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

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Thursday, 18.09.2025
07:30 – 08:30 Breakfast
08:30 – 08:45 Opening of the Symposium
For the Organizing Committee: Charlotte Niemeyer, Brigitte Strahm, Edoardo Muratore
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)

08:45 – 09:30 SAA beyond the state of the art
Invited experts: Brigitte Strahm (Freiburg, Germany), Carlo Dufour (Genova, Italy), Mirjam Belderbos (Utrecht, The Netherlands)
09:30 – 10:15 Well begun is half done: How to find the right diagnostic path in MDS
Invited experts: Marcin Wlodarski (Memphis, USA), Michael Dworzak (Vienna, Austria), Martina Rudelius (Munich, Germany)
10:15 – 10:30 Coffee Break
10:30 – 11:15 The journey begins with a single step: clonal evolution in germline predisposition and clinical management
Invited experts: Marlene Pasquet (Toulouse, France), Marc Bierings (Utrecht, The Netherlands), Henrik Hasle (Aarhus, Denmark)
11:15 – 12:00 JMML Preclinical Models: One size does not fit all – choosing the right model for the right question
Invited experts: Miriam Erlacher (Ulm, Gemany), Silvia Bresolin (Padova, Italy)
12:00 – 13:00 Lunch
13:00 – 14:30

Aplastic anemia I

Moderator: Tania Masmas (Copenhagen, Denmark), Michael Amrein (Zurich, Switzerland)

13:00 – 13:30 Keynote Lecture: The role of somatic T-cell mutations and immune dysregulation in SAA
Satu Mustjoki (Helsinki, Finland)
13:30 – 13:45 Alternative donor peripheral stem cell transplantation with TCRαβ/CD19 depletion for pediatric patients with bone marrow failure
Tim Olson*, C. W. Elgarten, J. H. Oved, L. Wray, K. Venella, P. Nicholas, S. Kadauke, Y. Wang, S. Grupp
*(Philadelphia, USA)
13:45 – 14:00 Outcome of second hematopoietic stem cell transplantation in pediatric patients with severe aplastic anemia
Nienke Wieringa*, M. Amrein, M. Dworzak, V. Labarque, M. Schmugge, J. Stary, F. Locatelli, O. Smith, M. Bierings, J. Buechner, K. Pawelec, B. Strahm
*(Utrecht, The Netherlands)
14:00 – 14:15 Late effects of immunosuppressive therapy in acquired bone marrow failure: Long-term follow-up of Czech patients with aplastic anemia and refractory cytopenia
Martina Sukova*, B. Cabalkova, E. Mejstrikova, M. Reiterova, P. Riha, P. Keslova, Z. Novak, O. Zapletal, L. Mastikova, I. Janotova, J. Stary
*(Prague, Czechia)
14:15- 14:30 Improved engraftment following a treosulfan-fludarabine conditioning regimen compared to thiotepa-fludarabine in patients with refractory cytopenia of childhood
Brigitte Strahm*, A. Yoshimi, I. Bodova, J. Buechner, A. Catala, V. De Haas, B. De Moerloose, M. Dworzak, H. Hasle, K. Kallay, F. Locatelli, R. Masetti, P. Noellke, M. Schmugge, O. Smith, J. Stary, D. Turkiewicz, M. Ussovicz, L. Vinci, M. Erlacher, C. M. Niemeyer
*(Freiburg, Germany)
14:30 – 15:30

Aplastic anemia II

Moderator: Brigitte Strahm (Freiburg, Germany); Julian Sevilla (Madrid, Spain)

Mini Panel:Long-term consequences of therapy in aplastic anemia
Moderator: Brigitte Strahm (Freiburg, Germany)

  • Setting the stage: Therapeutic concepts in pediatric SAA
    Carlo Dufour (Genova, Italy)
  • Relevance of clonal hematopoiesis following IST and HSCT
    Mirjam Belderbos (Utrecht, The Netherlands)
  • Risk of malignancy following IST and HSCT
    Akiko Shimamura (Boston, USA)
  • Risk of infertility
    Ayami Yoshimi (Freiburg, Germany)
  • How to design long term FUP study
    Jean Hugues Dalle (Paris, France)
15:30 – 16:00 Coffee break
16:00 – 17:30

