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 s...

Full description

Saved in:
Bibliographic Details
Published in:Blood 2005-09, Vol.106 (6), p.2102-2104
Main Authors: Führer, Monika, Rampf, Udo, Baumann, Irith, Faldum, Andreas, Niemeyer, Charlotte, Janka-Schaub, Gritta, Friedrich, Wilhelm, Ebell, Wolfram, Borkhardt, Arndt, Bender-Goetze, Christine
Format: Article
Language:English
Subjects:
Adolescent
Anemia, Aplastic - diagnosis
Anemia, Aplastic - drug therapy
Anemia, Aplastic - mortality
Antilymphocyte Serum - administration & dosage
Biological and medical sciences
Child
Child, Preschool
Cyclosporine - administration & dosage
Female
Granulocyte Colony-Stimulating Factor - administration & dosage
Hematologic and hematopoietic diseases
Humans
Immunosuppressive Agents - therapeutic use
Infant
Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis
Leukocyte Count
Male
Medical sciences
Neutrophils
Prognosis
Survival Rate
Treatment Outcome
Citations: Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary: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 × 109/L [> 200/μL]) and a very severe (< 0.2 × 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 × 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)
ISSN:0006-4971
1528-0020
DOI:10.1182/blood-2005-03-0874