New European study emphasizes need to enhance standard of haemophilia care in real life

Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO: SOBI) announces the results from a new European study that assessed the efficacy of haemophilia care in real life. The study, which was fully funded by Sobi, showed that treatment practice varied widely between countries and that patients treated on-demand and prophylactically both experienced bleeds, emphasizing the need for further enhancing standard of care.

The authors concluded that prophylaxis should be considered for people with frequent bleeds to avoid pain and deterioration of joint function, and the treatment regimen for people on prophylaxis needs improvement.

The study has been published in the on-line issue of Haemophilia, the official journal of the World Federation of Hemophilia.  

“The overall results indicate that there is a significant need to advance standard of care within haemophilia”, said Stefan Lethagen, Vice President Medical & Clinical Sciences, Haemophilia at Sobi. “Even when prophylaxis is the norm, it appears that prophylactic treatment is driven to the minimal acceptable level or even lower, which increases the risk of joint injury and limits the ability to live a full and active life.”

The retrospective study included 1346 people with haemophilia A and 312 people with haemophilia B, from seven European countries. The study was designed to provide insights into current haemophilia treatment practice and outcome.

Prophylaxis was overall found to be the most dominating treatment for people with severe haemophilia A. It was the most common treatment regimen among children and decreased with increasing age. On-demand treatment was reported to be most common in moderate haemophilia A, and there was no trend across age groups. For people with haemophilia B, prophylaxis was most common in four out of seven countries.

In this European study, the median ABR for patients with severe haemophilia A on prophylaxis was as high as four in some countries and some patients experienced more than 12 bleeds per year, likely reflecting insufficient therapy, inappropriate dose-interval, presence of target joints, poor adherence or difficulty of correctly assessing bleeds by some patients. The authors emphasised that there is also a need for improvement in individuals with moderate haemophilia A on prophylaxis, since bleeds were as frequent or more frequent than for those with severe disease on prophylaxis. The overall results of the study indicate that there is room for improvement of haemophilia therapy, even for patients currently on prophylactic treatment.