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| 37584291 | Spontaneous remission and loss of monosomy 7: a window of opportunity for young children with SAMD9L syndrome. | Haematologica | 2024 |
| 36657267 | Rare and potentially fatal - Cytogenetically cryptic TNIP1::PDGFRB and PCM1::FGFR1 fusion leading to myeloid/lymphoid neoplasms with eosinophilia in children. | Cancer Genet | 2023 |
| 36445482 | The International Consensus Classification (ICC) of hematologic neoplasms with germline predisposition, pediatric myelodysplastic syndrome, and juvenile myelomonocytic leukemia. | Virchows Archiv | 2023 |
| 37741518 | C-terminal variants in CDC42 drive type I interferon-dependent autoinflammation in NOCARH syndrome reversible by ruxolitinib. | Clin Immunol | 2023 |
| 37695474 | Azacitidine (Vidaza<sup>®</sup>) in Pediatric Patients with Relapsed Advanced MDS and JMML: Results of a Phase I/II Study by the ITCC Consortium and the EWOG-MDS Group (Study ITCC-015). | Paediatr Drugs | 2023 |
| 37216275 | Venetoclax-based therapies in pediatric advanced MDS and relapsed/refractory AML: a multicenter retrospective analysis. | | 2023 |
| 36775010 | European standard clinical practice - Key issues for the medical care of individuals with familial leukemia. | Eur J Med Genet | 2023 |
| 36823455 | Second allogeneic stem cell transplantation can rescue a significant proportion of patients with JMML relapsing after first allograft. | Bone Marrow Transplant | 2023 |
| 36764384 | Genetic susceptibility in children, adolescents, and young adults diagnosed with soft-tissue sarcomas. | Eur J Med Genet | 2023 |
| 36706428 | Complex Lymphatic Anomalies: Report on a Patient Registry Using the Latest Diagnostic Guidelines. | | 2023 |
| 34767620 | Gain-of-function mutations in RPA1 cause a syndrome with short telomeres and somatic genetic rescue. | Blood | 2022 |
| 35967575 | Abnormal B-Cell Maturation and Increased Transitional B Cells in CBL Syndrome. | Frontiers in Pediatrics | 2022 |
| 35767897 | International Consensus Classification of Myeloid Neoplasms and Acute Leukemias: integrating morphologic, clinical, and genomic data. | Blood | 2022 |
| 36271151 | Classification of rare pediatric myeloid neoplasia-Quo vadis? | Leukemia | 2022 |
| 36123612 | Immune dysregulation associated with co-occurring germline CBL and SH2B3 variants. | Human Genomics | 2022 |
| 36258014 | Correction to: Functional classification of RUNX1 variants in familial platelet disorder with associated myeloid malignancies. | Leukemia | 2022 |
| 36440328 | Leukoreductive response to the combination of sorafenib and chemotherapy in hyperleukocytosis of -ITD mutated pediatric AML. | Frontiers in Pediatrics | 2022 |
| 36253820 | Pearson syndrome: a multisystem mitochondrial disease with bone marrow failure. | Orphanet Journal of Rare Diseases | 2022 |
| 35717738 | Cytokine profile and brain biopsy in a case of childhood-onset central nervous system vasculitis in Noonan syndrome-like disorder due to a novel CBL variant. | Journal of Neuroimmunology | 2022 |
| 36551267 | Comprehensive Analyses of Coagulation Parameters in Patients with Vascular Anomalies. | | 2022 |
| 34866327 | Combinatorial effects of azacitidine and trametinib on NRAS-mutated melanoma. | Pediatr Blood Cancer | 2022 |
| 34854279 | Guideline for management of non-Down syndrome neonates with a myeloproliferative disease on behalf of the I-BFM AML Study Group and EWOG-MDS. | Haematologica | 2022 |
| 34660476 | Efficacy of Sirolimus in Patients Requiring Tracheostomy for Life-Threatening Lymphatic Malformation of the Head and Neck: A Report From the European Reference Network. | Frontiers in Pediatrics | 2021 |
| 34297046 | Response to upfront azacitidine in juvenile myelomonocytic leukemia in the AZA-JMML-001 trial. | Blood Adv | 2021 |
