An observational study in Hemophilia A With Inhibitor, sponsored by Haemnet. Completed at 1 site in United Kingdom. Open to male participants aged 8 Years to 100 Years. Per ClinicalTrials.gov, last updated 2024-08-19.
Sponsored by Haemnet · Observational
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.
866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.
This study's enrollment of 30 is below the median of 80 across 314 observational studies indexed under Hemophilia A.
Browse Hemophilia A studies →Haemnet is the lead sponsor of 5 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Male participants with Hameophilia A and inhibitors currently on emicizumab prophylaxis and a member of their family
Exclusion Criteria:
Qualitative interviews
Other: Qualitative Interview
A single one hour semi structured qualitative interview
Reduction in the Burden of the Condition Including Treatment Burden, Bleed Frequency, Pain, Control, Freedom and Missed Opportunities)
To capture the individual participant's and his family's experience of using emicizumab for haemophilia inhibitor therapy and see if there is any reduction in the burden of the condition.
Time frame: 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 & thematically analysed and reported.
Treatment Satisfaction (Do the Participants and Their Family Feel That the Change in Treatment Improved Their Condition Control and in What Ways)
To describe patient satisfaction with injections including frequency and numbers of injections, how to remember treatment dates, treatment comfort, bleed rate post switching.
Time frame: 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 & thematically analysed and reported.
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).
Time frame: 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 & thematically analysed and reported.
Treatment Impact
To describe the impact of the change in treatment on the extended family
Time frame: 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 & thematically analysed and reported.
Study participants will be people with inhibitors using emicizumab (prescribed by treating clinicians) in usual clinical care. Family members of participants will also be included.
| Milestone | Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members |
|---|---|
| Started | 30 |
| Completed | 28 |
| Not completed | 2 |
| Withdrew: Withdrawal by subject | 2 |
To capture the individual participant's and his family's experience of using emicizumab for haemophilia inhibitor therapy and see if there is any reduction in the burden of the condition.
| participants | Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members |
|---|---|
| Bleeds | 19 |
| Pain | 10 |
| Treatment Burden | 12 |
| Control | 9 |
| Freedom | 9 |
| Missed Opperyunities | 2 |
To describe patient satisfaction with injections including frequency and numbers of injections, how to remember treatment dates, treatment comfort, bleed rate post switching.
| participants | Individuals With Haemophilia A and With Inhibitors on Emicizumab |
|---|---|
| Treatment Burden | 9 |
| Control | 9 |
| Freedom | 9 |
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).
| participants | Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members |
|---|---|
| Increased sense of opportunity on new treatment | 26 |
| No improvement in opportunities | 2 |
To describe the impact of the change in treatment on the extended family
| participants | Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members |
|---|---|
| Increased control | 6 |
| Increased freedom | 6 |
Collected over Over the course of the 1 hour interview. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members | 0/28 (0%) | 0/28 (0%) | 0/28 (0%) |
16 people with haemophilia and inhibitors and 12 family members
| Age, Categorical(Participants) | Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members |
|---|---|
| <=18 years | 9 |
| Between 18 and 65 years | 19 |
| >=65 years | 0 |
| Sex: Female, Male(Participants) | Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members |
|---|---|
| Female | 12 |
| Male | 16 |
| Race and Ethnicity Not Collected(Participants) | Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members |
|---|
| Region of Enrollment(participants) | Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members |
|---|---|
| United Kingdom | 28 |
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Plan to share: No
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