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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
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
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
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
||Mark T. Reding, MD
Associate Professor of Medicine
Director, Center for Bleeding and Clotting Disorders
University of Minnesota Medical Center
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.
MD has no real or perceived financial relationships to
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
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
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