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Bleeding Disorders

 

Bleeding disorders are a group of disorders that share the inability to form a proper blood clot. They are characterized by extended bleeding after injury, surgery, trauma or menstruation. Sometimes the bleeding is spontaneous, without a known or identifiable cause. Improper clotting can be caused by defects in blood components such as platelets and/or clotting proteins, also called clotting factors.

 

Hemophilia occurs predominantly in males, but can occur in women. In about one-third of cases the disorder results from a spontaneous genetic mutation rather than by a family history of hemophilia. Hemophilia treatment may require life-long infusion of clotting factor based on the severity of the disorder. Average hemophilia treatment costs between $60,000 and $260,000 per year. Early recognition and prompt treatment of bleeds can help keep these costs down as well as reduce disability.

 

von Willebrand Disease is a bleeding disorder caused by a defect or deficiency of a blood clotting protein, called von Willebrand Factor. The disease is estimated to occur in 1% of the population; many are undiagnosed. vWD is a genetic disease that can be inherited from either parent. It affects males and females equally. A man or woman with VWD has a 50% chance of passing the gene on to his or her child. There are no racial or ethnic associations with the disorder. A family history of a bleeding disorder is the primary risk factor.

 

Other Factor Deficiencies: There are ten clotting factors that are necessary in forming a blood clot. Deficiencies in factors VIII and IX are well known to most people, but what of the other factor deficiencies? Not everyone is as familiar with these conditions because they are diagnosed so rarely. To date, deficiencies in eight of the lesser known coagulation factors have been documented in the medical literature. Many of these disorders were only discovered or described within the last 40 years.

News from HFA

  • Word from Washington: May 2023

    Lawmakers in May devoted close attention to the role that pharmacy benefits managers (PBMs) play in our health care system.  PBMs are middlemen that create and administer drug formularies on behalf of health insurers, self-insured employers, Medicaid, and other payers. PBMs choose which drugs a plan will cover, negotiate drug prices on behalf of payers, The post Word from Washington: May 2023 appeared first on Hemophilia Federation of America.

  • Pfizer Announces Positive Results from Hemophilia A and B Trial

    Pfizer Inc. today announced their Phase 3 BASIS clinical trial evaluating marstacimab has met its primary endpoints, having demonstrated statically significant and clinically meaningful effects. Marstacimab, a novel, investigational anti-tissue factor pathway inhibitor (anti-TFPI) being studied for the treatment of hemophilia A or B for people without inhibitors to Factor VIII (FVIII) or Factor IX The post Pfizer Announces Positive Results from Hemophilia A and B Trial appeared first on Hemophilia Federation of America.

  • FDA Finalizes Blood Donor Eligibility Guidance

    On May 11, 2023, the U.S. Food and Drug Administration released final guidance providing recommendations for evaluating blood and plasma donor eligibility using individual risk-based questions. Consistent with FDA’s January 2023 proposed guidance, the final guidance eliminates time-based donation deferrals and screening questions specific to men who have sex with men (MSM) and women who have The post FDA Finalizes Blood Donor Eligibility Guidance appeared first on Hemophilia Federation of America.

  • FDA issues response to Novo Nordisk’s request for concizumab approval

    Recently Novo Nordisk received a Complete Response Letter from the Food and Drug Administration for concizumab. Concizumab was submitted for regulatory approval in the U.S. for the treatment of hemophilia A and B with inhibitors in August 2022. In the letter, the FDA requested additional information related to the monitoring and dosing of patients to The post FDA issues response to Novo Nordisk’s request for concizumab approval appeared first on Hemophilia Federation of America.

  • The HELP Copays Act moves forward – now in the Senate as well as in the House 

    Protections for people who rely on copay assistance have been a top priority for HFA and many other patient groups for several years. That effort took a big step forward in April with the introduction of a first-ever Senate version (S. 1375) of the HELP Copays Act.   Sponsored by Sens. Kaine, Marshall, Markey, and Ernst, The post The HELP Copays Act moves forward – now in the Senate as well as in the House  appeared first on Hemophilia Federation of America.