Granulocyte macrophage colony-stimulating factor treatment of a patient in myasthenic crisis: effects on regulatory T cells.
BACKGROUND
In this study we describe a patient with a prolonged myasthenic crisis refractory to conventional immunomodulatory therapy who was treated with GM-CSF (granulocyte macrophage colony-stimulating factor, sargramostim).


METHODS
T-regulatory cell (Treg) suppressive function and Foxp3 expression were evaluated before and after treatment with GM-CSF.


RESULTS
Treatment with GM-CSF was associated with clinical improvement, expansion in the circulating numbers of Foxp3(+) cells, increase in Foxp3 expression levels in Tregs, early improvement in Treg suppressive capacity for AChR-α-induced T-cell proliferation, and subsequent enhancement in Treg suppression of polyclonal T-cell proliferation.


CONCLUSIONS
Although definitive conclusions cannot be drawn from a single case, the correlation with similar findings in GM-CSF-treated animals with experimental autoimmune myasthenia gravis suggests further exploration of the effects of GM-CSF in myasthenia gravis should be studied in a clinical trial setting.
Department of Neurology and Rehabilitation, University of Illinois Medical Center, 912 South Wood Street, 855N, M/C 796, Chicago, Illinois 60612, USA. rowin@uic.edu
Julie
J
Rowin
Muthusamy
M
Thiruppathi
Ebinehita
E
Arhebamen
Jianrong
J
Sheng
Bellur S
BS
Prabhakar
Matthew N
MN
Meriggioli
D000368:Aged; D049109:Cell Proliferation / Q000187:drug effects; D051858:Forkhead Transcription Factors / Q000378:metabolism; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000494:pharmacology / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D009157:Myasthenia Gravis / Q000188:drug therapy* / Q000276:immunology / Q000378:metabolism; D050378:T-Lymphocytes, Regulatory / Q000187:drug effects* / Q000276:immunology / Q000378:metabolism; D016896:Treatment Outcome
C418974:FOXP3 protein, human; D051858:Forkhead Transcription Factors; D016178:Granulocyte-Macrophage Colony-Stimulating Factor