Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major.

Abstract

The safety and efficacy of a preparation with treosulfan/thiotepa/fludarabine were explored in 20 thalassaemia patients given allogeneic marrow transplantation. Seventeen patients were transplanted from unrelated donors after receiving anti-thymocyte globulin. The regimen was well tolerated. Two patients experienced secondary graft failure; one died of acute graft-versus-host disease. Cumulative incidence (95% confidence interval, CI) of transplantation-related mortality and graft failure was 5% (95% CI, 0-34%) and 11% (95% CI, 3-43%), respectively. Two-year probability of survival and thalassaemia-free survival was 95% (95% CI, 85-100%) and 85% (95% CI, 66-100%), respectively. This regimen might find elective application in patients at high risk of developing life-threatening complications.

DOI: 10.1111/j.1365-2141.2008.07385.x
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@article{Bernardo2008TreosulfanbasedCR, title={Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major.}, author={Maria Ester Bernardo and Marco Zecca and Eugenia Piras and Adriana Vacca and Giovanna Giorgiani and Chiara Cugno and Giovanni Caocci and Patrizia Comoli and Angela Mastronuzzi and Pietro Merli and Giorgio La Nasa and Franco Locatelli}, journal={British journal of haematology}, year={2008}, volume={143 4}, pages={548-51} }