| 33914175 | Daratumumab therapy for post-HSCT immune-mediated cytopenia: experiences from two pediatric cases and review of literature. | Molecular and Cellular Pediatrics | 2021 |
| 33692461 | Functional classification of RUNX1 variants in familial platelet disorder with associated myeloid malignancies. | Leukemia | 2021 |
| 33480966 | Prevalence of SARS-CoV-2 Infection in Children and Their Parents in Southwest Germany. | JAMA Pediatr | 2021 |
| 33580918 | Severe adverse events during sirolimus "off-label" therapy for vascular anomalies. | Pediatric Blood and Cancer | 2021 |
| 33531590 | Long non-coding RNAs as novel therapeutic targets in juvenile myelomonocytic leukemia. | Scientific Reports | 2021 |
| 33894142 | iPSC modeling of stage-specific leukemogenesis reveals BAALC as a key oncogene in severe congenital neutropenia. | Cell Stem Cell | 2021 |
| 33837965 | Haematological characteristics and spontaneous haematological recovery in Pearson syndrome. | Br J Haematol | 2021 |
| 33781541 | Stem Cell Transplantation for Diamond-Blackfan Anemia. A Retrospective Study on Behalf of the Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplantation Group (EBMT). | Transplant Cell Ther | 2021 |
| 33565284 | High-resolution pediatric reference intervals for 15 biochemical analytes described using fractional polynomials. | Clin Chem Lab Med | 2021 |
| 33726626 | When to suspect GATA2 deficiency in pediatric patients: from complete blood count to diagnosis. | Pediatric Hematology and Oncology | 2021 |
| 34254124 | Functional assessment of two variants of unknown significance in TEK by endothelium-specific expression in zebrafish embryos. | Hum Mol Genet | 2021 |
| 34196171 | A novel classification of hematologic conditions in patients with Fanconi anemia. | Haematologica | 2021 |
| 34301409 | The variable biological signature of refractory cytopenia of childhood (RCC), a retrospective EWOG-MDS study. | Leuk Res | 2021 |
| 34300250 | Current Treatment of Juvenile Myelomonocytic Leukemia. | Journal of Clinical Medicine | 2021 |
| 34244664 | Hematopoietic stem cell transplantation in children and adolescents with GATA2-related myelodysplastic syndrome. | Bone Marrow Transplant | 2021 |
| 33139265 | International Consensus Definition of DNA Methylation Subgroups in Juvenile Myelomonocytic Leukemia. | Clin Cancer Res | 2021 |
| 33170215 | A distinct CD38+CD45RA+ population of CD4+, CD8+, and double-negative T cells is controlled by FAS. | J Exp Med | 2021 |
| 34469508 | Association of unbalanced translocation der(1;7) with germline GATA2 mutations. | Blood | 2021 |
| 32555368 | Synonymous GATA2 mutations result in selective loss of mutated RNA and are common in patients with GATA2 deficiency. | Leukemia | 2020 |
| 31906955 | Comprehensive assessments and related interventions to enhance the long-term outcomes of child, adolescent and young adult cancer survivors - presentation of the CARE for CAYA-Program study protocol and associated literature review. | BMC Cancer | 2020 |
| 32246016 | Oncogenic Kras causes myeloproliferation via NLRP3 inflammasome activation. | Nature Communications | 2020 |
| 31578451 | Hematopoietic stem cell transplantation for children with acute myeloid leukemia-results of the AML SCT-BFM 2007 trial. | Leukemia | 2020 |
| 32343795 | Favorable outcomes of hematopoietic stem cell transplantation in children and adolescents with Diamond-Blackfan anemia. | Blood advances | 2020 |
| 31768014 | Correction: Hematopoietic stem cell transplantation for children with acute myeloid leukemia-results of the AML SCT-BFM 2007 trial. | Leukemia | 2020 |
| 32285995 | TIM-3 deficiency presenting with two clonally unrelated episodes of mesenteric and subcutaneous panniculitis-like T-cell lymphoma and hemophagocytic lymphohistiocytosis. | Pediatric Blood and Cancer | 2020 |
| 32338640 | Loss of the Fanconi anemia-associated protein NIPA causes bone marrow failure. | Journal of Clinical Investigation | 2020 |