FBS0701: un nuovo chelante orale nel trattamento del paziente con sovraccarico trasfusionale di ferro

Background There is still a clinical need for a well-tolerated and safe iron chelator for the treatment of transfusional iron overload. The objective of this study was to assess the safety, tolerability and dose response of FBS0701, a novel oral iron chelator. Design and Methods This phase 2 study to assess the safety, tolerability, dose response of FBS0701, was conducted in 14 adult patients with iron overload consequent to transfusions.Two once-daily doses of 16 or 32 mg/Kg per day were randomly assigned to the study partecipants for 24 weeks. After then, higher doses up to 60 mg/Kg per day, were experimented in an extension phase for 72 weeks. Results There were no serious adverse events associated with FBS0701 and the drug was well tolerated at all dose levels. The difference in the mean LIC change at 24 weeks between treatment groups was statistically significant. Data concen the efficacy of higher doses experiemented in the second phase, cannot be well interpreted, because the different treatment duration and subjective patients response. Conclusions FBS0701 was well tolerated for 96 weeks in iron overloaded patients.A clear dose-response was observed for the first 24 week. The efficacy and safety of the drug should be investigated with a more extensive study.