title: Heterogeneity of pathogenetic mechanisms in aplastic anemia. Efficacy of therapy based on in-vitro results.
abstract: The mechanisms responsible for the bone marrow failure in 21 aplastic anemia patients were studied by the colony-forming units in culture assay (CFU-C). Twelve patients had no detectable in-vitro defect that could be responsible for the low CFU-C numbers. Three patients had suppressor T cells that inhibited CFU-C (p less than 0.001); one of two patients responded to antithymocyte globulin therapy and the third recovered spontaneously. Three patients had serum inhibitory immunoglobulins directed against their marrow CFU-C; plasmapheresis resulted in recovery of bone marrow function. Three patients had abnormalities at the colony-stimulating factor level: Two had inhibitors of colony-stimulating factor, corrected in vitro and in vivo by indomethacin and cholinergic agonists (p less than 0.01); and the third had colony-stimulating factor generation defect, corrected in vitro and in vivo by lithium. Testing for cellular or humoral suppressor factors directed against precursor cells or for abnormalities at the colony-stimulating factor level gives helpful guidelines to therapy in aplastic anemia.
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000209:etiology*; D001853:Bone Marrow / Q000473:pathology; D002648:Child; D003114:Colony-Forming Units Assay; D003115:Colony-Stimulating Factors / Q000502:physiology; D005260:Female; D006410:Hematopoiesis; D006801:Humans; D007958:Leukocyte Count; D008297:Male; D008875:Middle Aged; D013601:T-Lymphocytes / Q000502:physiology
chemical_list: D003115:Colony-Stimulating Factors