Male doctor smiling at male child patient

Hemophilia A

The characteristic phenotype of Hemophilia A is a tendency to bleed.1,2

Hemophilia A results from missing or defective clotting protein factor VIII (FVIII), and the frequency and severity of bleeding manifestations generally correlates with the degree of FVIII deficiency.1,2

Blood coagulation pathway educational diagram

Epidemiology

Hemophilia A is typically an X-linked recessive genetic disorder that most often affects males, with an incidence of approximately 1 in 5,000 male births.1,2 Between 2012 and 2018, there were an estimated 20,000 to 33,000 males with Hemophilia A living in the U.S.

Children can inherit Hemophilia A from a father with Hemophilia A, or from a mother who is a carrier.1,2,3 Around one-third of people with Hemophilia A have no prior family history (spontaneous). Females with hemophilia are rare, but may result in females with both X chromosomes affected or when one X chromosome is inactive — females account for 3% of people with Hemophilia A.

Acquired Hemophilia A (AHA), a rare autoimmune form of Hemophilia A in which an individual develops autoantibodies to FVIII, has an estimated incidence of 1.5 to 2 cases per million per year.4,5,6,7 It is more common in the elderly, with a median age of 73.9 at diagnosis, and can affect men and women equally.

Pathophysiology

FVIII is a glycoprotein synthesized mainly in hepatocytes.9,10,11,12 Von Willebrand Factor (VWF) stabilizes FVIII, protects it from premature proteolysis, and transfers it to the site of endothelial injuries.

In primary hemostasis, which involves the formation of a platelet plug after endothelial injury, VWF:FVIII complex is critical for the recruitment and activation of platelets, and binding to collagen.10,11,13 In this process, FVIII is freed from VWF, which activates FVIII. Activated FVIII plays a role in secondary hemostasis, which is the process of forming a stable fibrin clot.14

FVIII deficiencies, which can result from a variety of mutations to the FVIII gene, lead to varying degrees of inability to form a stable fibrin clot and thus stop bleeding.1,14

Diagnosis

In some cases, infant boys with genetic risk factors are tested shortly after birth.2 In other cases, testing is done after clinical suspicion of hemophilia due to symptoms.

Due to a variety of chromosomal abnormalities, the symptoms of hemophilia can vary greatly from one patient to another and they correlate to plasma FVIII levels.1 Severe Hemophilia A is typically diagnosed at a very young age due to joint bleeds or intramuscular bleeding, bleeding associated with a minor medical procedure (venipuncture, circumcision, central line placement, or heel prick), mucocutaneous bleeding, or extracranial bleeding.1,2

Individuals with mild Hemophilia A may rarely experience spontaneous bleeding, but can experience major bleeding with trauma or surgery.1 Individuals with moderate Hemophilia A may experience occasional spontaneous bleeding, and are at risk for prolonged bleeding with minor trauma or surgery. When including mild and moderate cases, 36% of Hemophilia A cases were expected to be undiagnosed in the U.S. based on data from 2016.8

Navigating Hemophilia A

Even a single bleed in an individual with Hemophilia A may be life-threatening or lead to debilitating joint disease.1,15,16 Therefore, prevention of all bleeds is the goal of optimal hemophilia patient care.17,18

One of the most serious complications of factor treatment is neutralizing antibodies to FVIII (FVIII inhibitors).1,2 Testing for inhibitors is required in any individual with hemophilia in the following circumstances:1

 

  • When clinical or laboratory response to concentrate is suboptimal;
  • Before and after a switch of treatment;
  • Before elective invasive procedures; and 
  • Within 4 weeks after intensive treatment (≥5 consecutive days of clotting factor administration) or before surgery

Medications

This resource provides information on Takeda medications available in the Hemophilia A category and is not intended to represent a complete list of therapeutic options.

Advate®

[Antihemophilic Factor (Recombinant)]

Adynovate®

[Antihemophilic Factor (Recombinant), PEGylated]

Feiba®

(anti-inhibitor coagulant complex)

Hemofil M®

[Antihemophilic Factor (Human), Method M, Monoclonal Purified]

Obizur®

[Antihemophilic Factor (Recombinant), Porcine Sequence]

Recombinate®

[Antihemophilic Factor (Recombinant)]

Scientific Congresses and Resources

This is not intended to be a comprehensive resource of all congresses and congress materials across therapeutic and disease areas. Congress materials may include information about investigational use(s) of compounds/products that are not approved for use by the U.S. Food and Drug Administration (FDA) and/or are inconsistent with the Prescribing Information. Takeda does not recommend the use of any Takeda product beyond the approved labeling. Any decisions regarding the usage of a Takeda product beyond the approved labeling are left to the discretion of the healthcare professional. Takeda makes no representations about whether investigational compounds or unapproved uses will be approved by the FDA.

