Deferasirox‐associated Fanconi syndrome in adult patients with transfusional iron overload

Background and Objectives Deferasirox is an oral chelator approved for iron overload, which has a potential side effect of renal Fanconi syndrome, with proximal tubular dysfunction and tubular acidosis. Monitoring of renal function is recommended, though no standard for monitoring exists. We aim to describe cases of deferasirox‐associated Fanconi syndrome in adults and the renal monitoring required to detect these findings. Materials and Methods We present a review of the literature and six cases from our institution of deferasirox‐associated partial Fanconi syndrome in adult patients with transfusional iron overload secondary to β‐thalassemia or Diamond Blackfan Anaemia. Results While prior cases in the literature occurred at high doses of deferasirox, our series included patients on doses as low as deferasirox 10 mg/kg who had been aggressively chelated. All patients had resolution of laboratory abnormalities with drug interruption. Conclusion Rather than chelating to normal iron levels, this series supports prior suggestions that deferasirox dose be reduced if ferritin <500–1000 ng/ml, and also supports dose reduction if liver iron content <3 mg iron per g dry weight or for those undergoing aggressive chelation with rapid decrease in iron. Monitoring with metabolic panel and urinalysis were sufficient to detect clinically significant proximal tubular dysfunction, but should be followed up with additional studies to confirm the diagnosis while deferasirox dose is decreased or held.