title: Unrelated donor stem cell transplantation in adult patients with thalassemia.
abstract: Allogeneic SCT remains the only potential cure for patients with thalassemia. However, most BMT candidates lack a suitable family donor and require an unrelated donor (UD). We evaluated whether BMT using UDs in high-risk adult thalassemia patients can offer a probability of cure comparable to that reported employing an HLA-compatible sibling as donor. A total of 27 adult thalassemia patients (15 males and 12 females, median age 22 years) underwent BMT from a UD selected by high-resolution HLA molecular typing. The conditioning regimen consisted of Busulphan (BU, 14 mg/kg) plus Cyclophosphamide (CY, 120 or 160 mg/kg) in 12 cases and BU (14 mg/kg), Thiotepa (10 mg/kg) and CY (120-160 mg/kg) in the remaining 15 cases. Cyclosporine-A and short-term Methotrexate were used for graft-versus-host disease (GVHD) prophylaxis. In all, 19 patients (70%) are alive and transfusion-independent after a median follow-up of 43 months (range 16-137). A total of 10 patients (37%) developed grade II-IV acute GVHD and six (27%) chronic GVHD. Eight patients (30%) died from transplant-related causes. UD-BMT can cure more than two-thirds of adult thalassemia patients, and is a particularly attractive option for patients who are not compliant with conventional treatment.
authors.affiliation: Unità Operativa Centro Trapianti di Midollo Osseo, Centro Regionale Trapianti, P.O. R. Binaghi, Cagliari, Italy. lanasa@tiscali.it
authors.forename: G
authors.initials: G
authors.lastname: La Nasa
authors.forename: G
authors.initials: G
authors.lastname: Caocci
authors.forename: F
authors.initials: F
authors.lastname: Argiolu
authors.forename: C
authors.initials: C
authors.lastname: Giardini
authors.forename: F
authors.initials: F
authors.lastname: Locatelli
authors.forename: A
authors.initials: A
authors.lastname: Vacca
authors.forename: M G
authors.initials: MG
authors.lastname: Orofino
authors.forename: E
authors.initials: E
authors.lastname: Piras
authors.forename: M C
authors.initials: MC
authors.lastname: Addari
authors.forename: A
authors.initials: A
authors.lastname: Ledda
authors.forename: L
authors.initials: L
authors.lastname: Contu
mesh_terms: D000293:Adolescent; D000328:Adult; D016026:Bone Marrow Transplantation; D002423:Cause of Death; D005260:Female; D006086:Graft vs Host Disease / Q000517:prevention & control; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods* / Q000401:mortality; D006650:Histocompatibility Testing / Q000379:methods; D006801:Humans; D008297:Male; D016019:Survival Analysis; D013789:Thalassemia / Q000401:mortality / Q000628:therapy*; D014019:Tissue Donors; D019172:Transplantation Conditioning / Q000379:methods; D016896:Treatment Outcome