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| Name | Class |
|---|---|
| Roche Chugai | INDUSTRY |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| individuals With Haemophilia A and with inhibitors on emicizumab | Qualitative interviews |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Qualitative Interview | Other | A single one hour semi structured qualitative interview |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. |
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Inclusion Criteria:
Exclusion Criteria:
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Male participants with Hameophilia A and inhibitors currently on emicizumab prophylaxis and a member of their family
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| Name | Affiliation | Role |
|---|---|---|
| Simon P Fletcher, MA | Researcher | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Oxford University Hospitals NHS Foundation Trust | Oxford | Oxfordshire | OX3 7LE | United Kingdom |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34878209 | Background | Fletcher S, Jenner K, Holland M, Khair K. The lived experience of a novel disruptive therapy in a group of men and boys with haemophilia A with inhibitors: Emi & Me. Health Expect. 2022 Feb;25(1):443-454. doi: 10.1111/hex.13404. Epub 2021 Dec 8. |
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Individual participants and a family member (if available and consented) were interviewed as a dyad pair.
Individual participants with no available family member were interviewed on their own
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members | Qualitative interviews Qualitative Interview: A single one hour semi structured qualitative interview |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
16 people with haemophilia and inhibitors and 12 family members
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| ID | Title | Description |
|---|---|---|
| BG000 | Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members | Qualitative interviews Qualitative Interview: A single one hour semi structured qualitative interview |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | 16 People with Haemophilia and inhibitors were interviewed 12 with family members 14 interviews took place online, one by telephone and one in person. 6 themes emerged: bleeds; pain; treatment burden; control; freedom (for both PwHi and family members) and missed potential. | Posted | Number | participants | 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. |
|
Over the course of the 1 hour interview
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members | Qualitative interviews Qualitative Interview: A single one hour semi structured qualitative interview |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Simon Fletcher | Oxford university Hospitals NHS Foundation Trust | 01865 225316 | simon.fletcher@ouh.nhs.uk |
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 4, 2019 | Jan 15, 2021 | Prot_000.pdf |
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| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| 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 & thematically analysed and reported. |
| 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 & thematically analysed and reported. |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race and Ethnicity Not Collected | Race and Ethnicity were not collected from any participant. | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
Qualitative interviews Qualitative Interview: A single one hour semi structured qualitative interview |
|
|
| Secondary | 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. | People with haemophilia and inhibitors and their family members | Posted | Number | participants | 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. |
|
|
|
| Secondary | 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). | people with haemophilia and inhibitors and their family members | Posted | Number | participants | 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. |
|
|
|
|
| Secondary | Treatment Impact | To describe the impact of the change in treatment on the extended family | Posted | Number | participants | 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. |
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| 0 |
| 28 |
| 0 |
| 28 |
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| 28 |
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| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| Title | Measurements |
|---|---|
|