Measuring levels of YKL-40 — a protein associated with inflammation and tissue remodeling — may help detect hemophilic arthropathy, the joint damage caused by repeated bleeding, in people with severe hemophilia A, a study found. The researchers said the results “suggest that YKL-40 has potential as a biomarker, with its…
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Switching to Hemlibra (emicizumab) was safe and effective in children with hemophilia A, including in those who had been minimally treated before or who had not been treated at all, a real-world study reported. Researchers noted that studies involving a larger number of patients are still warranted to…
Preventive treatment with Kovaltry (octocog alfa) is safe and effective at preventing bleeds, including joint bleeds, for up to two years in people with hemophilia A, a long-term extension study shows. Findings were reported in the study “BAY 81-8973 demonstrated efficacy, safety and joint status improvement…
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…
Patients with moderate to severe hemophilia A who switched to Kovaltry (octocog alfa) showed no worsening of their annual bleeding rate, according to real-world data from a Phase 4 study. Adhering to the therapy remained high and patients reported high treatment satisfaction. Overall, the study “confirms and extends clinical trial…
A lack of family history of disease can delay a diagnosis and timely initiation of preventive therapy in children with hemophilia A, increasing the need for intensive factor replacement therapy when a first bleeding event happens, a study shows. “These observations highlight the importance of an early diagnosis in…
Denecimig (Mim8), an investigational antibody therapy being developed by Novo Nordisk, continued to maintain low bleeding rates in children, adolescents, and adults with hemophilia A, with safety findings consistent with earlier studies, regardless of inhibitor status or dosing schedule. That’s according to interim data from FRONTIER4 (NCT05685238), an…
CSL Behring’s rVIII-SingleChain, a novel recombinant Factor VIII (rFVIII) treatment candidate, recently demonstrated effectiveness in surgery and in the control of bleeding in patients with severe hemophilia A. The Phase 1 to Phase 3 clinical trial results, recently published in the journal Blood, also demonstrated low annualized bleeding rates in patients…
A daily infusion of low-dose factor VIII (FVIII) improves trough levels in patients with severe hemophilia A — compared to infusions performed every other day — without increasing factor VIII consumption, or the amount of medication a patient must take, researchers have found. Trough levels are the lowest concentrations of a…
In the U.S., hemophilia A is estimated to affect one in every 5,000 male births, hemophilia B one in 25,000 male births, and hemophilia C one in every 100,000 people (males and females). Hemophilia A is thus four times more common than hemophilia B, and about 10 times more common than…