Growth disorders in polytransfused Beta-Thalassemia Patients

Abstract

Introduction: Beta-thalassemia is a chronic hereditary haemolytic anaemia characterized by a defect in the synthesis of beta-globin chains, which is particularly common in the Mediterranean region, South Asia, and the Middle East. Transfusion programs and chelation therapy have significantly extended the life expectancy of patients. This has led to an increase in the prevalence of complications related to iron overload. Growth retardation is extremely common in patients with transfusion-dependent thalassemia. Objectives: To describe the frequency of growth retardation and to evaluate the GH-IGF-I axis in a group of patients with transfusion-dependent beta-thalassemia. To study the relationship between growth retardation, ferritin levels, average transfusion volume, duration of transfusion therapy, splenectomy, and compliance with chelation therapy. Methods: This is a cross-sectional, descriptive, analytical, and monocentric study conducted in the pediatric department of Mustapha Bacha university hospital centre. The study involved 87 patients (46 girls and 41 boys) most of whom were regularly followed up for several years and treated with a transfusion regimen (more than 10 transfusions) combined with chelation therapy. All patients were assessed through clinical history, physical examination, measurement of height and weight, followed by blood tests for biochemical analysis, IGF1 assay, and a hand X-ray. Afterwards, we performed a dynamic test for patients with growth retardation. Results: Short stature (height <-2SD) was detected in 16 patients (18.4%). Among the 16 patients with short stature, 6 (37.5%) had severe growth retardation (<-3SD). 8 girls (50%) and 8 boys (50%) were affected by growth retardation. The mean age of patients with growth retardation was 16.3±5.6 years. Among the patients with short stature, three patients were diagnosed with growth hormone deficiency (GHD). There was a statistically significant correlation between serum ferritin levels and growth retardation (P=0.012), (OR=4.2). Conclusion: This study confirms the need to screen for short stature and the GH/IGF-I axis status in this group of patients. The presence of short stature does not seem to be correlated with the efficacy parameters of transfusion and chelation therapy. Therefore, other mechanisms, in addition to iron overload, may play a role in the pathogenesis of this clinical condition.

Keywords: Growth retardation, transfusion-dependent beta-thalassemia.