Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Meta-Analysis
. 2011 Apr 25;6(4):e18572.
doi: 10.1371/journal.pone.0018572.

First-line matched related donor hematopoietic stem cell transplantation compared to immunosuppressive therapy in acquired severe aplastic anemia

Frank Peinemann  1 , Ulrich Grouven, Nicolaus Kröger, Carmen Bartel, Max H Pittler, Stefan Lange
Affiliations
Meta-Analysis

First-line matched related donor hematopoietic stem cell transplantation compared to immunosuppressive therapy in acquired severe aplastic anemia

Frank Peinemann et al. PLoS One. .

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.

PubMed Disclaimer

Conflict of interest statement

Competing Interests: The authors have declared that no competing interests exist.

Figures

Figure 1
Figure 1. Literature search and study flow.
Abbreviations: HSCT: hematopoietic stem cell transplantation; IST: immunosuppressive therapy; SAA: severe aplastic anemia.
Figure 2
Figure 2. Meta-analysis of all studies with available data.
Meta-analysis of overall survival using hazard-ratio as effect measure after first-line HSCT vs. first-line IST. Pooled estimate not justified because of considerable heterogeneity and of not sufficient external validity. Abbreviations: HSCT: hematopoietic stem cell transplantation; IST: immunosuppressive therapy; SAA: severe aplastic anemia; SE: standard error.
Figure 3
Figure 3. Funnel plot using data from Figure 3 .
Distribution of estimates can be regarded as funnel-shaped and compatible with a moderate publication bias. Abbreviations: SE: standard error.
See this image and copyright information in PMC

References

    1. ORD . Office of Rare Diseases Terms: Aplastic anemia. Bethesda: National Institutes of Health (NIH); 2010.
    1. Kaufman DW, Kelly JP, Issaragrisil S, Laporte JR, Anderson T, et al. Relative incidence of agranulocytosis and aplastic anemia. Am J Hematol. 2006;81:65–67. - PubMed
    1. Brodsky RA, Jones RJ. Aplastic anaemia. Lancet. 2005;365:1647–1656. - PubMed
    1. Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006;108:2509–2519. - PMC - PubMed
    1. EBMT-AAWP. Guidelines for treating of aplastic anemia. London: European Group for Blood and Marrow Transplantation (EBMT) Aplastic Anaemia Working Party (AAWP); 2000.

MeSH terms

Substances