Factor VIII Inhibitors: Generating a National Blueprint for Future Research

Scientific Priorities and Innovative Implementation Strategies for
FVIII Inhibitor Clinical Trials
Chairs: Diane Nugent & Donna DiMichele

8:30 a.m. - 9:00 a.m. - Key Note Address
Clinical Trials in the Personalized Medicine Era: Strategies and Issues
Nicholas Schork, Ph.D.
Distinguished Professor of Quantitative Medicine, City of Hope/TGen
Professor and Director, Human Biology, J. Craig Venter Institute
Adjunct Professor of Psychiatry and Biostatistics, University of
California, San Diego

9:00 a.m. - 9:30 a.m. - Working Group 1 Report:
Blueprint for scientific priorities and innovative implementation
strategies for FVIII inhibitor clinical trials
Margaret Ragni & Lindsey George

9:30 a.m. - 10:15 a.m. General Discussion of Working Group Recommendations

Scientific Priorities and Strategies for 21st Century Data and
Specimen Collection and Observational FVIII Inhibitor Cohort
Studies
Chairs: Michael Soucie & Diane Nugent

10:45 a.m. - 11:15 a.m. - Key Note Address
Creating a Lens for Large-Scale Learning from Health Care
Charles Bailey, MD, Ph.D.
Associate Professor of Clinical Pediatrics, Perelman School of Medicine
University of Pennsylvania

11:15 a.m. - 11:45 a.m. - Working Group 2 Report:
Blueprint for scientific priorities and strategies for 21st century data
and specimen collection and observational FVIII inhibitor cohort
studies
Barbara Konkle & Michael Recht

11:45 a.m. - 12:30 p.m. - General Discussion of Working Group Recommendations

Scientific Priorities and Implementation Strategies for Acquiring an
Actionable Understanding of FVIII Immunogenicity and the
Immunology of Both the Host Immune Response and Tolerance
Chairs: Keith Hoots and Craig Hooper

1:30 p.m. - 2:00 p.m. - Key Note Address: FVIII Immunogenicity and Immune Response
Intervening in Antigen Presentation: A Strategy to Reduce
Immunogenicity of Replacement Therapeutics
Elizabeth Mellins, MD
Professor of Pediatrics (Human Gene Therapy), Lucile Salter Packard
Children’s Hospital, Stanford University

2:00 p.m. - 2:30 p.m. - Working Group 3 Report:
Scientific priorities and implementation strategies for acquiring an
actionable understanding of FVIII immunogenicity and the
immunology of both the host immune response and tolerance
Shannon Meeks & Roland Herzog  

2:30 p.m. - 3:15 p.m. - General Discussion of Working Group 3 Recommendations

Design of Pregnancy/Birth Longitudinal Cohorts That Leverage
‘Omics,’ Existing Phenotypic Data, and in Silico Modeling to Study
FVIII Immunogenicity, as well as Inhibitor Development and
Eradication
Chairs: Keith Hoots & Donna DiMichele

3:45 p.m. - 4:15 p.m. - Key Note Address: Novel Science and the Role of Hemophilia PUP
Cohorts in Solving the Problems of FVIII Immunogenicity
Heterogeneous Outcomes in Rare Genetic Disorders: Lessons From
3q29 Deletion Syndrome
Jennifer Mulle, Ph.D., MHS
Assistant Professor, Epidemiology, Rollins School of Public Health
Emory University

4:15 p.m. - 4:45 p.m. - Working Group 4 Report:
Design of pregnancy/birth longitudinal cohorts that leverage
‘omics,’ existing phenotypic data, and in silico modeling to study
FVIII immunogenicity, as well as inhibitor development and
eradication
Deborah Brown & Jill Johnsen

4:45 p.m. - 5:30 p.m. - General Discussion of Working Group 4 Recommendations

5:30 p.m. - 6:00 p.m. - Overview of the Day 1 and Preview of Day 2
Steven Pipe & Denise Sabatino

Scientific Priorities and Innovative Implementation Strategies for
Clinical Trials
Steven Pipe & Denise Sabatino

11:00 a.m. - 11:30 a.m. -  Working Group 1 Small Group Discussion:
Recap and presentation of a revised blueprint for the design and
Implementation of FVIII inhibitor clinical trials
Margaret Ragni & Lindsey George

11:30 a.m. - 12:00 p.m. - Final General Discussion

Scientific priorities and strategies for 21st century data and
specimen collection and observational cohort studies

12:00 p.m. - 12:30 p.m. - Working Group 2 Small Group Discussion:
Recap and presentation of a revised blueprint for future
data/specimen collection and cohort development for FVIII inhibitor
clinical research
Michael Recht & Barbara Konkle

12:30 p.m. - 1:00 p.m. Final General Discussion

Scientific Priorities and Implementation Strategies for Acquiring an
Actionable Understanding of FVIIII Immunogenicity and the
Immunogenicity and the Immunology of Both the Host Immune
Response and Tolerance
Steven Pipe & Denise Sabatino

2:00 p.m. - 2:30 p.m. - Working Group 3 Small Group Discussion:
Recap and presentation of a revised blueprint for future
mechanistic and translational research on FVIII immunogenicity
and the host immune response
Shannon Meeks

2:30 p.m. - 3:00 p.m. - Final General Discussion

Design of pregnancy/birth longitudinal cohorts that leverage
‘omics,’ existing phenotypic data, and in silico modeling to study
FVIII immunogenicity, inhibitor development and eradication

3:00 p.m. - 3:30 p.m. - Working Group 4 Small Group Discussion:
Recap and presentation of revised blueprint for data capture and
mechanistic studies required to build precision medicine-based
clinical decision-making algorithms to eradicate and prevent FVIII
inhibitors in hemophilia A PUPs
Jill Johnsen & Deborah Brown

3:30 p.m. - 4:00 p.m. - Final General Discussion

4:00 p.m. - 5:00 p.m. Workshop Summary and Next Steps
Steven Pipe & Denise Sabatino

5:00 p.m. - 5:15 p.m. Closing Comments
Keith Hoots & Donna DiMichele