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

IntroductionDue 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 MethodsTwelve 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.ResultsApart 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.ConclusionsOur 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.

FINAL HEIGHT AND ENDORINE COMPLICATIONS IN PATIENTS WITH Β-Thalasssemia INTERMEDIA: OUR EXPERIENCE IN NON-TRANSFUSED VERSUS INFREQUENTLY TRANSFUSED PATIENTS AND CORRELATIONS WITH LIVER IRON CONTENT

“…The uncomfortable side effects of therapy can have a negative impact on daily activities and well-being, which may affect adherence 45. A new tablet DFX formulation (Jadenu ® ) has been recently developed in an attempt to overcome tolerability issues and to improve the patients’ compliance 46…”

COVID‐19 in a pregnant patient with beta‐thalassemia major: A case report

“…The currently approved chelators are desferrioxamine (DFO), deferasirox (DFX), and deferiprone (DFP). 21,22 However, the safety profile for these agents is not well studied in pregnancy, and the usual recommendation is to hold them during pregnancy. Since holding chelating therapy for the duration of pregnancy may have important consequences on women, some researchers prefer to use DFO in the second and third trimesters as it is a large molecule and less likely to cross the placenta.…”

“…Iron chelating agents aim to excrete the accumulating iron through feces and/or urine. The currently approved chelators are desferrioxamine (DFO), deferasirox (DFX), and deferiprone (DFP) 21,22 . However, the safety profile for these agents is not well studied in pregnancy, and the usual recommendation is to hold them during pregnancy.…”

Recurrent spinal cord compression due to extramedullary hematopoiesis in thalassemia patient

“…[1] With increasing age, even patients with mild forms may develop complications from iron deposition in tissues, [2] with many subsequent health problems such as growth retardation, [3] fertility issues, [4] and hypoadrenalism, [5] which require chelation therapy. [6] Less frequently, patients may also develop complications secondary to massive erythroid expansion (known as extramedullary hematopoiesis "EMH"). [7] EMH is defined as a physiological compensatory production of red blood cells in tissues other than bone marrow secondary to chronic anemia.…”