New evidence from real-life clinical practice in the UK, presented today at the World Federation of Haemophilia (WFH) 2018 World Congress in Glasgow, shows that people with haemophilia A treated with an extended half-life (EHL) clotting factor have fewer bleeds than with previously available treatments.
The new findings, based on an analysis of the UK National Haemophilia Database, indicate that treatment with efmoroctocog alfa factor VIII (rFVIIIFc, Elocta® ) led to a reduction in bleeding episodes in patients living with haemophilia A. The median annualised bleed rate (ABR) among 70 haemophilia A patients fell from 1.5 a year when treated with conventional clotting factor VIII for a median of 58 weeks to 1.3 a year when treated with rFVIIIFc (p=0.6) for a median of 41 weeks. However, among 21 patients who had an ABR ≥5, the median ABR fell from 10.4 to 3.3 (p<0.01).
Additionally, the number of infusions among the 70 haemophilia A patients fell from a median of 3.5/week when treated with conventional clotting factor VIII to 2.4/week when treated with rFVIIIFc, a 33% reduction (p<0.0001), representing an average of 57 fewer infusions per year. A reduction in the number of infusions can be of great benefit to those living with haemophilia and their carers, reducing the burden of treatment and making it easier to go about day-to-day life without interruption.
Furthermore, a reduction of factor usage of 14% was seen among all haemophilia A patients in the study treated with rFVIIIFc, from a median of 95 IU/kg/week to 82 IU/kg/week (p<0.0001). This change was all the greater among those patients with an ABR ≥5, for whom factor usage decreased by 23% (p<0.01).
It has been found that most bleeding in haemophilia occurs internally, into the joints or muscles, which can cause pain and discomfort; even one joint bleed can cause permanent damage. Liz Carroll, CEO, The Haemophilia Society, said: “A survey of our members has told us that a reduction in joint problems, caused by bleeds, would be the most important factor for improving their quality of life, so it is reassuring to see that EHL treatments are having this effect. In families in which several members are living with haemophilia, the time saved from having fewer infusions also impacts on family life.”
Neil Dugdale, Vice-President and General Manager, Sobi UK and RoI, commented: “We’re delighted to see that the real world evidence matches what has already been seen in clinical trials, that treatment with our EHL product reduces the number of bleeding episodes for someone living with haemophilia A.”