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

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.

• 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)

Chemical References

• Immunosuppressive Agents

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