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CME: Hemophilia A: Therapeutic Updates, Emerging Therapies, and Barriers to Care


Activity Description / Statement of Need:

In this online, self-learning activity:


Hemophilia encompasses a small group of genetic disorders that impair the body’s hemostatic self-regulation mechanisms.  Hemophilias A and B, which describe deficiencies in clotting factors VIII and IX, respectively, occur in approximately 1 in 5,000 live male births.  Signs and symptoms are not always readily detectable and often escape notice for the first several months of life, but the effects can be life-threatening and may include intracranial hemorrhage, severe bleeding in other organ systems and superficial tissue, and muscoskeletal and intraarticular injury.


The mainstay of hemophilia treatment is clotting factor replacement therapy in the setting of comprehensive care.  Recombinant synthesis and blood donor screenings have significantly reduced the once common concern about transmission of infection through pooled donor products, although the development of clotting factors antibodies remain a concern.  Other treatments available or in development include other hemostatic and antifibrinolytic medicines and gene therapy.


One contemporary barrier to the optimal treatment of patients may be variability in care due to therapeutic controversies including when to initiate prophylactic therapy and may merit continuing educational programming for strategy elucidation.  Another gap is the challenge of continued adherence to therapy, and the healthcare professional may benefit from learning strategies aimed at maintaining and preserving the patient’s commitment to his or her own therapy in the face of obstacles including the necessity for frequent administration.

Target Audience:

The following healthcare professionals: hematology, primary care physicians, and pediatricians; physician assistants, nurse practitioners, nurses, and pharmacists who practice in hematology; and any other clinicians with an interest in hemophilia A.

This program is supported by an educational grant from Novo Nordisk.

Release Date: March 17, 2018 -- Expiration Date: March 17, 2020

Faculty: Doris Quon, MD PhD


Faculty introduction, disclosures

Updates in the epidemiology and diagnosis of hemophilia A

  • Statistics
  • The continuing diagnostic importance of joint pain
  • Genetics

Current and emerging treatment options for hemophilia A

  • Prophylaxis vs. On-Demand
    • Clinical trial evidence
    • Initiation and maintenance
    • Treatment Options
      • Clotting factor products, conventional and extended half-life
      • Nonfactor replacement therapies
      • Gene therapy
      • Emerging therapies
      • Individualizing treatments
        • Age and weight
        • Comorbidities
        • Physical activity levels
        • Patient pharmacokinetic profiles
        • Serum concentrations (troughs)
        • Patient case(s)

Challenges in care in hemophilia A

  • Inhibitors & immune tolerance reduction strategies
  • Adherence
  • Quality-of-life
  • Complications of the disease
    • Viral infections from transfusions and clotting concentrates prior to 1985
    • Arthritis
    • Weight loss
    • Cardiovascular disease
    • Cancer

Learning Objectives

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

  • Describe the pathophysiology of hemophilia A, with an emphasis on characteristics that play a role in determining the staging, prognosis, and diagnosis
  • Describe the benefits of prophylactic vs. on-demand clotting factor regimens in patients with hemophilia and apply them in patient cases
  • Describe the emerging therapies in the treatment of hemophilia A
  • Individualize treatments in patients with hemophilia A by taking into account treatment modalities, recent clinical trial evidence, and barriers to care , including inhibitors



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: Doris Quon, MD, PhD, Medical Director, Orthopaedic Hemophilia Treatment Center, Los Angeles, CA is a consultant and/or on a Speaker's bureau for Bayer, Bioverativ, Genentech, Novo Nordisk, Shire, Pfizer, Octapharma


Disclosures of Educational Planners: Charles Turck, PharmD has no relevent financial disclosures.


Commercial Support Disclosure: This program is supported by an educational grant from Novo Nordisk.


  • 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.

Additional Courses That Are Related To This Activity

Hemophilia B: Therapeutic Updates, Emerging Therapies, and Barriers to Care