Navigating Current and Emerging Approaches in the Treatment and Management of Hemophilia

A continuing medical education activity provided by NAMCP and AAMCN

This activity is an archive from the live session from the 2020 Fall Managed Care Forum. If you participated in the live session, you are not eligible for continuing education credits from this archive.

This activity is valid from February 1, 2021 to March 1, 2022


Instructions for CME/NCPD: Complete the pre-test, listen to the audio and view the slides, complete the post test, complete the evaluation form and hit submit. You will be asked to enter your name and email address on the pre-test, evaluation and post-test. If you close your internet browser without completing the post test, you will have ONE more opportunity to complete. A score of 70% must be achieved on the post test to receive continuing education credits. If you do not pass the post test after two attempts, you will not be eligible to try again. Once you complete the evaluation form and score 70% or higher on your post test, you will automatically be given your certificate.

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Audience: This activity is intended for healthcare professionals practicing in managed care environments.

This activity is supported by an educational grant from
BioMarin Pharmaceutical Inc.

Hemophilia is usually an inherited disorder and is caused by a defect in one of the X chromosomes. The number of people living with hemophilia is unknown; it’s believed that around 1 in 5,000 people are born with it every year. There are a couple of different types, but 9 out of 10 people with hemophilia have type A and are missing the clotting factor VIII. There are three levels of hemophilia, but 7 out of 10 sufferers of hemophilia are diagnosed with severe, which is having less than 1% of the normal clotting factor. Hemophilia is diagnosed through blood tests to see how long blood takes to clot, which clotting factor is low or missing, and the severity of the hemophilia. The main treatment is replacement therapy; concentrates of clotting factor VIII are dripped or injected into a vein. This concentrated clotting factor can be made from human blood or not. Having regular treatments is referred to as prophylactic, or preventive therapy, and helps to prevent bleeding episodes. There is also demand therapy, where the replacement therapy is done on an as-need basis. While demand therapy is less expensive, there is a risk that the delay in treatment can cause damage to joints and muscles.

Replacement therapy can be taught to a capable person, and infusions can be done at home, making for quicker treatment with less stress, less visits to the doctor, and less cost. However, it does require precise patient education and follow up. Another possibility for replacement therapy is having a vein access device implanted, which makes access easier. These devices can get infected and need proper care. Antibodies are the biggest complication for replacement therapy. These antibodies, or inhibitors, develop in around 20% of people with hemophilia A. This can be treated though either larger doses of the regular clotting factor, or sometimes by switching clotting factors. Novel treatments include longer lasting agents that will cut down on the number of infusions done per year, thus having the possibility of increasing patient compliance and decreasing the possibility of inhibitors forming.

Upon completion of this activity, participants will be able to:

  • Analyze recent clinical data on the efficacy, safety and mechanisms of current, novel, and emerging therapies

  • Discuss personalized strategies for treatments with and without inhibitors and how they may be integrated into the management of hemophilia

  • Assess the evolving role of gene therapies in the management of hemophilia

  • Explore evidence and strategies for appropriate patient selection of different therapies for hemophilia A

  • Identify utilization management and benefit design strategies for hemophilia therapies to promote appropriate prescribing and improve outcomes

  • Review appropriate solutions to the challenges of hemophilia management, including patient education on methods of administration and adherence promotion


Faculty: Mark T. Reding, MD
Associate Professor of Medicine
Director, Center for Bleeding and Clotting Disorders
University of Minnesota Medical Center


Dr. Reding serves on an advisory board for Bayer, CSL Behring, Novo Nordisk, Sanofi Genzyme, and Takeda. He serves as a consultant for Bayer, CSL Behring, Sanofi Genzyme, and Takeda. He has received grant/research support from Bayer and BioMarin. He serves on the speaker's bureau for Bayer, CSL Behring, Sanofi Genzyme, and Takeda. His presentation has been peer reviewed for any bias.
  Planning Committee:
Bill Williams, MD has no real or perceived financial relationships to disclose.
Jeremy Williams has no real or perceived financial relationships to disclose.
Jacqueline Cole, RN, MS, CMCN has no real or perceived financial relationships to disclose.

NAMCP and/or the presenter has copyright or has received permissions for use of materials provided in this activity.

Accreditation & Designation
This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of the National Association of Managed Care Physicians (NAMCP) and American Association of Managed Care Nurses (AAMCN). The National Association of Managed Care Physicians is accredited by the ACCME to provide continuing medical education for physicians.

NAMCP designates this enduring material for a maximum of 1 AMA PRA Category 1 credit(s)TM. Each
physician should claim credit commensurate with the extent of their participation in the activity.

The American Association of Managed Care Nurses is accredited as a provider of nursing continuing professional development by the American Nurses Credentialing Center's Commission on Accreditation.

Nurses who complete this activity and achieve a passing score will receive 1 hour in nursing continuing professional development.

This activity has been approved by the American Board of Managed Care Nursing for 1.0 contact hours toward CMCN recertification requirements.

This activity is supported by an educational grant from
BioMarin Pharmaceutical Inc.

NAMCP and/or this website does not provide medical advice, diagnosis or treatment. NAMCP does not endorse or imply endorsement of the content on any linked website. This website is to be used as an informational resource. With any health related concern, consult with your physician or healthcare professional.

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