Abstract
IntroductionAcquired 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.MethodsA 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.Results26 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.ConclusionsYoung 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.
Highlights
Acquired severe aplastic anemia (SAA) is a rare and progressive disease characterized by an immunemediated functional impairment of hematopoietic stem cells
Young age and recent year of treatment were identified as factors for improved survival in the hematopoietic stem cell transplantation (HSCT) group
In 19 studies (4,855 patients), summary statistics were sufficient to be included in meta-analysis
Summary
Acquired severe aplastic anemia (SAA) is a rare and progressive disease characterized by an immunemediated functional impairment of hematopoietic stem cells. Transplantation of these cells is a first-line treatment option if HLA-matched related donors are available. Acquired severe aplastic anemia (SAA) is a rare [1] and potentially fatal disease which is characterized by hypocellular bone marrow and pancytopenia, and mainly affects young adults. The treatment of SAA mainly includes immunosuppressive therapy (IST) with antithymocyte globulin (ATG)/antilymphocyte globulin (ALG) and cyclosporine A (CSA), or allogeneic hematopoietic stem cell transplantation (HSCT) [3,4,5]. Clinical treatment algorithms have been suggested to find a decision that meets individual conditions, personal preferences, and prognostic factors [9]
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