Unrelated donor stem cell transplantation in adult patients with thalassemia

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

Research output: Contribution to journalArticlepeer-review


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, 14mg/kg) plus Cyclophosphamide (CY, 120 or 160mg/kg) in 12 cases and BU (14mg/kg), Thiotepa (10mg/kg) and CY (120-160mg/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.

Original languageEnglish
Pages (from-to)971-975
Number of pages5
JournalBone Marrow Transplantation
Issue number11
Publication statusPublished - Dec 2005


  • Adult class 3
  • Thalassemia
  • Unrelated BMT

ASJC Scopus subject areas

  • Hematology
  • Transplantation


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