An Exploration of the Impact of Emicizumab on the Lives of People With Haemophilia and Inhibitors and Their Families

Study Overview
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Brief Summary
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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.

Official Title
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An Exploration of the Impact of Emicizumab on the Lives of People With Haemophilia and Inhibitors and Their Families

Conditions
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Hemophilia A With Inhibitor

Intervention / Treatment
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* Other: Qualitative Interview


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 8 Years
Maximum Age: 100 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| individuals With Haemophilia A and with inhibitors on emicizumab<br>Qualitative interviews | Other: Qualitative Interview<br>* A single one hour semi structured qualitative interview<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Real life experience | To capture the individual participant's and his family's experience of using emicizumab for haemophilia inhibitor therapy. | Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & analysed using a grounded theory approach. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Treatment satisfaction | To describe patient satisfaction with injections including frequency and numbers of injections, how to remember treatment dates, treatment comfort, bleed rate post switching. | Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & analysed using a grounded theory approach. | 
| Treatment expectations | To understand user's expectations of emicizumab and how they see future haemophilia care (less frequent injections, impact on home storage, number of bleeds, reduced hospitalisations etc). | Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & analysed using a grounded theory approach. | 
| Treatment impact | To describe the impact of the change in treatment on the extended family | Each participant & family member will take part in a 1 hour semi structured qualitative interview where experiences of his condition, previous treatment & current treatment with emicizumab will be discussed & analysed using a grounded theory approach. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Haemophilia, Quality of Life