Effect of stem cell factor on in vitro erythropoiesis in patients with bone
marrow failure syndromes
BP Alter, ME Knobloch, L He, AP Gillio, RJ O'Reilly, LK Reilly and RS Weinberg
Polly Annenberg Levee Hematology Center, Department of Medicine, Mount
Sinai School of Medicine, New York, NY.
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.
Volume 80,
Issue 12,
pp. 3000-3008,
12/15/1992
Copyright © 1992 by The American Society of Hematology
