −32.2 to −1.4) for “physical health.” Hemophilia Treatment Experience Measure and Haemophilia Patient Preference Questionnaire results favored concizumab prophylaxis over no prophylaxis or previous treatment.

Conclusion

PRO data from the phase 3 explorer8 study provided additional support for concizumab prophylaxis compared with no prophylaxis as a treatment option for patients with HA/HB.

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