Clausen Cecilie Maria Lüthje, Funding Eva, Tolver Anders, Jarden Mary
Copenhagen Haemophilia Centre, Department of Hematology, Copenhagen University Hospital Rigshospitalet, Copenhagen, Denmark.
Department of Clinical Medicine, University of Copenhagen, Copenhagen, Denmark.
Eur J Haematol. 2025 Jan;114(1):164-172. doi: 10.1111/ejh.14324. Epub 2024 Oct 10.
New treatments for patients with bleeding disorders (PWB) have emerged, including products with extended half-life and subcutaneous administration. These less frequent treatments can potentially enhance quality of life (QoL), but adherence becomes critically important.
To investigate adherence and QoL among PWB and explore the correlation between treatment adherence and QoL in adult patients with haemophilia A (HA), haemophilia B (HB) and Von Willebrand disease (vWD) in Denmark.
This survey used disease-specific patient-reported questionnaires: Veritas-PRO and Veritas-PRN to measure adherence, and Haemo-A-QoL and VWD-QoL to assess QoL.
Responses were obtained from 149 patients with HA, 32 with HB and 118 with vWD. Adherence was reported by 87.1% of patients on prophylaxis and 71.2% of patients treated on demand, according to Veritas-PRO and Veritas-PRN cut-off scores. High QoL was generally reported, decreasing with age in HA and HB, but not in vWD.
Danish patients with HA, HB and vWD reported high QoL and high adherence to prescribed treatments. There was no correlation between treatment adherence and QoL among the different patient groups. These findings highlight the need for further research to better understand adherence behaviours and identify opportunities to further improve QoL in PWB.
针对出血性疾病患者（PWB）出现了新的治疗方法，包括半衰期延长的产品和皮下给药方式。这些给药频率较低的治疗方法有可能提高生活质量（QoL），但依从性变得至关重要。
调查丹麦成年甲型血友病（HA）、乙型血友病（HB）和血管性血友病（vWD）患者的依从性和生活质量，并探讨治疗依从性与生活质量之间的相关性。
本调查使用了针对特定疾病的患者报告问卷：Veritas-PRO和Veritas-PRN来测量依从性，Haemo-A-QoL和VWD-QoL来评估生活质量。
共获得149例HA患者、32例HB患者和118例vWD患者的回复。根据Veritas-PRO和Veritas-PRN的截断分数，87.1%接受预防治疗的患者和71.2%按需治疗的患者报告了依从性。总体报告的生活质量较高，在HA和HB中随年龄下降，但在vWD中没有。
丹麦的HA、HB和vWD患者报告生活质量高，对规定治疗的依从性高。不同患者组之间的治疗依从性与生活质量之间没有相关性。这些发现凸显了进一步研究的必要性，以更好地理解依从行为，并确定进一步改善出血性疾病患者生活质量的机会。
