Unrelated donor stem cell transplantation in adult patients with thalassemia.

Abstract:

:Allogeneic SCT remains the only potential cure for patients with thalassemia. However, most BMT candidates lack a suitable family donor and require an unrelated donor (UD). We evaluated whether BMT using UDs in high-risk adult thalassemia patients can offer a probability of cure comparable to that reported employing an HLA-compatible sibling as donor. A total of 27 adult thalassemia patients (15 males and 12 females, median age 22 years) underwent BMT from a UD selected by high-resolution HLA molecular typing. The conditioning regimen consisted of Busulphan (BU, 14 mg/kg) plus Cyclophosphamide (CY, 120 or 160 mg/kg) in 12 cases and BU (14 mg/kg), Thiotepa (10 mg/kg) and CY (120-160 mg/kg) in the remaining 15 cases. Cyclosporine-A and short-term Methotrexate were used for graft-versus-host disease (GVHD) prophylaxis. In all, 19 patients (70%) are alive and transfusion-independent after a median follow-up of 43 months (range 16-137). A total of 10 patients (37%) developed grade II-IV acute GVHD and six (27%) chronic GVHD. Eight patients (30%) died from transplant-related causes. UD-BMT can cure more than two-thirds of adult thalassemia patients, and is a particularly attractive option for patients who are not compliant with conventional treatment.

journal_name

Bone Marrow Transplant

authors

La Nasa G,Caocci G,Argiolu F,Giardini C,Locatelli F,Vacca A,Orofino MG,Piras E,Addari MC,Ledda A,Contu L

doi

10.1038/sj.bmt.1705173

subject

Has Abstract

pub_date

2005-12-01 00:00:00

pages

971-5

issue

11

eissn

0268-3369

issn

1476-5365

pii

1705173

journal_volume

36

pub_type

临床试验,杂志文章,多中心研究
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