News

A population-based pharmacokinetic approach for tailored treatment regimens of hemophilia A patients may contribute to an increase in treatment adherence and a decrease in annual bleeding episodes, a study shows. The study, “Clinical outcomes in hemophilia A patients undergoing tailoring of prophylaxis based on population-based pharmacokinetic dosing,” was published…

Treatment of hemophilia A with bypassing agents is associated with significantly higher costs than with factor replacement therapy, according to a real-world study in the U.S. The study, “Healthcare costs among patients with hemophilia A treated with factor replacement or bypassing agents,” appeared in…

New evidence suggests that a parameter called FVIII-EC50 has potential as a new way to predict the response of an individual with severe hemophilia to preventive treatment with FVIII, according to a small study. The study “Evaluation of EC50 of factor VIII as predictor of prophylaxis efficacy in patients…

Regular preventive treatment is able to at least partially prevent bleeding episodes in adolescent and adult males with severe hemophilia A and without inhibitors, according to a real-world study. The study, “Bleeding and safety outcomes in persons with haemophilia A without inhibitors: Results from a prospective…

People with severe hemophilia A living in Europe can now receive treatment with Hemlibra (emicizumab-kxwh) to prevent bleeding episodes. The European Commission approved the prophylactic use of Hemlibra in patients of all ages who do not have factor VIII inhibitors, announced the treatment’s manufacturer, Roche. Hemlibra is a man-made antibody…

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bath dissertation, she analyzed Brexit’s long-term impact…

More than half of adults who either have hemophilia or are family members affected by the disease support the use of preconception and prenatal genetic screens, a survey reveals. However, 90 percent of this population do not agree with ending pregnancies due to hemophilia, the results also showed. Findings…

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…

Erectile dysfunction, more commonly known as male impotence, is associated with older age in men with hemophilia, especially those who smoked long term and have high blood pressure, a study reports. The study, titled “Erectile dysfunction in patients with haemophilia,” was published in Haemophilia. Hemophilia is…

Advate is an effective and well-tolerated treatment among patients with hemophilia A, according to an observational, long-term, Japanese study. The treatment showed better results when used as a preventive, rather than an on-demand, therapy. The findings were reported in an article titled “Inhibitor development, safety and efficacy of…