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.

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., Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major, <<BRITISH JOURNAL OF HAEMATOLOGY>>, 2008; 143 (4): 548-551. [doi:10.1111/j.1365-2141.2008.07385.x] [https://hdl.handle.net/10807/258301]

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

Locatelli, Franco
2008

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.
2008
Inglese
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., Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major, <<BRITISH JOURNAL OF HAEMATOLOGY>>, 2008; 143 (4): 548-551. [doi:10.1111/j.1365-2141.2008.07385.x] [https://hdl.handle.net/10807/258301]
File in questo prodotto:
File Dimensione Formato  
Treosulfan‐based conditioning regimen for allogeneic haematopoietic stem cell.pdf

accesso aperto

Tipologia file ?: Versione Editoriale (PDF)
Licenza: Creative commons
Dimensione 100.98 kB
Formato Adobe PDF
100.98 kB Adobe PDF Visualizza/Apri

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10807/258301
Citazioni
  • ???jsp.display-item.citation.pmc??? 15
  • Scopus 59
  • ???jsp.display-item.citation.isi??? 50
social impact