The path to clonal myeloid malignancy I

Moderator: Kirsi Jahnukainen (Helsinki, Finland), Barbora Cabalkova (Prague, Czech Republic)

16:00 – 16:15 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)
16:15 – 16:30 MMolecular surveillance in bone marrow failure: Predictive value of clonal hematopoiesis
Nathan Gray*, E. Attardi, M. Boals, S. Lewis, P. Shaker, K. Ray, J. Uhrich, M. Hale, A. Kennedy, M. Wlodarski
*(Memphis, USA)
16:30 – 16:45 Refractory cytopenia of childhood with normal karyotype: Long-term results of an observational approach
Beatrice Drexler*, S. Schwarz-Furlan, I. Baumann, M. Rudelius, P. Noellke, D. Lebrecht, S. Ramamoorthy, N. Rotari, A. Karow, S. Hirabayashi, F. Beier, Y. L. Behrens, G. Goehring, R. Kalb, M. Wlodarski, B. Strahm, M. Erlacher, C. M. Niemeyer, A. Yoshimi
*(Basel, Swizerland)
16:45 – 17:00 SSingle-cell and spatial transcriptomics of pediatric myelodysplastic syndrome with refractory cytopenia of childhood reveal a disrupted bone marrow niche driving impaired hematopoiesis
Patrycja Fryzik *, L-T. Chen, E.S. Hanemaaijer, I. J. Kal, T. Candelli, B. M. TePas, N. Epskamp, V. De Haas, M. Scheijde-Vermeulen, W. J. De Jonge, T. Margaritis, M. Belderbos
*(Utrecht, The Netherlands)
17:00 – 17:15 Concordance of somatic genetic testing in blood vs. bone marrow in Shwachman-Diamond syndrome
Felicia Andresen*, K. C. Myers, A. Guttierez, D. Schwarz, B. Goldberg, E. Weller, C. Reilly, K. Brundige, S. Loveless, L. Cheng, C. Lindsley, A. Shimamura
*(Boston, USA)
17:15 – 17:30 In-vivo mouse model of acute myeloid leukemia development in congenital neutropenia
Jeremy Haaf*, M. Ritter, P. A. Tutusaus, S. Kandabarau, M. Klimiankou, J. Skokowa
*(Tübingen, Germany)
17:30-19:00

The path to clonal myeloid malignancy II

Moderator: Miriam Erlacher (Ulm, Germany), Kirsten Thus (Utrecht, The Netherlands)

Keynote Lecture: How risky is clonal hematopoiesis?
Luca Malcovati (Pavia, Italy)
Mini Panel: Clonal hematopoiesis in aging and germline predisposition: Is it the same?
Moderator: Miriam Erlacher (Ulm, Germany)

  • Impulse statement
    Luca Malcovati (Pavia, Italy)
  • Battle of clones: Somatic genetic rescue in congenital bone marrow failure disorders
    Akiko Shimamura (Boston, USA)
  • Genetic outfit and modes of stem cell aging
    Julia Skokowa (Tübingen, Germany)
  • Clonal evolution in aplastic anemia – what is different?
    Marcin Wlodarski (Memphis, USA)

End of scientific program
19:00 – 21:00 Welcome reception
Friday, 19.09.2025
07:30 – 08:30 Breakfast
08:30 – 10:00

Inherited predispositions to myeloid neoplasms and their clinical implications

Moderator: Shlomit Barzalai (Tikva, Israel), Marena Niewisch (Ulm, Germany)

08:30 – 09:00 Keynote Lecture: GATA2 deficiency: from pathophysiology to clinical care
Marlene Pasquet (Toulouse, France)
09:00 – 09:15 St. Jude.org/GATA2: online catalogue of 900 individuals with germline GATA2 mutations
Lili Kotmayer*, K. Gangwani, B. Csaba, Z. Xin, M. Wlodarski
*(Memphis, USA)
09:15 – 09:30 Mitotic defects impaired CD34+ proliferation in GATA2 deficiencyMaria Magallon-Mosella*, D. Romero-Moya, C. Calvo, J. Pera, E. Torralba-Sales, M. Erlacher, O. Molina, A. Giorgetti
*(Hospitalet de Llobregat, Spain)
09:30 – 09:45 Superior survival following hematopoietic stem cell transplantation for high-risk marrow features in patients with Shwachman Diamond syndrome
Kasiani Myers*, F. Andresen, B. Goldberg, E. Weller, C. Reilly, T. Nakano, A. Bertuch, A. Geddis, M. Joos, K. Coyne, K. Brundige, S. Loveless, L. Cheng, A. Shimamura
*(Cincinnati, USA)
09:45 – 10:00 Impulse Statement: Tailoring pre- and post HSCT therapy in advanced MDS
Ricardo Massetti (Bologna, Italy)
10:00 – 11:30 Poster Viewing – Coffee served

