Hemophilia Resources

Hemophilia Resources 

Hemophilia is a disorder where the blood does not clot properly. This is due to the body not having enough blood-clotting proteins. This can lead to spontaneous bleeding as well as bleeding following injuries or surgery. They are two types of hemophilia. Hemophilia A and Hemophilia B. Hemophilia A is caused by the lack of low level or clotting factor VIII. Hemophilia B is caused by the lack or low level of clotting factor IX. 

Signs and symptoms of hemophilia include: 

• Bleeding into the joints, mainly knees, elbows, and ankles
• Bleeding into the skin (bruising) or muscle and soft tissue which in turn causes a buildup of blood in the area (hematoma)
• Bleeding of the mouth and gums
• Nosebleeds without a known cause
• Unusual bleeding after vaccinations
• Blood in urine or stool 

Hemophilia is usually inherited, which means that a person is born with the disorder. The faulty gene for hemophilia is with the X chromosome. Hemophilia also can be acquired as well. 

It can be associated with 

• Pregnancy
• Autoimmune Conditions
• Cancer
• Multiple Sclerosis
• Drug Reactions’ 

To diagnose hemophilia, doctors perform a blood test to show if the blood is clotting properly. If not, clotting factor tests are used to show the type of hemophilia and severity. To treat hemophilia A and B is to replace the missing blood clotting factor. This is done by infusing commercially prepared clotting concentrates. By performing prophylaxis, the individual can prevent most bleeding episodes. 

Journal of Managed Care + Specialty Pharmacy

• A Systematic Review of Cost-Effectiveness Analyses of Gene Therapy for Hemophilia Type A and B. Journal of Managed Care + Specialty, 2024, Sep;30(10):1178-1118.
• Health Care Costs and Resource Use of Managing Hemophilia A: A Targeted Literature Review. Journal of Managed Care + Specialty, 2023 Jun;29(6):647-658.
• Assessing Health Care Resource Use, Outcomes, and Costs Among Medicaid Beneficiaries Receiving Factor IX Prophylaxis for Hemophilia B. Journal of Managed Care + Specialty, 2024 Jun;30(6):1095-1105.
• Evaluation of Clinical Characteristics, Health Care Resource Utilization, and Cost Outcomes of Hemophilia A Carriers and Noncarriers in the United States: A Real-World Comparative Analysis. Journal of Managed Care + Specialty, 2023 Jun;29(6):626-634. 
• Health Care Utilization and Expenditures of Parents of Children With and Without Hemophilia A. Journal of Managed Care + Specialty, 2022 Apr;28(4):529-537. 

Additional Publications 

• Gene Therapy for Hemophilia. Nathwani A C. Hematology, American Society of Hematology, Educational Program, 2022, Sep. doi: 10.1182/hematology.2022000388.
• Hemophilia Gene Therapy: First, Do No Harm. Valentio L A, Kaczmarek R, Pierce G F, et al. Journal of Thrombosis and Haemostasis: JTH, 2023, Sep. doi: 10.1016/j.jtha.2023.06.016.
• Diagnosis and Laboratory Monitoring of Acquired Hemophilia A. Platton S, Sivapalaratnam S, Raheja P, et al. Hematology. American Society of Hematology Education Program, 2023, Dec. doi: 10.1182/hematology.2023000460.
• Foundations of Hemophilia and Epidemiology. Paris L Q, et al. Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis, 2023, Jun. doi: 10.1097/MBC.0000000000001222.
• Clinical Burden of Hemophilia in Older Adults: Beyond Bleeding Risk. Hodroj M H, Hasbani G E, Al-Shamsi H O, et al. Blood Reviews, 2022, May. doi: 10.1016/j.blre.2021.100912. 

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