Highlights on Deferasirox Use and its Renal Toxicity in Children with Beta Thalassemia Major

Abstract

Background: Thalassemia is a hereditary disorder due to imbalance between α/β-globin chains. This leads to early hemolysis. It is of two type, α and β-thalassemia which is more sever needs lifelong follow up, treatment, by RBCs transfusion, to compensate for the drop in hemoglobin. This leads to increased iron stores and overload, excess iron deposited in different organs and kidneys. This leads to overproduction of free radicals; that causes organ damages.Objectives: This study aimed to highlight the prevalence of renal injury in patients with β -thalassemia major, comparing subcutaneous and new oral iron chelating agents, and their effects on the renal functions. Patients and methods: This case control and observational study included a total of 60 thalassemia major patients aged between 5 and 24 years who were following up at the Hematology Outpatient Clinic, Department of Pediatrics, Cairo University Hospitals. All patients were regularly transfused and receiving regular chelation program. Patients were divided between three groups. Results: There were no significant differences between the three groups regarding glomerular filtration rate (p> 0.05), and between Deferiprone and Deferoxamine groups regarding the age, serum ferritin, serum cystatin C and urinary β2-MG (p> 0.05). There were significant differences between Deferasirox group and both Deferiprone and Deferoxamine groups regarding serum ferritin, serum cystatin C and Urinary β2-MG (p< 0.05). Conclusion:Younger patients who are on iron chelation therapy with deferasirox needs regular assessment of renal dysfunction markers as having relatively higher levels of serum ferritin, serum cystatin C and urinary β2-MG.

Novel biochemical markers for the prediction of renal injury in beta-thalassemia major pediatric patients

“…Additionally, elevated levels of NGAL in both serum and urine may be attributed to various underlying factors associated with renal injuries, such as chronic hypoxia, toxicity from iron chelation treatment, or tubular damage caused by iron deposition. The exact cause of this increase remains unclear, but it suggests a potential link to these factors [8]. Badeli et al found no statistically significant association between renal damage and NGAL levels in the three groups of thalassemia patients who had received deferoxamine therapy [46].…”

“…Chronic doi: https://doi.org/10.15407/ubj95.05.041 anemia and hypoxia result in renal fibrosis and sclerosis, lowering systemic vascular resistance while increasing hyperfiltration, renal plasma flow, apoptosis, and tubular cell damage. Hypoxia and chronic anemia also lead to free radical production, lipid peroxidation, and oxidative stress [8]. Additionally, renal damage in thalassemic patients significantly contribu tes to the pathophysiology of iron overload.…”