JW Pharmaceutical said on 6 that a study published in the international journal TH Open found children and adolescents with hemophilia A who received Hemlibra (emicizumab) maintained a low risk of bleeding even while taking part in a range of physical activities.
Hemlibra is a novel drug designed to mimic the function of clotting factor VIII, which is lacking in people with hemophilia. The company said it is the only hemophilia A treatment that can be used in both patients who have developed antibodies that make standard factor VIII products ineffective and those who have not. It is given by subcutaneous injection, with preventive effects lasting up to once every four weeks.
The study was conducted at 50 medical institutions in Japan over about 97 weeks. Researchers analyzed physical activity, bleeding, safety and changes in quality of life after patients switched to Hemlibra prophylaxis.
Before Hemlibra, patients experienced an average of 1.5 to 2.0 bleeding episodes over the most recent 12 or 24 weeks. After switching treatment, the median annualized bleeding rate was 0.53.
During the study, researchers recorded 172 physical activities: 44 high-risk, 70 moderate-risk and 42 low-risk. One traumatic bleed related to physical activity was reported, and no significant link was found between activity intensity and bleeding.
Quality-of-life measures also improved. In caregiver surveys, 43.8% reported their child was more active, and 56.3% reported less anxiety about bleeding. On safety, no intracranial hemorrhage (ICH) or thromboembolism — concerns in infants and toddlers — were reported.
* This article has been translated by AI.
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