Tom Nijenhuis, theme Renal disorders, received this grant for his research project “Salt supplementation in Gitelman syndrome: A simple intervention using an innovative N-of-1 trial approach”.In patients with this rare kidney disease characterized by urinary salt wasting, the effect of pharmacological and supraphysiological sodiumchloride supplementation on the individual patient’s symptoms, quality of life and electrolyte imbalances (particularly hypokalemia) will be determined. N-of-1 trials are single subject randomized crossover trials performed within an individual patient. This approach enables the use of personalized outcome parameters and requires much lower patient numbers than a randomized controlled trial, while individual N-of-1 trials can be aggregated to allow conclusions at group level. This personalized approach will also enable clinical application of the results in the individual patient directly after the result of their N-of-1 trial is available. The project eventually aims to optimize personalized treatment of the individual patient with Gitelman syndrome and possibly other rare renal tubular salt-losing disorders.
Tom Nijenhuis, internist-nephrologist, coordinates the Radboudumc/ErasmusMC national referral Center for Tubular Disorders, embedded in the Radboudumc Expertise Center for Rare Kidney Disorders, a tertiary referral center for renal tubular disorders including Gitelman syndrome.
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