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CME: Hemophilia B: Optimizing Pharmacotherapeutic Management Strategies


Activity Description / Statement of Need:

In this online CME self-learning activity:

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.

Commercial Support Disclosure: This activity is supported by an educational grant from Sanofi / Genzyme

Learners may participate in this activity free of charge.

Release Date: June 10, 2020 -- Expiration Date: June 10, 2022

Faculty: Lisa Boggio, MD


Faculty introduction, disclosures

Updates in the epidemiology and diagnosis of hemophilia B

  • History of hemophilia
  • Incidence
  • Congenital vs. acquired
  • Pathophysiology, genetics of hemostasis, hemophilia, and the coagulation cascade
  • Severity categories, sites of bleeding
  • Complications: neurological, musculoskeletal, infectious disease (including with transfusion), allo-antibodies/inhibitors
  • Diagnosis: median age at diagnosis by category, screening tests and relevant lab assays

Current and emerging treatment options for hemophilia B

  • History of hemophilia treatment
  • Treatment options
    • Immune tolerance induction: risks, benefits, and success rate
    • Pharmacokinetics of FIX
    • Fresh frozen plasma, cryoprecipitate
    • Factor concentrate, conventional and extended half-life
    • Nonfactor replacement therapies: DDAVP
    • Gene therapy
    • Emerging therapies
  • Individualizing treatments: replacement factor dosing
    • Weight and desired factor level
    • Dosing guidelines
    • Guideline recommendations for patients who qualify for primary prophylaxis
    • Treatment goal(s)
    • Literature-demonstrated benefits of primary prophylaxis
  • Barriers to care: adherence, IV access, imposition of travel to treatment centers
  • Summary of factors to consider in individualizing therapy: age, activities, lifestyle, pharmacokinetics (including lab tests and monitoring), bleeding phenotype, adherence, target joints and status, time of infusion
  • Inhibitors
    • Statistics
    • When to suspect them
    • Risk factors and complications
    • Treatment: acute bleed treatment and long-term therapy
  • Patient case(s)

Summary, best practice recap, conclusion


Learning Objectives

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.


ACCME Activity #201806134


Faculty Disclosure and Resolution of COI

As a provider of continuing medical education, it is the policy of ScientiaCME to ensure balance, independence, objectivity, and scientific rigor in all of its educational activities. In accordance with this policy, faculty and educational planners must disclose any significant relationships with commercial interests whose products or devices may be mentioned in faculty presentations, and any relationships with the commercial supporter of the activity. The intent of this disclosure is to provide the intended audience with information on which they can make their own judgments. Additionally, in the event a conflict of interest (COI) does exist, it is the policy of ScientiaCME to ensure that the COI is resolved in order to ensure the integrity of the CME activity. For this CME activity, any COI has been resolved thru content review ScientiaCME.

Faculty Disclosure: Lisa N. Boggio, MD, MS, Professor, Rush University Hemophilia and Thrombophilia Center, has no relevant financial disclosures.

Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, President of ScientiaCME, has no relevant financial disclosures.
Commercial Support Disclosure: This activity is supported by an educational grant from Sanofi / Genzyme


  • Read the learning objectives above
  • Take the Pre-Test (optional). Completion of the pre-test will help us evaluate the knowledge gained by participating in this CME activity.
  • View the online activity. You may view this is in more than one session, and may pause or repeat any portion of the presentation if you need to.
  • Minimum participation threshold: Take the post-test. A score of 70% or higher is required to pass and proceed to the activity evaluation.
  • Complete the activity evaluation and CME registration. A CE certificate will be emailed to you immediately.

Cultural/Linguistic Competence & Health Disparities

System Requirements

Windows 7 or above
Internet Explorer 8
*Adobe Acrobat Reader
Mac OS 10.2.8
Safari or Chrome or Firefox
*Adobe Acrobat Reader
Internet Explorer is not supported on the Macintosh

*Required to view Printable PDF Version

Perform Pre-Test (optional)

Please take a few minutes to participate in the optional pre-test. It will help us measure the knowledge gained by participating in this activity.

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