Rome Time (CEST)
13.25|13.30 Welcome to participants and opening remarks
Aurelio Maggio (Italy)-Khaled Musallam (UK)-Vijay Sankaran (USA)
SESSION 1
Moderators: Aurelio Maggio (Italy); Antonis Kattamis (Greece)
13.30|13.50 Ali Taher (Lebanon)
Modern understanding of clinical forms and determinants of outcomes in thalassemia
- Conventional and new classifications
- The role of anemia/transfusions, iron overload/chelation in shaping disease prospect
13.50|14.10 Baba Inusa (UK)
Pathophysiology and clinical correlates in SCD: status quo
- Overview of key novel insights into pathophysiology of SCD
- Clinical observations in children and adults
14.10|14.30 Vijay Sankaran (USA)
Thalassemia and SCD from the eye of molecular pathology
- Correlations between genotypes and hematologic/clinical profiles
- Key discoveries in molecular pathways that reshaped our understanding of disease
14.30|14.45 Q&A - Interactive discussion on the topics of the current session (Faculty)
SESSION 2
Moderators: Giuliana Ferrari (Italy); Vijay Sankaran (USA)
14.45|15.05 John Tisdale (USA)
Bone marrow transplant in hemoglobinopathies: what have we achieved over the years?
- Review of outcomes data
- Developments in strategy and technique
15.05|15.25 Franco Locatelli (Italy)
Gene therapy: progress so far
- Available approaches and protocols
- Current data and ongoing/future trials
15.25|15.45 Josu De La Fuente (UK)
Gene editing: the new kid on the block
- Available approaches and protocols
- Current data and ongoing/future trials
15.45|16.05 Gianluca Forni (Italy)
Decisional algorithm for gene therapy: recommendations from “SITE”
- Clinical profile of the ideal candidate for gene therapy
- Main recommendations for enrollment
16.05|16.20 Q&A - Interactive discussion on the topics of the current session (Faculty)
SESSION 3
Moderators: Khaled Musallam (UK); Ali Taher (Lebanon)
16.20|16.40 Vip Viprakasit (Thailand)
Targeting ineffective erythropoiesis and iron dysregulation in thalassemia: evolving strategies
- Rationale and pathways
- Updates from animal studies and ongoing/future trials of novel agents
16.40|17.00 Maria Cappellini (Italy)
Luspatercept: optimal integration into clinical practice
- Overall program direction and recent data
- Real-life practical implementation
17.00|17.20 Elliott Vichinsky (USA)
Advances in management of SCD: agents on the horizon
- Updates on experience with HU
- New drugs in development
17.20|17.35 Q&A - Interactive discussion on the topics of the current session (Faculty)
SESSION 4
Moderator: Michael Angastiniotis (Cyprus)
17.35|17.55 Mehran Karimi (Iran)
Hemoglobinopathies and Covid-19: should we be concerned?
- Experience and challenges
- Strategies and solutions
17.55|18.00 Q&A - Interactive discussion on the topics of the current session (Faculty)
18.00|18.05 Take home messages and concluding remarks
Aurelio Maggio (Italy), Khaled Musallam (UK), Vijay Sankaran (USA)