Diamond-Blackfan anemia: promotion of marrow erythropoiesis in vitro by
recombinant interleukin-3
DS Halperin, Z Estrov and MH Freedman
Division of Hematology/Oncology, Hospital for Sick Children, Toronto,
Ontario, Canada.
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 possibility of IL-3 as a therapeutic
agent in Diamond- Blackfan anemia.
Volume 73,
Issue 5,
pp. 1168-1174,
04/01/1989
Copyright © 1989 by The American Society of Hematology
