Scientific Program
The European Working Group of Myelodysplastic Syndrome Childhood (EWOG-MDS) and severe aplastic anemia (EWOG-SAA) is a consortium of National Working Groups of clinicians and researchers of 20 European Countries dedicated to diagnose and treat young individuals with acquired and inherited bone marrow failure, predisposition syndromes, MDS and rare MPD in young people.
Biannually, EWOG-MDS/SAA organizes an International Symposium to bring together renowned experts in the fields. The upcoming 11th International Symposium of EWOG-MDS/SAA will be held September 18 – 20, 2025 in Berlin, Germany.
National Coordinators and reference laboratories are the backbone of the EWOG and contribute to the Symposium.
The Symposium is a Hybrid Meeting. Content will be available for all registered participants until March 1, 2026.
Program
| Thursday, 18.09.2025 | |||||||||||
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| 07:30 – 08:30 | Breakfast | ||||||||||
| 08:30 – 08:45 | Opening of the Symposium For the Organizing Committee: Charlotte M. Niemeyer, Brigitte Strahm, Edoardo Muratore |
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| 08:45 – 12:00 | Education Session – organized by Young EWOG
Moderators Part I: Marc Bierings (Utrecht, The Netherlands), Felicia Andresen (Freiburg, Germany) Moderators Part II: Barbara de Moerloose (Ghent, Belgium), Edoardo Muratore (Bologna, Italy) |
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| 12:00 – 13:00 | Lunch | ||||||||||
| 13:00 – 14:30 |
Aplastic Anemia I |
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| Keynote Lecture: The role of somatic T-cell mutations and immune dysregulation in SAA Satu Mustjoki (Helsinki, Finland) Alternative Donor Peripheral Stem Cell Transplantation with TCRαβ/CD19 depletion for Pediatric Patients with Bone Marrow Failure Outcome of second hematopoietic stem cell transplantation in pediatric patients with severe aplastic anemia Late effects of immunosuppressive therapy in acquired bone marrow failure: Long-term follow-up of Czech patients with aplastic anemia and refractory cytopenia Improved engraftment following a treosulfan-fludarabine conditioning regimen compared to thiotepa-fludarabine in patients with refractory cytopenia of childhood |
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| 14:30 – 15:30 |
Aplastic Anemia II |
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| Mini Panel: Long-term Consequences of Therapy in Aplastic Anemia Moderator: Brigitte Strahm (Freiburg, Germany)
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| 15:30 – 16:00 | Coffee break | ||||||||||
| 16:00 – 17:30 |
The Path to Clonal Myeloid Malignancy I |
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| Assessment of genetic testing following current comprehensive clinical diagnostic evaluation for pediatric severe aplastic anemia Helen Reed*, J. De Jong, M. Gaviria, A. Koppayi, Y. Ahmed, I. Atkinson, M. Joos, M. Malsch, M. McClung, P. Nicholas, M. Shah, E. Sullivan, H. Xie, S. Zavarella, Y. Zhou, B. Zorman, L. Godley, T. Nakano, T. Olson, A. Bertuch, A. Shimamura *(Boston, USA) Molecular surveillance in bone marrow failure: Predictive value of clonal hematopoiesis Refractory cytopenia of childhood with normal karyotype: Long-term results of an observational approach Single-cell and spatial transcriptomics of pediatric myelodysplastic syndrome with refractory cytopenia of childhood reveal a disrupted bone marrow niche driving impaired hematopoiesis Concordance of Somatic Genetic Testing in Blood vs. Bone Marrow in Shwachman-Diamond Syndrome In-vivo mouse model of acute myeloid leukemia development in congenital neutropenia |
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| 17:30-19:00 |
The Path to Clonal Myeloid Malignancy II |
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| Keynote Lecture: How Risky is Clonal Hematopoiesis? Luca Malcovati (Pavia, Italy) Mini Panel: Clonal hematopoiesis in aging and germline predisposition: Is it the same?
End of Scientific Program |
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| 19:00 – 21:00 | Welcome Reception | ||||||||||
| Friday, 19.09.2025 | |
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| 07:30 – 08:30 | Breakfast |
| 08:30 – 10:00 |
Inherited Predispositions to Myeloid Neoplasms and their Clinical Implication |
| Keynote Lecture: GATA2 Deficiency: From Pathophysiology to Clinical Care Marlene Pasquet (Toulouse, France) St. Jude.org/gata2: Online catalogue of 900 individuals with germline GATA2 mutations Mitotic defects impaired CD34+ proliferation in GATA2 deficiency Superior survival following hematopoietic stem cell transplantation for high-risk marrow features in patients with Shwachman Diamond syndrome Impluse Statement: Tailoring Pre- and Post HSCT Therapy in Advanced MDS |
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| 10:00 – 11:30 | Poster Viewing (coffee served) |
| 10:10 | Poster Walk 1: SAA (inkl. Therapy) Poster Walk 2: Predisposition Poster Walk 3: Therapy (other than SAA) |
| 10:40 | Poster Walk 4: Classical bone marrow failure Poster Walk 5: MDS Poster Walk 6: JMML |
| 11:30 – 13:00 |
Inherited Bone Marrow Failure and Telomeropathies |
