From the voices of people with haemophilia A and their caregivers: Challenges with current treatment, their impact on quality of life and desired improvements in future therapies

Abstract

IntroductionHaemophilia A is a chronic disease requiring frequent intravenous infusions of recombinant factor VIII. Previous studies have shown that challenges associated with current treatments may have significant impacts on quality of life (QoL) that are as important as the health outcomes conferred by the therapy. Emerging therapeutic innovations offer the potential to mitigate treatment‐related challenges, and it is therefore important to develop a better understanding of patient and caregiver experiences with existing haemophilia A treatments in order to characterize the full value of new treatments.AimTo gather firsthand perspectives from people with haemophilia A (PWHA) and caregivers on the challenges with current treatment, their impact on QoL and desired improvements in future therapies.MethodsQualitative insights were gathered from 20 non‐inhibitor PWHA or caregivers of PWHA across Canada through one‐on‐one interviews; insights were further explored through focus group sessions to uncover overarching themes and prioritize issues with current treatments.ResultsPWHA and caregivers identified several challenges, including administration of intravenous infusions, coordination of treatment schedules and ensuring adequate medication and supplies. Participants described how these challenges impact psychosocial well‐being, physical health, personal/social life and work. Alternate modes of administration and longer‐lasting treatment effects were identified as desired improvements over current treatments.ConclusionThis study emphasizes the impact that existing haemophilia A treatments have on psychological well‐being, employment opportunities and adherence to treatment regimens. These considerations may help to inform decision‐making for policymakers and health systems around the true value of new therapies entering the haemophilia market.

Health‐related quality of life and caregiver burden of emicizumab in children with haemophilia A and factor VIII inhibitors—Results from the HAVEN 2 study

“…Haemophilia A (HA), a congenital bleeding disorder characterized by deficiency of coagulation protein factor (F)VIII, has a negative impact on the health-related quality of life (HRQoL) of affected people. [1][2][3] A clinical hallmark of severe HA is recurrent spontaneous bleeds, particularly into joints, and a substantial corresponding impact on physical health and HRQoL. [4][5][6][7][8] Approximately 30% of previously untreated persons with haemophilia A (PwHA) develop one of the most challenging complications of haemophilia treatment: neutralizing alloantibodies (inhibitors) against FVIII, [9][10][11] preventing effective FVIII prophylaxis.…”

<p>Clinical Evidence and Safety Profile of Emicizumab for the Management of Children with Hemophilia A</p>

“…It induces financial, technical, and educational challenges that considerably impact their quality of life. 60,61 Subsequently, it may affect adherence to prescribed treatments, 62,63 which may lead to an increased risk of bleeding and joint damage. 62 In addition, it has also been shown that frequent bleedings, especially in joints, and their long-term complications, alter the HA patients' and caregivers' health-related quality of life (HRQOL), especially on emotional, social, and physical aspects.…”

“…Alternate mode of administration and longer-lasting treatment effects were identified as being required to overcome the challenges represented by administration of IV infusion, and coordination of treatment schedules. 63 Therefore, no one can deny that the arrival of non-factor therapies such as emicizumab is revolutionizing the landscape of hemophilia.…”

A systematic review evaluating the efficacy and factor consumption of long-acting recombinant factor VIII products for the prophylactic treatment of hemophilia A

“…Frequent dosing can be a burden for patients and can negatively affect their quality-of-life, potentially resulting in a lack of treatment adherence that can compromise treatment effectiveness 5,6 . Maintaining FVIII trough levels above 1% is associated with a reduction in bleeding rate and so is often a target for prophylactic therapy.…”