Krishnan S, Vietri J, Furlan R, Duncan N
Biogen Idec, Weston, MA, USA.
Haemophilia. 2015 Jan;21(1):64-70. doi: 10.1111/hae.12533. Epub 2014 Dec 2.
Severe haemophilia is associated with bleeding into joints and development of arthropathy. Prophylactic treatment with infusion of replacement clotting factor is known to prevent bleeding, preserve joint functioning and result in higher health-related quality of life (HRQoL) than episodic treatment; however, adhering to standard prophylaxis schedules can be difficult, and little is known about the relationship between adherence to prophylactic treatment and outcomes. The aim of this study was to assess the relationship between self-reported adherence to prophylaxis and health outcomes, including HRQoL and bleeding episodes. Adults with haemophilia (n = 55) and caregivers of children with haemophilia (n = 55) in Australia, Canada, and the United States completed an online questionnaire which included measures of HRQoL (SF-12v2 for adults and SF-10 for caregivers of children), self-reported bleeding episodes, and the VERITAS-Pro measure of adherence to prophylaxis in haemophilia. Regression analysis was used to test the association between VERITAS-Pro total score and outcomes. Poorer adherence (higher VERITAS-Pro scores) was associated with a greater number of self-reported bleeding episodes in the past year among adults (p < 0.01), more days of work/school missed among paediatric patients (p < 0.01), and lower physical health status scores among paediatric patients (p < 0.05). This study highlights the benefits of adherence to prophylaxis among those with severe haemophilia and provides evidence for the utility of the VERITAS-Pro by demonstrating a relationship between adherence and outcomes.
重度血友病与关节出血及关节病的发展有关。已知输注替代凝血因子进行预防性治疗可预防出血、保持关节功能，并比按需治疗带来更高的健康相关生活质量（HRQoL）；然而，坚持标准的预防方案可能很困难，而且对于坚持预防性治疗与治疗结果之间的关系知之甚少。本研究的目的是评估自我报告的预防治疗依从性与健康结果之间的关系，包括HRQoL和出血事件。澳大利亚、加拿大和美国的成年血友病患者（n = 55）及血友病患儿的照料者（n = 55）完成了一份在线问卷，其中包括HRQoL测量（成人用SF-12v2，患儿照料者用SF-10）、自我报告的出血事件，以及血友病预防治疗依从性的VERITAS-Pro测量。采用回归分析来检验VERITAS-Pro总分与结果之间的关联。依从性较差（VERITAS-Pro得分较高）与成人过去一年中自我报告的出血事件数量较多（p < 0.01）、儿科患者错过工作/上学的天数较多（p < 0.01）以及儿科患者的身体健康状况得分较低（p < 0.05）相关。本研究强调了重度血友病患者坚持预防治疗的益处，并通过证明依从性与治疗结果之间的关系，为VERITAS-Pro的效用提供了证据。
