Long term outcome of Fanhdi® in Haemophilia A patients in London
Research type
Research Study
Full title
Long term outcome of Fanhdi® in Haemophilia A patients in London
IRAS ID
157221
Contact name
Savita Rangarajan
Contact email
Sponsor organisation
Hampshire Hospitals NHS Foundation Trust
Research summary
FVIII factor replacement therapy has revolutionised the care of patients with Haemophilia (PWH) in successfully treating and preventing bleeding episodes in these patients. FVIII concentrates have traditionally been derived from plasma of healthy donors. In the 1970-80s the epidemic in the transmission of blood born viruses from virally inactivated plasma derived coagulation factors drove forward the development and use of recombinant products which have now been routine for the last 10 years.
Whilst plasma derived Factor VIII concentrate (pdFVIII) (Fanhdi®) remains the mainstay of factor replacement in many parts of the world, the use of such a product long term in the UK has been overshadowed by the use of recombinant FVIII products. Despite this, pdFVIII products still have a vital role in the modern management of PWH. For example, it has often been suggested that FVIII product type may influence the risk of inhibitor development and the success rate of immune tolerance induction (ITI). For these reasons, pdFVIII is frequently considered in select groups of patients. Hence it is important to review our long-term data on Fanhdi®, a product that is likely to continue to have a role in management of PWH in the foreseeable future. Furthermore, at the time there was little experience with the newer recombinant forms.
Unique to St Thomas’ Hospital in London, there are around 60 patients who did not swap to a recombinant form of FVIII concentrate and have remained on a pdFVIII concentrate (Fanhdi®) for prophylaxis as well as treatment of bleeding events. The aim of the study is to determine the safety, efficacy and tolerability record and perceived inhibitor risk of Fanhdi® therapy in these patients. This will be determined by retrospective case note analysis and completion of the Quality of Life Questionnaire and Haemophilia Activities List.REC name
East of England - Cambridgeshire and Hertfordshire Research Ethics Committee
REC reference
14/EE/1070
Date of REC Opinion
22 Jul 2014
REC opinion
Favourable Opinion