Prophylaxis use of clotting factor replacement products in people with non‐severe haemophilia: A review of the literature

Abstract

Introduction: People with non-severe haemophilia appear to be under-treated in many countries, and this may lead to joint damage and worsen quality of life.Aim: To review literature for clotting factor replacement prophylaxis in people with non-severe haemophilia A and B (HA/HB) in relation to long-term outcomes to support clinical decision-making.Methods: A targeted literature search was performed to identify studies published between 2000 and 2021 that included prophylaxis in people with non-severe HA/HB and long-term outcomes, including annualized bleeding rates, joint health and quality of life.Results: Although eligible articles included 2737 and 2272 people with mild or moderate HA, respectively, only 22% (n = 609) and 29% (n = 668) reported treatment regimens. A total of 549 people with moderate HA were treated with factor replacement prophylaxis and were from high-income countries. On the contrary, nearly all people with mild HA received desmopressin (n = 599). Details of treatment regimens for women with haemophilia and people with HB were sparse. Three studies provided long-term outcomes for people with moderate haemophilia who received prophylaxis with factor concentrate, supporting early prophylaxis in people with a frequent bleeding phenotype regardless of their endogenous clotting factor level to preserve joint health. Conclusion:There remain large knowledge gaps when considering how to provide optimal treatment for people with non-severe haemophilia. Nonetheless, there is a strong rationale that prophylaxis should be considered early in life according to similar strategies as for severe haemophilia for those with a frequent severe bleeding phenotype. K E Y W O R D Sarthropathy, bleeding, episodic/on-demand treatment, non-severe (mild or moderate) haemophilia A or B, prophylactic FVIII or FIX, women with haemophilia (symptomatic carriers)This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.

The Evaluation of Clot Waveform Analyses for Assessing Hypercoagulability in Patients Treated with Factor VIII Concentrate

“…Regular prophylactic therapy including EHL-FVIII and emicizumab is common [20,21]. Treatment with emicizumab has become increasingly widespread [22,23], as emicizumab is quick and easy to inject.…”

Nonsevere Hemophilia: The Need for a Renewed Focus and Improved Outcomes

“…22 47 Evidence and knowledge gaps relating to use of factor prophylaxis in PWNSH have been the subject of recent discussion, with some clinicians describing a “strong rationale” for early prophylaxis in individuals experiencing frequent severe bleeds. 48 It is also appropriate to note that while recommendations for prophylaxis extend to those with a severe bleeding phenotype, 2 without formal definition of the term, it is important to ensure this does not inadvertently create a barrier to prophylaxis. It may, for instance, be needed to cover higher risk of bleeding, such as periods of anticoagulation/antiplatelet therapy in patients with mild hemophilia.…”

Blood-Induced Arthropathy: A Major Disabling Complication of Haemophilia

“…The primary goal of anti-haemophilic prophylaxis is to reduce and prevent joint bleeds and preserve joint health [ 84 ]. A study using data from the British Haemtrack registry, which involved 273 patients with haemophilia A or B, both adults and children, examined the occurrence of haemarthrosis during prophylaxis over one year [ 85 ].…”