Effect of Stem Cell Factor on In Vitro Erythropoiesis in Patients With Bone Marrow Failure Syndromes

• Published in: Blood Vol. 80; no. 12; pp. 3000 - 3008
• Main Authors: Alter, Blanche P., Knobloch, Mary Ellen, He, Liya, Gillio, Alfred P., O’Reilly, Richard J., Reilly, Laura K., Weinberg, Rona S.
• Format: Journal Article
• Language: English
• Published: Washington, DC Elsevier Inc 15.12.1992; The Americain Society of Hematology
• Subjects: Adolescent; Adult; Anemias. Hemoglobinopathies; Biological and medical sciences; Blood Transfusion; Bone Marrow - drug effects; Bone Marrow - pathology; Bone Marrow Diseases - blood; Bone Marrow Diseases - pathology; Bone Marrow Diseases - therapy; Cells, Cultured; Child; Child, Preschool; Colony-Forming Units Assay; Diseases of red blood cells; Erythropoiesis - drug effects; Erythropoietin - pharmacology; Fanconi Anemia - blood; Fanconi Anemia - pathology; Fanconi Anemia - therapy; Female; Hematologic and hematopoietic diseases; Hematologic Diseases - blood; Hematologic Diseases - pathology; Hematologic Diseases - therapy; Hematopoietic Cell Growth Factors - pharmacology; Hematopoietic Stem Cells - cytology; Hematopoietic Stem Cells - drug effects; Hematopoietic Stem Cells - pathology; Humans; Infant; Male; Medical sciences; Recombinant Proteins - pharmacology; Reference Values; Stem Cell Factor; Erythropoiesis; Human; Fanconi anemia; Deficiency; Aplastic anemia; Bone marrow; Exploration; Hemopathy; In vitro
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood.V80.12.3000.3000

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. © 1992 by The American Society of Hematology.