Impact of Emicizumab on Mental Liberation in Adult Patients with Severe and Moderate Factor VIII Deficiency

The treatment landscape of hemophilia has been evolving rapidly over the past few decades. One of these innovations is emicizumab (Hemlibra®), a bispecific antibody mimicking the cofactor activity of coagulation Factor VIII (FVIII). Unlike classical replacement therapy with FVIII concentrates, emicizumab is administered subcutaneously on a weekly basis or every 2 to 4 weeks, providing stable hemostatic activity. Several advantages of emicizumab have been reported: improved access to prophylaxis for persons with hemophilia (PWH) unable to perform regular intravenous infusions, including those with an inhibitor, better hemostatic control as reflected by a lower bleeding rate, and a positive impact on pain and quality of life.

Beyond these physical benefits, emicizumab should allow PWH to be mentally liberated from the disease. The concept of a "hemophilia-free mind" has been recently proposed to describe how new treatments for hemophilia could liberate patients not only from the physical but also the mental burden of the disease and its treatment. In routine clinical practice, asking PWH if they currently experience days when their minds are not preoccupied by hemophilia has been proposed as a first step to evaluate and validate this concept.

A survey was recently conducted among all 80 adult patients (>18) with severe (69) and moderate (11) hemophilia A treated with emicizumab for at least 6 months, representing 80 % of the total number of severe and moderate PWH followed at the Hemophilia Treatment Center of the Cliniques universitaires Saint-Luc in Brussels. An online questionnaire via SurveyMonkey was sent to 74 patients reachable by email. Fifty patients (67 %) provided anonymous responses that were collectively analyzed. Thirty-nine patients (78%) reported experiencing days without thinking about hemophilia and its treatment. The number of "hemophilia-free mind" days was 5.2/5.5 (mean/median) per week and 23.2/27.5 (mean/median) per month. The main obstacles that prevented PWH from being totally mentally liberated were musculoskeletal pain (26/50, 52%), restricted articular ranges of motion (14/50, 28%), fear of bleeding (6/50, 12%), bleeding events (1/50, 2%), and other non-specified limitations imposed by hemophilia (3/50, 6%). The ability of emicizumab to provide more freedom to perform daily tasks and activities was rated as weak (6/50, 12%), important (25/50, 50%), or major (19/50, 38%).

This study clearly demonstrates for the first time the major potential of a new treatment such as emicizumab to liberate adult PWH not only from the physical but also the mental burdens of hemophilia and supports the value of recording the number of "hemophilia-free mind" days. Musculoskeletal pain and functional limitations represent major obstacles to this ambition, emphasizing the importance of pain and musculoskeletal management and care in the era of new therapies. This potential for mental liberation should be evaluated in young patients on emicizumab as well as in patients of all ages currently benefiting from other new treatment options of hemophilia (rebalancing agents, ultra-long FVIII, and gene therapy).

References :

Krumb E and Hermans C. Living with a "hemophilia-free mind" - The new ambition of hemophilia care? Res Pract Thromb Haemost. 2021 Aug 3;5(5):e12567

Hermans C and Pierce GF. Towards achieving a haemophilia-free mind. Haemophilia. 2023 Jul;29(4):951-953

Hermans C, Krumb E, Rotellini D, Pierce GF. The underevaluated impacts of the therapeutic revolution of hemophilia on women and girls. J Thromb Haemost. 2024 Apr;22(4):915-918