Differences in longitudinal growth patterns of children and adolescents with transfusion-dependent hemoglobin E/β-thalassemia and those achieving successful hematopoietic stem-cell transplantation

Background: Short stature is a very common endocrinopathy among children with transfusion-dependent (TD) thalassemia. Hematopoietic stem-cell transplantation (HSCT) is the only effective curative treatment for TD thalassemia. This study aimed to identify and compare the longitudinal growth patterns of children with TD hemoglobin E (Hb E)/β-thalassemia against those of children successfully undergoing HSCT. Materials and methods: We reviewed the medical records of 39 patients with TD Hb E/β-thalassemia receiving regular blood transfusions, and 39 post-HSCT patients. Longitudinal weight and height Z-scores at each year of age were recorded for TD patients, and longitudinal weight and height Z-scores at each year before and after HSCT were obtained for post-HSCT patients. Results: The mean weight and height Z-scores of TD patients decreased gradually and were lowest at age 13. However, post-HSCT subjects saw significant improvement in their mean weight and height Z-scores 6 and 3 years after HSCT, respectively, relative to pre-HSCT baseline values. Conclusions: Longitudinal growth patterns differed between patients successfully undergoing HSCT and children and adolescents with TD Hb E/β-thalassemia. HSCT significantly improved height outcomes of children and adolescents with TD Hb E/β-thalassemia.