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.
Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major / M. E., B., M., Z., E., P., A., V., G., G., C., C., G., C., P., C., Mastronuzzi, A., P., M., G. L., N., F., L.. - In: BRITISH JOURNAL OF HAEMATOLOGY. - ISSN 0007-1048. - 143:(2008), pp. 548-551. [10.1111/j.1365-2141.2008.07385.x]
Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major.
MASTRONUZZI, ANGELA;F. Locatelli
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.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
