BACKGROUND Patients with paroxysmal nocturnal hemoglobinuria harbor clonal glycosylphosphatidylinositol-anchor deficient cells arising from a multipotent hematopoietic stem cell acquiring a PIG-A mutation. Many patients with aplastic anemia and myelodysplastic syndromes also harbor small populations of glycosylphosphatidylinositol-anchor deficient cells. Patients with aplastic anemia often evol...

Few therapeutic options are available for patients with aplastic anemia who are ineligible for transplantation or refractory to immunosuppressive therapy. Eltrombopag was recently shown to produce trilineage responses in refractory patients. However, the effects of real-life use of this drug remain unknown. This retrospective study (2012-2016) was conducted by the French Reference Center for Ap...

The bone marrow failure states, aplastic anemia and myelodysplastic syndrome, are characterized by reticulocytopenic anemia, with variable neutropenia and thrombocytopenia. The bone marrow biopsy is very hypocellular in aplastic anemia, but it is usually hypercellular in myelodysplastic syndrome. Marrow cytogenetic abnormalities are present in approximately half of myelodysplastic syndrome pati...

9. Zhang F, Zhang L, Jing L, et al. High-dose cyclophosphamide compared with antithymocyte globulin for treatment of acquired severe aplastic anemia. Exp Hematol. 2013;41(4):328-334. 10. Bhatnagar N, Wynn R, Velangi M, et al. Upfront matched and mismatched unrelated transplantation in paediatric aplastic anemia: a United Kingdom multicenter retrospective experience. In: BMT ed. EBMT annual meet...

Inherited bone marrow failure syndromes (BMFs) comprise at least one-fourth of children with aplastic anemia, and perhaps up to 10% of adults. The most common syndrome is Fanconi's anemia (FA), with more than 1,000 reported cases. FA is autosomal recessive, with birth defects in approximately 75% of patients. It is a DNA repair syndrome, diagnosed by finding chromosomal aberrations in cells tre...

Eighteen patients, twelve men and six women, with aplastic anemia underwent allogenic bone marrow transplantation (BMT) from HLA-matched siblings during the period of 1990 to 1996. The conditioning regimen was cyclophosphamide with or without busulfan, depending on the cause of aplasia. Antilymphocyte globulin (ALG) and cyclosporine were used for rejection and acute GVHD prophylaxis, respe...