Sansanee Jitpirasakun1, Julaporn Pooliam2, Charotorn Sriwichakorn3, Kleebsabai Sanpakit3, Pairunyar Nakavachara4. 1. Division of Pediatric Endocrinology, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, 10700, Thailand. 2. Research Group and Research Network Division, Research Department, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand. 3. Division of Pediatric Hematology and Oncology, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand. 4. Division of Pediatric Endocrinology, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, 10700, Thailand. pairunyar.nak@mahidol.ac.th.
Abstract
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.
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.
Authors: M Caruso-Nicoletti; V De Sanctis; M Capra; G Cardinale; L Cuccia; F Di Gregorio; A Filosa; M C Galati; A Lauriola; R Malizia; A Mangiagli; F Massolo; C Mastrangelo; A Meo; M F Messina; G Ponzi; G Raiola; L Ruggiero; G Tamborino; A Saviano Journal: J Pediatr Endocrinol Metab Date: 1998 Impact factor: 1.634