Inherited fibrinogen disorders
Research type
Research Study
Full title
Evaluating the effects of concentrated fibrinogen replacement therapies in the management of inherited fibrinogen disorders.
IRAS ID
350394
Contact name
Nicola Curry
Contact email
Sponsor organisation
Oxford University Hospitals NHS Foundation Trust
Duration of Study in the UK
1 years, 11 months, 30 days
Research summary
Patients with the rare inherited bleeding disorders, afibrinogenaemia and dysfibrinogenaemia commonly require concentrated fibrinogen therapy to treat or prevent bleeding. Recent studies have highlighted that each type of fibrinogen treatment works a little differently, in particular in the degree to which a strong blood clot, that doesn't dissolve rapidly, is formed. We have recently studied the different effects of two commercial fibrinogen concentrates (Riastap®and Fibryga®) on clot formation in a patient with afibrinogenaemia. Our report suggests that the specific choice of fibrinogen treatment may be an important consideration for a clinician and their patient.
In-depth laboratory investigation is not routinely conducted for patients with inherited fibrinogen deficiency and is of particular importance in the setting of dysfibrinogenaemia. Dysfibrinogenemia is a highly variable condition, that can increase a patient's risk of thrombosis as well as bleeding (or neither). This variability increases the complexity of treatment decisions, particularly for surgery, where patients are exposed to bleeding risks, and where the treatment may increase their thrombosis risk. Post-operatively, all patients will have an increase from their baseline for thrombosis, and the overall balance of thrombosis vs. bleeding risk is often unclear.
Clinicians need different tools to help them decide which treatment to give patients according to the patient's condition, the bleeding and thrombosis risk, and the type of fibrinogen treatment. This study will evaluate the effects of two fibrinogen concentrates, and cryoprecipitate, on the clotting ability of the plasma of patients registered at the Oxford Haemophilia & Thrombosis Centre, with inherited fibrinogen disorders to learn how their condition responds to treatments.REC name
North West - Greater Manchester East Research Ethics Committee
REC reference
25/NW/0109
Date of REC Opinion
20 May 2025
REC opinion
Further Information Favourable Opinion