PROGRAMME
08:30 – 09:00 - Gather together and On-site Registration
09:00 – 09:10 - Introduction
Sumathi Iyengar - Amir Kedar, WAS organizations and Fabio Candotti, CHUV, Lausanne, Switzerland
09:10 – 09:25 – Families Stories
Family story – Daniel Wheeler, UK, Young Adult post GT and HSCT story
09:25 – 09:55 - Plenary Session 1
Markus G. Seidel - Deputy Head of the Division of Pediatric Hematology-Oncology, Medical University Graz, Austria
"Malignancies in IEI/PID
09:55 – 10:50 - Research Session 1
Chair: Fabio Candotti (Lausanne University Hospital, Lausanne, Switzerland)
09:55 - 10:10 Ismail Shakir - King Abdullah University of Science and Technology, Saudi Arabia
“WASP Regulates the Nucleolus and Macrophage Inflammatory Response via NPM1”
10:10 - 10:25 Rhaissa Calixto Vieira - Karolinska Intitute, Solna, Sweden
“Small compound PTW08 restores megakaryocyte and platelet production in Wiskott-Aldrich Syndrome”
10:25 - 10:40 Lia G Pinho - Karolinska Institute, Solna, Sweden
“A systematic approach to understanding how patient variants affect the activity of Wiskott-Aldrich syndrome protein”
10:40 - 10:50 Questions & answers and discussion
10:50 – 11:10 – Break
11:10 – 11:40 - Plenary Session II
Loïc Dupre- co-head of team ‘Integrative Mapping of Lymphocyte Signaling and Function INFINITy’ Toulouse Institute for Infectious and Inflammatory Diseases – INSERM UMR 1291 – France
"WASP and co-actors as remodelers of the actin networks sustaining T cell function"
11:40 – 12:30 –Research Session 2
Chair: Lisa Westerberg (Karolinska Institute, Solna, Sweden)
11:40 - 11:55 Jordan Chill - Bar Ilan University, Israel
“A Molecular View of WAS Protein in Health and Disease: The Case of Hotspot Mutation R86C”
11:55 - 12:10 Aisha Siddique - King Abdullah University of Science and Technology, Saudi Arabia
“Beyond the Cytoskeleton: Isogenic iPSC Models Reveal New Mechanisms of Wiskott-Aldrich Syndrome”
12:10 - 12:25 TBA - Karolinska Intitute, Solna, Sweden
"The actin regulator WASp associates with chromatin and regulates RNA polymerase II transcription in B cells"
12:25 - 12:30 Questions & answers and discussion
12:30 – 13:10 – Gene Therapy Session
Chair: Hans Ochs (University of Washington School of Medicine in Seattle, Seatle, USA)
12:30 - 12:45 Sabina Cenciarelli - San Raffaele Hospital, Milan, Italy
“Megakaryocytes alterations and Thrombopoietin levels in patients affected by Wiskott-Aldrich Syndrome before and after Gene Therapy with Etuvetidigene Autotemcel”
12:45 - 13:00 Michaela Semeraro - Hôpital Necker Enfants malades APHP, Paris, France
“Incomplete myeloid correction drives late IL-1–sensitive inflammation after WAS Gene Therapy”
13:00 - 13:10 Questions & answers and discussion
13:10 – 14:15 - Lunch
14:15 – 14:30 – Families Story
Family story – Alon Cand, Israel. Young Adult Future decision-making considerations
14:30 – 15:00– Gene Therapy Panel
Chair: Michael Albert (DKNS, Munich, Germany)
“Challenges of extending access to gene therapy for WAS”
Participants: Alessandro Aiuti, Francesca Ferrua, Fabio Candotti, Catriona Crombie, Sheela Upadhyaya, Sumathi Iyengar.
15:00 – 15:30 - Plenary Session C
Fabio Candotti - CHUV, Lausanne, Switzerland
“IgA nephropathy and WAS”
15:30 – 16:20 –Clinical Session 1
Chair: Francesca Ferrua (San Raffaele Hospital, Milan, Italy)
15:30 - 15:45 Maja Stojanović – President, Serbian Group for Primary Immunodeficiencies, University of Belgrade, Belgrade, Serbia. (Invited speaker)
“Challenges of diagnosing and treating Wiskott-Aldrich Syndrome (WAS) in resource-constrained settings”.
15:45 - 16:00 Silvia Juliani - University of Brescia, ERN Rita Center, Brescia, Italy
“Retrospective-Prospective immunological study of Class I and Class II Wiskott- Aldrich patients”
16:00 - 16:15 Anna Shcherbina - Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia
“Thrombopoietin receptor agonists (TPO-RA) and spleen tyrosine kinase inhibitor (SYKi) for the treatment of thrombocytopenia in Wiskott-Aldrich syndrome (WAS) patients”
16:15 - 16:20 Questions and answers and discussion
16:20 – 16:40 –Break
16:40 – 17:30 –Clinical Session 2
Chair: Suhag Parikh
16:40 - 16:55
Monirujjaman Biswas - National Institute of Respiratory Diseases, New Delhi, India.
“Early Identification, Clinical Outcomes and Predictors of Autoimmune Complications in Wiskott–Aldrich Syndrome”
16:55 - 17:10 Deepti Suri - Postgraduate Institute of Medical Education and Research, Chandigarh, India.
“Renal injury in patients with Wiskott Aldrich Syndrome: an underrecognized entity”
17:10 - 17:25 Lea Makhlouf - Necker-Enfants Malades Hospital, AP-HP, Paris,
“Nearly Two Decades of Allogeneic HSCT for Wiskott-Aldrich Syndrome: A Single-Center Experience”
17:25 - 17:30 Questions and answers and discussion
17:30 – 18:00 - Clinical Session Panel
Chair: Anders Fasth (Sweden)
“Delphi Consensus statement for WAS”
Participants: Fabio Candotti, Alessandro Aiuti, Michael Albert, Sumathi Iyengar
18:00– 18:10 – Closing and Summary of the day
Fabio Candotti, Michael Albert, Francesca Ferrua, Lisa Westerberg
18:10 – 19:00 – Reception
An informal get-together to renew acquaintances and meet new colleagues will be held at the Meeting Venue.
All registered participants are invited to join.
“Thrombopoietin receptor agonists (TPO-RA) and spleen tyrosine kinase inhibitor (SYKi) for the treatment of thrombocytopenia in Wiskott-Aldrich syndrome (WAS) patients”