Trends in prescribing practices for management of haemophilia: 1999–2021

• Published in: Haemophilia : the official journal of the World Federation of Hemophilia Vol. 29; no. 3; pp. 761 - 769
• Main Authors: Curtis, Randall, Roberts, Jonathan C., Crook, Nicole, Decker‐Palmer, Marquita, Khainar, Rahul, Baker, Judith R., Ullman, Megan, Koerper, Marion A., Wu, Joanne, Nichol, Michael B.
• Format: Journal Article
• Language: English
• Published: England Wiley Subscription Services, Inc 01.05.2023
• Subjects: Adult; Antibodies, Bispecific - therapeutic use; Child; clotting factor dose trend; Dosage; emicizumab; Factor IX deficiency; Factor VIII - therapeutic use; Gene therapy; haemophilia; Hemophilia; Hemophilia A - drug therapy; Hemophilia B - drug therapy; Hemorrhage - drug therapy; Hemostasis; Humans; inhibitor; Polls & surveys; prescribing practices; Prophylaxis; Surveys; Thrombosis; Trends; United States > US; emicizumab; haemophilia; clotting factor dose trend; inhibitor; prophylaxis; prescribing practices
• ISSN: 1351-8216; 1365-2516
• DOI: 10.1111/hae.14769

Introduction 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. Aim The objective of this study is to describe trends in clinician prescribing practices for the management of haemophilia in the United States (US). Methods 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. Results 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. Conclusion 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.