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Stem cell transplantation of matched sibling donors compared with immunosuppressive therapy for acquired severe aplastic anaemia: a Cochrane systematic review.
BMJ Open. 2014 Jul 15; 4(7):e005039.BO

Abstract

OBJECTIVES

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.

SETTING

Specialised stem cell transplantations units in primary care hospitals.

PARTICIPANTS

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.

INTERVENTIONS

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+Show Affiliations

Children's Hospital, University of Cologne, Cologne, Germany.Center for Outcomes Research, University of Illinois College of Medicine, Peoria, Illinois, USA.

Pub Type(s)

Comparative Study
Journal Article
Research Support, Non-U.S. Gov't
Review
Systematic Review

Language

eng

PubMed ID

25031191

Citation

Peinemann, Frank, and Alexander Michael Labeit. "Stem Cell Transplantation of Matched Sibling Donors Compared With Immunosuppressive Therapy for Acquired Severe Aplastic Anaemia: a Cochrane Systematic Review." BMJ Open, vol. 4, no. 7, 2014, pp. e005039.
Peinemann F, Labeit AM. Stem cell transplantation of matched sibling donors compared with immunosuppressive therapy for acquired severe aplastic anaemia: a Cochrane systematic review. BMJ Open. 2014;4(7):e005039.
Peinemann, F., & Labeit, A. M. (2014). Stem cell transplantation of matched sibling donors compared with immunosuppressive therapy for acquired severe aplastic anaemia: a Cochrane systematic review. BMJ Open, 4(7), e005039. https://doi.org/10.1136/bmjopen-2014-005039
Peinemann F, Labeit AM. Stem Cell Transplantation of Matched Sibling Donors Compared With Immunosuppressive Therapy for Acquired Severe Aplastic Anaemia: a Cochrane Systematic Review. BMJ Open. 2014 Jul 15;4(7):e005039. PubMed PMID: 25031191.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Stem cell transplantation of matched sibling donors compared with immunosuppressive therapy for acquired severe aplastic anaemia: a Cochrane systematic review. AU - Peinemann,Frank, AU - Labeit,Alexander Michael, Y1 - 2014/07/15/ PY - 2014/7/18/entrez PY - 2014/7/18/pubmed PY - 2014/7/18/medline KW - CHEMOTHERAPY KW - STATISTICS & RESEARCH METHODS KW - TRANSPLANT MEDICINE SP - e005039 EP - e005039 JF - BMJ open JO - BMJ Open VL - 4 IS - 7 N2 - OBJECTIVES: 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. SETTING: Specialised stem cell transplantations units in primary care hospitals. PARTICIPANTS: 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. INTERVENTIONS: 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. SN - 2044-6055 UR - https://www.unboundmedicine.com/medline/citation/25031191/Stem_cell_transplantation_of_matched_sibling_donors_compared_with_immunosuppressive_therapy_for_acquired_severe_aplastic_anaemia:_a_Cochrane_systematic_review_ L2 - https://bmjopen.bmj.com/lookup/pmidlookup?view=long&pmid=25031191 DB - PRIME DP - Unbound Medicine ER -