10:00 – 10:45

Moderator: Joanne Yacobovich (Tikva, Israel), Pawelec Katarzyna (Warsaw, Poland)

P1 C. Dufour OUTCOMES OF IMMUNOSUPPRESSIVE AND TRANSPLANT THERAPIES IN PEDIATRIC PATIENTS WITH MODERATE APLASTIC ANEMIA: A SYSTEMATIC LITERATURE REVIEW
P2 C. Kelaidi FAVORABLE LONG-TERM OUTCOMES WITH IMMUNOSUPPRESSIVE THERAPY IN CHILDREN WITH SEVERE APLASTIC ANEMIA
P3 E. Picotti CLINICAL AND LABORATORY CHARACTERISTICS OF IMMUNE SUPPRESSION THERAPY RESPONDERS AND NON-RESPONDERS IN SWISS PEDIATRIC PATIENTS WITH APLASTIC ANEMIA OR HYPOCELLULAR REFRACTORY CYTOPENIA OF CHILDHOOD
P4 D. Montes BONE MARROW FAILURE SYNDROMES AND APLASTIC ANAEMIA: GENOMIC LANDSCAPE AND OUTCOMES – ANALYSIS FROM A TERTIARY CARE CENTER OF WESTERN INDIA
P5 D. Dulla HEPATITIS-ASSOCIATED VS IDIOPATHIC PAEDIATRIC APLASTIC ANAEMIA: CLONAL ARCHITECTURE AND DISEASE TRAJECTORY
P6 K. Ray BUILDING RESOURCES FOR DISCOVERY AND TREATMENT: THE INSIGHT-HD BIOBANKING INITIATIVE FOR PEDIATRIC BONE MARROW FAILURE AND MYELODYSPLASTIC SYNDROMES
P7 M. Komonova FULMINANT HEPATITIS ASSOCIATED APLASTIC ANEMIA PRESENTED CONCOMITANTLY WITH LIVER INJURY AND AFFECTED BY LIVER TRANSPLANTATION: A CASE SERIES
P8 A. Sharathkumar ROMILOSTIM FOR TRTEATMENT OF CHILDREN AND YOUNG ADULTS WITH SEVERE APLASTIC ANEMIA AND MYELODYPLASTIC SYNDROME: A SINGLE CENTRE PILOT STUDY
P9 A. Delgado Beltran CLINICAL CHARACTERISTICS AND OUTCOMES IN CHILDREN WITH SEVERE APLASTIC ANEMIA WHO RECEIVED IMMUNOMODULATORY THERAPY AND/OR TRANSPLANTATION AT THE MISERICORDIA PEDIATRIC HOSPITAL FOUNDATION 2015 TO 2024

Moderator: Marena Niewisch (Ulm, Germany), Mikael Sundin (Stockholm, Sweden)

