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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
Cystic fibrosis (CF), also known as mucoviscidosis, is a genetic,
autosomal recessive disorder that affects mostly the lungs but also
the pancreas, liver, kidneys and intestine. It is caused by the
presence of mutations in both copies of the gene for the protein
cystic fibrosis transmembrane conductance regulator (CFTR). Those
with a single working copy are carriers and otherwise mostly normal.
CFTR is involved in production of sweat, digestive fluids, and
mucus. When CFTR is not functional, secretions which are usually
thin instead become thick. The condition is diagnosed by a sweat
test and genetic testing. Long-term issues include difficulty
breathing and coughing up sputum as a result of frequent lung
infections. Other symptoms include sinus infections, poor growth,
fatty stool, clubbing of the finger and toes, and infertility in
males among others. According to the Cystic Fibrosis Foundation,
about 30,000 people are currently living with cystic fibrosis, and
approximately 1,000 new cases of CF are diagnosed each year. There
is no cure for cystic fibrosis, but recent treatment breakthroughs
have the ability to provide a greater quality of life and improved
outcomes for patients with CF, especially in combination therapy.
Upon completion of this
activity, participants will be able to:
Compare and contrast current and
emerging CFTR-based therapeutic options, including combination
therapies, in terms of efficacy, safety, and clinical potential
to improve pulmonary and non-pulmonary outcomes of patients with
Explore clinical trial data and the
role of newer combination CFTR modulator therapies, including
triple combination regimens, and discuss ways to integrate them
into the evolving treatment paradigm
Identify cystic fibrosis patients
who would potentially benefit from a CFTR modulator therapy
Describe challenges and
opportunities in CF disease management, including patient
adherence, quality of life and total cost of care
Examine the importance of newborn
screening, accurate diagnosis, genomic evaluation, and early
pharmacologic intervention in the management of cystic fibrosis
Assess the managed care
considerations on CFTR modulator therapies, including
combination options, and explore where these agents fit in the
cystic fibrosis management paradigm
||Patrick A. Flume, MD,
Professor of Medicine and Pediatrics
Director, Adult Cystic Fibrosis Program
Powers-Huggins Endowed Chair for Cystic Fibrosis
Medical University of South Carolina
serves as a consultant for Aureus Clinical, Corbus
Pharmaceuticals, Eloxx Pharmaceuticals, Insmed, and
Janssen. He has received grant/research support from
AbbVie, Corbus Pharmaceuticals, the Cystic Fibrosis
Foundation Therapeutics, Insmed, and the National
Institute of Health. 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
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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