Trial Outcomes & Findings for An Exploration of the Impact of Emicizumab on the Lives of People With Haemophilia and Inhibitors and Their Families (NCT NCT04723693)

Results Overview

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.

Recruitment status

COMPLETED

Target enrollment

30 participants

Primary outcome timeframe

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.

Results posted on

2024-08-19

Participant Flow

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.

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

Participant milestones

Participant milestones
Measure	Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members Qualitative interviews Qualitative Interview: A single one hour semi structured qualitative interview
Overall Study STARTED	30
Overall Study COMPLETED	28
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members Qualitative interviews Qualitative Interview: A single one hour semi structured qualitative interview
Overall Study Withdrawal by Subject	2

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members n=28 Participants Qualitative interviews Qualitative Interview: A single one hour semi structured qualitative interview
Age, Categorical <=18 years	9 Participants n=28 Participants
Age, Categorical Between 18 and 65 years	19 Participants n=28 Participants
Age, Categorical >=65 years	0 Participants n=28 Participants
Sex: Female, Male Female	12 Participants n=28 Participants
Sex: Female, Male Male	16 Participants n=28 Participants
Region of Enrollment United Kingdom	28 participants n=28 Participants

PRIMARY outcome

Timeframe: 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.

Population: 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.

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.

Outcome measures

Outcome measures
Measure	Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members n=28 Participants Qualitative interviews 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) Bleeds	19 participants
Reduction in the Burden of the Condition Including Treatment Burden, Bleed Frequency, Pain, Control, Freedom and Missed Opportunities) Pain	10 participants
Reduction in the Burden of the Condition Including Treatment Burden, Bleed Frequency, Pain, Control, Freedom and Missed Opportunities) Treatment Burden	12 participants
Reduction in the Burden of the Condition Including Treatment Burden, Bleed Frequency, Pain, Control, Freedom and Missed Opportunities) Control	9 participants
Reduction in the Burden of the Condition Including Treatment Burden, Bleed Frequency, Pain, Control, Freedom and Missed Opportunities) Freedom	9 participants
Reduction in the Burden of the Condition Including Treatment Burden, Bleed Frequency, Pain, Control, Freedom and Missed Opportunities) Missed Opperyunities	2 participants

SECONDARY outcome

Timeframe: 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.

Population: People with haemophilia and inhibitors and their family members

To describe patient satisfaction with injections including frequency and numbers of injections, how to remember treatment dates, treatment comfort, bleed rate post switching.

Outcome measures

Outcome measures
Measure	Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members n=28 Participants Qualitative interviews Qualitative Interview: A single one hour semi structured qualitative interview
Treatment Satisfaction (Do the Participants and Their Family Feel That the Change in Treatment Improved Their Condition Control and in What Ways) Treatment Burden	9 participants
Treatment Satisfaction (Do the Participants and Their Family Feel That the Change in Treatment Improved Their Condition Control and in What Ways) Control	9 participants
Treatment Satisfaction (Do the Participants and Their Family Feel That the Change in Treatment Improved Their Condition Control and in What Ways) Freedom	9 participants

SECONDARY outcome

Timeframe: 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.

Population: people with haemophilia and inhibitors and their family members

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).

Outcome measures

Outcome measures
Measure	Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members n=28 Participants Qualitative interviews Qualitative Interview: A single one hour semi structured qualitative interview
Treatment Expectations Increased sense of opportunity on new treatment	26 participants
Treatment Expectations No improvement in opportunities	2 participants

SECONDARY outcome

Timeframe: 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.

To describe the impact of the change in treatment on the extended family

Outcome measures

Outcome measures
Measure	Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members n=12 Participants Qualitative interviews Qualitative Interview: A single one hour semi structured qualitative interview
Treatment Impact Increased control	6 participants
Treatment Impact Increased freedom	6 participants

Adverse Events

Individuals With Haemophilia A and With Inhibitors on Emicizumab and Family Members

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Simon Fletcher

Oxford university Hospitals NHS Foundation Trust

Phone: 01865 225316

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place