TY - JOUR
T1 - Diamond-Blackfan anemia
T2 - Promotion of marrow erythropoiesis in vitro by recombinant interleukin-3
AU - Halperin, D. S.
AU - Estrov, Z.
AU - Freedman, M. H.
N1 - Copyright:
Copyright 2020 Elsevier B.V., All rights reserved.
PY - 1989
Y1 - 1989
N2 - To clarify the defective erythropoiesis in eight patients with Diamond-Blackfan anemia, we studied their bone marrow response in vitro to recombinant human interleukin-3 (IL-3) and recombinant granulocyte-macrophage colony-stimulating factor (GM-CSF). In an erythropoietin-containing assay system, specimens from six of the eight patients yielded low numbers of erythroid colonies compared to control values, and in five of these no erythropoietin dose-response could be elicited. Addition of IL-3, GM-CSF or both to cultures from the six patients had no effect on CFU-E-derived colonies. In contrast, IL-3 but not GM-CSF induced a marked increase in the number (183%) and size of the BFU-E-derived colonies in five of the six cases and partially corrected the impaired dose-response to erythropoietin in four. Bone marrow from the other two patients yielded numbers of CFU-E and BFU-E colonies comparable to controls and manifested similar increments in colonies with increasing concentrations of erythropoietin. When IL-3 was added to these cultures, further increments were observed in the number and size of BFU-E colonies. We conclude that IL-3 enhanced the marrow erythropoiesis in most of the patients and exerted a corrective effect on the aberrant colony formation in the presence of erythropoietin. The data raise the possiblity of IL-3 as a therapeutic agent in Diamond-Blackfan anemia.
AB - To clarify the defective erythropoiesis in eight patients with Diamond-Blackfan anemia, we studied their bone marrow response in vitro to recombinant human interleukin-3 (IL-3) and recombinant granulocyte-macrophage colony-stimulating factor (GM-CSF). In an erythropoietin-containing assay system, specimens from six of the eight patients yielded low numbers of erythroid colonies compared to control values, and in five of these no erythropoietin dose-response could be elicited. Addition of IL-3, GM-CSF or both to cultures from the six patients had no effect on CFU-E-derived colonies. In contrast, IL-3 but not GM-CSF induced a marked increase in the number (183%) and size of the BFU-E-derived colonies in five of the six cases and partially corrected the impaired dose-response to erythropoietin in four. Bone marrow from the other two patients yielded numbers of CFU-E and BFU-E colonies comparable to controls and manifested similar increments in colonies with increasing concentrations of erythropoietin. When IL-3 was added to these cultures, further increments were observed in the number and size of BFU-E colonies. We conclude that IL-3 enhanced the marrow erythropoiesis in most of the patients and exerted a corrective effect on the aberrant colony formation in the presence of erythropoietin. The data raise the possiblity of IL-3 as a therapeutic agent in Diamond-Blackfan anemia.
UR - http://www.scopus.com/inward/record.url?scp=0024590087&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=0024590087&partnerID=8YFLogxK
U2 - 10.1182/blood.v73.5.1168.1168
DO - 10.1182/blood.v73.5.1168.1168
M3 - Article
C2 - 2649168
AN - SCOPUS:0024590087
SN - 0006-4971
VL - 73
SP - 1168
EP - 1174
JO - Blood
JF - Blood
IS - 5
ER -