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

Abstract

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.

Test–retest reliability of a mobile application of the patient reported outcomes burdens and experiences (PROBE) study

“…1 There are several comorbidities associated with haemophilia; the presence and severity of these comorbidities and the impact on health-related quality of life (HRQoL) increase with the more severe forms of hemophilia. 2,3 Typical treatment for the more severe forms of haemophilia has consisted of prophylaxis with clotting factor concentrates, or nonfactor therapies in wealthier countries. Gene therapy has been recently approved for both haemophilia A and B, but has not been a common therapy, yet.…”

“…Bleeds typically affect the joints, and repeated joint bleeds can result in arthrosis 1 . There are several comorbidities associated with haemophilia; the presence and severity of these comorbidities and the impact on health‐related quality of life (HRQoL) increase with the more severe forms of hemophilia 2,3 . Typical treatment for the more severe forms of haemophilia has consisted of prophylaxis with clotting factor concentrates, or nonfactor therapies in wealthier countries.…”

Transitioning from emicizumab prophylaxis to valoctocogene roxaparvovec gene therapy: A simulation study for individuals with severe haemophilia A

“…The mean trough concentration for all three dosing regimens is approximately 40 µg/mL 12 and is hypothesized to correspond to a similar level of haemostatic control as FVIII activity levels of 10–15 IU/dL 2,13 . Emicizumab is an approved therapy for SHA in the US and Europe that has been widely adopted for routine prophylaxis regardless of FVIII inhibitor status 14–16 …”

“…2,13 Emicizumab is an approved therapy for SHA in the US and Europe that has been widely adopted for routine prophylaxis regardless of FVIII inhibitor status. [14][15][16] Valoctocogene roxaparvovec is a gene therapy approved for SHA in the US and Europe 17,18 that uses an adeno-associated virus serotype 5 vector to transfer a B-domain-deleted human FVIII-coding sequence controlled by a liver-selective promoter. 19,20 In a phase 3, singlearm, open-label study (NCT03370913; GENEr8-1), 134 participants with SHA were treated with a single valoctocogene roxaparvovec infusion that supported endogenous FVIII production and reduced bleeding and FVIII use compared with FVIII prophylaxis.…”