对艾法奈索托可α的治療滿意度和患者偏好優於先前的A型血友病治療：XTEND-1第3期臨床試驗結果
Treatment satisfaction and patient preference for efanesoctocog alfa over previous hemophilia A treatment: Results from the XTEND-1 phase 3 clinical trial.
摘要(中文译文)
血友病A的治疗包括凝血因子Ⅷ（FVIII）替代疗法的预防性治疗。采用标准半衰期和延长半衰期疗法时，患者仍会发生出血，且需每周给药2~4次，影响治疗依从性。Efanesoctocog alfa是一种每周一次的FVIII替代疗法，已获批用于治疗和预防所有年龄段血友病A患者的出血。旨在分析关键III期XTEND-1研究（NCT04161495）中，成人及青少年重型血友病A患者对每周一次efanesoctocog alfa与入组前预防性治疗方案的治疗偏好及满意度。通过治疗偏好调查评估第52周时的治疗偏好，并通过9项药物治疗满意度问卷（TSQM-9，涵盖总体满意度、有效性和便利性领域）评估基线和至第52周时的患者满意度，作为探索性事后患者报告结局分析。同时探讨治疗满意度及偏好与出血和地区之间的关系。在完成偏好调查的130例患者中，90.0%偏好efanesoctocog alfa而非其既往预防性治疗方案。按地区分析观察到相似趋势。最常见的原因包括“治疗频率更低”、“出血及出血相关并发症减少”和“感觉受到更好的保护”。在完成第52周TSQM-9评估的115例患者中，所有三个领域较基线均有统计学显著改善。至研究结束时，所有TSQM-9条目中选择“最佳应答”的患者比例均有所增加。第52周治疗满意度的改善与生活质量改善或出血事件之间无强相关性。大多数患者偏好预防性efanesoctocog alfa而非既往预防性治疗方案，且第52周时患者满意度得到改善。
Abstract
Treatments for Hemophilia A include prophylaxis with factor VIII (FVIII) replacement therapy. With standard and extended half-life therapies, patients still experience bleeds, and administration 2-4 times a week is needed, which affects adherence. Efanesoctocog alfa is a once-weekly FVIII replacement therapy approved to treat and prevent bleeding in patients with hemophilia A for all age groups. To analyze patient treatment preferences and satisfaction comparing once-weekly efanesoctocog alfa with pre-study prophylaxis in adults and adolescents with severe hemophilia A in the pivotal Phase 3 XTEND-1 study (NCT04161495). Exploratory post-hoc patient-reported outcomes were assessed by treatment preference at Week 52 (via a treatment preference survey) and patient satisfaction at baseline and up to Week 52 (via the 9-item Treatment Satisfaction Questionnaire for Medication [TSQM-9], covering: global satisfaction, effectiveness and convenience domains). Relationships between treatment satisfaction and preference versus bleeding and region were also explored. Of 130 patients who completed the preference survey, 90.0% preferred efanesoctocog alfa versus their previous prophylaxis regimen. Similar trends were observed by region. The most common reasons were 'less frequent treatment', 'bleeds and bleed-related complications reduction', and 'feel better protected'. Of 115 patients who completed the TSQM-9 at Week 52, there was a statistically significant improvement from baseline in all three domains. By study end, an increase in the proportion of patients selecting the "best response" for all TSQM-9 items was observed. There were no strong correlations with treatment satisfaction improvements at Week 52 and improvement in quality of life or bleeding episodes. Prophylactic efanesoctocog alfa was preferred to prior prophylactic regimens by most patients and improved patient satisfaction at 52 weeks.
如何引用
AMA
Emil Spasov, Doris Quon, Katsuyuki Fukutake, Annemieke Willemze, Jennifer Dumont, Helena Palmborg, et al. Treatment satisfaction and patient preference for efanesoctocog alfa over previous hemophilia A treatment: Results from the XTEND-1 phase 3 clinical trial.. Current therapeutic research, clinical and experimental. 2026; doi:10.1016/j.curtheres.2026.100839.
APA
Emil Spasov, Doris Quon, Katsuyuki Fukutake, Annemieke Willemze, Jennifer Dumont, Helena Palmborg, et al (2026). Treatment satisfaction and patient preference for efanesoctocog alfa over previous hemophilia A treatment: Results from the XTEND-1 phase 3 clinical trial.. Current therapeutic research, clinical and experimental. https://doi.org/10.1016/j.curtheres.2026.100839
GB/T 7714
Emil Spasov, Doris Quon, Katsuyuki Fukutake, Annemieke Willemze, Jennifer Dumont, Helena Palmborg, et al. Treatment satisfaction and patient preference for efanesoctocog alfa over previous hemophilia A treatment: Results from the XTEND-1 phase 3 clinical trial.[J]. Current therapeutic research, clinical and experimental, 2026 doi:10.1016/j.curtheres.2026.100839.
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