Granulocytic macrophage colony stimulating factor restores in vitro growth of granulocyte-macrophage bone marrow hematopoietic progenitors in dyskeratosis congenita.

In vitro 14-day cultures of bone marrow from a patient with dyskeratosis congenita showed virtually no growth of colonies. The addition of recombinant granulocyte-macrophage colony stimulating factor (rGM-CSF) promoted a significant increase in the number of GM colonies (CFU-GM). Interleukin 3 also increased GM colony formation but to a lesser extent. GM-CSF may have a therapeutic implication for pancytopenia in dyskeratosis congenita.