title: Immunosuppressive therapy for aplastic anemia in children: a more severe disease predicts better survival.
abstract: Severe acquired aplastic anaemia (SAA) is a life-threatening disease characterized by pancytopenia and hypoplastic bone marrow. Autologous T lymphocytes are thought to cause bone marrow failure by immune-mediated excessive apoptosis of stem and progenitor cells. The disease is subclassified into a severe (neutrophil count, > 0.2 x 10(9)/L [> 200/microL]) and a very severe (< 0.2 x 10(9)/L [< 200/microL]) (vSAA) form. We report the results of a prospective multicenter trial with a combined immunosuppressive regimen of cyclosporin A (CSA), anti-thymocyte globulin (ATG) and, in cases with neutrophil counts fewer than 0.5 x 10(9)/L (< 500/microL), granulocyte colony-stimulating factor (G-CSF) for treatment of SAA in children. Children with vSAA showed a higher rate of complete response than did children with SAA (68% versus 45%; P = .009), as well as better survival (93% versus 81%; P < .001). Thus, in children with SAA a more severe disease stage at diagnosis indicates a favorable outcome with immunosuppressive therapy.
authors.affiliation: Children's University Hospital, Department of Hematology and Oncology, Ludwig-Maximilians-University, Munich, Germany. monika.fuehrer@med.uni-muenchen.de
authors.forename: Monika
authors.initials: M
authors.lastname: Führer
authors.forename: Udo
authors.initials: U
authors.lastname: Rampf
authors.forename: Irith
authors.initials: I
authors.lastname: Baumann
authors.forename: Andreas
authors.initials: A
authors.lastname: Faldum
authors.forename: Charlotte
authors.initials: C
authors.lastname: Niemeyer
authors.forename: Gritta
authors.initials: G
authors.lastname: Janka-Schaub
authors.forename: Wilhelm
authors.initials: W
authors.lastname: Friedrich
authors.forename: Wolfram
authors.initials: W
authors.lastname: Ebell
authors.forename: Arndt
authors.initials: A
authors.lastname: Borkhardt
authors.forename: Christine
authors.initials: C
authors.lastname: Bender-Goetze
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality; D000961:Antilymphocyte Serum / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000008:administration & dosage; D005260:Female; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D007958:Leukocyte Count; D008297:Male; D009504:Neutrophils; D011379:Prognosis; D015996:Survival Rate; D016896:Treatment Outcome
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016179:Granulocyte Colony-Stimulating Factor; D016572:Cyclosporine