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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

  • BioMarin Update: FDA Extends PDUFA Target Action Date to June 30, 2023

    SAN RAFAEL, Calif., March 6, 2023 — BioMarin Pharmaceutical Inc., a global biotechnology company dedicated to transforming lives through genetic discovery, announced that it received notice this afternoon from the U.S. Food and Drug Administration (FDA) that the agency has extended review of the company’s Biologics License Application (BLA) for ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy for adults The post BioMarin Update: FDA Extends PDUFA Target Action Date to June 30, 2023 appeared first on Hemophilia Federation of America.

  • The Complexity of Unwinding Medicaid

    The long-heralded Medicaid “unwinding” has begun. As previously reported, state Medicaid programs have, since the start of the COVID-19 pandemic, operated under a law that prohibited them from disenrolling Medicaid enrollees for the duration of the public health emergency (PHE). Congress changed that requirement in December 2022, delinking the Medicaid continuous enrollment obligation from the PHE. The post The Complexity of Unwinding Medicaid appeared first on Hemophilia Federation of America.

  • FDA Approves Sanofi’s Once-Weekly ALTUVIIIO™

    Source: Sanofi Press Release Sanofi announces that the U.S. Food and Drug Administration approved ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-vWF-XTEN Fusion Protein-ehtl], previously known as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy for adults and children with hemophilia A.  Sanofi states that with ALTUVIIIO, they are looking to raise the bar for people with The post FDA Approves Sanofi’s Once-Weekly ALTUVIIIO™ appeared first on Hemophilia Federation of America.

  • Dear Addy: Project CALLS

    Dear Addy,  I recently attended an event and learned about submitting issues with my insurance to HFA’s Project CALLS. I have an insurance issue, however, I still have some questions about how CALLS works and what happens when I submit an issue. Can you explain the process?  Signed,  CALLS Quandary   Dear Quandary,  You are not The post Dear Addy: Project CALLS appeared first on Hemophilia Federation of America.

  • Finding Strength Through Diversity

    This Black History Month, HFA asks our community to think back on the family members they’ve loved and the moments they created. We ask our community to think on the diversity of our community, the stories we’ve shared, and the people of color who helped make the bleeding disorder community as strong as it is The post Finding Strength Through Diversity appeared first on Hemophilia Federation of America.