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

Abstract

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 ^® is a new oral formulation of deferasirox (Exjade ^®) tablets for oral suspension. While Exjade ^® is a dispersible tablet that must be mixed in liquid and taken on an empty stomach, Jadenu ^® can 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 with β-thalassemia major (BMT). The aim of this study was to evaluate 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 red cell transfusion therapy to keep their pre-transfusional hemoglobin (Hb) level not less than 9 g/dL. They were on Exjade ^® therapy (30 mg/kg per day) for two years or more before starting Jadenu® therapy (14–28 mg/kg/day). The reason for shifting from Deferasirox ^® to Jadenu ^® therapy was lack of tolerability, as described by patients, such as nausea, vomiting, diarrhea, stomach pain. Most of them also reported that Deferasirox ^® was not palatable. Lab investigations included monthly 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 ^®. Thyroid function, vitamin D and serum parathormone, before and one year after starting Jadenu ^® 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 Jadenu ^® compared to Exjade ^® therapy in 8 out of 12 BMT patients. A non-significant decrease in LIC measured by FerriScan ^® and serum ferritin levels was observed after one year of treatment with Jadenu ^®. A significant positive correlation was found between serum ferritin level and LIC measured by the FerriScan ^® method. LIC and serum ferritin level correlated significantly with ALT level (r = 0.31 and 0.45 respectively, p < 0.05). No significant correlation was detected between LIC and other biochemical or hormonal parameters.

Conclusions

Our study shows that short-term treatment with Jadenu ^® 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 longterm outcomes of new deferasirox formulation.