Unrelated bone marrow transplantation for beta-thalassemia patients: The experience of the Italian Bone Marrow Transplant Group.
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.
La Nasa
Giorgio
G
Centro Trapianti Midollo Osseo, Ospedale "R. Binaghi" ASL 8, via Is Guadazzonis 3, 09126 Cagliari, Italy. lanasa@tiscali.it
Argiolu
Franca
F
Giardini
Claudio
C
Pession
Andrea
A
Fagioli
Franca
F
Caocci
Giovanni
G
Vacca
Adriana
A
De Stefano
Piero
P
Piras
Eugenia
E
Ledda
Antonio
A
Piroddi
Antonio
A
Littera
Roberto
R
Nesci
Sonia
S
Locatelli
Franco
F
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