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Welcome | |
SESSION I – STEM CELL BIOLOGY: FROM STRESS TO FAILURE Chairs: Tim Brümmendorf and Markus Manz |
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Introduction | |
Haemopoietic stem cell activation and failure Andreas Trumpp (Heidelberg) |
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Genotoxic stress and repair in stem cells Ketan J. Patel (Cambridge) |
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Inflammageing of the haemopoietic system Markus Manz (Zürich) Question from Marc Raaijmakers: Answer from Markus Manz: |
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Immunologic Basis to Poor Graft Function Post Allogeneic Transplantation Ashvind Prabahran (Melbourne) |
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Discussion | |
SESSION II – CONSTITUTIONAL MARROW FAILURE 1: FANCONI ANAEMIA (FA) Chairs: Carlo Dufour and Inderjeet Dokal |
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Introduction | |
Fanconi anaemia : diagnostic and natural history in the bone marrow Jean Soulier (Paris) |
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Classical and new treatment of Fanconi anaemia Carlo Dufour (Genova) |
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Gene therapy for Fanconi anaemia Juan Bueren (Madrid) |
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Genetic Rescue of Fanca -/- HSPCs with Evi-1 Overexpression Suggests a Mechanistic Link with DNA Damage Response Susanne Lux (Heidelberg) |
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Discussion |
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SESSION III – CONSTITUTIONAL MARROW FAILURE 2: RIBOSOME DISEASES Chairs: Marc H. G. P. Raaijmakers and Alan Warren |
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Introduction | |
Diamond-Blackfan anaemia (DBA), ribosomes and haemopoiesis Vijay Sankaran (Boston) |
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Shwachman-Diamond syndrome (SDS) and related ribosomopathies Alan Warren (Cambridge) |
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Management of ribosomopathies Yigal Dror (Toronto) |
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A Single Base Change Makes the Cure: Correcting the Most Common SBDS Mutation in Shwachman-Diamond Syndrome Chi-Yuan Zhang (Boston) |
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Discussion | |
SESSION IV – CONSTITUTIONAL MARROW FAILURE 3: TELOMERE BIOLOGY DISEASES Chairs: Inderjeet Dokal and Tim Brümmendorf |
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Introduction | |
Telomeres and telomerase in health and ageing Alison Bertuch (Houston) |
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Dyskeratosis congenita and related haematological diseases Inderjeet Dokal (London) |
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Diagnosis and clinical presentation of adult onset telomeropathies Fabian Beier (Aachen) |
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Natural History and Outcomes Depending on Organ System Involvement in Patients with Clinically-Relevant Short Telomeres Abhishek Mangaonkar (Rochester) |
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Discussion | |
Poster walks Leaders: Tim Brümmendorf and Marc H.G.P. Raaijmakers |
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SATELLITE SYMPOSIUM: Beyond current endpoints: a patient-centric approach to PNH treatment Chair: Régis Peffault de Latour Speakers: Pascale Burmester, Talha Munir and Jens Panse This symposium is organized by Roche |
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SESSION V – IMMUNE APLASTIC ANAEMIA AND PAROXYSMAL NOCTURNAL HAEMOGLOBINURIA (PNH) 1. PATHOPHYSIOLOGY AND DIAGNOSIS Chairs: Neal Young and Regis Peffault de Latour |
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Introduction | |
Pathophysiology of immune aplastic anaemia Neal Young (Bethesda) |
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Pathophysiology of PNH Rosario Notaro (Florence) |
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Modern diagnosis of aplastic anaemia and PNH Austin Kulasekararaj (London) |
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Single Cell eQTL Mapping Identifies Cell-Type Specific Control of Autoimmune Disease Kirsten Fairfax (Hobart) |
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Discussion In pediatrics patients, <18 years do you recomend use Eltrombopag? To Austin (and everybody): somatic mutations are frequent in AA, but emergind data clearly show that in addition to “myeloid cancer genes” they may affect “immune-privilege genes”, such as PIGA or HLA. Can we conclude that we are looking at standard rate of mutations, keeping in mind that these 2 types are simply the ones that we may detect because they lead to expansions of mutant clones? Thus, are we overestimating the risk of MDS or leukemia by simply looking to NGS data? Antonio For panel (from Olaia Naveiras, Lausanne Switzerland): Have you observed a clinical overlap of SAA and late onset telomere biology disorders? Else how would you interpret this case? Young (29yo) patient with classic presentation of SAA (with autoimmune hepatitis) and partial response to hATG/Cs/eltrombopag (full response for neutrophils), Telomere attrition was detected and confirmed in the parents. No donor available. He remained fully dependent on platelet transfussions and partially transfussion-dependent for RBCs. He was started in danazol. Platelet transfussion independency was reached 18 months