1. Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major
- Author
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Pietro Merli, Adriana Vacca, Angela Mastronuzzi, Marco Zecca, Maria Ester Bernardo, Giovanna Giorgiani, Giovanni Caocci, Patrizia Comoli, Eugenia Piras, Giorgio La Nasa, Chiara Cugno, Franco Locatelli, Bernardo, M. E., Zecca, M., Piras, E., Vacca, A., Giorgiani, G., Cugno, C., Caocci, G., Comoli, P., Mastronuzzi, A., Merli, P., La Nasa, G., and Locatelli, F.
- Subjects
Adult ,Graft Rejection ,Male ,medicine.medical_specialty ,Transplantation Conditioning ,Treosulfan ,Adolescent ,medicine.medical_treatment ,Graft vs Host Disease ,Hematopoietic stem cell transplantation ,ThioTEPA ,Young Adult ,Conditioning regimen ,Internal medicine ,medicine ,Humans ,Cumulative incidence ,Child ,Busulfan ,Toxicity ,business.industry ,beta-Thalassemia ,Haematopoietic stem cell transplantation ,Hematopoietic Stem Cell Transplantation ,Infant ,Thalassaemia major ,Hematology ,Myeloablative Agonists ,medicine.disease ,Surgery ,Fludarabine ,Transplantation ,Regimen ,Hemoglobinopathy ,Treatment Outcome ,Child, Preschool ,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 ,Drug Therapy, Combination ,Female ,business ,Thiotepa ,Vidarabine ,medicine.drug - 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 The Authors.
- Published
- 2008