Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in patients with thalassaemia major.
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.
Bernardo
Maria Ester
ME
Oncoematologia Pediatrica, Fondazione IRCCS Policlinico San Matteo, Università di Pavia, Italy. mebernardo@gmail.com
Zecca
Marco
M
Piras
Eugenia
E
Vacca
Adriana
A
Giorgiani
Giovanna
G
Cugno
Chiara
C
Caocci
Giovanni
G
Comoli
Patrizia
P
Mastronuzzi
Angela
A
Merli
Pietro
P
La Nasa
Giorgio
G
Locatelli
Franco
F
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*
D019653:Myeloablative Agonists; D013852:Thiotepa; C018404:treosulfan; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine