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CME: Gaps in the identification and treatment of hemophilia B: what are the missing factors?


Activity Description / Statement of Need:

In this online, 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. Unfortunately, the literature shows that not only do clinicians struggle with the classification of hemophilia severity and that there are gaps in knowledge present that contribute to delayed diagnosis and treatment, with an attendant increase in morbidity and mortality. Challenges in diagnosis and classification are only the first of several gaps in care that patients with hemophilia face.

Target Audience:

The following HCPs: hematologists and pediatricians; physician assistants, nurse practitioners, and pharmacists who practice in hematology, and other HCPs who practice in hemophilia treatment center; and any other clinicians with an interest in or who clinically encounter patients with hemophilia B.


Commercial Support Disclosure: This program is supported by an educational grant from CSL Behring.

This activity is free of charge.

Release Date: April 23, 2022 -- Expiration Date: April 23, 2024

Faculty: Christopher Walsh, MD


Faculty introduction, disclosures

Hemophilia B introductory content

·       Statistics

·       Pathophysiology, primary vs. secondary hemostasis, and the role of genetics

·       Challenges in the identification of disease, its complications, and healthcare burden

Therapeutic considerations and emerging treatment options for hemophilia B

·       Goals of care: bleeding episode elimination, quality of life improvement, and “health equity”

·       Prophylactic therapy optimization

o   When to initiate, at what dose, prophylaxis in active patients, and duration

o   Moderate vs. severe hemophilia

o   Clinical trial evidence

·       Pharmacokinetics and -dynamics

o   Pharmacokinetic studies using patient cross-over designs to compare extended half-life FIX products

o   Understanding the mechanism of action

§  The central role in the cycle of coagulation factor activation

§  Fibrinogen’s conversion into fibrin and clot stabilization

§  The potential importance of FIX’s presence in the extravascular space

·       Treatment considerations

o   Management of acute bleeds

o   Individualizing regimens, including consideration of age and weight, comorbidities, and physical activity levels

o   Special clinical circumstances and populations, including in orthopedic surgery

·       New & investigational hemophilia B treatments

o   Extended half-life FIX products

o   Gene therapy

·       Challenges in care in hemophilia B

o   Hemophilia management during the COVID-19 pandemic

o   Quality-of-life and addressing hemophilia’s the psychosocial aspects: depression, anxiety, and pain and their impact on treatment adherence

o   Inhibitors & immune tolerance reduction strategies

·       Patient case(s)

Summary, best practice recap, conclusion

Learning Objectives

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

  • Identify challenges in the challenges in the optimal identification and treatment of hemophilia B
  • Explain goals of care that comprise best practice in the treatment of hemophilia B
  • Recall present and investigational therapies in the treatment of hemophilia B
  • Compose a plan of care for a patient with hemophilia B


ACCME Activity #202289989


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 by ScientiaCME.

Faculty Disclosure:

Christopher E. Walsh, MD, PhD, Mount Sinai School of Medicine, New York, NY, has received financial compensation for consulting work for CSL Behring, Novonordisk, Takeda, Genentech, and Sanofi.

Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, President of ScientiaCME, has no relevant financial disclosures.

Faculty will not discuss off-label uses.

All relevant financial relationships have been mitigated.

ScientiaCME adheres to the ACCME’s Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CE activity, including faculty, planners, reviewers or others are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity.

Commercial Support Disclosure: This program is supported by an educational grant from CSL Behring.


  • 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.