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

Maria Ester Bernardo, Marco Zecca, Eugenia Piras, Adriana Vacca, Giovanna Giorgiani, Chiara Cugno, Giovanni Caocci, Patrizia Comoli, Angela Mastronuzzi, Pietro Merli, Giorgio La Nasa, Franco Locatelli

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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.

Original languageEnglish
Pages (from-to)548-551
Number of pages4
JournalBritish Journal of Haematology
Volume143
Issue number4
DOIs
Publication statusPublished - 1 Nov 2008

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Keywords

  • Conditioning regimen
  • Haematopoietic stem cell transplantation
  • Thalassaemia major
  • Toxicity
  • Treosulfan

ASJC Scopus subject areas

  • Hematology

Cite this

Bernardo, M. E., Zecca, M., Piras, E., Vacca, A., Giorgiani, G., Cugno, C., Caocci, G., Comoli, P., Mastronuzzi, A., Merli, P., La Nasa, G., & Locatelli, F. (2008). Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major. British Journal of Haematology, 143(4), 548-551. https://doi.org/10.1111/j.1365-2141.2008.07385.x