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Beyond itch: Exploring the clinical benefits of cholestasis treatment in children
Presented by: Mirum Pharma - Medical
Date/time: Thursday, October 13, 19:15–20:45 EDT (7:15 PM - 8:45 PM)
Room: Sebastian L 1/2

Program

19:15–19:25   Welcome and introduction William Balistreri, MD
                         Cincinnati Children’s Hospital Medical Center, USA

19:25–19:55  Ileal bile acid transporter (IBAT) inhibitors for cholestasis: Beyond itch control?
                       Noelle Ebel, MD
                       Stanford University, USA

                      Binita Kamath, MBBChir, MRCP, MTR
                      Hospital for Sick Children, Canada

                      Regino Gonzalez-Peralta, MD
                      AdventHealth for Children and AdventHealth Transplant Institue, USA

                       William Balistreri, MD

19:55–20:25 Exploring the benefits of cholestasis treatment: Clinical case studies
                      Regino Gonzalez-Peralta, MD
                      Noelle Ebel, MD
                      Binita Kamath, MBBChir, MRCP, MTR
                      William Balistreri, MD

20:25–20:35 Q&A

20:35–20:45 Concluding remarks William Balistreri, MD

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Progress in the Management of Acid Sphingomyelinase Deficiency: Improving Diagnosis and Evaluating the Role of Enzyme Replacement Therapy 
Presented by: Catalyst Med Ed for Sanofi
Date/time: Thursday, October 13, 7:15 PM - 9:15 PM
Room: Sebastian L 3/4

Description: Enzyme replacement therapy (ERT) with olipudase alfa has recently been approved internationally for acid sphingomyelinase deficiency (ASMD), a rare lysosomal storage disorder with predominant hepatic and gastroenterologic manifestations. The availability of this first-ever disease-specific therapy underscores the importance of prompt disease recognition and differential diagnosis to ensure individuals can access treatments that may change their disease course. Patients with undiagnosed chronic ASMD often present to gastroenterology, hepatology, and related clinical practice settings because hepatosplenomegaly, accompanied by other gastrointestinal/hepatic symptoms, often prompts patients to seek medical care. This accredited continuing medical education symposium will outline strategies that hepatologists, gastroenterologists, and other healthcare providers (HCPs) can implement to lower their threshold of suspicion for ASMD when confronted with a patient with suggestive symptoms. This activity will also discuss the principles of ASMD care, taking into consideration emerging ERT and supportive care measures. Finally, a unique case-based approach will highlight the burden of misdiagnosis and review practical methods to navigate challenges in the differential diagnosis of ASMD.

Registration link:  catalystmeded.com/ASMD2  

Speakers: 
Melissa Wasserstein, MD (Course Director) 
Chief, Division of Pediatric Genetic Medicine 
Professor of Pediatrics and Genetics 
The Children's Hospital at Montefiore 
The University Hospital for Albert Einstein College of Medicine 
Bronx, New York   

Roberto Giugliani, MD, PhD 
Professor, Department of Genetics 
Federal University of Rio Grande do Sul 
Porto Alegre, Brazil  

Margaret McGovern, MD, PhD 
Yale School of Medicine 
Deputy Dean for Clinical Affairs 
CEO, Yale Medicine 
New Haven, Connecticut  

LEARNING OBJECTIVES 

• Recognize ASMD as a potential etiology for hepatosplenomegaly and other hepatic/gastroenterologic manifestations
• Identify multisystem clinical manifestations that are suggestive of ASMD
• Apply diagnostic algorithms and appropriate assays to rule out confounding diagnoses and make a differential diagnosis of ASMD
• Evaluate the efficacy and safety of ERT for pediatric and adult patients with ASMD
• Implement strategies to optimize multidisciplinary, individualized management of ASMD, considering the use of ERT and supportive care

CE Dinner Symposium
Jointly provided by AKH Inc., Advancing Knowledge in Healthcare and Catalyst Medical Education, LLC.

