Sixty thalassemia patients (median age, 7 years; range, 1-37) underwent allogeneic hematopoietic stem cell transplantation (HSCT) after a preparation combining thiotepa, treosulfan, and fludarabine. Before HSCT, 27 children were assigned to risk class 1 of the Pesaro classification, 17 to class 2, and 4 to class 3; 12 patients were adults. Twenty patients were trans-planted from an HLA-identical sibling and 40 from an unrelated donor. The cumulative incidence of graft failure and transplantation-related mortality was 9% and 7%, respectively. Eight patients experienced grade II-IV acute GVHD, the cumulative incidence being 14%. Among 56 patients at risk, 1 developed limited chronic GVHD. With a median follow-up of 36 months (range, 4-72), the 5-year probability of survival and thalassemia-free survival are 93% and 84%, respectively. Neither the class of risk nor the donor used influenced outcome. This treosulfan-based preparation proved to be safe and effective for thalassemia patients given allogeneic HSCT. (Blood. 2012;120(2):473-476

Bernardo, M. E., Piras, E., Vacca, A., Giorgiani, G., Zecca, M., Bertaina, A., Pagliara, D., Contoli, B., Pinto, R. M., Caocci, G., Mastronuzzi, A., La Nasa, G., Locatelli, F., Allogeneic hematopoietic stem cell transplantation in thalassemia major: results of a reduced-toxicity conditioning regimen based on the use of treosulfan, <<BLOOD>>, 2012; 120 (2): 473-476. [doi:10.1182/blood-2012-04-423822] [https://hdl.handle.net/10807/245635]

Allogeneic hematopoietic stem cell transplantation in thalassemia major: results of a reduced-toxicity conditioning regimen based on the use of treosulfan

Locatelli, Franco
2012

Abstract

Sixty thalassemia patients (median age, 7 years; range, 1-37) underwent allogeneic hematopoietic stem cell transplantation (HSCT) after a preparation combining thiotepa, treosulfan, and fludarabine. Before HSCT, 27 children were assigned to risk class 1 of the Pesaro classification, 17 to class 2, and 4 to class 3; 12 patients were adults. Twenty patients were trans-planted from an HLA-identical sibling and 40 from an unrelated donor. The cumulative incidence of graft failure and transplantation-related mortality was 9% and 7%, respectively. Eight patients experienced grade II-IV acute GVHD, the cumulative incidence being 14%. Among 56 patients at risk, 1 developed limited chronic GVHD. With a median follow-up of 36 months (range, 4-72), the 5-year probability of survival and thalassemia-free survival are 93% and 84%, respectively. Neither the class of risk nor the donor used influenced outcome. This treosulfan-based preparation proved to be safe and effective for thalassemia patients given allogeneic HSCT. (Blood. 2012;120(2):473-476
2012
Inglese
Bernardo, M. E., Piras, E., Vacca, A., Giorgiani, G., Zecca, M., Bertaina, A., Pagliara, D., Contoli, B., Pinto, R. M., Caocci, G., Mastronuzzi, A., La Nasa, G., Locatelli, F., Allogeneic hematopoietic stem cell transplantation in thalassemia major: results of a reduced-toxicity conditioning regimen based on the use of treosulfan, <<BLOOD>>, 2012; 120 (2): 473-476. [doi:10.1182/blood-2012-04-423822] [https://hdl.handle.net/10807/245635]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10807/245635
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