P10 D. Karapinar A FAMILY WITH MYSM1 MUTATION
P11 J. Fernandez-Orth EPIGENETIC MECHANISMS AND THERAPEUTIC VULNERABILITIES IN A GATA2 HAPLOINSUFFICIENT MOUSE MODEL
P12 M. Alcaide Miranda EXPLORING THE IMPACT OF GATA2 HAPLOINSUFFICIENCY ON DNA DAMAGE REPAIR IN MURINE HEMATOPOIETIC CELLS
P13 N. Lelli WHEN STANDARD TESTS FALL SHORT: SOLVING A COMPLEX CASE OF INHERITED BONE MARROW FAILURE SYNDROME THROUGH WHOLE-EXOME SEQUENCING
P14 A. Frisanco Oliveira GATA2 GENE HAPLOINSUFFICIENCY: A CLINICAL AND LABORATORY CHARACTERIZATION OF BRAZILIAN PATIENTS REGISTERED IN GCB-SMD-PED.
P15 P. Leal-Anaya GENETIC LANDSCAPE OF INHERITED BONE MARROW FAILURE SYNDROMES IN PEDIATRIC MEXICAN PATIENTS: A NATIONAL REGISTRY OVERVIEW
P16 M. Vasileva INHERITED BONE MARROW FAILURE WITH A PREDISPOSITION TO MYELODYSPLASTIC SYNDROMES/ACUTE MYELOID LEUKEMIA IN CHILDREN
P17 P. Shaker THE ROLE OF GENETIC COUNSELORS IN BONE MARROW FAILURE: A CASE-BASED APPROACH TO SRP72 AND DHX34 VARIANTS OF UNCERTAIN SIGNIFICANCE
P18 V. Pastor Loyola INTEGRATION OF A BONE MARROW FAILURE GERMLINE PANEL INTO THE COMPREHENSIVE CLINICAL GENOMICS PIPELINE AT ST. JUDE CHILDREN’S RESEARCH HOSPITAL: INSIGHTS FROM 22 MONTHS’ EXPERIENCE
P19 B. Urbański CANCER AND CANCER PREDISPOSITION SYNDROMES IN A POLISH INHERITED THROMBOCYTOPENIA COHORT
P20 J.B. Lang BONE MARROW FAILURE IN LIGASE IV DEFICIENCY CURED BY ALLOGENEIC HSCT
P21 E. Sebastián WHEN A “ZERO” CAN BE A DIAGNOSIS IN PEDIATRICS
P22 M. Muñoz CLONAL EVOLUTION AND SOMATIC GENETIC RESCUE IN SAMD9 SYNDROME: PRESENTATION OF FOUR CASES FROM THE SPANISH REGISTRY OF PEDIATRIC MYELODYSPLASTIC SYNDROMES
P23 N.S. Malyasova CO-EXITANCE OF BIALLELIC GERMLINE AND ACQUIRED SOMATIC RAD50 VARIANTS IN PEDIATRIC PATIENT WITH COMBINED PRIMARY IMMUNODEFICIENCY WITH SIGNS OF BONE MARROW FAILURE
P24 J. Nyiro HIGH PREVALENCE OF GERMLINE PREDISPOSITION IN PAEDIATRIC MYELODYSPLASTIC SYNDROME: A SINGLE-CENTRE RETROSPECTIVE ANALYSIS

Moderator: Kristian Juul-Dam (aarhus, Denmark), Kavicic Marko (Ljubljana, Slovenia)

P25 S. Hirabayashi NATIONWIDE RETROSPECTIVE STUDY OF GATA2 DEFICIENCY IN JAPAN: CLINICAL SPECTRUM AND TRANSPLANT OUTCOMES
P26 D. Bresters IMPROVED DISEASE FREE SURVIVAL IN JMML. THE DUTCH EXPERIENCE.
P27 A. Frisanco Oliveira HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH POST-TRANSPLANT CYCLOPHOSPHAMIDE FOR CHILDREN WITH JUVENILE MYELOMONOCYTIC LEUKEMIA
P28 R. Balceiro AZACITIDINE FOR PEDIATRIC PATIENTS WITH ADVANCED MYELODYSPLASTIC SYNDROME: EXPERIENCE FROM BRAZILIAN COOPERATIVE GROUP OF PEDIATRIC MYELODYSPLASTIC SYNDROME (GCB-SMD-PED).
P29 M. Maurer-Granofszky A PILOT STUDY FOR MUTATION- BASED MRD ASSESSMENT TO GUIDE POST-TRANSPLANT THERAPEUTIC INTERVENTION IN JUVENILE MYELOMONOCYTIC LEUKEMIA.
P30 P. Riha FOUR CASES OF DYSKERATOSIS CONGENITA TINF2 PATIENTS DIAGNOSED WITH BMF AND TRANSPLANTED IN CZECHIA BETWEEN 2002 AND 2024.
P31 L. Xiaolan A SINGLE-CENTER RETROSPECTIVE ANALYSIS OF TRANSPLANTATION OUTCOMES IN PATIENTS WITH JUVENILE MYELOMONOCYTIC LEUKEMIA
P32 L. Xiaolan IMPROVED OUTCOMES FOR PEDIATRIC PATIENTS WITH DE NOVO CHRONIC MYELOID LEUKEMIA IN BLAST PHASE BY EARLY STAGE ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION
P33 X. Qin RISK-ADAPTED THERAPY FOR JUVENILE MYELOMONOCYTIC LEUKEMIA: A MULTICENTER, PROSPECTIVE STUDY IN CHINA
P34 M. Kastamoulas HEMATOPOETIC STEM CELL TRANSPLANTATION (HSCT) IN PEDIATRIC PATIENTS WITH SEVERE APLASTIC ANEMIA (SAA): THE GREEK EXPERIENCE
10:45 – 11:30

