Immunomodulation therapy is a mainstay modality of cure or improvement for the
patients with aplastic anemia who can not have opportunity for bone marrow
transplantation. The response rate of ALG or ATG therapy varies between 40-70%, but
increasing response has reported by introducing of cyclosporine A. We performed
immunodulation therapy (ALG or ATG+CsA) including recombinant human Granulocyte
Macrophage Colony Stimulating Factor(rhGM-CSF) to 22 patients with aplastic anemia
from January 1992;[14 of Group I (duration of follow up>3 months/rhGM-CSF, Sandoz,
Swiss), 8 of Group Ⅱ (F.U<3 months/rhGM-CSF, Lucky co.,Korea)]. Recombinant
human GM-CSF (5ug/kg) was administered as a subcutaneous injection daily after or
during ALG or ATG therapy. 6 patients(42.8%) had complete response(CR) and 7
patients(50%) had partial response(PR)among the Group 1. There was no difference in
rate of CR with previously reported ALG or ATG+CSA group. Because of temporary
improvements were seen in granulocyte counts, the number of partial response had
higher increment. No effect on raising of platelets were observed. All responses were
initiated within 6 months from the begining of the ALG or ATG therapy in Group Ⅰ.
There was only minimal toxicity consisting of transient general weakness, arthralgia,
low grade fever except 2 cases had generalized urticarial rash and dyspnea. Our data at
least gave us a conclusion that rhGM-CSF is well tolerated and is likely to result in
elevations of partial response rate so that decreasing of serious infection on
immunomodulation therapy for patients with aplastic anemia is documentated, but role of
rhGM-CSF in immunodulatory therapy remains to be determined because of short
termed observation. The recent advances in various growth actors such as G-CSF, IL-3,
or thrombopoletin(IL-6 etc) are highly evaluable to be investigated.

Keywords: Aplastic anemia, ALG, ATG, Cyclospome A, rhGm-CSF