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., Bernardo; M., Zecca; E., Piras; A., Vacca; G., Giorgiani; C., Cugno; G., Caocci; P., Comoli; Mastronuzzi, Angela; P., Merli; G. L., Nasa; F., Locatelli. - 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.
2008
Adolescent, Adult, Busulfan; adverse effects/analogs /&/ derivatives/therapeutic use, Child, Child; Preschool, Drug Therapy; Combination, Female, Graft Rejection, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Humans, Infant, Male, Myeloablative Agonists; adverse effects/therapeutic use, Thiotepa; adverse effects/therapeutic use, Transplantation Conditioning; adverse effects/methods, Treatment Outcome, Vidarabine; adverse effects/analogs /&/ derivatives/therapeutic use, Young Adult, beta-Thalassemia; drug therapy/therapy
01 Pubblicazione su rivista::01a Articolo in rivista
Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major / M. E., Bernardo; M., Zecca; E., Piras; A., Vacca; G., Giorgiani; C., Cugno; G., Caocci; P., Comoli; Mastronuzzi, Angela; P., Merli; G. L., Nasa; F., Locatelli. - In: BRITISH JOURNAL OF HAEMATOLOGY. - ISSN 0007-1048. - 143:(2008), pp. 548-551. [10.1111/j.1365-2141.2008.07385.x]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/759356
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