Allogeneic hematopoietic stem cell transplantation in thalassemia major: results of a reduced-toxicity conditioning regimen based on the use of treosulfan.
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 transplanted 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.
Department of Pediatric Hematology-Oncology, Istituto di Ricovero e Cura a Carattere Scientifico, Bambino Gesù Children's Hospital, Piazzale S Onofrio, Rome, Italy. mariaester.bernardo@opbg.net
Maria Ester
ME
Bernardo
Eugenia
E
Piras
Adriana
A
Vacca
Giovanna
G
Giorgiani
Marco
M
Zecca
Alice
A
Bertaina
Daria
D
Pagliara
Benedetta
B
Contoli
Rita Maria
RM
Pinto
Giovanni
G
Caocci
Angela
A
Mastronuzzi
Giorgio
G
La Nasa
Franco
F
Locatelli
D000293:Adolescent; D000328:Adult; D002066:Busulfan / Q000031:analogs & derivatives* / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods* / Q000401:mortality; D006801:Humans; D007223:Infant; D008297:Male; D019653:Myeloablative Agonists / Q000627:therapeutic use; D012307:Risk Factors; D013852:Thiotepa / Q000627:therapeutic use; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D014740:Vidarabine / Q000031:analogs & derivatives / Q000627:therapeutic use; D055815:Young Adult; D017086:beta-Thalassemia / Q000628:therapy*
D019653:Myeloablative Agonists; D013852:Thiotepa; C018404:treosulfan; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine