Antihemophilic factor
New research suggests prophylaxis with a recombinant factor VIII Fc fusion protein (rFVIIIFc) can improve joint health over time in patients with hemophilia A.
Patients saw continuous improvement in joint health over a nearly 3-year period, regardless of prior treatment regimen, severity of joint damage, or target joints.
“Gradual joint destruction, which is the leading cause of morbidity for people with hemophilia, remains a significant challenge in the treatment of hemophilia A,” said Johannes Oldenburg, MD, of University Clinic Bonn in Germany.
“This is the first study to show that functional joint health can continue to improve using prophylactic treatment with an extended half-life factor therapy, even for those who have severe joint disease at the start of treatment.”
Dr Oldenburg and his colleagues reported these findings in Haemophilia. The research was sponsored by Biogen/Bioverativ and Sobi, the companies marketing rFVIIIFc (or efmoroctocog alfa) as Eloctate or Elocta.
This interim post hoc analysis was an evaluation of joint health in adults and adolescents who received rFVIIIFc prophylaxis in the A-LONG and ASPIRE studies.
In A-LONG, patients age 12 and older who had severe hemophilia A received rFVIIIFc at 25-65 IU/kg every 3 to 5 days (arm 1), at 65 IU/kg weekly (arm 2), or as episodic treatment (arm 3). Patients who completed A-LONG could then enroll in the ASPIRE extension study.
For the current analysis, Dr Oldenburg and his colleagues assessed joint health in ASPIRE enrollees using a modified version of the Hemophilia Joint Health Score (mHJHS). This tool grades joints by specific domains, including swelling, muscle atrophy, alignment, range of motion, joint pain, strength, and global gait.
The researchers examined mHJHS measurements (a decrease in score reflecting improvement) taken at A-LONG baseline, ASPIRE baseline, and annually thereafter for roughly 2.8 years of treatment.
There were 47 patients who had mHJHS data at both study baselines, ASPIRE year 1, and ASPIRE year 2.
These patients had a mean improvement in joint health score of -4.1 at ASPIRE year 2, compared with A-LONG baseline (P=0.001).
The mean improvement was -2.4 (P=0.09) for patients who received pre-study prophylaxis and -7.2 (P=0.003) for those who received pre-study episodic treatment.
The mean improvement was -5.6 (P=0.005) in patients with target joints and -8.8 (P=0.02) in those with severe joint destruction.
The mHJHS components with the greatest improvement at ASPIRE year 2 were swelling (-1.4, P=0.008), range of motion (-1.1, P=0.03), and strength (-0.8, P=0.04).
