Unrelated bone marrow transplantation in thalassemia. The experience of the Italian Bone Marrow Transplant Group (GITMO).
OBJECTIVE
Allogeneic bone marrow transplantation (BMT) is a widely accepted therapeutic approach in homozygous beta-thalassemia. However, the majority of patients do not have a genotypically identical donor within the family. This prompted us to conduct a pilot study to investigate the feasibility of matched unrelated bone marrow transplantation in thalassemia. The major drawback was the high risk of immunologic and transplant-related complications, mainly graft-versus-host disease (GvHD) and graft failure.


METHODS
Our aim was to reduce this risk through careful selection of donor/recipient pairs. HLA haplotypes that show a high linkage disequilibrium among their class I, class II and class III alleles are considered extended or ancestral haplotypes.


RESULTS
These haplotypes are conserved and can be shared by apparently unrelated individuals. Our study shows that matching for these haplotypes significantly improves the outcome of unrelated bone marrow transplantation in thalassemia. In fact, results were comparable to those obtained in transplants using HLA-identifical family donors.


CONCLUSIONS
Better results were obtained in patients with lesser iron overload and when the donor shared an identity for the DPB1 alleles.
La Nasa
G
G
Centro Trapianti Midollo Osseo, Ospedale R. Binaghi, Cattedra di Genetica Medica, Università di Cagliari, Italy. lanasa@tiscalinet.it
Giardini
C
C
Locatelli
F
F
Argiolu
F
F
Vassallo
E
E
Prete
A
A
Caocci
G
G
Floris
R
R
Garau
P
P
Littera
R
R
Mantovani
D
D
Oppi
S
S
Piras
E
E
De Stefano
P
P
Sanna
M A
MA
Mulargia
M
M
Carcassi
C
C
Contu
L
L
D000293:Adolescent; D000328:Adult; D016026:Bone Marrow Transplantation / Q000276:immunology / Q000706:statistics & numerical data*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006650:Histocompatibility Testing; D006801:Humans; D007558:Italy; D008297:Male; D012189:Retrospective Studies; D018570:Risk Assessment; D014184:Transplantation, Homologous / Q000706:statistics & numerical data*; D016896:Treatment Outcome; D017086:beta-Thalassemia / Q000276:immunology / Q000628:therapy*