Haematopoietic stem cell transplantation from related donors in severe beta thalassaemia

• Published in: Bone marrow transplantation (Basingstoke) Vol. 43; no. S1; p. S232
• Main Authors: Ghavamzadeh, A, Hamidieh, A.A, Jahani, M, Alimoghaddam, K, Mousavi, A, Iravani, M, Bahar, B, Khodabandeh, A, Jalili, M
• Format: Journal Article
• Language: English
• Published: Nature Publishing Group 01.03.2009
• Subjects: Care and treatment; Health aspects; Hematopoietic stem cells; Histocompatibility antigens; HLA histocompatibility antigens; Patient outcomes; Thalassemia; Transplantation; Iran
• ISSN: 0268-3369

Objective: Follow-up of patients with Severe Beta Thalassemia transplanted in Hematology-Oncology and Stem Cell Transplantation Research Center, Tehran, Iran. Methods: 381 patients (165 female, 216 male) received allogeneic Hematopoietic Stem Cell Transplantation (HSCT) between 1991 and 2008. 358 patients were thalassemia major (106 patients in class I, 132 patients in class 2 and 120 patients in class 3) and 23 patients were thalassemia intermediate. 366 patients received stem cell from healthy full human leukocyte antigen (HLA) matched siblings, 5 patients from other related (HLA-matched confirms with high resolution method) and 10 patients from other related with one antigen mismatch. The graft source was bone marrow in 165 patients, peripheral blood stem cells (PBSC) in 207 patients and cord blood in 9 patients. For 10 patients second transplantation was performed. Median age at HSCT was 6 years (range, 2-28 years). 272 patients were conditioned with Busulfan and Cyclophosphamide, 59 patients with antithymocyte globulin (ATG), cyclophosphamide and Busulfan and 50 patients with ATG, Busulfan and Fludarabine. No radiation therapy was given. For graft-versus-host disease (GVHD) prophylaxis, we used cyclosporine and Methotrexate. Results: Median follow-up was 2.5 years. 319 (83.7%) patients are alive and 62 (16.3%) patients died. Most common causes of death include GVHD in 25 patients, infection in 11 patients and graft failure in 7 patients. 5-year overall survival and disease free survival was 80% (se = 2.4%) and 70% (se = 2.8%) respectively. Median time to ANC engraftment (ANC>0.5 x [10.sup.9]/I) was 15 days (range 6-73 days) and for platelets engraftment (PLT >20 x [10.sup.9]/l) was 22 days (range 6-92 days). Acute GvHD was seen in 67% of patients. Conclusions: Allogeneic HSCT remains the only cure for severe beta thalassaemia. Now we are the second after Pesaro team, regarding large number of transplanted thalassemia patients. In order to make this treatment available to more patients, we should enlarge the donor pool. As there are marriages among relatives in some countries, we may find HLA-identical relatives. It should consider that patients with thalassemia who lack sibling donor but have HLA-identical relatives should be transplanted before they are in class 3.