Moderator: Krisztián Kállay (Budapest, Hungary), Shinsuke Hirabayashi (Hokkaido, Japan)

P35 T. Aksu TELOMERE BIOLOGY DISORDERS: PHENOTYPE AND GENETIC FINDINGS
P36 A. Bataev THE EFFECT OF THROMBOPOIETIN MIMETIC ELTROMBOPAG (EPAG) ON EX VIVO HAEMATOPOIETIC PROGENITORS CELLS PROLIFERATION AND DIFFERENTIATION IN DIAMOND BLACKFAN ANEMIA (DBA) PEDIATRIC PATIENTS
P37 A. Catala FANCONI ANEMIA AND T-LYMPHOBLASTIC LYMPHOMA. REPORT OF TWO CASES
P38 M. Nasri CRISPR/CAS9-BASED GENE EDITING FOR ELANE-ASSOCIATED CONGENITAL NEUTROPENIA: A PATH TO CURE AND LEUKEMIA PREVENTION
P39 N. Karadas MALIGNANCY IN FANCONI APLASTIC ANEMIA; SINGLE CENTER EXPERIENCE
P40 M. Yousef OUTCOMES OF RABBIT VS. HORSE ANTI-THYMOCYTE GLOBULIN IN IMMUNOSUPPRESSIVE THERAPY FOR REFRACTORY CYTOPENIA OF CHILDHOOD
P41 M. Boals A CASE OF SEVERE CONGENITAL THROMBOCYTOPENIA WITH HETEROZYGOUS PATHOGENIC NBEAL2 MUTATION.
P42 F. Pegoraro CSF3R MUTATIONS IN CONGENITAL NEUTROPENIA, A LONG STORY: DATA FROM THE ITALIAN REGISTRY
P43 H. Reed GENETIC RE-EVALUATION OF SHWACHMAN-DIAMOND SYNDROME-LIKE AND NEUTROPENIA CONDITIONS
P44 A.Kennedy PARADIGM: ST. JUDE INITIATIVE TO ADVANCE DEVELOPMENT OF INDIVIDUALIZED BASE AND PRIME EDITING THERAPIES FOR BONE MARROW FAILURE SYNDROMES
P45 A.Droste FANCE MISSENSE VARIANT WITH MILD PHENOTYPE BUT RETAINS HIGH MMC SENSITIVITY

Moderator: Sarah Basali (Ulm, Germany), Pierre Goncalves (Lisboa, Portugal)

P46 N. Karadas A RARE BONE MARROW FINDING IN CHILDHOOD: MYELOFIBROSIS
P47 L. Kotmayer REPORT OF A FIRST CASE OF PEDIATRIC MDS WITH VEXAS SYNDROME
P48 S.M. Makkeyah CLINICAL SPECTRUM OF AN EGYPTIAN COHORT OF CHILDREN WITH MYELODYSPLASTIC SYNDROME
P49 M. Pavlovic HUMAN HERPES VIRUS 6 INFECTION WITH BACTERIAL SEPSIS MIMICKING MYELODISPLASTIC SYNDROME IN AN INFANT
P50 T. Aksu MYELODYSPLASTIC SYNDROME IN CHILDHOOD: THE HACETTEPE COHORT

Moderator: Marek Ussowicz (Wroclaw, Poland), Ingrid Furlan (Ulm, Germany)

