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, FACG, FACP, AGAF, FAASLD

​

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