• Anthea Tsatsaronis, Mark Tiong, Kathleen Nicholls, and Irene Ruderman.
• Department of Nephrology, The Royal Melbourne Hospital, Melbourne, Victoria, Australia.
• Intern Med J. 2024 Dec 24.

BackgroundFabry disease is a rare X-linked multisystem disease, with progressive proteinuric kidney disease contributing significantly to morbidity and mortality of these patients. Evidence shows that sodium-glucose cotransporter 2 inhibitors (SGLT2Is) can reduce proteinuria and slow progression to end-stage kidney disease in both diabetic and non-diabetic kidney disease.AimEvaluate the effects of SGLT2I on kidney function and albuminuria in patients with Fabry disease.MethodsSingle-centre real-world case series reviewing electronic medical records of patients with Fabry disease who initiated therapy with dapagliflozin or empagliflozin (n = 11). Changes in urine albumin-creatinine ratio (uACR) and creatinine before and after treatment with SGLT2I were analysed using Wilcoxon signed-rank test. Two-tailed P-values <0.05 were considered significant.ResultsEleven patients were followed for up to 19 months after commencement of SGLT2I. An overall significant reduction in albuminuria (P = 0.05) was seen with SGLT2I use in the Fabry cohort. Median uACR before SGLT2I was 76 mg/mmol (interquartile range (IQR) 47-141) and after SGLT2I was 39 mg/mmol (IQR 18-95) (P = 0.05). All patients with uACR >100 mg/mmol had reduction in albuminuria over the study period. SGLT2Is were well tolerated overall, with only one case resulting in cessation of treatment due to adverse effects.ConclusionThese results suggest SGLT2Is can significantly reduce albuminuria in a portion of patients with Fabry-related kidney disease and offer additional treatment for Fabry nephropathy. Given the nature of the study design and small case numbers, further long-term controlled studies are required to evaluate the long-term efficacy of this medication class in both cardiac and renal outcomes in Fabry disease.© 2024 Royal Australasian College of Physicians.

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