Bone marrow transplantation for thalassemia. Experience in Pesaro, Italy.
OBJECTIVE
We reviewed the results of transplanting allogeneic marrow from HLA-identical donors in patients with beta-thalassemia. Among the 484 consecutive patients who have received transplants since 1981, survival and disease-free survival rates leveled off at approximately 1 year after transplantation, at 82 and 75%, respectively.


METHODS
Clinical characteristics of patients before transplant have been studied to determine their impact on survival, disease-free survival, and graft rejection. By multivariate analysis, portal fibrosis, hepatomegaly, and a history of inadequate chelation therapy were identified as risk factors. The patients were then divided into three classes of risk.


RESULTS
The rate of prolonged disease-free survival was 98% and 87% for class 1 and class 2 patients. This rate of disease-free survival is 70% with the use of our last conditioning protocol for class 3 patients. Older patients (17-32 years) have a 79% probability of prolonged disease-free survival.


CONCLUSIONS
We conclude that for patients with thalassemia major, transplantation of bone marrow from a human leukocyte antigen-identical donor offers a high probability of disease-free survival, particularly for those patients in early stages of their disease.
Giardini
C
C
Divisione Ematologica di Muraglia, Centro Trapianto Midollo Osseo, Ospedale di Pesaro, Italy.
Angelucci
E
E
Lucarelli
G
G
Galimberti
M
M
Polchi
P
P
Baronciani
D
D
Bechelli
G
G
D000293:Adolescent; D000328:Adult; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D006086:Graft vs Host Disease / Q000209:etiology; D006801:Humans; D007223:Infant; D007558:Italy; D015996:Survival Rate; D017086:beta-Thalassemia / Q000401:mortality / Q000628:therapy*