title: Stem cell transplantation of matched sibling donors compared with immunosuppressive therapy for acquired severe aplastic anaemia: a Cochrane systematic review.
abstract: OBJECTIVE
  Acquired severe aplastic anaemia is a rare and potentially fatal disease. The aim of this Cochrane review was to evaluate the effectiveness and adverse events of first-line allogeneic haematopoietic stem cell transplantation of human leucocyte antigen (HLA)-matched sibling donors compared with first-line immunosuppressive therapy.
  
  
  METHODS
  Specialised stem cell transplantations units in primary care hospitals.
  
  
  METHODS
  We included 302 participants with newly diagnosed acquired severe aplastic anaemia. The age ranged from early childhood to young adulthood. We excluded studies on participants with secondary aplastic anaemia.
  
  
  METHODS
  We included allogeneic haematopoietic stem cell transplantation as the test intervention harvested from any source of matched sibling donor and serving as a first-line therapy. We included immunosuppressive therapy as comparator with either antithymocyte/antilymphocyte globulin or ciclosporin or a combination of the two. PRIMARY AND SECONDARY OUTCOME MEASURES PLANNED AND FINALLY MEASURED: The primary outcome was overall mortality. Secondary outcomes were treatment-related mortality, graft failure, graft-versus-host disease, no response to immunosuppressive therapy, relapse after initial successful treatment, secondary clonal disease or malignancies, health-related quality of life and performance scores.
  
  
  RESULTS
  We identified three prospective non-randomised controlled trials with a study design that was consistent with the principle of 'Mendelian randomisation' in allocating patients to treatment groups. All studies had a high risk of bias due to the study design and were conducted more than 15 years. The pooled HR for overall mortality for the donor group versus the no donor group was 0.95 (95% CI 0.43 to 2.12, p=0.90).
  
  
  CONCLUSIONS
  There are insufficient and biased data that do not allow any firm conclusions to be made about the comparative effectiveness of first-line allogeneic haematopoietic stem cell transplantation of HLA-matched sibling donors and first-line immunosuppressive therapy of patients with acquired severe aplastic anaemia.
authors.affiliation: Children's Hospital, University of Cologne, Cologne, Germany.
authors.forename: Frank
authors.initials: F
authors.lastname: Peinemann
authors.affiliation: Center for Outcomes Research, University of Illinois College of Medicine, Peoria, Illinois, USA.
authors.forename: Alexander Michael
authors.initials: AM
authors.lastname: Labeit
mesh_terms: D000293:Adolescent; D000741:Anemia, Aplastic / Q000188:drug therapy* / Q000209:etiology / Q000601:surgery*; D002648:Child; D002675:Child, Preschool; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D012720:Severity of Illness Index; D035781:Siblings; D014019:Tissue Donors; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D007166:Immunosuppressive Agents
keywords: CHEMOTHERAPY; STATISTICS & RESEARCH METHODS; TRANSPLANT MEDICINE