Liver enzyme levels may rise again as corticosteroid doses are gradually reduced in people with hemophilia B treated with Hemgenix, a case report suggests. Elevated liver enzymes are a known side effect of the gene therapy that can signal liver inflammation or damage. Corticosteroids are used to treat…
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By educating and empowering patients and their families, clinicians can help improve access to accurate diagnoses and care for women and girls affected by hemophilia. That’s the argument made by a pair of doctors in Belgium in a paper titled, “How I Identify and Diagnose Carriers of Hemophilia…
Treatment disruptions during the height of the COVID-19 pandemic were a substantial driver of psychological distress for people with hemophilia in China, causing more stress than the virus itself, a study found. The researchers said the findings underscore the importance of reliable access to treatments, especially self-administered ones, to…
Inno8, a potential oral treatment to prevent bleeds in people with hemophilia A, appears more potent and long-lasting than currently available injectable treatments in preclinical laboratory and animal studies, a study suggested. The candidate therapy, being developed by Novo Nordisk, is undergoing clinical testing and has completed…
More people are being identified with hemophilia B across the eastern Mediterranean region, suggesting improvements in hemophilia diagnosis and reporting, according to a study analyzing 10 years of data from 14 countries in North Africa, the Middle East, and parts of Asia. Yet…
Blood tests can give different readings of factor VIII (FVIII) activity after treatment with Altuvoct (efanesoctocog alfa), marketed as Altuviiio in the U.S., a study found. The differences were greater when FVIII levels were low or several days had passed since an infusion, highlighting the need for careful interpretation;…
Bleeding that persists or restarts after dental procedures may be an important warning sign of previously undiagnosed mild hemophilia, a systematic review of published case reports suggests. Across seven reports involving eight people, delayed and persistent bleeding — in most cases after a tooth extraction — prompted further testing…
Artificial intelligence (AI) shows promise for helping doctors diagnose hemophilia, detect joint complications, and predict disease severity and other clinical outcomes, a review of published studies suggested. “However, methodological [diversity], limited external validation, and the scarcity of studies … remain important barriers to routine clinical implementation,” the researchers wrote.
Preventive treatment with low-dose Hemlibra (emicizumab-KXWH) significantly reduced bleeding and improved quality of life in young children with hemophilia A, regardless of inhibitor status, a real-world study in China found. But ultrasound scans showed signs of worsening joint health despite fewer clinically apparent bleeds, suggesting that lower doses may not…
More than half of people with hemophilia receiving prophylactic (preventive) factor replacement therapies at two treatment centers in Brazil did not collect enough medication to follow their prescribed treatment regimen, a real-world study has found. This gap between “prescription and practice” was evident even though Brazil provides universal,…
