Universität zu Köln

Peinemann, Frank ORCID: 0000-0002-4727-1313 and Labeit, Alexander Michael (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). LONDON: BMJ PUBLISHING GROUP. ISSN 2044-6055

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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.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Peinemann, Frank UNSPECIFIED orcid.org/0000-0002-4727-1313 UNSPECIFIED Labeit, Alexander Michael UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-449872
DOI:	10.1136/bmjopen-2014-005039
Journal or Publication Title:	BMJ Open
Volume:	4
Number:	7
Date:	2014
Publisher:	BMJ PUBLISHING GROUP
Place of Publication:	LONDON
ISSN:	2044-6055
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language BONE-MARROW-TRANSPLANTATION; MENDELIAN RANDOMIZATION; ANTITHYMOCYTE GLOBULIN; CHILDREN; DISEASE; IST; AA Multiple languages Medicine, General & Internal Multiple languages
URI:	http://kups.ub.uni-koeln.de/id/eprint/44987