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…
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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,…
Hemophilia A patients rack up high hospital bills, driving costs for the healthcare system higher, a study from Canada showed. “This study confirms that even with improved [treatment to reduce bleeding], hemophilia A has a non-negligible impact on healthcare system costs,” researchers wrote in the study, “Population-Level Real-World…
A branch of the European Medicines Agency (EMA) has recommended that the experimental therapy denecimig be approved to treat adults and children with hemophilia A, with or without inhibitors. The recommendation from the EMA’s Committee for Medicinal Products for Human Use (CHMP) will be reviewed by the European…
Heavy periods in teenage girls should prompt further evaluation for an underlying bleeding disorder, such as hemophilia, rather than simply being considered a normal part of adolescence, according to a call to action from an international group of experts. Heavy menstrual bleeding, or HMB, can sometimes be the first — or…
A gene therapy that delivers instructions to produce a longer-lasting version of the clotting protein missing or deficient in hemophilia B may improve treatment effectiveness while maintaining a favorable safety profile, a study in mice suggests. Researchers in Belgium, working in a lab, combined the genetic instructions for a…
Nearly a quarter of hemophilia patients aged 50 and older in a prospective screening program at a large UK hemophilia center met criteria for frailty, a study found. All patients with frailty had other health conditions, and nearly 80% were taking five or more regular medications. Hemophilic arthropathy, or…
Caring for a child with hemophilia can bring emotional strain and disrupt family life even as advances in treatment make it easier to manage the disease, a study in the Netherlands found. Caregivers interviewed for the study generally valued the care they received at specialized hemophilia centers, but identified important…
In children with severe hemophilia A who are undergoing treatment to remove inhibitors, adding Hemlibra (emicizumab-KXWH) was associated with faster immune tolerance and fewer treated bleeds. Adding Hemlibra also did not increase direct treatment costs during immune tolerance induction, study data showed. The work was partially funded by…
Alvotech and Lotus Pharmaceutical are teaming up on plans to commercialize AVT87, a proposed biosimilar to Hemlibra (emicizumab). Lisa Graver, CEO of Alvotech, said in a company press release that the companies “look forward to working together” to broaden access to high-quality biologics. Hemlibra is an…