P51 A.K. Gupta THE GENOMIC LANDSCAPE OF JUVENILE MYELOMONOCYTIC LEUKEMIA ON WHOLE EXOME SEQUENCING
P52 J. Wang MACROPHAGE POLARIZATION AND CD47 BLOCKADE IN PTPN11-MUTATED JUVENILE MYELOMONOCYTIC LEUKEMIA
P53 A.Frisanco Oliveira FLOW CYTOMETRY IMMUNOPHENOTYPING AND JUVENILE MYELOMONOCYTIC LEUKEMIA: JUST FOR BLAST COUNT?
P54 D. Yamashita GENETIC MUTATIONAL PROFILE INFLUENCES THE MORPHOLOGICAL CHARACTERISTICS OF ABNORMAL MONOCYTES IN PATIENTS WITH JUVENILE MYELOMONOCYTIC LEUKEMIA
P55 Z. Wehbe MODELING JUVENILE MYELOMONOCYTIC LEUKEMIA USING PATIENT-DERIVED INDUCED PLURIPOTENT STEM CELLS TO UNRAVEL EPIGENETIC AND METABOLIC INTERPLAY
P56 S. Jin CHARACTERIZATION OF MURINE STEM AND PROGENITOR CELLS HARBORING ONCOGENIC PTPN11 OR KRAS MUTATIONS
P57 R. Majhi SH2B3 GENE MUTATION IN JUVENILE MYELOMONOCYTIC LEUKEMIA (JMML)- A FREQUENT CO-EXISTING MUTATION WITH RAS MUTATIONS
P58 J. Rajak SILENT CONVERSATIONS: JMML’S INTERPLAY WITH T CELL
P59 S. Frahia Bento da Silva MOLECULAR PROFILING OF JUVENILE MYELOMONOCYTIC LEUKEMIA: CHARACTERIZATION OF PATIENTS WITH MULTIPLE ONCOGENIC VARIANTS USING A TARGETED NGS PANEL
P60 E. Muratore COAGULATIVE ABNORMALITIES IN PATIENTS WITH CBL SYNDROME
P61 J. Werner CELLULAR IMMUNOTHERAPY TARGETING CLL-1 FOR JUVENILE MYELOMONOCYTIC LEUKEMIA
11:30 – 13:00

Inherited bone marrow failure and telomeropathies

Moderator: Sophia Polychronopoulou (Athens, Greece), Juncal Fernandez-Orth (Ulm, Germany)

11:30 – 12:00 Keynote Lecture:germline disorders linked to telomere maintenance and dysfunction
Sharon Savage (Bethesda, USA)
12:00- 12:15 Functional analysis of non-canonical FANCJ variants in Fanconi anemia
Reinhard Kalb*, A. Droste, A. Repczynska, P. Bydgoszcz, B. Fiebig, J. Vodopiutz, A-R. Janecke, J. Knaup, R. Hark, R. Kulka, A. Sobeck *(Würzburg, Germany)
12:15- 12:30 Clonal hematopoiesis in congenital neutropenia: Insights from somatic mutation analysis
Maksim Klimiankou*, S. Kandabarau, C. Zeidler, S. Kadah, D. Pogozhykh, Y.L. Behrens, D. Dale, V. Makaryan, M. Kelley, A. Bolyard, M. Mezger, C. Gruenes, P. Lang, C. Lengerke, K. Welte, J. Skokowa
*(Tübingen, Germany)
12:30 – 12:45 Transcriptional contingency in the early hematopoiesis of inherited bone marrow failure syndromes
Alfredo de Jesús Rodríguez Gómez*, A. De La Cruz, P. Siliceo, P. Leal Anaya
*(Mexico City, Mexico)
12:45- 13:00 In vitro model of stage-specific evolution of clonal hematopoiesis in congenital neutropenia to CN/AML
Natalia Alejandra Borbaran Bravo*, A-S Hellmuth, S. Kandabarau, M. Klimiankou, J. Skokowa
*(Tübingen, Germany)
13:00 – 13:30 Lunch + grab a coffee
13:30 – 14:30

(Epi-)genomic Landscape in JMML

Moderator: Anupuma Rao (London, UK), Maximilian Schönung (Heidelberg, Germany)

