Young Hemophilia Patients Adhere Well to Prophylactic Treatment, Study Finds

Most young people with moderate or severe hemophilia A and B in France receive long-term prophylactic (preventive) treatment — an approach linked to a lower rate of bleeding episodes than among patients treated on demand.

The study, “Choice of factor VIII/IX regimen in adolescents and young adults with severe or moderately severe haemophilia. A French national observational study (ORTHem 15-25),” also dispersed concerns that young patients adhere poorly to long-term prophylaxis, showing that 97 percent of patients have excellent or good treatment compliance. The work was published in the journal Thrombosis Research.

Since hemophilia prophylactic treatment is a demanding practice, researchers are concerned that young patients may not adhere well. But no information exists about real-life treatment in young hemophiliacs, so researchers at the Haemophilia Treatment Centre at Louis Pradel Hospital in France decided to study the topic — exploring treatment patterns in patients ages 15 to 25.

Researchers targeted patients who kept a treatment and bleeding diary, and also accessed data from their medical records. Among the 212 patients included, 169 were receiving long-term prophylaxis, while 40 were treated with factor replacement on demand. An additional three patients were on other treatment regimens.

Of the 209 patients relying on either prophylaxis or on-demand treatment, 82.8 percent had complete diaries and 13.4 percent had partial diaries. Information was missing for the remaining 3.8 percent.

While patients receiving prophylactic treatment were of all ages, most in the on-demand group were older, 21 to 25 years of age. Those in the continuous treatment group started therapy at an earlier age, and had their first joint bleed earlier, suggesting they had more severe disease.

Treating physicians of patients on continuous treatment rated adherence as excellent in 86.4%, good in 10.7% and poor in only 3.0% of patients.

Most often, patients stated a lack of frequent or serious bleeding episodes, or related ill health, as reasons for taking factor replacement on demand. It was, however, most often the patient’s physician who had proposed a treatment regimen. The majority of continuous treatment patients never, or rarely, changed their regimen. In contrast, 82.5 percent of patients receiving on-demand treatment changed their treatment twice during the three-year study.

Bleeding episodes were about twice as common among those receiving on-demand treatment compared to continuous treatment, with annualized bleeding rates of 6.33 and 3.07, respectively. Joint bleeds were also more common in the on-demand group, with an annualized rate of 1.20 versus 0.79, but the difference was not statistically significant.