A Prospective Study of Tubular Dysfunction in Pediatric Patients with Beta Thalassemia Major Receiving Deferasirox

Background: Beta thalassemia major is a lifelong transfusion-dependent disorder. Transfusion-dependent thalassemia patients are prone to develop renal dysfunction due to iron overload, chronic anemia, and/or chelation therapy.

Methods: In this prospective study, thalassemia patients who fitted inclusion and exclusion criteria received Deferasirox 20 mg/kg/day. A complete biochemistry analysis of serum and 24-hour-urine specimens was performed before and after treatment. Estimated glomerular filtration rate (eGFR), Fractional excretion of sodium (FENA), potassium (FEK), uric acid (FEUA), and the maximum ratio of tubular reabsorption of phosphorus to eGFR (TmP/GFR) at baseline and after treatment was calculated and compared.

Results: A total of 30 patients with mean age of 4.9 ± 3.2 years were recruited. The mean serum creatinine increased significantly after 6 months of treatment (0.54 ± 0.08 vs. 0.67 ± 0.16, P < .001) while eGFR was decreased (104.36 ± 19.62 vs. 86.00 ± 16.92, P < .001). Mean potassium level in serum was increased after treatment, while serum calcium, magnesium, and uric acid levels decreased significantly (P > .05). A significant increase was confirmed for mean urinary β2-microglobulin (β2-MG), protein, uric acid, calcium, and magnesium (P > .05).

Conclusion: Our findings highlighted tubular nephropathy induced by Deferasirox in patients with beta thalassemia, and confirmed the necessity for diligent monitoring of renal function in thalassemia patients receiving Deferasirox.