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Blood, 15 June 2002, Vol. 99, No. 12, pp. 4350-4356
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Unrelated donor bone marrow transplantation for thalassemia:
the effect of extended haplotypes
Giorgio La Nasa,
Claudio Giardini,
Francesca Argiolu,
Franco Locatelli,
Marcella Arras,
Piero De Stefano,
Antonio Ledda,
Antonella Pizzati,
Maria A. Sanna,
Adriana Vacca,
Guido Lucarelli, and
Licinio Contu
From the Centro Trapianti Midollo Osseo, Ospedale
Binaghi-Cattedra di Genetica Medica, and the Dipartimento di Scienze
Biomediche e Biotecnologie, Università di Cagliari, Italy; the
Unità Operativa di Ematologia, Centro Trapianti di Midollo Osseo
di Muraglia, Ospedale San Salvatore, Pesaro, Italy; and the
Oncoematologia Pediatrica, Università di Pavia, IRCCS Policlinico
San Matteo, Pavia, Italy.
Allogeneic bone marrow transplantation (BMT) from a genotypically
identical family donor is an accepted therapeutic option for homozygous
beta-thalassemia. However, only a minority of patients have access to
this curative procedure. The aim of this study is to explore the
feasibility of matched unrelated transplants in thalassemia and the
possibility of reducing the risk of immunologic complications through
careful selection of donor/recipient pairs. Since November 1992, 32 patients (age range, 2-28 years) have been enrolled. There were 4 patients assigned to risk-class I, 11 to risk-class II, and 17 to
risk-class III of the Pesaro classification. Extended haplotype
analysis and family segregation studies were employed for
identification of suitable donors. Of the 32 donor/recipient pairs, 24 were identical for HLA-A, B, C, DRB1, DRB3, DRB4, DRB5, DQA1, and DQB1
loci; 7 pairs were identical for 2 extended haplotypes, and 15 pairs
shared one extended haplotype. Grade II-IV acute graft-versus-host
disease (GVHD) developed in 11 cases (41%) and chronic GVHD in 6 (25%) out of 24 patients at risk. There are 22 patients (69%) who are
alive and transfusion-independent after a median follow-up of 30 months
(range, 7-109 months). There were 6 patients (19%) who engrafted and
subsequently died from transplant-related complications. In 4 cases (12.5%) graft rejection was observed within 30 days and it was
followed by autologous reconstitution. Out of 22 patients with a donor
identical for at least one extended haplotype, there are 19 who
survived, 17 of them being transfusion-independent. Among the 10 recipients who did not share any extended haplotype with the donor,
only 5 are alive without thalassemia and 3 patients died. Of the 6 patients who died, 5 belonged to risk-class III and only 1 to
risk-class II. BMT from well-selected unrelated donors may offer
results comparable to those obtained in transplantations using
HLA-identical family donors, especially for patients with lesser iron overload.
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