Elocta Found to Reduce Elbow, Knee Bleeds in Severe Hem A Patients
One year of treatment with Elocta (efmoroctocog alfa) — an extended half-life replacement therapy known as Eloctate in the U.S. — reduced the frequency of bleeds in the elbows and knees of adults with severe hemophilia A, a Spanish study showed.
Reduced pain intensity in the elbows also was reported, along with improved strength in the hamstring muscles at the back of the legs.
“Continued prophylaxis [preventive treatment] with [such therapies] may improve joint health, health-related quality of life, activity, and pain in patients with severe haemophilia, regardless of age and previous joint damage,” the researchers wrote.
The team noted, however, that longer follow-up periods are needed to assess the long-term impact of this treatment on joint health among those with hemophilia A.
The study, “LongHest project: A prospective, observational study of extended half-life treatment in the musculoskeletal health of patients with severe haemophilia A,” was published in the journal Haemophilia.
Joint bleeds are a major complication of hemophilia A, a condition caused by the lack of a blood-clotting protein called factor VIII (FVIII). Such bleeds can lead to permanent joint damage — a condition known as hemophilic arthropathy — causing disability and functional limitations.
Replacement therapy is one of the standard treatments for hemophilia A. It involves giving a form of FVIII to patients, via infusions, to prevent or stop bleeds.
More recently, extended half-life (EHL) blood clotting factors have become available. These products are modified in such a way as to increase the time they remain active in the body, leading to fewer infusions and reducing the treatment burden.
Now, researchers in Spain launched an observational study (NCT03914209) to assess the efficacy of such preventive EHL treatment. Specifically, they examined whether prophylaxis would reduce joint bleeds in adults with severe hemophilia A over the course of one year.
Secondary objectives included assessing joint health, pain intensity, range of joint movement, and associated muscle strength.
A total of 44 individuals diagnosed with severe hemophilia A —with a diagnosis of hemophilic arthropathy in at least three joints — were assessed. Patients with neutralizing antibodies against FVIII replacement therapies (inhibitors) were excluded from the study.
All participants were treated with efmoroctocog alfa, which is sold as Eloctate in the U.S. and Canada, among other countries, and as Elocta in Europe. The medication contains a lab-made version of FVIII that is attached to an Fc protein, with the purpose of lasting longer in the body.
Overall, there was a significant decrease in weekly EHL dosage over one year, dropping from a mean of 6,434.78 international units (IU) to 5,913.04 IU. Likewise, significantly fewer weekly infusions were needed, decreasing from a mean of 2.35 to 1.83 days per week.
In the elbow joint, there was a significant reduction in the mean frequency of joint bleeds, which dropped from a mean of 0.66 to 0.04. There also was a significant decrease in pain intensity, as measured by a 0–10 visual analog pain scale. Pain intensity dropped from a mean of 3.08 to 2.67.
Joint health, as assessed by the Hemophilia Joint Health Score (HJHS), as well as the range of joint movement and the strength of the upper arm muscles (triceps and biceps), were unaffected.
In the knees, there were significantly fewer bleeds over the year, dropping from a mean of 0.55 to 0.33. Here, the strength of the hamstrings — the muscles that run down the back of the legs — also significantly improved. There were no changes in knee joint health, pain, range of movement, or the strength of other muscles associated with the knees.
No significant changes were seen across all assessments of the ankle joint.
“Prophylaxis with EHL treatment improves the frequency of [joint bleeds]” — meaning it lessens them — “in elbows and knees in adult patients with haemophilia,” the researchers wrote. “Continued administration of EHL can reduce the intensity of joint pain in patients with elbow haemophilic arthropathy.”
“It is necessary to carry out studies with longer follow-up periods to assess the impact of prophylaxis with EHL treatment in adult patients with haemophilic arthropathy,” the researchers concluded.
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