Patients with moderate or severe hemophilia A can benefit from early prophylactic antihemophilic factor (recombinant) (rAHF), according to an interim analysis presented at the 2021 British Society for Haematology annual meeting.
The international AHEAD study is a 6 year non-interventional, prospective trial looking at joint health in patients receiving prophylactic or on-demand rAHF. Joint health was assessed using the Gilbert score, an evaluation system developed by the World Federation of Hemophilia.
A total of 707 patients were enrolled across 92 sites. The trial included children as young as 2 years old, as well as adolescents and adults.
Children and adolescents had consistently lower Gilbert scores than adults. A total of 52 children had Gilbert score data available, all of whom received prophylactic rAHF. Their scores remained low throughout the observation period, ranging from a mean of 0.8 to 0.2.
Adults aged 18 and older receiving prophylactic rAHF had consistently lower Gilbert scores than those receiving on-demand therapy. The mean Gilbert score at year 1 for prophylactic rAHF in adults was 2.7 (n=86) vs 3.6 for adults receiving on-demand therapy (n=30). At year 6, adults with prophylactic rAHF had a mean score of 1.4 (n=8) vs 6.3 for on-demand (n=2).
There were no new safety signals in the AHEAD trial. A total of 57.9% of patients reported adverse events, with 19.7% of patients reporting severe adverse events.
Of the 707 enrolled patients, Gilbert score data was available for 187 patients during year 1 and 14 patients by year 6. The lower number of patients available at the end of the study and the ongoing nature of the study made it difficult for the researchers to draw firm conclusions.
However, the results of the trial suggested that early prophylactic rAHF may help preserve joint health over time in patients with hemophilia A.
Disclosure: Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.
Ozelo MC, Klamroth R, Guillet B, et al. Joint health outcomes in patients with haemophilia A receiving antihemophilic factor (recombinant) in a real-world setting: results of a 6-year interim analysis of the AHEAD international study. Poster presented at: 2021 British Society for Haematology annual meeting; April 25-28, 2021; virtual.