aplastic anemia

[Haematologica] The study comprises children with persistent cytopenias, myelodysplastic syndrome, aplastic anemia, or suspected inherited bone marrow failure syndromes, who were referred for genetic evaluation from all pediatric hematology centers in Israel during 2016-2019.

[Journal of Clinical Investigation] TET inhibition in TET2 mutant malignant myeloid cells prevented neoplastic clonal evolution, in vitro and in vivo. This new mechanism of action may offer a restorative therapeutic index and provide a scientific rationale to treat selected patients with TET2-mutant or TET deficiency associated myeloid malignancies.

[Advanced Science] Using Pich knock-out mouse models, the authors provided evidence for a mechanistic link between PICH deficiency and defective hematopoiesis.

[Leukemia] Investigators reported that FZR1 was downregulated in severe aplastic anemia HSCs compared with healthy control and was associated with decreased quiescence of HSCs.

[Annals of Hematology] The authors compared transplantation outcomes between a fludarabine (Flu)- and melphalan-based regimen and a Flu- and cyclophosphamide-based regimen

[Blood] The authors performed single-cell transcriptome analysis of residual hematopoietic stem and progenitor cells and T cells to identify the molecular players from patients with aplastic anemia.