AGENDA
Thursday, February 23, 2023
1:00 – 2:00 PM
Registration
2:00 – 3:45 PM Session I
Clinical assessment of alpha-1 antitrypsin (AAT) deficiency related liver disease (LD)
Moderated by: Ginger Clark, MD and Scott Santarella
2:00 – 2:10 PM
Welcome remarks
Rohit Loomba, MD, MHSc
2:10 – 2:25 PM
Epidemiology, natural history, and diagnosis of adult AAT deficiency related LD
Ginger Clark, MD
2:25 – 2:40 PM
Pathogenesis of AAT deficiency related LD
David Brenner, MD
2:40 – 2:55 PM
Liver histologic assessment in AAT deficiency related LD
Cynthia Behling, MD
2:55 – 3:10 PM
Fibroscan, ARFI, SWE, and ultrasound-based modalities in AAT deficiency related LD
Raj Vuppalanchi, MD
3:10 – 3:25 PM
Update on clinical trials in AAT LD
Monica Tincopa, MD, MS
3:25 – 3:45 PM
Panel discussion and Q&A
3:35 – 4:15 PM
Break and poster viewing
4:15 – 6:00 PM Session II
Mechanisms of fibrosis progression in the liver and lungs
Moderated by: David Brenner, MD and Pavel Strnad, MD
4:15 – 4:30 PM
Unique and common mechanisms of fibrosis progression in AAT deficiency related LD
David Brenner, MD
4:30 – 4:45 PM
Current Alpha-1 Foundation programs
Scott Santarella
4:45 – 5:00 PM
How to enrich recruitment into trials
Pavel Strnad, MD
5:00 – 5:15 PM
Treatment endpoints in AAT deficiency related LD
Rohit Loomba, MD, MHSc
5:15 – 5:30 PM
Debate: Who should be treated? Only those with stage 3 and 4 disease versus all fibrosis stages
For Don Rockey and against Mattias Mandorfer
5:30 – 5:45 PM
Opportunities to collaborate with FDA and understanding the process
Ruby Mehta, MD
5:45 – 6:00 PM
Panel discussion and Q&A
6:00 – 7:00 PM
Welcome reception
*** LD: liver disease
***AAT: alpha-1 antitrypsin
Friday, February 24, 2023
7:00 – 8:00 AM
Breakfast
8:00 – 9:35 AM Session III
Biomarkers of alpha-1 antitrypsin (AAT) deficiency related liver disease (LD)
Moderated by: Pavel Strnad, MD and Mark Brantly, MD
8:00 – 8:15 AM
AI based assessment of liver pathology in AAT
Janani Iyer, PhD (PATH AI)
8:15 – 8:30 AM
MRI imaging-based biomarkers of AAT deficiency related LD
Veeral Ajmera, MD
8:30 – 8:45 AM
Recent updates from AAT LD: three-center prospective study
Jeffrey Teckman, MD
8:45 – 9:00 AM
Should we screen all patients with ZZ lung disease for liver disease? How should they be screened and what are the opportunities for collaboration with the biomarker consortium?
Jeanine D'Armiento, MD
9:00 – 9:15 AM
How to set up collaboration between a pulmonary clinic and a liver clinic
Ginger Clark, MD
9:15 – 9:35 AM
Panel discussion and Q&A
9:35 – 10:00 AM
Break and poster viewing
10:00 – 11:30 AM Session IV
Emerging therapies and clinical drug development endpoints
Moderated by: Alice Turner, MBChB (Hons), MRCP, PGCE (MedEd), PhD and Aleksander Krag, MD
10:00 – 10:15 AM
How to assess pulmonary safety of IP in an AAT LD trial: issues and proposed solutions
Mark Brantly, MD
10:15 – 10:30 AM
How to engage the FDA and EMA regulatory pathways for clinical drug development in AAT deficiency related LD
Rohit Loomba, MD, MHSc
10:30 – 10:45 AM
Real world experience: why, when, and how? What can we learn from other disease areas?
Michael Fried, MD
10:45 – 11:00 AM
Economic burden and patient reported outcome assessment in AAT deficiency related LD
Zobair Younossi, MD, MPH
11:00 – 11:15 AM
Role of AASLD, EASL, and societies in building consensus
Aleksander Krag, MD
11:15 – 11:30 AM
Concluding remarks for the open session
Rohit Loomba, MD, MHSc
11:30 AM – 12:15 PM
Break and lunch
12:15 PM
Meeting concludes
SPEAKERS
Click on a speaker's name to learn more about them!
UC San Diego Health
San Diego, CA
Cynthia Behling, MD
University of California – San Diego
La Jolla, CA
University of Florida College of Medicine
Gainesville, FL
CEO Sanford Burnham Prebys
La Jolla, CA
Ginger Clark, MD, MS
University of Florida Health
Gainesville, FL
Columbia University
New York, NY
UNC School of Medicine
Chapel Hill, NC
PathAi, Inc.
Boston, MA
University of Southern Denmark
Odense, Denmark
UC San Diego Health
San Diego, CA
Medical University of Vienna
Wien, Austria
U.S. Food and Drug Administration
Silver Spring, MD
Medical University of South Carolina
Charleston, SC
CEO of Alpha 1 Foundation
Coral Gables, FL
University Hospital RWTH Aachen
Aachen, Germany
Saint Louis University School of Medicine
St. Louis, MO
UC San Diego Health
San Diego, CA
Alice Turner, MBChB (Hons), MRCP,
PGCE (MedEd), PhD
University of Birmingham
Edgbaston, Birmingham
Indiana University School of Medicine
Indianapolis, IN
Inova Liver Disease Services
Fairfax, VA