“REEL” Time Patient Counseling: Optimal Approaches for Managing and Monitoring Patients With LC-FAOD

Release Date: April 28, 2021
Expiration Date: April 28, 2022

Activity Overview

In this CME-certified activity, Jerry Vockley, MD, PhD, is joined by Suzanne Hollander, MS, RD, to explore the diagnosis and management of long-chain fatty acid oxidation disorders (LC-FAODs). These disorders are recessively inherited inborn errors of mitochondrial fatty acid oxidation. LC-FAODs are caused by defects in the metabolic pathway that converts stored long-chain fatty acids into energy within muscles and other cells, leading to a deficiency in mitochondrial energy production during times of physiologic stress and fasting. LC-FAODs cause serious clinical complications, including cardiomyopathy, recurrent episodes of rhabdomyolysis, weakness, hepatic failure, hypoglycemia, and hyperammonemia. These complications persist in patients despite decades of dietary therapy, a diet rich in even-carbon, medium-chain triglycerides (MCT), or a combination of both. Premature mortality may reach 60% to 90% in patients with these diseases.

In this activity, Dr Vockley discusses key issues of pathophysiology, early diagnosis, and optimal management of patients with LC-FAODs, while Ms. Hollander talks with an actor playing the role of a parent of a child with an LC-FAOD. They demonstrate some of the common concerns of patients and their families, and how health care providers can offer information and assistance in a timely and developmentally appropriate manner.

Acknowledgement of Commercial Support

This activity is supported by educational grants from Ultragenyx Pharmaceutical Inc.

Target Audience

This educational activity is directed toward pediatricians, primary care providers, metabolic dietitians, endocrinologists, neurologists, including neuromuscular specialists, nephrologists, geneticists and genetic counselors, nurse practitioners, physician assistants, and other health care professionals involved in the care of patients with metabolic disorders.

Learning Objectives

Upon successful completion of this activity, you should be better prepared to:

• Determine clinical signs and testing approaches to facilitate diagnosis of long-chain fatty acid oxidation disorders (LC-FAOD)
• Identify approaches for optimal, individualized management and monitoring of patients with LC-FAOD
• Assess data regarding the mechanism of action, safety, and efficacy results from recent clinical trials that evaluated emerging strategies among patients with LC-FAOD

Faculty, Staff, and Planners’ Disclosures

The staff of Physicians’ Education Resource®, LLC, have no relevant financial relationships with ineligible entities.

Faculty

Suzanne Hollander, MS, RDN, LDN
Clinical Nutrition Specialist
Division of Genetics and Genomics
Boston Children’s Hospital
Boston, Massachusetts

Disclosures: Consultant: Vitaflo USA.

Jerry Vockley, MD, PhD
University of Pittsburgh
Cleveland Family Endowed Chair in Pediatric Research
Professor of Human Genetics
UPMC Children’s Hospital of Pittsburgh
Chief of Medical Genetics
Director of the Center for Rare Disease Therapy
Pittsburgh, Pennsylvania

Disclosures: Grant/Research Support: Reno Pharmaceuticals, Ultragenyx Pharmaceuticals.

PER^® mitigated all COI for faculty, staff, and planners prior to the start of this activity by using a multistep process.

Off-Label Disclosure and Disclaimer

This activity may or may not discuss investigational, unapproved, or off-label use of drugs. Learners are advised to consult prescribing information for any products discussed. The information provided in this accredited activity is for continuing education purposes only and is not meant to substitute for the independent clinical judgment of a health care professional relative to diagnostic, treatment, or management options for a specific patient’s medical condition. The opinions expressed in the content are solely those of the individual faculty members and do not reflect those of PER^® or any company that provided commercial support for this activity.