ECHO: Expanding Communications on Hemophilia-A Outcomes
Research type
Research Study
Full title
A prospective, international, longitudinal, observational disease registry of patient-reported outcomes (PROs), and the association with Hemophilia A and its treatment in patients with moderate to severe Hemophilia A.
IRAS ID
173689
Contact name
Andrew Nolan
Contact email
Sponsor organisation
Bayer Plc
Duration of Study in the UK
5 years, 0 months, 0 days
Research summary
Haemophilia affects the body’s ability to clot blood and stop bleeding. In a person without haemophilia, clotting factors circulate in the blood accumulate at the site of a cut or injury, and form a blood clot. In people with haemophilia, there isn’t enough of these factor to form a blood clot to stop the bleeding.
There are several types of clotting factor and people with haemophilia A do not make enough clotting factor VIII, whereas people with haemophilia B do not have enough factor IX.
Haemophilia A can be classed as mild, moderate or severe.
People with severe haemophilia usually bleed frequently into their muscles or joints. They may bleed one to two times per week. Bleeding is often spontaneous, which means it happens for no obvious reason.
People with mild and moderate haemophilia bleed less frequently, about once a month. They may bleed for a long time after surgery, a bad injury, or dental work. A person with mild or moderate haemophilia will rarely experience spontaneous bleeding.
Haemophilia A is a manageable condition, because the majority of sufferers are treated by replacing the missing clotting factor VIII in the blood through an intravenous infusion of clotting factor.
Limited research has been completed to understand how patients view these treatments and the impact they have upon their lives. Therefore the purpose of this registry is to characterise the treatment burden of haemophilia upon patients with moderate to severe Haemophilia A.
This will be determined by collecting information about a patient's well-being such as quality of life, physical impairment and bleeding patterns.
The registry will also collect data to help understand how often patients are seen by a healthcare professional and the clinical resources needed to treat their illness.
REC name
East of England - Cambridgeshire and Hertfordshire Research Ethics Committee
REC reference
15/EE/0301
Date of REC Opinion
6 Oct 2015
REC opinion
Further Information Favourable Opinion