To characterize glycosylphosphatidylinositol-anchored protein-deficient and HLA-class I allele-lacking hematopoietic stem progenitor cells in acquired aplastic anemia (AA), researchers studied the peripheral blood of 56 AA patients in remission.
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Yoroidaka, T., Hosokawa, K., Imi, T., Mizumaki, H., Katagiri, T., Ishiyama, K., Yamazaki, H., Azuma, F., Nanya, Y., Ogawa, S., & Nakao, S. (2021). Hematopoietic stem progenitor cells lacking HLA differ from those lacking GPI-anchored proteins in the hierarchical stage and sensitivity to immune attack in patients with acquired aplastic anemia. Leukemia, 1–11. https://doi.org/10.1038/s41375-021-01202-8 Cite
IFNγ was required for increased expression of the chemokine receptor CCR5 on MΦs. CCR5 antagonism in murine SAA improved survival, correlating with increased platelets and significantly increased platelet-biased CD41hi HSCs.
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Scientists evaluated the effectiveness and safety of reduced-dose cyclophosphamide, 20 mg/kg for 13 patients in matched sibling donor hematopoietic stem cell transplantation (HSCT) cohort and 25 mg/kg for 22 patients in haplo-HSCT cohort, on days +3, +4 combined with cotransplantation of peripheral blood stem cells and human umbilical cord-derived MSCs for severe aplastic anemia (SAA).
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Zu, Y., Zhou, J., Fu, Y., Fang, B., Liu, X., Zhang, Y., Yu, F., Zuo, W., Zhou, H., Gui, R., Li, Z., Liu, Y., Zhao, H., Zhang, C., & Song, Y. (2021). Feasibility of reduced-dose posttransplant cyclophosphamide and cotransplantation of peripheral blood stem cells and umbilical cord-derived mesenchymal stem cells for SAA. Scientific Reports, 11(1), 253. https://doi.org/10.1038/s41598-020-80531-7 Cite
Scientists provide an overview of clinical results obtained through the use of haploidentical transplantation in severe aplastic anemia, mainly focusing on current advances and future challenges.
[Bone Marrow Transplantation]
Investigators found that reprogramming transcription during hematopoietic differentiation resulted in an overload of genotoxic stress, which caused aborted differentiation and depletion of Fanconi anemia mutant progenitor cells.
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The authors designed a combination of haplo‐hematopoietic stem cells transplantation with allogenic mesenchymal stem cells for treatment of severe aplastic anemia in pediatric and adolescent patients and evaluated its effects.
[Stem Cells Translational Medicine]
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Researchers analyzed blood samples of 70 aplastic anemia (AA) and 18 dyskeratosis congenita (DKC) patients to demonstrate that their epigenetic age predictions were overall increased, albeit not directly correlated with telomere length. Aberrant DNA methylation was observed in the gene PRDM8 in DKC and AA as well as in other diseases with premature aging phenotype, such as Down syndrome and Hutchinson-Gilford-Progeria syndrome.
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Researchers retrospectively analyzed the outcomes of 214 severe aplastic anemia patients who underwent allogeneic hematopoietic stem cell transplantation with r-ATG or ATG-Fresenius.
[Biology of Blood and Marrow Transplantation]
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Investigators report the first case of an adult patient with severe aplastic anemia who was successfully transplanted with a UM171‐expanded cord blood graft. After a conditioning of rabbit anti‐thymocyte globulin, fludarabine, cyclophosphamide and total body irradiation, a UM171 expanded graft of 3.29 x 106 CD34+ cells/kg was infused.
[European Journal of Haematology]
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Claveau, J.-S., Cohen, S., Ahmad, I., Delisle, J.-S., Kiss, T., Lachance, S., Sauvageau, G., Busque, L., Brito, R.-M., Bambace, N., Bernard, L., Roy, D. C., & Roy, J. (n.d.). Single UM171-expanded cord blood transplant can cure severe idiopathic aplastic anemia in absence of suitable donors. European Journal of Haematology, n/a(n/a). https://doi.org/10.1111/ejh.13504 Cite