an exploration of the impact of emicizumab on the lives of people with haemophilia and inhibitors and their families

Conditions: Hemophilia A With Inhibitor

Summary: this study aims to examine the real-life experience and impact of using emicizumab in a cohort of patients with haemophilia and inhibitors, who were prescribed emicizumab as part of the early access to medicine schema (eams),those who have been in clinical trials and those now receiving emicizumab as part of routine haemophilia care. the investigators also intend to capture the impact of emicizumab use on the lives of close family members (parents/carers/children/partners/siblings). each participant and his family members will be deemed a study 'dyad'. this is a prospective, observational cohort qualitative research study to be conducted among patients using emicizumab in routine clinical practice. the study is designed to allow english-speaking patients and their families to tell their own life stories through narrative accounts. the narratives represent a true sharing of experiences and therefore offers insight into how these patients and families cope with haemophilia.

emicizumab offers patients protection from bleeding with fewer injections, this should reduce treatment burden. while patient expectation is high (as evidenced by the conversations on uk haemophilia social media sites) there are risks that patients may forget to treat and thus experience bleeds. during clinical trials participants have been monitored closely and, through questionnaire completion, have described improvement in quality of life \[mancuso et al, 2018, oldenburg et al 2019\]. what is also apparent to clinical teams is the dramatic change to life experience of not just the patient but his family - the ability to travel, to plan attending events knowing that bleeds will not occur, even potentially to have another child because of the reduction in treatment burden, particularly the time needed for treatment and rehabilitation. this therapy represents a substantial shift in the entire life experience of living with and managing haemophilia with inhibitors. as such, there is a need to look beyond the quantitative data collected in clinical trials and to assess the real impact of therapy on the everyday lives of patients and their families, gathered using qualitative research techniques. emicizumab has been available in the uk in clinical trials and as part of an early access to medicines scheme (eams) for non-trial eligible patients since spring 2018. it was recently licensed in the uk and is now available for routine clinical care for any person with haemophilia a and an inhibitor. we hypothesise that patients and their carers/families will be excited about this new treatment option and that for most, if not all, the promise of improved care and quality of life will be a reality.

Eligibility criteria:
inclusion criteria: * males * a diagnosis of haemophilia a with inhibitors * using emicizumab (prescribed by treating clinicians) in usual clinical care. exclusion criteria: * no history of a factor viii inhibitor * not currently being treated with emicizumab, * does not speak english (for the interviews) * does not consent to take part.

Keywords: haemophilia, quality of life