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Build your nutrition toolbox with innovative solutions for practical management of your pediatric patients
Presented by: Nutricia North America
Date/time: Friday, October 14, 6:30 am - 8:00 am
Room: Sebastian L 1/2

Learning objectives:

• Recognize clinical scenarios in which ready to feed amino acid-based formula may be indicated.
• Identify a novel, evidence-based way of managing term infants with malnutrition and growth failure.
• Explore GI symptom management with enteral patients using whole food formula.

Moderator:
Ann O. Scheimann, M.D., M.B.A
Associate Professor of Pediatrics
Division of Pediatric Gastroenterology and Nutrition
Johns Hopkins Hospital

Speakers:
Alison Cassin, MS, RD, CSP, LD
Cincinnati Children’s Hospital Medical Center

Nishant Patel, MD
Pediatric Gastroenterologist
Arnold Palmer Hospital

Sharon Weston, MS, RD, CSP, LDN
Sr. Clinical Nutrition Specialist

Register here: nlc.pub/NASPGHAN22

This program is CE-eligible for dietitians and nurses in the U.S.

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Updates in Eosinophilic Esophagitis: Exploring Underlying Mechanisms and Integrating Novel Targeted Therapy to Improve Patient Care
Presented by: PeerView for Sanofi and Regeneron Alliance
Date/time: Friday, October 14, 6:00 pm - 7:30 pm
Room: Sebastian L 3/4

Description: Eosinophilic esophagitis (EoE) is an increasingly prevalent, chronic, T2 inflammatory disease triggered by food and/or environmental allergens. Due to underlying tissue inflammation, patients suffer diminished quality of life due to symptoms such as weight loss, difficulty swallowing, and heartburn. Patients can experience these symptoms for a long time before receiving a proper diagnosis. Once diagnosed, treatment options have historically been limited. Fortunately, therapies that treat other type 2 disorders are in development or are approved to treat EoE. In this one-hour interactive activity using patient cases and consultation, participants will benefit from expert instruction in the art of diagnosis (especially identifying comorbid T2 disorders such as asthma), the underlying causes, and the newest treatments for EoE, including biologic therapies. Through peer coaching, participants will learn how to integrate these newer therapies into management plans to ease symptoms and manage the disease.

Chair & Presenter:
Sandeep K. Gupta, MD
Community Health Network
Riley Hospital for Children
Indiana University School of Medicine
Indianapolis, Indiana

Presenter: 
Gregory M. Constantine, MD
Clinician-Scientist - Eosinophilic Disorders
Allergy/Immunology
Bethesda, Maryland

Presenter:
Amanda B. Muir, MD
The Children’s Hospital of Philadelphia
Philadelphia, Pennsylvania

Registration link: www.PeerView.com/EoEOrlando22-Live

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Cholestatic Pruritus Across the Age Spectrum: What Are You Missing?
Presented by: The Chronic Liver Disease Foundation
Date: Friday, October 14th 6:30 – 8:00 PM
Room: Sebastian L 1-2

Objectives:

• Explain how pruritus in pediatric patients may indicate a cholestatic, progressive disease (PFIC) that should lead to further testing and interventions
• Analyze the role of genetic testing in cholestatic disorders and link genotype with other clinical parameters, including serum bile acid, to PFIC patient prognosis
• Identify the needs in caring for pediatric vs. adult patients with cholestatic disease to properly transition care
• Discuss the importance of gene sequencing in adult-onset cholestatic disease

Faculty:
Naim Alkhouri, MD
VP of Academic Affairs
Chief of Transplant Hepatology
Director of the Fatty Liver and Metabolic Liver Diseases
Arizona Liver Health (ALH)
Glendale, Arizona 

Nitika Gupta, MD, DCH, DNB, MRCPCH
Pediatric Gastroenterology Professor
Emory University School of Medicine
Atlanta, Georgia 

Miriam Vos, MD, MSPH
Co-Director, Center for Clinical and Translational Research (CCTR)
Professor of Pediatrics
Division of GI, Hepatology and Nutrition &
Nutrition Health Sciences, Graduate Division
Emory University School of Medicine
The Mason Transplant and Wellness Program Director
Children’s Healthcare of Atlanta
Atlanta, Georgia

Register now: https://mfuhrer100.wufoo.com/forms/q6fa9k809ljqr4/