Patricia Inácio, PhD, science writer —

Preventive treatment with Hemlibra (emicizumab) improves health-related quality of life (HQRoL) in children with hemophilia A who develop inhibitors, or neutralizing antibodies, against engineered factor VIII (FVIII), an essential blood-clotting protein, a new analysis of a Phase 3 trial shows. Such Hemlibra therapy also reduces the burden on…

Long-term treatment with Esperoct (turoctocog alfa pegol) prevented bleeding in one-fifth of children with severe hemophilia A over the course of a Phase 3 clinical trial, according to final results from the study. Treatment was effective in both spontaneous and traumatic bleeds, and appeared to be…

An upcoming episode of the TV series “Advancements,” hosted by Ted Danson, will be dedicated to current and future research and therapeutics for inheritable bleeding disorders. The new episode will feature work developed by the National Hemophilia Foundation (NHF) and explore the non-profit’s programs in bleeding diseases, such…

The National Hemophilia Foundation (NHF) has opened a grant program to support the local groups and chapters that work with bleeding disorder families, and have watched as their revenue sources dry up during the COVID-19 pandemic. Called the COVID-19 Fund Bridge Grant program, the effort is open to…

Starting factor VIII (FVIII) preventive therapy before the age of 2.5 years improves joint outcomes in young adults with hemophilia A compared to starting treatment later in childhood, a clinical trial shows. The study, “ Young adult outcomes of childhood prophylaxis for severe hemophilia A:…

Under-the-skin, preventive treatment with Dalcinonacog alfa (DalcA) led to sustained and protective levels of factor IX (FIX) in people with severe hemophilia B, final data from a Phase 2b trial show. The findings, Phase 2b Trial to Evaluate the Safety and Factor IX Levels of a Daily Subcutaneous…

Europeans with severe hemophilia are largely satisfied with available treatments and able to maintain active lives, according to a survey of healthcare professionals by the biopharmaceutical company Sobi. Results also showed that 23 countries across Africa, South America, and Asia rely entirely on the World Federation of Hemophilia…

Switching from on-demand to preventive treatment — or prophylaxis — significantly reduces bleeding in adults with hemophilia A, including the elderly, a real-life study shows. The study, “Long‐term analysis of the benefit of prophylaxis for adult patients with severe or moderate haemophilia A,”…

Blood visibly present in urine — called macroscopic hematuria — considerably increases the risk of hypertension in hemophilia patients with a family history of high blood pressure, and is not a “benign” condition for this patient group, a study reports. The study, “Macroscopic hematuria as a risk…

In contrast to observations in the general population, blood type and levels of von Willebrand factor (VWF) don’t appear to influence factor VIII (FVIII) levels in people with non-severe hemophilia A, a study reports. Moreover, age was found to play only a minor role in FVIII levels in…