ABSTRACT Posttransplant cyclophosphamide (PTCy) to prevent graft‐versus‐host disease (GVHD) improves outcomes in recipients of HLA mismatched unrelated donor (MMUD) allogeneic hematopoietic cell transplantation (allo HCT). Outcomes of MMUD HCT using PTCy in patients requiring reduced intensity or non‐myeloablative conditioning (RIC/NMA) are not well ...
Brian C. Shaffer +38 more
wiley +1 more source
Mitoxantrone in combination with clofarabine (MITCL) in children, adolescents and young adults with relapsed/refractory acute leukaemia: final results of a phase I/II trial. [PDF]
Hochberg J +10 more
europepmc +1 more source
ABSTRACT Chromosomal abnormalities have a major clinical impact on hematological malignancies, particularly with regard to treatment strategies. To preserve and consolidate fundamental knowledge in this rapidly evolving field, the Francophone Group of Hematological Cytogenetics (Groupe Francophone de Cytogénétique Hématologique, GFCH) conducted a ...
Florence Nguyen‐Khac +16 more
wiley +1 more source
Prognostic significance of morphology markers of chromosomal instability in acute leukaemia and myelodysplastic syndrome. [PDF]
Khairwa A, Kotru M, Dewan P, Narang S.
europepmc +1 more source
ABSTRACT Glucocorticoid resistance syndrome (GRS) is a rare hereditary disorder caused by pathogenic variants in NR3C1, characterized by marked phenotypic heterogeneity and frequent misdiagnosis as primary aldosteronism or subclinical Cushing's syndrome.
Sufang Yun +7 more
wiley +1 more source
Invasive fungal disease and antifungal prophylaxis in children with acute leukaemia: a multicentre retrospective Australian cohort study. [PDF]
Yeoh DK +11 more
europepmc +1 more source
ABSTRACT Heterozygous germline variants in CBL disrupt its function as a negative regulator of the Ras/MAPK pathway, classically predisposing to Juvenile myelomonocytic leukemia (JMML) and moyamoya. We describe two affected siblings carrying a paternally inherited CBL variant (c.1210 T> C, p.
Michal Bar‐Hakim +12 more
wiley +1 more source
Graft-versus-host disease is not associated with reduced incidence of relapse following haploidentical stem cell transplantation with post-transplant cyclophosphamide for acute lymphoblastic leukaemia: A study on behalf of the Acute Leukaemia Working Party of European Society for Blood and Marrow Transplantation. [PDF]
Shimoni A +16 more
europepmc +1 more source
Concurrent Germline RB1 & Mosaic TP53 in a Child With Multiple Childhood Cancers
ABSTRACT We report a patient with a pathogenic germline variant (PGV) in RB1 and somatic mosaicism for a pathogenic TP53 variant who developed three distinct types of childhood cancer: retinoblastoma, osteosarcoma, and myelodysplastic syndrome (MDS) before the age of 6 years.
Ole Haubjerg Nielsen +8 more
wiley +1 more source
A synthetic biology platform was established for the complete refactoring of complex anthracycline biosynthetic pathways in Streptomyces coelicolor using standardized, chemically synthetic DNA parts. This modular construction enabled streamlined combinatorial biosynthesis, yielding novel analogs that decouple DNA damage from chromatin damage (histone ...
Rongbin Wang +15 more
wiley +2 more sources

