Unrelated donor stem cell transplantation in adult patients with thalassemia.
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.
La Nasa
G
G
Unità Operativa Centro Trapianti di Midollo Osseo, Centro Regionale Trapianti, P.O. R. Binaghi, Cagliari, Italy. lanasa@tiscali.it
Caocci
G
G
Argiolu
F
F
Giardini
C
C
Locatelli
F
F
Vacca
A
A
Orofino
M G
MG
Piras
E
E
Addari
M C
MC
Ledda
A
A
Contu
L
L
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