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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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IMMUNOBIOLOGY Brief report
Immunosuppressive therapy for aplastic anemia in children: a more severe disease predicts better survival
Monika Führer,
Udo Rampf,
Irith Baumann,
Andreas Faldum,
Charlotte Niemeyer,
Gritta Janka-Schaub,
Wilhelm Friedrich,
Wolfram Ebell,
Arndt Borkhardt,
Christine Bender-Goetze, for the German/Austrian Aplastic Anemia Working Group
From the Children's University Hospital, Department of Hematology and Oncology, Ludwig-Maximilians-University, Munich, Germany; the Department of Pathology, University of Erlangen, Erlangen, Germany; the Institut for Medizinische Biometrie, Epidemiologie und Informatik, Johannes Gutenberg University, Mainz, Germany; the Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany; the Children's University Hospital, Department of Hematology and Oncology, Hamburg, Germany; the Department of Pediatrics, University of Ulm, Ulm, Germany; and the Department of Pediatrics, Charite, Campus Virchow Klinikum, Humboldt University, Berlin, Germany.
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 109/L [> 200/µL]) and a very severe (< 0.2 x 109/L [< 200/µL]) (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 109/L (< 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 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. (Blood. 2005;106:2102-2104)

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