Hemophilia is a genetic disorder that prevents blood clotting. Most common in men, there are two main types of hemophilia: hemophilia A and B. Below is a curated list of recommended reads for family members, friends and carers of patients with hemophilia, with help from Good Reads and Alibris.
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Hemophilia A is a genetic disorder that impairs blood clotting and is the most common form of hemophilia, accounting for 80% of cases. It is caused by a mutation on the X chromosome leading to a deficiency in factor VIII. While there is no cure, treatments like factor replacement therapy can manage symptoms and prevent severe bleeding. Research is exploring new treatments, including targeting the activated protein C enzyme and studying real-world data on managing hemophilia A with inhibitors. Support groups and resources are available for those affected.
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A first patient in the U.S. has enrolled in a multinational study to collect real-world data on different approaches in managing hemophilia A in people with inhibitors, with an aim of understanding how treatment decisions affect patients. The investigator-led MOTIVATE study (NCT04023019; EudraCT No. 2019-003427-38) is recruiting…
Hemophilia A is a rare genetic disorder that affects the blood’s ability to clot properly. It is the most common form of hemophilia, responsible for 80% of all cases.
An enzyme called activate protein C (APC), which prevents the formation of blood clots and therefore promotes bleeding could be targeted to treat hemophilia, suggests a study conducted by researcher at the University of Cambridge and the Children’s Hospital of Philadelphia.
Approximately 30,000 males have hemophilia in the U.S., according to estimates from a recent study, which also found that patients with hemophilia are generally young and that the disease is more common in Northeastern and Midwestern states. The study, “Occurrence rates of haemophilia among males in…
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Explaining Hemophilia A and B
https://www.youtube.com/watch?v=S_YMGmHqIW4 This video from Claire Blatt shares a lecture about the effects of two blood-clotting disorders: hemophilia A and hemophilia B. MORE: A study shows that NovoSeven counters bleeding in children with hemophilia. Though the lecture is aimed at nurses, it’s helpful for anyone looking to learn about these…
In hemophilia A, the most significant therapeutic complication is the development of antibodies against factor VIII (FVIII) that inhibit its coagulant activity and make the therapy ineffective. The plasma-derived porcine FVIII product represents a therapy with low levels of autoantibodies and successful management of bleeding when compared to human plasma-derived products.
https://www.youtube.com/watch?v=pTIVORH7anc This video from Wochit News is all about a new treatment for severe hemophilia A. The Children’s Hospital of Los Angeles, California, has developed a breakthrough treatment that can reduce the bleeding episodes in people living with hemophilia A by up to 90 percent. MORE: Emicizumab shows…
The once-weekly injection therapy marstacimab is being considered for approval in both the U.S. and Europe for people with hemophilia A and hemophilia B who don’t have inhibitors. The U.S. Food and Drug Administration (FDA) is expected to decide on it in late 2024, while a decision from the European…