Hematopoietic Stem Cell Transplantation in Thalassemia Major. Report from the Ebmt Hemoglobinopathy Registry

Abstract Abstract 905 Background. Hemopoietic allogeneic stem cell transplantation (HSCT) still remains the only available curative option for β-thalassaemia major. At today more than 3000 transplants have been performed worldwide and registered. We analyze transplant activity and outcome after 20 years of the European Hemoglobinopathy Bone Marrow Transplant (EBMT) Registry. Methods. Data have been analyzed from promise MED-A form. Survival data have been reported with the method of Kaplan-Meier in patients with complete survival data. Results. One hundred and 34 centres from 28 different countries reported their data to the registry. Three thousands and 821 (3821) consecutive first HSCT for patients affected by a hemoglobinopathy have been registered. There were only 364 (10.5%) patients aged 18 years or older. Further analyses were restricted to patients with thalassemia major and to transplants performed after January 1st, 2000. In this latter period 1493 transplants have been performed (median age 7.2 years, range 0.3–45.1) with 133 (9.8%) patients aged 18 years or older. During the last decade 70% of the transplants have been performed in Europe and 30% in Asia and Africa with a trend to an increase activity in these countries compared to previous decades. Of the 1493 transplants performed after January 2000, 1061 (71%) have been performed from an HLA identical sibling donors, 129 (8.6%) from a matched other relative, 57 (3.8%) from a mismatched relative, 210 (14%) from an unrelated donor and in 36 (2.4%) cases donor information were missing. Source of stem cells was bone marrow in 1011 patients (67.7%), peripheral blood in 303 patients (20.3%), cord blood in 60 patients (4%), mixed in 82 patients (5.5%). Source of stem cell was missing in 37 (2.5%). In matched sibling donor transplant recipients overall survival (OS) and thalassemia free survival (TFS) plotted at 91 ± 0.01% and 83 ± 0.01%, respectively. Significant differences have been reported in OS and TFS when related to different age groups as reported in Table 1 (P < 0.001). Conclusion. This analysis confirms allogeneic transplantation is a worldwide diffuse high success curative procedure for β-thalassaemia major. Bone marrow still remains the principal source of stem cells. The large majority of transplants have been performed from a matched sibling donor with a marginal role of other donors. In addition these data demonstrate the trend to not transplant adult patients. In the univariate analyses 14 years old appears as threshold age for best transplant strategy. Disclosures: No relevant conflicts of interest to declare.