Hematopoietic stem cell transplantation for beta-thalassemia major: experience in south of Iran.
ABSTRACT Allogeneic stem cell transplantation as a curative treatment for thalassemia major was established in Shiraz in 1993. In this article we describe our results of 10 years experience with allogeneic bone marrow transplantation for thalassemia major.
From June 1993 to January 2003, 112 cases of beta-thalassemia major underwent allogeneic marrow transplantation from HLA-identical or one antigen-mismatched related donors. Conditioning chemotherapy included busulfan (14 to 15 mg/kg), cyclophosphamide (200 mg/kg), and antithymocyte globulin (ATG; 40 mg/kg). Prophylaxis for graft-versus-host disease consisted of cyclosporine, prednisolone, and methotrexate.
One hundred twelve patients with a diagnosis of beta-thalassemia major underwent allogeneic marrow transplantation during this period. The mean age of the patients was 9.5 years with the range of 2 to 20 years. The distribution of cases according to the Lucarelli classification were: 27 cases class I, 38 cases class II, and 47 cases class III. Eighty-seven of 112 patients (77.6%) with diagnosis of beta-thalassemia major are living with full engraftment at a median follow-up of 6 years (range 2 to 119 months).
Allogeneic bone marrow transplantation has changed the outcome of disease dramatically. According to our results stem cell transplantation is the treatment of choice for class I and II (Lucarelli risk groups). Also, we recommend transplantation as a curative method for treatment of class III beta-thalassemic patients.
- [show abstract] [hide abstract]
ABSTRACT: Yesilipek MA, Ertem M, Cetin M, Öniz H, Kansoy S, Tanyeli A, Anak S, Kurekci E, Hazar V. HLA-matched family hematopoetic stem cell transplantation in children with beta thalassemia major: The experience of the Turkish Pediatric Bone Marrow Transplantation Group. Abstract: From January 1991 to June 2009, 245 children with beta thalassemia major who underwent their first allogeneic HSCT in Turkey and who were followed for a minimum of one yr post-transplantation were enrolled this study. The median age of the patients was 6.6 yr old (range, 1-22 yr). The distribution of Pesaro risk class I, II, and III categories was 41, 130, and 63 children, respectively. The median serum ferritin level was 2203 ng/mL. Eighty-eight patients received bone marrow (BM) stem cells; 137, peripheral blood (PB) stem cells; and 20, cord blood (CB) stem cells. The donors were HLA-matched siblings or parents. Median engraftment times were shorter in PBSCT patients compared with the BMT group (p < 0.001). Grade II-IV acute GvHD was observed in 33 children (13.5%), while cGvHD was observed in 28 patients (12.5%), eight of whom had the extensive form. Thalassemic reconstitution was observed in 43 (17%) of the transplant patients. Post-transplant aplasia occurred in three patients, and the TRM rate was 7.75%. Seventeen patients were lost after 100 days. The thalassemia-free survival and OS rates were 68% (95% CI, 61.8-74.2) and 85.0% (95% CI, 80.2-89.8), respectively. We believe that this study is important because it is the first multicenter national data for children with beta thalassemia major receiving HSCT.Pediatric Transplantation 08/2012; · 1.50 Impact Factor
- [show abstract] [hide abstract]
ABSTRACT: Several clinical trials have shown the superiority of high-dose therapy (HDT) and autologous stem cell transplantation (ASCT) over conventional dose therapy for patients with multiple myeloma. There is limited experience with non-cryopreserved autologous hematopoietic stem cell transplantation. Between January 2004 and February 2010, we treated 38 patients with myeloma (mean age = 50.6) with a preparative regimen of Melphalan 140-200 mg/m(2) and non-cryopreserved ASCT. All the apheresis products were kept in a conventional blood bank refrigerator at 4°C for 2 d before infusion. The median time to platelet count of >20 × 10(9) /L was 13 d (range 10-31). Also, the median time to absolute neutrophil count >0.5 × 10(9) /L was 11 d (range 9-21). All the 38 patients were engrafted and there was not graft failure in this study group. Twenty-nine of 38 (76.3%) patients are alive and without disease activity after a median follow-up of 31 months (range 6-77). Responses (complete and partial response) were seen in all the 38 patients. The median progression-free survival was 27 months with 95% confidence interval 23.52-30.48, whereas the median overall survival was 30 months. One hundred days transplant-related mortality was 0%. HDT and autologous transplantation without cryopreservation is an effective and safe method which simplifies the procedure and is feasible and cost saving in our patients.Clinical Transplantation 09/2011; 26(1):117-22. · 1.63 Impact Factor