Hemlibra found to ease pain, improve life quality in hemophilia A
Study shows improvements after 3 months, benefits sustained almost 1.5 years
Hemlibra (emicizumab), a preventive treatment for hemophilia A, reduced pain and improved quality of life for adults and adolescents with the disease, including those with recurrent joint bleeds, a study found.
Significant improvements in pain-related quality of life were seen 13 weeks, or about three months, after starting Hemlibra, and benefits were sustained through 78 weeks, or nearly 1.5 years.
“Improvements may reflect a reduction in chronic pain, a crucial outcome for [people with hemophilia A] with established joint damage,” the researchers wrote, adding that the study’s results “highlight the clinical benefits of [Hemlibra] prophylaxis beyond bleed prevention.”
The study, “Pain-Related Quality of Life Outcomes in People With Haemophilia A Receiving Emicizumab: A Post Hoc Analysis of the HAVEN 1, 3 and 4 and STASEY Studies,” was published in Haemophilia.
Hemophilia A is a rare, genetic condition in which the blood does not clot properly due to a deficiency in a clotting protein called factor VIII (FVIII). As a result, people with hemophilia A experience frequent and uncontrolled bleeding episodes, particularly in the joints. These bleeding episodes can cause acute pain as well as chronic pain, which is typically associated with long-term joint damage from repeated joint bleeds.
Mimicking FVIII to reduce bleeding, ease pain
Hemlibra is an antibody-based therapy that works by mimicking the function of FVIII to help the blood clot more effectively. The therapy is also effective in patients who have FVIII inhibitors, or neutralizing antibodies that can render standard FVIII replacement therapies less effective.
Clinical trials have shown that Hemlibra safely and effectively reduced bleeding episodes in hemophilia A patients of different ages, with and without FVIII inhibitors.
“Joint bleed prevention and reduction … demonstrated across the phase 3 clinical trials and subsequent post-marketing phase 3b trials, may be expected to improve acute and chronic pain and, consequently, pain-related [quality of life],” the researchers wrote.
The researchers analyzed data from four clinical trials of Hemlibra involving 504 patients with severe hemophilia A, with the goal of assessing the impact of the therapy on pain-related quality of life.
The trials — HAVEN 1 (NCT02622321), HAVEN 3 (NCT02847637), HAVEN 4 (NCT03020160), and STASEY (NCT03191799) — enrolled patients ages 12 to 80, with varying levels of joint damage and treatment histories. Participants received preventive treatment with Hemlibra, and researchers evaluated their pain and quality of life through patient-reported questionnaires over as long as 78 weeks.
By week 13, 61% of patients reported that joint pain occurred never or rarely, up from 30% at the study’s start. Those who reported that swelling pain occurred never or rarely rose to 84% from 37% over the same period. The proportion of patients who had no or slight pain or discomfort also increased, to 81% from 71%.
All improvements in pain-related quality of life were maintained through weeks 73-78.
Patients of all ages, with and without FVIII inhibitors, and with different treatment histories benefited from Hemlibra.
Younger participants saw the most dramatic improvements in analyses in which patients were stratified by age. The percentage of patients ages 12 to 17 who reported no or rare swelling pain rose to 93% at week 13 from 43% at the study’s start.
Older patients, particularly those ages 50 and older, also experienced improvements, though their gains were smaller. This difference may be due to older patients accumulating more severe joint damage over their lifetimes, the researchers noted.
Pain-related quality of life improvements were also seen in patients with target joints, those in which repeated bleeding episodes occurred within a short period of time, making them particularly prone to chronic damage.
“Improvements in pain were observed regardless of target joint status at study entry or prior treatment approach,” the researchers wrote.
While the study highlights promising results, it also had limitations. The analysis relied on general quality-of-life questionnaires, rather than tools specifically designed to measure pain. The scientists said more research is needed to confirm these findings in real-world settings and to explore the long-term effects of Hemlibra on pain and mobility.
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