Abstract | INTRODUCTION: Acquired severe aplastic anemia (SAA) is a rare and progressive disease characterized by an immune-mediated functional impairment of hematopoietic stem cells. Transplantation of these cells is a first-line treatment option if HLA-matched related donors are available. First-line immunosuppressive therapy may be offered as alternative. The aim was to compare the outcome of these patients in controlled trials. METHODS: A systematic search was performed in the bibliographic databases MEDLINE, EMBASE, and The Cochrane Library. To show an overview of various outcomes by treatment group we conducted a meta-analysis on overall survival. We evaluated whether studies reported statistically significant factors for improved survival. RESULTS: 26 non-randomized controlled trials (7,955 patients enrolled from 1970 to 2001) were identified. We did not identify any RCTs. Risk of bias was high except in 4 studies. Young age and recent year of treatment were identified as factors for improved survival in the HSCT group. Advanced age, SAA without very severe aplastic anemia, and combination of anti-lymphocyte globulin with cyclosporine A were factors for improved survival in the IST group. In 19 studies (4,855 patients), summary statistics were sufficient to be included in meta-analysis. Considerable heterogeneity did not justify a pooled estimate. Adverse events were inconsistently reported and varied significantly across studies. CONCLUSIONS: Young age and recent year of treatment were identified as factors for improved survival in the transplant group. Advanced age, SAA without very severe aplastic anemia, and combination of anti-lymphocyte globulin with cyclosporine A were factors for improved survival in the immunosuppressive group. Considerable heterogeneity of non-randomized controlled studies did not justify a pooled estimate. Adverse events were inconsistently reported and varied significantly across studies.
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Authors | Frank Peinemann, Ulrich Grouven, Nicolaus Kröger, Carmen Bartel, Max H Pittler, Stefan Lange |
Journal | PloS one
(PLoS One)
Vol. 6
Issue 4
Pg. e18572
(Apr 25 2011)
ISSN: 1932-6203 [Electronic] United States |
PMID | 21541024
(Publication Type: Comparative Study, Journal Article, Meta-Analysis)
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Chemical References |
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Topics |
- Adolescent
- Adult
- Aged
- Anemia, Aplastic
(drug therapy, mortality)
- Female
- Graft vs Host Disease
(chemically induced, etiology)
- Hematopoietic Stem Cell Transplantation
(adverse effects)
- Histocompatibility Testing
- Humans
- Immunosuppressive Agents
(adverse effects, therapeutic use)
- Male
- Middle Aged
- Publication Bias
- Survival Analysis
- Tissue Donors
- Treatment Outcome
- Young Adult
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