13:30 – 13:45 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)
13:45 – 14:00 The watch-and-wait approach in patients with juvenile myelomonocytic leukemia (JMML): Results of the French national cohort
Neven Quentin*, C. Arfeuille, A. Caye Eude, P. Durand, E. Lainey, B. Nelken, M. Nolla, A. Sterin, C. Thomas, C. Khouri, M-E. Dourthe, M. Fahd, F. Millot, B. Neven, A. Petit, J-H. Dalle, A. Baruchel, H. Cavé, M. Strullu,
*(Paris, France)
14:00 – 14:15 Long-term outcome of an observational approach in patients with NRAS-mutated juvenile myelomonocytic leukemia and absence of high risk features
Jolien De Waele*, M. Hofmans, A. Fischer, A. Catala, M. Dworzak, M. Erlacher, H. Hasle, R. Masetti, M. Schmugge, M. Ussowicz, S. Keogh, O. Tufekci, M. A. Yesilipek, A. Yoshimi, D. Lebrecht, M. Schönung, P. Nöllke, B. De Moerloose, C. M. Niemeyer
*(Ghent, Belgium)
14:15 – 14:30 Single-cell trajectories of monosomy 7 driven KRAS-mutant JMML
Lili Kotmayer*, S. Sahoo, Y. Masanori, A. Frisanco, F. Andresen, C. Flotho, M. Erlacher, C. M. Niemeyer, M. Wlodarski
*(Memphis, USA)
14:30-15:15

JMML risk factors for outcome

Moderator: Christian Flotho (Freiburg, Germany), Daniel Lipka (Heidelberg, Germany)

Mini Panel: Towards an international consensus on risk criteria
Moderator: Christian Flotho(Freiburg, Germany), Daniel Lipka (Heidelberg, Germany)

  • One size fits all? Risk criteria and genetic groups
    Hélène Cavé (Paris, France)
  • Is a DNA methylation score mandatory?
    Hideki Muramatsu (Nagoya, Japan)
  • What is the role of subclonal mutations in risk definition?
    Elliot Stieglitz (San Francisco, USA)
  • How many parameters do we need?
    Edoardo Muratore (Bologna, Italy)
14:00 – 15:00

Meeting of reference pathologists EWOG pathology: MDS/MPN and MPN– clear cut or overlapping?

Room: Strasbourg, Estrel Hotel
Martina, Rudelius (Munich, Germany)

Pathology-Workshop: General Information
15:15 – 15:30 Coffee break
15:30 – 16:30

Classification and clinical approach to overlap syndromes
and rare MPDs

Moderator: Valérie De Haas (Utrecht, The Netherlands), Chyzyński Bartosz (Warsaw, Poland)

15:30 – 15:45 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)
15:45 – 16:00 Predicting the unpredictable: chimerism as a relapse indicator in JMML post-haematopoietic stem cell transplant
Susanne Kricke Orszulik*, A. Rao, E. Louka, S. Adams
*(London, UK)
16:00 – 16:15 Myeloproliferative neoplasms (MPN) diagnosed in childhood and adolescence – prospective data on clinical and genetic characteristics from the German national MPN childhood registry
Axel Karow*, Y.L. Behrens, T. Reinkens, W. Hofmann, A. Gumann, R. Strasser, P. Wanjek, N. Di Donato, L. Seitz, M. Pontones, M-P. Hitz, Z. Wotschofsky, B. Schlegelberger, T. Ripperger
*(Erlangen, Germany)
16:15 – 16:30 Impluse statement:classification of overlap syndromes and rare MPD
Martina Rudelius (Munich, Germany)

End of scientific program
17:00 Departure for evening program
Saturday, 20.09.2025
07:30 – 08:30 Breakfast
07:30 – 08:30 Closed EWOG Meeting
08:30 – 10:15

Advanced MDS – from clones to therapy

Moderator: Jochen Buechner (Oslo, Norway), Hila Dias-Polak (Haifa, Israel)

