Management of Endocrine Complications Affecting Survival and Quality of Life in Children and Adolescents with Thalassemia Major: A Single Center Experience

Abstract

BACKGROUND/AIMS: Although advances for treating beta-thalassemia major have improved the survival of patients, endocrine complications are still quite common in these patients. These complications continue to negatively affect the survival and quality of life of patients with thalassemia major. This study aimed to determine the frequency and distribution of endocrine complications and to investigate the relationship between these complications and serum ferritin levels in children and adolescents followed up with the diagnosis of beta-thalassemia major MATERIALS AND METHODS: Fifty-eight children and adolescents (female/male: 27/31, mean age 12.6±5.16 years) diagnosed beta-thalassemia major were included in the study. Anthropometric (body weight and height standard deviation score), laboratory and radiological evaluations according to International Network of Endocrine Complications in Thalassemia (ICET) recommendations were performed. RESULTS: The median serum ferritin level was 2,969 ng/mL (562–10,251 ng/mL). In this study, the rate of at least one endocrine complication was 83% (n=48). Fifteen of them (31%) were under 10 years of age. The most common endocrine complications were vitamin D deficiency (53%), short stature (45%), osteopenia (34%), pubertal disorder (mean 25% for each sex) and osteoporosis. Ferritin levels did not correlate with anthropometric, laboratory parameters and endocrine complications. CONCLUSION: Regular monitoring of growth, vitamin D status, puberty, and all other endocrine functions is important to improve the quality of life of patients with thalassemia major. Increasing the quality of life and survival of these patients may be possible with early diagnosis and treatment of endocrine complications.