Effect of stem cell factor on in vitro erythropoiesis in patients with bone marrow failure syndromes

Abstract

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.

In vitro progenitor analysis in a Diamond Blackfan anaemia patient who responded once but not twice to interleukin‐3 therapy, using short‐term and long‐term cultures and c‐kit analysis

“…However, even addition of SCF appears to normalize erythroid numbers in only a proportion of our patients. Other reports have also shown SCF is more effective in increasing erythroid colony numbers than IL-3 (Casadevall et al, 1994;Alter et al, 1992). Although there has been much emphasis recently that in vitro and in vivo response to IL-3 cannot be correlated, in vitro response to IL-3 is relatively low and quite variable compared to the response we have seen in our patients to SCF .…”

Clonal chromosomal abnormalities in Fanconi's anaemia: what do they really mean?

“…Three of the 11 samples adequate for cytogenetics were abnormal (Table 11). The first patient was in clinical group 3 (Alter et al, 1992). with a cellular marrow with minimal dysmyelopoiesis.…”

“…His blood counts were beginning to decline, and his marrow was myelodysplastic. The second patient is on no treatment and is in group 5 (Alter et al, 1992), with a hypocellular marrow. In 1991, two of 1 0 cells had an abnormal chromosome 3; 8 months later all of 2 3 cells were normal.…”

“…The third case presented in 1992 with aplastic anaemia and a hypocellular marrow (group 4, Alter et al 1992). Eight of nine metaphases had a derivative chromosome 2.…”

In Vitro Proliferation and Differentiation of Erythroid Progenitors of Cord Blood

“…Many investigators have reported the effects of SCF on erythropoiesis, some using bone marrow cells [26,27] and some using adult peripheral blood MNC [4,28]. In this study, we evaluated responses of human cord blood cells to SCF and EPO.…”