08:30 – 09:00 Keynote Lecture: Therapy-induced clonal hematopoiesis
Sam Behjati (Cambridge, UK)
09:00 – 09:15 Genetic origins and clonal trajectories of monosomy 7 in pediatric MDS
Sushree Sahoo*, V. P. Pastor, M. van Roosmalen, D. Lebrecht, S. Ramamoorthy, C.G. Harris, A. van Leeuwen, L. Kotmayer, K. Schratz, A. Yoshimi, B. Strahm, P. Noellke, A. Gebert, R. van Boxtel, C. M. Niemeyer, M. Erlacher, M. Wlodarski
*(Memphis, USA)
09:15 – 09:30 Genetic landscape of myelodysplastic syndrome in children
Masataka Hasegawa*, K. Mimura, R. Ono, D. Keino, S. Tsujimoto, K. Isobe, T. Deguchi, H. Iwafuchi, H. Moritake, A. Manabe, S. Ogawa, K. Yoshida, D. Hasegawa,
*(Tokyo, Japan)
09:30 – 09:45 Genetic landscape of primary myelodysplastic syndromes with excess of blasts (MDS-EB) in children and adolescents
Davide Leardini*, L. Vinci, B. De Moerloose, H. Hasle, K. Heitink-Polle, M. Kavčič, K. Thus, D. Turkiewicz, C.M. Niemeyer, M. Erlacher, R. Masetti, M. Dworzak, and EWOG-MDS National Coordinators
*(Bologna, Italy)
09:45 – 10:00 Genetic landscape and outcomes after hematopoietic stem cell transplantation in children and adolescents with therapy-related myeloid neoplasms
Luca Vinci*, S. Ramamoorthy, E-V. Kornemann, V. De Haas, B. De Moerloose, M. Dworzak, M. Erlacher, G. Goehring, H. Hasle, K. Jahnukainen, K. Kallay, R. Masetti, P. Noellke, M. Schmugge, J. Stary, D. Turkiewicz, M. Ussowicz, F. Locatelli, T. N. Masmas, P. Sedlacek, M. Wlodarski, A. Yoshimi, M. Zecca, C. M. Niemeyer, B. Strahm
*(Freiburg, Germany)
10:00 – 10:15 Advanced myelodysplastic syndrome. The Dutch experience
Katja Heitink-Polle*, K. Thus, D. Bresters, V. De Haas, A. Vissers, A. Kors, M. Belderbos, L. Kester, E. Waanders, M. Bierings
*(Utrecht, The Netherlands)
10:15 – 10:35 Coffee break
10:35 – 11:45

Innovations in therapy and diagnostics for MDS

Moderator: Dominik Turkiewicz (Lund, Sweden), Wolfgang Novak (Vienna, Austria)

10:35 – 11:00 Integrated immunophenotyping and drug response profiling in pediatric MDS-EB: The Zurich-Vienna experience
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
11:00 – 11:15 BH3-mimetic treatment reveals subtype-specific BCL-2 protein dependency in pediatric MDS with excess blasts
Sheila Bohler*, A. Yoshimi-Noellke, N. Scheidegger, B. Haladik, B. Bornhauser, M.N. Dworzak, K. Boztug , M. Schmugge, C. M. Niemeyer, M. Erlacher
*(Freiburg, Germany)
11:15 – 11:30 Stratus Prime™ GMP compliant process to create high numbers of HLA compatible definitive HSPCS from IPSCS using Piezo1 agonists
Michael P Cooke*, A. Das, G. Singh, Z-J. Liu, Q. Lin, S.K. Patel, J. Li, V. G.Tirunagaru
*(Cambridge, USA)
11:30 – 11:45 Impulse statement: Myeloid targets in CAR T-cell therapy
Tobias Feuchtinger (Freiburg, Germany)
11:45 – 13:00

Advances in molecular diagnostics for bone marrow failure

Moderator: Marcin Wlodarski (Memphis, USA), Markus Schmugge (Zurich, Switzerland)

Keynote Lecture: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

  • Setting the stage: Germline testing concepts in hematologic diseases
    Piers Blombery (Melbourne, Australia)
  • Germline testing using nails and buccal swabs: Lessons from JMML
    Hideki Muramatsu (Nagoya, Japan)
  • Dual platform (WGS and WES) testing from direct skin biopsy
    Victor Pastor Loyola (Memphis, USA)
  • Hair follicles NGS: Fast and reliable method for confirmation of germline status
    Dirk Lebrecht (Freiburg, Germany)

End of Scientific Program
13:00
 Adjourn
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Young EWOG

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:

Förderverein für krebskranke Kinder e.V. Freiburg i.Br.

Deutsche Kinderkrebsstiftung

Stiftung Lichterzellen

We kindly acknowledge support from the following sponsors:

Bristol Myers Squibb

Medac GmbH

Pfizer Pharma GmbH

Neovii Biotech GmbH

Novartis Pharma GmbH

Platinum Sponsor

Gold Sponsor

Silver Sponsor

Bronze Sponsor

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