Long-term granulocyte-macrophage colony-stimulating factor and immunosuppression in the treatment of acquired severe aplastic anemia.
OBJECTIVE
Seven children with newly diagnosed acquired severe aplastic anemia (SAA) were treated with a combination of long-term granulocyte-macrophage colony-stimulating factor (GM-CSF) and immunosuppression to assess the potential for GM-CSF to induce sustained neutrophil recovery, reduce the incidence of infection, and enhance the therapeutic efficacy of immunosuppression.


METHODS
Patients received a 14-day course of i.v. antithymocyte globulin 15 mg/kg/day with oral prednisone 1 mg/kg/day, long-term daily oral cyclosporine A 10 mg/kg/day, and long-term daily s.c. GM-CSF 5 micrograms/kg/day.


RESULTS
All seven children recovered an absolute neutrophil count of > 1.0 x 10(9)/L within 3.5 months of diagnosis (mean 60 days). Of the six children followed throughout their entire illness (follow-up 10-27 months), five are platelet and red cell transfusion independent (three off-therapy, two on tapering therapy) and one continues on therapy with a diminishing transfusion requirement. Compared with seven children treated previously with immunosuppression alone, children who received GM-CSF spent fewer days in the hospital and were less likely to develop infection.


CONCLUSIONS
The addition of GM-CSF to immunosuppressive therapy appears to be beneficial in the treatment of children with acquired SAA with GM-CSF stimulating granulopoiesis. The children are better protected from infectious complications while immunosuppressive agents achieve full therapeutic potential.
Hord
J D
JD
Department of Pediatrics, Vanderbilt University Medical Center, Nashville, Tennessee 37232-2588, USA.
Gay
J C
JC
Whitlock
J A
JA
Janco
R L
RL
Edwards
J R
JR
Greer
J P
JP
Lukens
J N
JN
D000293:Adolescent; D000741:Anemia, Aplastic / Q000188:drug therapy*; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D005260:Female; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D011241:Prednisone / Q000627:therapeutic use
D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016178:Granulocyte-Macrophage Colony-Stimulating Factor; D016572:Cyclosporine; D011241:Prednisone