Current Results of Bone Marrow Transplantation in Patients with Acquired Severe Aplastic Anemia
Report of the European Group for Blood and Marrow TransplantationBacigalupo A. · Oneto R. · Bruno B. · Socié G. · Passweg J. · Locasciulli A. · van Lint M.T. · Tichelli A. · McCann S. · Marsh J. · Ljungman P. · Hows J. · Marin P. · Schrezenmeier H.
on behalf of the Working Party on Severe Aplastic Anemia of the European Group for Blood and Marrow Transplantation, and the Medical Statistical Department, University of Leiden, The Netherlands
We have analyzed 2,002 patients grafted in Europe between 1976 and 1998 from an identical twin (n = 34), from an HLA-identical sibling (n = 1,699) or from an alternative donor (n = 269), which included unrelated and family mismatched donors. The proportions of patients surviving in these three groups are, respectively, 91, 66 and 37%: major causes of failure were acute graft-versus host disease (GvHD) (11%), infection (12%), pneumonitis (4%), rejection (4%). In multivariate Cox analysis, factors predicting outcome were patient’s age (p < 0.0001), donor type (p < 0.0001), interval between diagnosis and bone marrow transplantation (BMT) (p < 0.0005), year of BMT (p = 0.0005) and female donor for a male recipient (p = 0.02). Patients were then divided in two groups according to the year of BMT: up to or after 1990. The overall death rate dropped from 43 to 24% (p < 0.00001). Improvements were seen mostly for grafts from identical siblings (from 54 to 75%, p < 0.0001), and less so for alternative-donor grafts (from 28 to 35%; p = 0.07). Major changes have occurred in the BMT protocol: decreasing use of radiotherapy in the conditioning regimen (from 35 to 24%; p < 0.0001) and increasing use of cyclosporin (with or without methotrexate) for GvHD prophylaxis (from 70 to 98%; p < 0.0001). In conclusion, the outcome of allogeneic BMT for patients with severe aplastic anemia has considerably improved over the past two decades: young patients, grafted early after diagnosis from an identical sibling, have currently an over 80% chance of long-term survival. Transplants from twins are very successful as well. The risk of complications with alternative donor transplants is still high.
© 2000 S. Karger AG, Basel
Second Department of Haematology
Ospedale San Martino, Servizio Radioterapia IST
I–16132 Genoa (Italy)
Tel. +39 010 355 469, Fax +39 010 355 583,
Number of Print Pages : 7
Number of Figures : 1, Number of Tables : 2, Number of References : 20
Affiliated with Molecular Biology of Hematopoiesis Symposium
Vol. 103, No. 1, Year 2000 (Cover Date: Released March 2000)
Journal Editor: I. Ben-Bassat, Tel Hashomer
ISSN: 0001–5792 (print), 1421–9662 (Online)
For additional information: http://www.karger.com/journals/aha