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Reversible Fanconi syndrome in a pediatric patient on deferasirox
Michelle N Rheault1, Heather Bechtel, Joseph P Neglia
1Department of Pediatrics, University of Minnesota Amplatz Children's Hospital, Minneapolis, Minnesota 55455, USA. rheau002@umn.edu
Insights
Deferasirox can cause kidney problems, including reversible Fanconi syndrome, in patients with iron overload. Regular monitoring of kidney and proximal tubule function is recommended during deferasirox therapy.
Area of Science:
- Nephrology
- Pharmacology
- Pediatric Hematology
Background:
- Deferasirox is an oral iron chelator used for transfusional iron overload.
- Common side effects include increased serum creatinine, but proximal tubule effects are less understood.
Observation:
- A pediatric case of reversible Fanconi syndrome linked to long-term deferasirox use.
- Another patient experienced mild proximal tubular dysfunction.
Findings:
- Deferasirox therapy may be associated with proximal tubule dysfunction.
- Fanconi syndrome is a potential, reversible adverse effect.
Implications:
- Periodic monitoring of renal and proximal tubule function is crucial for patients on deferasirox.
- Early detection of tubular dysfunction can guide treatment adjustments.
- This highlights the importance of pharmacovigilance for iron chelators.
Abstract:
Deferasirox (Exjade®, Novartis) is a widely used oral iron chelator for the treatment of patients with iron overload due to chronic transfusion therapy for diseases such as β-thalassemia and sickle cell disease. Renal side effects of deferasirox are common and include non-progressive increases in serum creatinine, however, the effect of deferasirox on proximal tubule function is unclear. We report one pediatric patient with reversible Fanconi syndrome associated with long-term deferasirox therapy and one patient with mild proximal tubular dysfunction. Kidney and proximal tubular function should be periodically monitored in patients receiving deferasirox throughout their course of therapy.
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