College of Pharmacy, Yonsei Institute of Pharmaceutical Sciences, Yonsei University, Incheon, South Korea.
CONNECT-AI Research Center, Yonsei University College of Medicine, Seoul, South Korea.
Haemophilia. 2021 Jan;27(1):e12-e21. doi: 10.1111/hae.14143. Epub 2020 Sep 7.
Haemophilia A patients with factor VIII inhibitors (HAPI) experience frequent spontaneous bleeding, approximately once a week, and require expensive bypassing agent (BPA) treatments to control bleeding over their lifetime. According to the HAVEN 1 trial, weekly emicizumab (Hemlibra®) prophylaxis injection reduces annualized bleeding rates (ABR) by 87% compared with BPA on-demand treatment (BPA-OD) administered at the time of bleeding. Our study aimed to assess the cost-effectiveness of emicizumab prophylaxis in HAPI in Korea.
Using a lifetime Markov model with health states of 'alive with bleeds' and 'dead', we simulated the experience of HAPI receiving emicizumab prophylaxis (treatment arm) or BPA-OD (control arm) and estimated expected clinical and economic outcomes under each treatment arm. Model parameters included comparative effectiveness, clinical and epidemiologic characteristics of Korean HAPI, costs of drug treatment and medical events and utility for 'alive with bleeds' state under each treatment. We utilized local data, including National Health Insurance claims data, national statistics, literature and expert surveys with haematologists.
Base-case analysis results showed that compared with BPA-OD, lifetime emicizumab prophylaxis prevented 807 bleedings, extended 3.04 quality-adjusted life-years and reduced costs by 2.6 million US dollars. Thus, emicizumab prophylaxis is a dominant treatment option with better effectiveness and lower costs than BPA-OD. A series of one-way sensitivity analyses consistently showed dominant results, confirming that lifetime emicizumab prophylaxis is a cost-saving intervention for HAPI.
Emicizumab prophylaxis is an excellent treatment choice reducing ABR, improving quality of life and reducing costs.
患有因子 VIII 抑制剂（HAPI）的血友病 A 患者经常自发性出血，大约每周一次，并且需要昂贵的旁路剂（BPA）治疗来控制其一生中的出血。根据 HAVEN 1 试验，与按需给予出血时的 BPA（BPA-OD）相比，每周给予emicizumab（Hemlibra®）预防注射可使年化出血率（ABR）降低 87%。本研究旨在评估emicizumab 在韩国 HAPI 中的成本效益。
使用具有“有出血生存”和“死亡”健康状态的终生马尔可夫模型，我们模拟了接受emicizumab 预防治疗（治疗组）或 BPA-OD（对照组）的 HAPI 的经历，并根据每种治疗方案估计了预期的临床和经济结果。模型参数包括比较疗效、韩国 HAPI 的临床和流行病学特征、药物治疗和医疗事件的成本以及每种治疗方案下“有出血生存”状态的效用。我们利用了包括国家健康保险索赔数据、国家统计数据、文献和血液病专家调查在内的当地数据。
基于案例分析结果显示，与 BPA-OD 相比，终生 emicizumab 预防可预防 807 次出血，延长 3.04 个质量调整生命年，并降低 260 万美元的成本。因此，emicizumab 预防是一种优于 BPA-OD 的治疗选择，具有更好的疗效和更低的成本。一系列单因素敏感性分析结果始终表明，emicizumab 预防是一种节省成本的 HAPI 干预措施。
emicizumab 预防是一种降低 ABR、改善生活质量和降低成本的优秀治疗选择。
