title: Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major.
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.
authors.affiliation: Oncoematologia Pediatrica, Fondazione IRCCS Policlinico San Matteo, Università di Pavia, Italy. mebernardo@gmail.com
authors.forename: Maria Ester
authors.initials: ME
authors.lastname: Bernardo
authors.forename: Marco
authors.initials: M
authors.lastname: Zecca
authors.forename: Eugenia
authors.initials: E
authors.lastname: Piras
authors.forename: Adriana
authors.initials: A
authors.lastname: Vacca
authors.forename: Giovanna
authors.initials: G
authors.lastname: Giorgiani
authors.forename: Chiara
authors.initials: C
authors.lastname: Cugno
authors.forename: Giovanni
authors.initials: G
authors.lastname: Caocci
authors.forename: Patrizia
authors.initials: P
authors.lastname: Comoli
authors.forename: Angela
authors.initials: A
authors.lastname: Mastronuzzi
authors.forename: Pietro
authors.initials: P
authors.lastname: Merli
authors.forename: Giorgio
authors.initials: G
authors.lastname: La Nasa
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
mesh_terms: D000293:Adolescent; D000328:Adult; D002066:Busulfan / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D004359:Drug Therapy, Combination; D005260:Female; D006084:Graft Rejection; D006086:Graft vs Host Disease; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D008297:Male; D019653:Myeloablative Agonists / Q000009:adverse effects / Q000627:therapeutic use; D013852:Thiotepa / Q000009:adverse effects / Q000627:therapeutic use; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods*; D016896:Treatment Outcome; D014740:Vidarabine / Q000009:adverse effects / Q000031:analogs & derivatives / Q000627:therapeutic use; D055815:Young Adult; D017086:beta-Thalassemia / Q000188:drug therapy / Q000628:therapy*
chemical_list: D019653:Myeloablative Agonists; D013852:Thiotepa; C018404:treosulfan; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine