Hemophilia B: Optimizing Pharmacotherapeutic Management Strategies

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Hemophilia is a genetic disease caused by mutation of one of the genes for coagulation proteins leading to dangerous, uncontrolled bleeding. In hemophilia B, a mutation in the gene for factor IX (FIX) leads to an endogenous deficiency in the clotting factor. The incidence of hemophilia B is the same in all geographic regions, populations, and ethnic groups,affecting approximately 1 out of every 30,000 male births. The condition is diagnosed by measuring FIX activity, and patients with severe hemophilia have levels of 1% or less.

Patients with severe hemophilia B are at risk for spontaneous, life-threatening bleeding episodes. Untreated, the life expectancy is approximately 20 years,and painful or even life-threatening morbidities include intracranial hemorrhage, severe bleeding in other organ systems, musculoskeletal injury, and joint injury. In contrast, in people with moderate or mild hemophilia, abnormal bleeding usually occurs after minor trauma or surgery.

Physical therapy can ease symptoms if internal bleeding has damaged a patient’s joints, and surgery may be necessary if internal bleeding has caused severe damage. However, the current standard of therapy for hemophilia B is intravenous infusion of therapeutic factor concentrates. Through the reduction in the number of bleeding incidences and improvement in quality of life, factor replacement therapy has significantly reduced the morbidity and mortality of hemophilia. Furthermore, prophylactic therapy has the demonstrated benefit of reducing the development of hemophilic arthropathy.

Target Audience:

The following healthcare professionals: hematology, primary care physicians, and pediatricians; physician assistants, nurse practitioners, nurses, and pharmacists who practice in hematology as well as other Hemophilia Treatment Center HCPs; and any other clinicians with an interest in hemophilia B.

By the end of the session the participant will be able to:

  • Describe the risk factors and occurrence of hemophilia B.
  • Identify available prophylactic and treatment options for hemophilia B and apply them to a patient case.
  • Identify the new treatment options for hemophilia B.
  • Identify adherence barriers in and deliver effective treatment counseling to patients with hemophilia B.

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