BACKGROUND An alternative reduced-toxicity conditioning regimen for allogeneic transplantation, based on treosulfan and fludarabine, has recently been identified. The rationale for this study was to investigate the efficacy and safety of this regimen prospectively in patients with a primary myelodysplastic syndrome. DESIGN AND METHODS A total of 45 patients with primary myelodysplastic syndro...

GATA-2 deficiency was recently described as common cause of overlapping syndromes of immunodeficiency, lymphedema, familiar myelodysplastic syndrome or acute myeloid leukemia. The aim of our study was to analyze bone marrow and peripheral blood samples of children with myelodysplastic syndrome or aplastic anemia to define prevalence of the GATA2 mutation and to assess whether mutations in GATA-...

The myelodysplastic Syndromes (MDS) are a group of clonal hematopoietic stem cell diseases characterized by cytopenia(s), dysplasia in one or more of the major myeloid cell lines, ineffective hematopoiesis, and increased risk of development of acute myeloid leukemia. The classification and the diagnostic criteria have been redefined by the recent World Health Organization Classification of Tumo...

transfusion. Primary refiactcry anemia is distinguished from secondary refractory anemia in that there is no associated infection, renal or l cpatic disease, malignancy or The etiology, clinical and laboratory findings, therapy, and prognosis of acquired ap!astic anemia have been reviewed recently by Scott and coworkers2 and by Mohier and Leavell.3 Studies of iron metabolism, generally either t...

BACKGROUND Autoimmune hemolytic anemia is rare in children. First-line therapies for this disease consist of corticosteroids and intravenously administered immunoglobulin that are effective in most patients. However, a small proportion of cases (5 to 10%) is refractory to these therapies and may represent a medical emergency, especially when hemolysis is due to warm immunoglobulin M. Recently, ...

By CiIAnLI s M. HUGULEY, Jn., JAMES A. BAIN, SHIRLEY L. RIVERS AND ROBERT B. ScoGcINs T HE MAJORITY of anemias associated with a megalohiastic hone marrow respond to vitamin B,2 parenterally. Those which do not respond usually fall into well defined categories of nutritional deficiency, intestinal malahsorption syndromes, pernicious anemia of pregnancy, megaloblastic anemia of infancy or reacti...