Treatment of severe aplastic anemia (SAA) in Europe between 1970 and 1986 is reviewed. 487 patients received an HLA-identical BMT: results are encouraging and currently suggest a 65% survival. However, many patients cannot be offered this procedure because of the absence of an appropriate donor. Forty-five patients were given a non-HLA identical BMT: results are dependent on the degree of misma...

Aplastic anemia, the failure of the pluripotent stem cell system, is characterized by pancytopenia and hypoplasia of bone marrow. The patient exhibits signs and symptoms of neutropenia and thrombocytopenia. Though the disease is rare in children, when it is seen, the peak age of incidence is between 3 and 5 years. The acquired form may occur as a toxic reaction to medications (e.g., antiinflamm...

Survival in severe aplastic anemia (SAA) has markedly improved in the past 4 decades because of advances in hematopoietic stem cell transplantation, immunosuppressive biologics and drugs, and supportive care. However, management of SAA patients remains challenging, both acutely in addressing the immediate consequences of pancytopenia and in the long term because of the disease's natural history...

AcquiredSAAis regardedas the resultof an immune-mediateddestruction of hematopoietic cells, at least in a proportion of patients. The emergence of late clonal disorders in10%to20%ofpatients after immunosuppressive therapy (IST) raises the questions of whether some patients with SAA actually have a premalignant disease and whether IST is just postponing the inevitable. Support for this view has ...

We investigated the efficacy of arsenic trioxide (ATO) in patients with refractory severe aplastic anemia (SAA). A total of 5 consecutive adults were enrolled. The patients received ATO at a dose of 0.15 mg/kg intravenously daily for 5 days every week for 8 weeks. If necessary, a second course was performed after an interval of one week. All patients achieved clinically significant responses to...

Clonal hematopoiesis (CH) in aplastic anemia (AA) has been closely linked to the evolution of late clonal disorders, including paroxysmal nocturnal hemoglobinuria and myelodysplastic syndromes (MDS)/acute myeloid leukemia (AML), which are common complications after successful immunosuppressive therapy (IST). With the advent of high-throughput sequencing of recent years, the molecular aspect of ...

We report a case of a 51-year old man with a severe aplastic anemia who developed an invasive trichosporonosis to Trichosporon faecale with fungemia and skin lesions during severe neutropenia. The treatment was successful before neutrophil recovery with a combination of voriconazole and liposomal amphotericin B.