JADENU? SUBSTITUTING EXJADE? IN IRON OVERLOADED Β- THALASSEMIA MAJOR (BTM) PATIENTS: A PRELIMINARY REPORT OF THE EFFECTS ON THE TOLERABILITY, SERUM FERRITIN LEVEL, LIVER IRON CONCENTRATION AND BIOCHEMICAL PROFILES

摘要

. Introduction : Due to the chronic nature of chelation therapy and the adverse consequences of iron overload, patient adherence to therapy is an important issue. Jadenu sup? /supis a new oral formulation of deferasirox (Exjade sup?/sup) tablets for oral suspension. While Exjadesup? /supsup?/supis a dispersible tablet that must be mixed in liquid and taken on an empty stomach, Jadenu sup? /supcan be taken in a single step, with or without a light meal, simplifying administration for the treatment of patients with chronic iron overload. This may significantly improve the compliance to treatment of patients withβ-thalasemia major (BMT). The aim of this study was to evalute the drug tolerability and the effects of chelation therapy on serum ferritin concentration, liver iron concentration (LIC) and biochemical profiles in patients with BMT and iron overload. Patients and Methods: Twelve selected adult patients BMT (mean age: 29 years; range:15-34 years) were enrolled in the study. All patients were on monthly regular packed cell transfusion therapy to keep their pre-transfusional hemoglobin (Hb) level not less than 9 g/dL. They were on Exjade sup?/sup therapy (30 mg/kg per day) for 2 years or more before starting Jadenu ? therapy (14-28 mg/kg/day). The reason for shifting from Deferasirox sup?/sup to Jadenu sup? /suptherapy was lack of tolerability, since most of the patients described Deferasirox sup?/sup as not palatable. Lab investigations included montly urine analysis and measurement of their serum concentrations of creatinine, fasting blood glucose (FBG), serum ferritin, alkaline phosphatase (ALP), alanine transferase (ALT), aspartate transferase (AST) and albumin concentrations. LIC was measured using FerriScan sup?/sup. Thyroid function, vitamin D and serum parathormone, before and one year after starting Jadenu sup?/sup therapy, were also assessed. Results: Apart from some minor gastrointestinal complaints reported in 3 BMT patients that did not require discontinuation of therapy, other side effects were not registered during the treatment. Subjectively, patients reported an improvement in the palatability of Jadenusup? /supcompared to Exjade sup?/sup therapy in 8 out of 12 BMT patients. A non-significant decrease in LIC and serum ferritin levels was observed after 1 year of treatment with Jadenu sup? /sup. A positive significant correlation was found between serum ferritin level and LIC measured by FerriScan sup?/sup method. LIC and serum ferritin level correlated significantly with ALT level (r = 0.31 and 0.45 respectively, p ?/sup is safe but is associated with a non-significant decrease in LIC and serum ferritin levels. Therefore, there is an urgent need for adequately-powered and high-quality trials to assess the clinical efficacy and the long-term outcomes of new deferasirox formulation.