Abstract Number: PB0684
Meeting: ISTH 2021 Congress
Background: Compared with people with Haemophilia A, people with haemophilia A and inhibitors (PWHi) have been disproportionately affected by their condition, suffering more orthopaedic complications, bleeding and pain. The advent of emicizumab as a prophylactic treatment has led to a reduction in bleed frequency and a significant improvement in overall quality of life. No research at this time has examined the nature of this improvement.
Aims: The Emi and Me study aims to capture the real-life experience of using emicizumab for PWHi and their families.
Methods: Participants were recruited through treatment centres, social media and word of mouth. Each participant and a family member, if available, took part in a semi-structured qualitative interview. All interviews were recorded, transcribed verbatim and analysed thematically.
All elements of the study were reviewed by local statutory authorities and informed consent was sought from all participants.
Results: 15 participants, mean age 27.2 years (range 8-63 years), along with a family member, participated in a single qualitative interview online (n=13), by telephone (n=1) or in person (n=1). Mean time on emicizumab was 2.22 years (range 1-5 years).
Six themes emerged: a reduction in bleed frequency; a reduction in treatment burden; an increased sense of freedom (for both PWHi and family members); decreased pain; enhanced wellbeing; and unachieved potential.
Emicizumab prophylaxis has delivered significant improvements in the lives of the participants. Despite this, some participants felt that pre-existing physical disabilities, as well as a lack of physiotherapy support has prevented them achieving the levels of functional ability they expected these improvements to facilitate.
Conclusions: This study suggests a more detailed understanding of the impact of treatments on the lives of PWHi and their families is required. Such an understanding is needed to determine patients’ true needs and may also help to identify more appropriate treatment outcomes to guide medical management.
To cite this abstract in AMA style:Fletcher S, Holland M, Jenner K, Khair K. Emi and Me: The Real-world Impact of Emicizumab on the Lives of People with Haemophilia A with Inhibitors and their Families [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/emi-and-me-the-real-world-impact-of-emicizumab-on-the-lives-of-people-with-haemophilia-a-with-inhibitors-and-their-families/. Accessed November 29, 2023.
« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/emi-and-me-the-real-world-impact-of-emicizumab-on-the-lives-of-people-with-haemophilia-a-with-inhibitors-and-their-families/