Immunosuppressive therapy for aplastic anemia in children: a more severe disease predicts better survival.
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.
Führer
Monika
M
Children's University Hospital, Department of Hematology and Oncology, Ludwig-Maximilians-University, Munich, Germany. monika.fuehrer@med.uni-muenchen.de
Rampf
Udo
U
Baumann
Irith
I
Faldum
Andreas
A
Niemeyer
Charlotte
C
Janka-Schaub
Gritta
G
Friedrich
Wilhelm
W
Ebell
Wolfram
W
Borkhardt
Arndt
A
Bender-Goetze
Christine
C
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
D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016179:Granulocyte Colony-Stimulating Factor; D016572:Cyclosporine