Effect of stem cell factor on in vitro erythropoiesis in patients with bone marrow failure syndromes.
Stem cell factor (SCF) enhances normal hematopoiesis. We examined its effect in vitro on bone marrow and blood progenitors from patients with inherited bone marrow failure syndromes, including 17 patients each with Diamond-Blackfan anemia (DBA) and Fanconi's anemia (FA), 3 with dyskeratosis congenita (DC), and 1 each with amegakaryocytic thrombocytopenia (amega) and transient erythroblastopenia of childhood (TEC). Mononuclear cells were cultured with erythropoietin (Ep) alone or combined with SCF or other factors. SCF increased the growth of erythroid progenitors in cultures from 50% of normal controls, 90% of DBA, 70% of FA, 30% of DC, and the amega and TEC patients; normal numbers were reached in 25% of DBA studies. Improved in vitro erythropoiesis with SCF in all types of inherited marrow failure syndromes does not suggest a common defect involving kit or SCF, but implies that SCF may be helpful in the treatment of hematopoietic defects of varied etiologies.
D000293:Adolescent; D000328:Adult; D001803:Blood Transfusion; D001853:Bone Marrow / Q000187:drug effects / Q000473:pathology*; D001855:Bone Marrow Diseases / Q000097:blood* / Q000473:pathology* / Q000628:therapy; D002478:Cells, Cultured; D002648:Child; D002675:Child, Preschool; D003114:Colony-Forming Units Assay; D004920:Erythropoiesis / Q000187:drug effects*; D004921:Erythropoietin / Q000494:pharmacology; D005199:Fanconi Anemia / Q000097:blood* / Q000473:pathology* / Q000628:therapy; D005260:Female; D006402:Hematologic Diseases / Q000097:blood* / Q000473:pathology* / Q000628:therapy; D016298:Hematopoietic Cell Growth Factors / Q000494:pharmacology*; D006412:Hematopoietic Stem Cells / Q000166:cytology / Q000187:drug effects* / Q000473:pathology; D006801:Humans; D007223:Infant; D008297:Male; D011994:Recombinant Proteins / Q000494:pharmacology; D012016:Reference Values; D019089:Stem Cell Factor
D016298:Hematopoietic Cell Growth Factors; D011994:Recombinant Proteins; D019089:Stem Cell Factor; D004921:Erythropoietin