Immunosuppressive therapy for aplastic anemia in children: a more severe disease predicts better survival.
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Immunosuppressive therapy for aplastic anemia in children: a more severe disease predicts better survival. / Führer, Monika; Rampf, Udo; Baumann, Irith; Faldum, Andreas; Niemeyer, Charlotte; Janka-Schaub, Gritta; Friedrich, Wilhelm; Ebell, Wolfram; Borkhardt, Arndt; Bender-Goetze, Christine.
In: BLOOD, Vol. 106, No. 6, 6, 2005, p. 2102-2104.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Immunosuppressive therapy for aplastic anemia in children: a more severe disease predicts better survival.
AU - Führer, Monika
AU - Rampf, Udo
AU - Baumann, Irith
AU - Faldum, Andreas
AU - Niemeyer, Charlotte
AU - Janka-Schaub, Gritta
AU - Friedrich, Wilhelm
AU - Ebell, Wolfram
AU - Borkhardt, Arndt
AU - Bender-Goetze, Christine
PY - 2005
Y1 - 2005
N2 - 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.
AB - 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.
M3 - SCORING: Zeitschriftenaufsatz
VL - 106
SP - 2102
EP - 2104
JO - BLOOD
JF - BLOOD
SN - 0006-4971
IS - 6
M1 - 6
ER -