American Thrombosis and Hemostasis Network Virtual Data Summit (ATHN), 2025

October 21 - 23, 2025 | Link to Event

Brings together leading experts in blood disorders research and top clinicians to share their perspectives through ground-breaking presentations and robust Q&A sessions. Together, we’ll explore how ATHN’s unique data infrastructure supports research projects that leverage data to answer scientifically and clinically meaningful questions.

American Society of Hematology (ASH), 2025

December 6 - 9, 2025 | Link to Event

The premier global congress from the world's largest professional society serving both clinicians and scientists working in malignant and classical hematology.

Thrombosis and Hemostasis Societies of North America (THSNA), 2026

March 19 - 21, 2026 | Link to Event

The THSNA Summit is a collaboration of the 13 leading non-profit organizations in the fields of Thrombosis and Hemostasis. The Summit provides a focused forum for over 1,000 attendees with an interest in bleeding and clotting disorders to network, learn, and share across disciplines and disease states. The educational programming is organized in a series of plenary presentations, educational track session, oral abstract presentations and digital poster sessions.

American Society of Pediatric Hematology/Oncology (ASPHO), 2026

April 29 - May 2, 2026 | Link to Event

Conference delivers vital education and networking opportunities to subspecialist investigators and practitioners from every sector of the field as well as hospitalists, physician assistants, advanced nurse practitioners, other advanced practice providers, and allied healthcare professionals.

BIC International Conference (BIC International), 2025

September 12 - 14, 2025

The BIC International Conference is traditionally addressed to a relatively narrow selection of medical specialties since it is meant to feature only the most novel advances of basic science and clinical research in haemophilia, rare inherited coagulation disorders, von Willebrand factor and disease, gene therapy and thrombotic microangiopathies.

Adzynma (ADAMTS13, recombinant-krhn)

  • Recombinant ADAMTS13 Prophylaxis for the Treatment of Pediatric Patients with Congenital Thrombotic Thrombocytopenic Purpura: Pooled Outcomes from Two Phase 3 Studies

International Society on Thrombosis and Haemostasis (ISTH), 2025

June 21 - 25, 2025

Global congress featuring the world’s leading experts on thrombosis, hemostasis and vascular biology presenting the most recent advances, the latest science, and the newest clinical applications designed to improve patient care.

Academy of Managed Care Pharmacy (AMCP), 2025

March 30 - April 3, 2025

Annual event with more than 2,500 members and non-members of the AMCP to engage on the latest innovations and most intentional networking in managed care pharmacy.

National Comprehensive Cancer Network® (NCCN), 2025

March 28 - 30, 2025

Congress focusing on the practical management of patients living with or at risk for cancer and best practices for cancer care delivery.

Hemostasis and Thrombosis Research Society (HTRS), 2025

March 13 - 15, 2025

Scientific meeting open to all medical, research, and allied health professionals interested in disorders of hemostasis and thrombosis.

American Society of Hematology (ASH), 2024

December 7 - 10, 2024

The premier global congress from the world's largest professional society serving both clinicians and scientists working in malignant and classical hematology.

Adzynma (ADAMTS13, recombinant-krhn)

  • Pharmacodynamic Activity Of Recombinant Adamts13 Versus Plasma-Based Therapies In Congenital Thrombotic Thrombocytopenic Purpura: Interim Results Of A Phase 3 Randomized, Controlled, Open-Label Study
  • Mutation Analysis of the ADAMTS13 gene in patients with Congenital Thrombotic Thrombocytopenic Purpura from the rADAMTS13 Phase 3 study

American Thrombosis and Hemostasis Network Data Summit (ATHN Data Summit), 2024

October 22 - 23, 2024

Annual meeting discussing important research areas of hemophilia, health equity, thrombosis, Von Willebrand disease, rare blood disorders, and data management.

Academy of Managed Care Pharmacy Nexus (AMCP Nexus), 2024

October 14 - 17, 2024

Annual event with more than 2,500 members and non-members of the AMCP to engage on the latest innovations and most intentional networking in managed care pharmacy.

Videos

Watch videos focused on Hemophilia.

GOAL-Hēm

Learn about GOAL-Hēm, a hemophilia-specific patient-centered outcome measure and clinical engagement tool to aid in individualized goal setting.

  

   

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