after danazol introduction. Ciclosporine and eltrombopag have not yet been fully tampered due to neutrophil dependency. Cytogenetics are normal at the last BM control (24 months after danazol introduction). Thank you for the great session! To Neal Young: Thank you for the outstanding review! Why – if evasion of IFNg competition for the MBL-receptor binding by eltrombopag is one of the major mechanisms of action in AA patients does romiplostim also seem to work, even in patients not responding to eltrombopag. And is the timing of the addition of eltrombopag to IST essential? Jens Panse @Neal: excellent talk ! Do you thinks the the lower risk of clonal evolution for patients under IST + ELT as opposed to ELT monotherapy (and probably also IST alone) would also hold true fror the combination of CSA + ELT (compared to ELT or CSA mono therapy) in elderly, frail AA patients (who are in any way at higher risk of clonal evolution ? Thanks Tim Brümmendorf What would you recommend for a 25 y/o male who relapsed 2 years post sib allogeneic HCT, did not respond to salvage with eltrombopag and cyclosporine and had a second allogeneic HCT from the same sibling. no GVHD – prolong CSP duration? add sirolimus? Is it possible to accelerate clonal evolution with immunosuppressive therapy in severe aplastic anemia? (Abhishek Mangaonkar – Mayo Clinic) |
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SATELLITE SYMPOSIUM: A journey through the SAA and PNH treatment landscapes Chair: Régis Peffault de Latour and Antonio Risitano Speakers: Régis Peffault de Latour and Antonio Risitano This symposium is organized by Novartis |
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SESSION VI – IDIOPATHIC APLASTIC ANAEMIA AND PAROXYSMAL NOCTURNAL HAEMOGLOBINURIA (PNH) 2. DIFFERENTIAL DIAGNOSIS AND CLONAL EVOLUTION Chairs: Neal Young and Jaroslaw P. Maciejewski |
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Introduction | |
Keynote Presentation: Bone marrow failure and somatic mutations: the PNH paradigm Lucio Luzzatto (Dar-es-Salaam) |
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Discussion | |
Innate immunity and clonal evolution in bone marrow failure Alan List (Tampa) |
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Impact of clonal haemopoiesis in aplastic anaemia Jaroslaw P. Maciejewski (Cleveland) |
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Health-related quality of life and its assessment in aplastic anaemia and paroxysmal nocturnal haemoglobinuria Jens Panse (Aachen) |
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The Metabolomic Status of the Differentiating Myeloid Lineage in MDS with Low and High Bone Marrow Blast Counts Aikaterini Poulaki (Athens) |
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Discussion | |
SATELLITE SYMPOSIUM: Current unmet need and the future of complement inhibition in PNH Chair: Peter Hillmen and Antonio Risitano Speakers: Peter Hillmen and Antonio Risitano This symposium is organized by Apellis |
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SESSION VII – TREATMENT OF IMMUNE APLASTIC ANAEMIA (AA) Chairs: Andrea Bacigalupo and Carlo Dufour |
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Introduction | |
Immunosuppression and TPO agonists Régis Peffault de Latour (Paris) |
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Transplantation for acquired SAA: are we making progress? Andrea Bacigalupo (Rome) |
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How to handle moderate aplastic anaemia Britta Höchsmann (Ulm) |
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Blockade of Common Gamma Chain Cytokines with REGN7257, an Interleukin 2 Receptor Gamma (IL2RG) Monoclonal Antibody, Protected Mice from T Cell-Mediated Disease Audrey Le Floc’h (Tarrytown) |
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Discussion | |
SESSION VIII – TREATMENT OF PAROXYSMAL NOCTURNAL HAEMOGLOBINURIA (PNH) Chairs: Regis Peffault de Latour and Hubert Schrezenmeier |
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Introduction | |
Classical treatment of PNH Hubert Schrezenmeier (Ulm) |
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Challenging situations Peter Hillmen (Leeds) |
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Novel complement inhibitors Antonio Risitano (Naples) |
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Results of the Pegasus Phase 3 Randomized Trial Demonstrating Superiority of the C3 Inhibitor, Pegcetacoplan, Compared to Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria Regis Peffault de Latour (Paris) |
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Discussion | |
SESSION IX – EMERGING AREAS AND NEW CHALLENGES Chairs: Marc H. G. P. Raaijmakers and Lucy Godley |
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Introduction | |
Genetic editing of haemopoietic stem cells. Are we getting there? Matthew Porteus (Stanford) |
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Unraveling the biology of normal and malignant haemopoietic stem and progenitor cells at the single cell level Sten Eirik Jacobsen (Stockholm) |
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Genetic predisposition for myeloid malignancies: clinical management Lucy Godley (Chicago) |
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Prevalence of Inherited Predisposition Syndromes in Young Patients with Acute Myeloid Leukemia and Aberrant Karyotypes Fabian Beier (Aachen) |
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Discussion | |
Closing remarks |