| Keynote Lecture: Germline Disorders Linked to Telomere Maintanance and Dysfunction Sharon Savage (Bethesda, USA) Functional analysis of non-canonical fancj variants in Fanconi anemia Clonal hematopoiesis in congenital neutropenia: Insights from somatic mutation analysis Transcriptional contingency in the early hematopoiesis of inherited bone marrow failure syndromes In vitro model of stage-specific evolution of clonal hematopoiesis in congenital neutropenia to CN/AML |
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| 13:00 – 13:30 | Lunch + grab a coffee |
| 13:30 – 14:30 |
(Epi-)genomic Landscape in JMML |
| Comprehensive characterization of somatic PTPN11-mutated JMML Edoardo Muratore*, V. De Haas, D. Bresters, C. Flotho, P. Goncalves, M. Hofmans, M. Kavcic, T. Lammens, D.B. Lipka, L. Petrikkos, C. M. Niemeyer for the EWOG-MDS JMML Working Group, P. Noellke, S. Ramamoorthy, D. Lebrecht, R. Masetti, M. Schoenung, and EWOG-MDS National Coordinators for EWOG-MDS *(Bologna, Italy) The watch-and-wait approach in patients with juvenile myelomonocytic leukemia (JMML): Results of the French national cohort Long-term outcome of an observational approach in patients with NRAS-mutated juvenile myelomonocytic leukemia and absence of high risk features Single-cell trajectories of monosomy 7 driven KRAS-mutant JMML |
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| 14:30-15:15 |
JMML Risk Factors for Outcome |
| Mini Panel: Towards an International Consensus on Risk Criteria Moderator: Christian Flotho(Freiburg, Germany), Daniel Lipka (Heidelberg, Germany)
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| 14:00 – 15:00 |
Meeting of reference pathologists EWOG Pathology:
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| 15:15 – 15:30 | Coffee break |
| 15:30 – 16:45 |
Classification and Clinical Approch to Overlap Syndromes and rare MPDs |
| Biological and clinical implications of subclonal genetic events in juvenile myelomonocytic leukemia revealed via error-corrected next-generation sequencing Daichi Sajiki*, H. Muramatsu, M. Wakamatsu, K. Ogawa, D. Yamashita, A. Yamamori, K. Narita, S. Kataoka, Y. Takahashi *(Nagoya, Japan) Predicting the unpredictable: Chimerism as a relapse indicator in JMML post-haematopoietic stem cell transplant Myeloproliferative neoplasms (MPN) diagnosed in childhood and adolescence – prospective data on clinical and genetic characteristics from the German national MPN childhood registry Impluse statement: Classification of Overlap Syndromes and rare MPD End of Scientific Program |
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| 17:00 | Departure for evening program |
| Saturday, 20.09.2025 | |
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| 07:30 – 08:30 | Breakfast |
| 07:30 – 08:30 | Closed EWOG Meeting |
| 08:30 – 10:00 |
Advanced MDS – from Clones to Therapy |
| Keynote Lecture: Therapy-induced clonal hematopoiesis Sam Behjati (Cambridge, UK) Genetic origins and clonal trajectories of monosomy 7 in pediatric MDS Genetic landscape of myelodysplastic syndrome in children Genetic landscape of primary myelodysplastic syndromes with excess of blasts (MDS-EB) in children and adolescents Genetic landscape and outcomes after hematopoietic stem cell transplantation in children and adolescents with therapy-related myeloid neoplasms Advanced myelodysplastic syndrome. The Dutch experience |
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| 10:00 – 10:20 | Coffee break |
| 10:20 – 11:45 |
Innovations in Therapy and Diagnostics for MDS |
| Integrated immunophenotyping and drug response profiling in pediatric MDS-EB : The Zurich-Vienna experince Zürich (Switzerland): Nastassja Scheidegger-Egloff, S. Gutnik, C. Moeller, F. Steffen, A. Arpagaus, L. Schori, S. Schuehle, S. Bohler, B. Haladik, A. Hurt, C.M. Koebl, M. Maurer-Granofszky, B. De Moerloose, H. Hasle, C.M. Niemeyer, M. Ussowicz, L. Vinci, A. Yoshimi-Noellke, K. Boztug, M.N. Dworzak, J-P. Bourquin, M. Erlacher, M. Schmugge, B. Bornhauser Vienna (Austria): Ben Haladik, M. Maurer-Granofszky, A. Hurt, C.M. Koelbl, N. Scheidegger, S. Gutnik, F. Steffen, B. De Moerloose, H. Hasle, M. Ussowicz, L. Vinci, A. Yoshimi-Noellke, J-P. Bourquin, C.M. Niemeyer, M. Schmugge, B. Bornhauser, M. Erlacher, M.N. Dworzak, K. Boztug BH3-mimetic treatment reveals subtype-specific BCL-2 protein dependency in pediatric MDS with excess blasts Stratus Prime™ GMP compliant process to create high numbers of HLA compatible definitive HSPCS from IPSCS using Piezo1 agonists Impulse statement: Myeloid targets in CAR T-Cell therapy |
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| 11:45 – 13:00 |
Advances in Molecular Diagnostics for Bone Marrow Failure |
| Keynote Lecture: Establishing a Diagnostic Pipeline for Bone Marrow Failure and Inherited Predisposition Syndromes in Australia Piers Blombery (Melbourne, Australia) Mini Panel: The Future in Germline Diagnostics
End of Scientific Program |
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| 13:00 |
Adjourn |
Dear young colleagues, newcomers and researchers interested in EWOG topics,
We are looking forward to meeting you at the Symposium On the Verge to Malignancy: Cytopenia and Beyond of the European Working Group of Myelodysplastic Syndromes (EWOG-MDS) and Severe Aplastic Anemia (EWOG-SAA) in Childhood. The group of Young EWOG will prepare an educational session that will be the part of the sientific program.
Marc Bierings for the organizing team of the educational session
The Symposium would not have been possible without the financial support from:
We kindly acknowledge support from the following sponsors:
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