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Blood, 15 September 2005, Vol. 106, No. 6, pp. 2102-2104.
Prepublished online as a Blood First Edition Paper on June 2, 2005; DOI 10.1182/blood-2005-03-0874.
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Submitted March 2, 2005
Accepted May 14, 2005
Immunosuppressive therapy for aplastic anaemia in children: a more severe disease predicts better survival
Monika Fuehrer*, Udo Rampf, Irith Baumann, Andreas Faldum, Charlotte Niemeyer, Gritta Janka-Schaub, Wilhelm Friedrich, Wolfram Ebell, Arndt Borkhardt, and Christine Bender-Goetze
Children's University Hospital, Department of Hematology and Oncology, Ludwig-Maximilians University, Munich, Germany
Department of Pathology, University of Erlangen, Erlangen, Germany
Institut fur Medizinische Biometrie, Epidemiologie und Informatik, Johannes Gutenberg University, Mainz, Germany
Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany
Department of Hematology and Oncology, Children's University Hospital, Hamburg, Germany
Department of Pediatrics, University of Ulm, Ulm, Germany
Department of Pediatrics, Charite, Campus Virchow Klinikum, Humboldt University, Berlin, Germany
* Corresponding author; email: monika.fuehrer{at}med.uni-muenchen.de.
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 sub-classified into a severe (neutrophils >200/µl) and a very severe (< 200/µl) (vSAA) form. We report the results of a prospective multicentre trial with a combined immunosuppressive regimen of cyclosporin A (CSA), anti-thymocyte-globulin (ATG) and, in cases with neutrophils < 500/µl, granulocyte colony-stimulating factor (G-CSF) for treatment of SAA in children. Children with vSAA showed a higher rate of complete response than children with SAA (68% vs. 45%; p = 0.009), as well as better survival (93% vs. 81%; p = 0.0266). Thus, in children with SAA a more severe disease stage at diagnosis indicates a favourable outcome with immunosuppressive therapy.
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