Versiti Blood Research Institute, Milwaukee, Wisconsin.
Medical College of Wisconsin, Milwaukee, Wisconsin.
Am J Hematol. 2020 Aug;95(8):960-965. doi: 10.1002/ajh.25844.
With licensure of extended half-life (EHL) factor products and the changing landscape of available hemophilia products, patients and providers have options for less treatment-intense prophylaxis. The impact of these products in clinical practice to date remains understudied. We aimed to quantify the use of EHL products in prophylaxis in the US using the ATHN-dataset, a database of 145 ATHN-affiliated hemophilia treatment centers (HTCs). Further, we aimed to quantify the impact of EHL on key hemophilia indicators including annualized bleed rates (ABRs), hemophilia joint health scores (HJHS) and quality of life (QOL) metrics. The use of EHL vs standard half-life (SHL) products in severe hemophilia was compared between June 2018 and March 2019 using the ATHN-dataset. A cohort of patients was also recruited from seven participating HTCs in order to compare ABR, HJHS and QOL between product classes. By March 2019 the number of individuals with severe Hemophilia A (SHA) receiving EHLs remained relatively stable (28.4%), whereas the number of prescribed non-factor products increased to 7.1%, with a diminishing majority of patients (64.0%) continuing to receive SHLs. The majority of patients with severe hemophilia B (SHB) received treatment with EHLs including 57.5% by March 2019. There was a trend toward lower ABR with use of EHLs in SHA and SHB, although this did not result in improved HJHS nor QOL. EHL use in the United States in severe hemophilia continues to increase, although at a slower rate in SHA with the availability of non-factor therapy. The impact of the EHL therapies in clinical practice should continue to be examined prospectively.
随着长效（EHL）因子产品的许可和可用血友病产品的不断变化，患者和提供者有了更少治疗强度预防的选择。迄今为止，这些产品在临床实践中的影响仍在研究之中。我们旨在使用 ATHN 数据集（一个由 145 个 ATHN 附属血友病治疗中心组成的数据库）来量化美国 EHL 产品在预防治疗中的使用情况。此外，我们旨在量化 EHL 对关键血友病指标的影响，包括年化出血率（ABR）、血友病关节健康评分（HJHS）和生活质量（QOL）指标。使用 ATHN 数据集比较了 2018 年 6 月至 2019 年 3 月期间严重血友病患者中 EHL 与标准半衰期（SHL）产品的使用情况。还从七个参与的 HTC 招募了一个患者队列，以比较产品类别之间的 ABR、HJHS 和 QOL。到 2019 年 3 月，接受 EHL 治疗的严重血友病 A（SHA）患者人数相对稳定（28.4%），而处方非因子产品的数量增加到 7.1%，大多数患者（64.0%）继续接受 SHL。大多数严重血友病 B（SHB）患者接受 EHL 治疗，其中 57.5%的患者在 2019 年 3 月接受 EHL 治疗。尽管这并没有改善 HJHS 或 QOL，但在 SHA 和 SHB 中，使用 EHL 与 ABR 降低有关。在美国，严重血友病中 EHL 的使用继续增加，尽管随着非因子治疗的出现，SHA 的使用速度较慢。EHL 疗法在临床实践中的影响应继续进行前瞻性检查。
