title: Unrelated bone marrow transplantation for beta-thalassemia patients: The experience of the Italian Bone Marrow Transplant Group.
abstract: Bone marrow transplantation (BMT) remains the only potentially curative treatment for patients with thalassemia major. However, most candidates for BMT do not have a suitable family donor. In order to evaluate whether BMT from an HLA-matched unrelated volunteer donor can offer a probability of cure comparable to that obtained when the donor is a compatible sibling, we carried out a study involving 68 thalassemia patients transplanted in six Italian BMT Centers. Thirty-three males and 35 females (age range, 2-37 years; median age, 15) were transplanted from unrelated volunteer donors, all selected using high-resolution molecular typing of both HLA class I and II loci. Fourteen patients were classified in risk class 1; 16 in risk class 2; and 38 in risk class III of the Pesaro classification system. Nine patients (13%) had either primary or secondary graft failure. Fourteen patients (20%) died from transplant-related causes. Grade II-IV acute graft-versus-host disease (GVHD) developed in 24 cases (40%), and chronic GVHD in 10 cases (18%). Overall survival (OS) in the cohort of 68 patients was 79.3% (CI 67-88%), whereas the Kaplan-Meier estimates of disease-free survival (DFS) with transfusion independence was 65.8% (CI 54-77%). In the group of 30 thalassemic patients in risk classes 1 and 2, the probability of OS and DFS were 96.7% (CI 90-100%) and 80.0% (CI 65-94%), respectively, whereas in the 38 patients in class 3 OS was 65.2% (CI 49-80%) and DFS was 54.5% (CI 38-70%). These data show that when donor selection is based on stringent compatibility criteria, the results of unrelated transplantation in thalassemia patients are comparable to those obtained when the donor is a compatible sibling.
authors.affiliation: Centro Trapianti Midollo Osseo, Ospedale "R. Binaghi" ASL 8, via Is Guadazzonis 3, 09126 Cagliari, Italy. lanasa@tiscali.it
authors.forename: Giorgio
authors.initials: G
authors.lastname: La Nasa
authors.forename: Franca
authors.initials: F
authors.lastname: Argiolu
authors.forename: Claudio
authors.initials: C
authors.lastname: Giardini
authors.forename: Andrea
authors.initials: A
authors.lastname: Pession
authors.forename: Franca
authors.initials: F
authors.lastname: Fagioli
authors.forename: Giovanni
authors.initials: G
authors.lastname: Caocci
authors.forename: Adriana
authors.initials: A
authors.lastname: Vacca
authors.forename: Piero
authors.initials: P
authors.lastname: De Stefano
authors.forename: Eugenia
authors.initials: E
authors.lastname: Piras
authors.forename: Antonio
authors.initials: A
authors.lastname: Ledda
authors.forename: Antonio
authors.initials: A
authors.lastname: Piroddi
authors.forename: Roberto
authors.initials: R
authors.lastname: Littera
authors.forename: Sonia
authors.initials: S
authors.lastname: Nesci
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
mesh_terms: D000293:Adolescent; D000328:Adult; D001803:Blood Transfusion; D016026:Bone Marrow Transplantation / Q000401:mortality / Q000706:statistics & numerical data; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D018572:Disease-Free Survival; D005260:Female; D006084:Graft Rejection / Q000453:epidemiology; D006086:Graft vs Host Disease / Q000453:epidemiology; D006648:Histocompatibility; D006801:Humans; D007165:Immunosuppression Therapy; D015994:Incidence; D007558:Italy; D015993:Life Tables; D019520:Living Donors; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D018570:Risk Assessment; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000401:mortality; D014184:Transplantation, Homologous / Q000401:mortality / Q000706:statistics & numerical data; D016896:Treatment Outcome; D017086:beta-Thalassemia / Q000235:genetics / Q000401:mortality / Q000601:surgery* / Q000628:therapy