ESPE Abstracts

Introduction: β-thalassemia is a chronic hereditary hemolytic anemia characterized by a defect in the synthesis of beta-globin chains, particularly common in the Mediterranean region, southern Asia, and the Middle East. Transfusion programs and chelation therapy have considerably 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 polytransfused patients with thalassemia.

Objectives: To describe the frequency of growth retardation and to evaluate the GH-IGF-I axis in a group of patients with polytransfused β-thalassemia. To study the relationship between growth retardation and serum ferritin level, mean transfusion volume (VTM), duration of transfusion treatment, splenectomy, and compliance with chelation treatment.

Method: This is a descriptive, analytical and monocentric cross-sectional study which was carried out in the CHU Mustapha pediatrics department and which involved 87 patients (46 girls and 41 boys), followed regularly for several years for the most part, and treated by a transfusion regimen (more than 10 transfusions) combined with chelation therapy. All patients were assessed by clinical history, somatic examination, measurement of height and weight, followed by blood samples for biochemical assessment, IGF1 assay, and hand X-ray for all patients.. Secondly, 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 (<-3DS). 8 girls (50%) and 8 boys (50%) are affected by short stature; The average age of patients with growth retardation is 16.3 ± 5.6 years. among the patients with short stature, three patients were labeled as growth hormone deficiency (GHD). There is a statistically significant correlation between serum ferritin level and growth retardation (P=0.012), (OR=4.2).

Conclusion: This study confirms the need to screen for short stature and GH/IGF-I axis status in this group of patients. The presence of short stature does not appear to be correlated with efficacy parameters of transfusion and chelation therapy. Other mechanisms, in addition to iron overload, may therefore play a role in the pathogenesis of this clinical condition.