Emi and me
Research type
Research Study
Full title
Emi and Me. An Exploration of the Impact of Emicizumab on the Lives of People with Haemophilia and Inhibitors and their Families
IRAS ID
248511
Contact name
Simon Fletcher
Contact email
Sponsor organisation
Haemnet
Duration of Study in the UK
0 years, 11 months, 31 days
Research summary
Summary of Research
Haemophilia A is a rare disorder caused by a gene defect resulting in a deficiency in factor VIII (FVIII) which is important in blood clotting. Treatment includes replacing the missing FVIII to treat and prevent bleeds. Unfortunately approximately 30% of people who are treated with FVIII develop an antibody (or inhibitor) to it, as their body sees this as a 'foreign protein'; this means that the factor is rendered inactive. Treatment of inhibitors is with very intensive treatment which works for about 70% of those affected.
For the remaining 30% treatment of bleeds is extremely difficult, using 'bypassing agents' which bypass FVIII in the way the blood clots; despite this many people still experience severe bleeding, predominantly in to the joints which is painful and causes early arthritic damage. All treatment has to be given into a vein, usually daily, which causes treatment burden for the person with haemophilia and his caregivers.
Emicizumab is a newly available drug which is used for inhibitor treatment, it is given as an injection under the skin as little as once a month and offers protection from bleeding with fewer injections. This should reduce treatment burden. This therapy represents a substantial change in the entire experience of living with and managing haemophilia with inhibitors for the patient and his family. The results from clinical trials of Emicizumab are astounding with reported improved quality of life for patients measured using quality of life questionnaires.
In this study we will look beyond the data collected about patients in clinical trials to assess the real impact of Emicizumab therapy on the everyday lives of patients and their families. We will do this using face-to-face interviews of patients/family groups. The interviews with be analysed to describe the impact of this new treatment for this group of patients and their families.
Summary of Results
Fifteen people with haemophilia and thirteen family members took part in a single interview online, ia telephone or in person.
Six major themes emerged: bleeds: pain; treatment burden; control; freedom and missed potential. Emicizumab prophylaxis delivered significant improvements in the lives of participants but despite these improvements some participants felt that their pre-existing physical disabilities and the lack of physiotherapy provision had prevented them achieving similar improvements in their functional ability.
The study has shown that in reducing bleeds, pain and treatment burden, emicizumab has given people with haemophilia and inhibitors greater control over their condition, allowing a sense of freedom they had not experienced with FVIII or bypassing agent prophylaxis. For emicizumab to be truly effective, there is a need to ensure the continued availability and accessibility of robust multidisciplinary support services. without this, it is unlikely that people with haemophilia and inhibitors will realise the life changing potential offered either by emicizumab or any other novel treatment approach.REC name
London - Chelsea Research Ethics Committee
REC reference
19/LO/1592
Date of REC Opinion
7 Jan 2020
REC opinion
Further Information Favourable Opinion