Results of a real-world, retrospective study suggest there may be a need to improve the standard of care for hemophilia A and B in some European countries.
Hemophilia treatment practices varied widely among the 7 countries studied, as did annual bleeding rates (ABRs).
Some countries had low median ABRs in hemophilia A and B patients receiving prophylaxis—in the range of 1.0 to 2.0.
However, the median ABR was as high as 8.0 for some patients, despite receiving prophylaxis. And these were patients with moderate disease.
The study, which was sponsored by Sobi, was published in Haemophilia.
“The overall results indicate that there is a significant need to advance standard of care within hemophilia,” 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 study was designed to provide insight into current hemophilia treatment practice and outcomes in 7 European countries—Belgium, France, Germany, Italy, Spain, Sweden, and the UK.
The researchers analyzed data on 1346 patients with hemophilia A and 312 with hemophilia B treated in these countries.
Treatment type, dosing
Prophylaxis was, overall, the most dominating treatment for patients with severe hemophilia 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 hemophilia A, and there was no trend across age groups.
For patients with hemophilia B, prophylaxis was the most common treatment in 4 out of the 7 countries (France, Germany, Sweden, and UK).
Overall, a majority of patients received a recombinant antihemophilic factor product as opposed to a plasma-derived product. The one exception was patients with hemophilia B in Germany. A majority of these patients (66%) received a plasma-derived product.
The mean prescribed prophylactic treatment ranged from 67.9 IU kg-1 per week (Belgium) to 108.4 IU kg-1 per week (Germany) for hemophilia A and 32.3 IU kg-1 per week (Belgium) to 97.7 IU kg-1 per week (France) for hemophilia B.
Most patients on prophylaxis were treated 3 or more times a week if they had hemophilia A and 2 times a week if they had hemophilia B.
ABRs across countries
For hemophilia A patients on prophylaxis, the median ABR ranged from 1.0 (Belgium, Italy, Sweden) to 4.0 (France, UK) for patients with severe disease and from 2.0 (Sweden) to 8.0 (Belgium) for patients with moderate disease.
The researchers pointed out that ABRs were higher for patients with moderate hemophilia A, but low patient numbers should be taken into account when interpreting these data.
The median ABRs for hemophilia A patients who received on-demand treatment ranged from 4.5 (Sweden) to 18.0 (UK, Belgium) for patients with severe disease and from 1.0 (Spain, Sweden) to 12.0 (UK) for patients with moderate disease.
For hemophilia B patients on prophylaxis, the median ABRs ranged from 1.0 (Germany, Sweden) to 6.0 (Belgium) for patients with severe disease and from 1.5 (Sweden) to 8.0 (Belgium) for patients with moderate disease. Again, ABRs were higher for patients with moderate disease.
The median ABRs for hemophilia B patients who received on-demand treatment ranged from 1.5 (Germany) to 14.0 (UK) for patients with severe disease and from 1.0 (Belgium, France, Germany, Italy) to 6.5 (UK) for patients with moderate disease.
The researchers said the high number of bleeds observed in some patients likely reflects insufficient therapy, inappropriate dose-interval, presence of target joints, poor adherence, or patient difficulty in correctly assessing bleeds.
The team said the overall results of the study suggest there is room for improvement of hemophilia therapy, even for patients currently on prophylactic treatment.
