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. 2019 May;25(3):433-440.
doi: 10.1111/hae.13754. Epub 2019 Apr 23.

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

Ryan E Wiley  1 , Charles P Khoury  1 , Adrian W K Snihur  1 , Marni Williams  1 , David Page  2 , Nicole Graham  3 , Lori Laudenbach  4 , Cindy Milne-Wren  5 , Jayson M Stoffman  6
Affiliations

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

Ryan E Wiley et al. Haemophilia. 2019 May.

Abstract

Introduction: Haemophilia 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.

Aim: To 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.

Methods: Qualitative 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.

Results: PWHA 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.

Conclusion: This 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.

Keywords: adherence; employment; haemophilia; psychological well-being; quality of life; treatment.

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Conflict of interest statement

The study was sponsored by Hoffmann‐La Roche Limited (Canada). JMS has performed consultancy work for Bayer Inc and Hoffmann‐La Roche Ltd. Shift Health consults with organizations across the health and life sciences sector, including Hoffmann‐La Roche Limited. Authors from Shift Health (REW, CPK, AWKS, MW) were employed under contract with Hoffmann‐La Roche Limited (Canada) for the purposes of this study. All other authors have no competing interests.

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