title: Long-term granulocyte-macrophage colony-stimulating factor and immunosuppression in the treatment of acquired severe aplastic anemia.
abstract: 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.
authors.affiliation: Department of Pediatrics, Vanderbilt University Medical Center, Nashville, Tennessee 37232-2588, USA.
authors.forename: J D
authors.initials: JD
authors.lastname: Hord
authors.forename: J C
authors.initials: JC
authors.lastname: Gay
authors.forename: J A
authors.initials: JA
authors.lastname: Whitlock
authors.forename: R L
authors.initials: RL
authors.lastname: Janco
authors.forename: J R
authors.initials: JR
authors.lastname: Edwards
authors.forename: J P
authors.initials: JP
authors.lastname: Greer
authors.forename: J N
authors.initials: JN
authors.lastname: Lukens
mesh_terms: 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
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016178:Granulocyte-Macrophage Colony-Stimulating Factor; D016572:Cyclosporine; D011241:Prednisone