Factor VIII Computing, Berkeley, California, USA.
Bleeding & Clotting Disorders Institute, Peoria, Illinois, USA.
Haemophilia. 2023 May;29(3):761-769. doi: 10.1111/hae.14769. Epub 2023 Mar 15.
People with haemophilia rely on specialists for their care, yet the specific dosing regimens of treatments prescribed by these specialists have not been widely studied.
The objective of this study is to describe trends in clinician prescribing practices for the management of haemophilia in the United States (US).
We administered surveys to members of the Hemostasis and Thrombosis Research Society via paper surveys at its in-person annual symposia in 1999 and 2015, and an online survey in 2021. The surveys collected information on haemophilia treatments including factor dosing, inhibitor therapy and gene therapy.
Clinicians treating haemophilia for more than 50% of their practice time have increased from 37.5% of respondents in 1999 to 46.3% in 2021. Clinicians prescribing factor concentrates at >40 units/kg for routine bleeding events increased from 0% in 1999 to 29.3% in 2021 in haemophilia A (HA) and from 22.5% to 87.8% in haemophilia B (HB). In 2021, the clinicians reported prescribing emicizumab to treat HA patients (>89.5% paediatric, >85.7% adult) with or without inhibitors at least some of the time. Approximately 78.0% of respondents reported that they expected to recommend gene therapy at least some of time.
These data indicate changing trends in prescribing practices among US haemophilia specialists during the past 22 years. Preference for high doses of factor (>40 units/kg) has increased during this period. Emicizumab prophylaxis has been prescribed for patients with and without HA inhibitors. Clinicians expect gene therapy to have value for some haemophilia patients.
血友病患者依赖专家进行治疗，但这些专家开具的治疗方案的具体剂量方案尚未得到广泛研究。
本研究旨在描述美国（US）血友病专家管理实践中的临床医生处方趋势。
我们通过纸质调查向血栓止血研究学会成员进行了调查，该调查于 1999 年和 2015 年在其现场年会以及 2021 年的在线调查中进行。该调查收集了有关血友病治疗的信息，包括因子剂量、抑制剂治疗和基因治疗。
在过去 50%的时间里治疗血友病的临床医生的比例从 1999 年的 37.5%增加到 2021 年的 46.3%。在 2021 年，为常规出血事件开大于 40 单位/公斤的因子浓缩物的医生在 A 型血友病（HA）中从 1999 年的 0%增加到 29.3%，在乙型血友病（HB）中从 22.5%增加到 87.8%。2021 年，报告称至少在某些时候为有或无抑制剂的 HA 患者（>89.5%的儿科患者，>85.7%的成人患者）开具emicizumab。大约 78.0%的受访者报告称，他们预计至少在某些时候会推荐基因治疗。
这些数据表明，在过去的 22 年中，美国血友病专家的处方实践趋势发生了变化。在此期间，对高剂量因子（>40 单位/公斤）的偏好增加了。emicizumab 预防治疗已用于有和无 HA 抑制剂的患者。临床医生希望基因治疗对一些血友病患者有价值。
