Weekly issue 2026-W43 ·
Empagliflozin Therapy in Autosomal Dominant Polycystic Kidney Disease: A Feasibility Randomized Controlled Trial
Plain language
SGLT2 inhibitors such as empagliflozin protect kidney function in many forms of chronic kidney disease, but people with autosomal dominant polycystic kidney disease (ADPKD) were excluded from the large landmark trials. In this 12-month pilot randomized trial (NCT05510115), 50 adults aged 18–55 with high-risk ADPKD who were not taking tolvaptan received either empagliflozin (25 mg daily) or placebo. The medicine was generally well tolerated, with 96% of participants in each group tolerating the full dose. However, total kidney volume grew faster in the empagliflozin group than in the placebo group (10.5% vs 5.1% per year), while after an expected initial dip at 1 month, the rate of change in kidney function (eGFR) from month 1 to month 12 did not differ significantly between groups. This small pilot trial shows why SGLT2 inhibitors should not be assumed to slow cyst growth in ADPKD.
Clinical note
Pilot and feasibility randomized, placebo-controlled trial (J Am Soc Nephrol; NCT05510115) in n = 50 adults aged 18–55 years with ADPKD at high risk of rapid progression, not receiving tolvaptan, randomized 1:1 to empagliflozin 25 mg/day vs placebo for 12 months (mean age 42 ± 7 years, baseline eGFR 70 ± 17 mL/min/1.73 m²). Primary outcomes (safety/tolerability): 96% in each arm tolerated the full dose; adverse event rates were similar (UTI in 2 empagliflozin vs 0 placebo; AKI in 1 placebo). Secondary outcomes: height-adjusted TKV growth was significantly greater with empagliflozin than placebo (10.5%/year [95% CI 8.2 to 12.7] vs 5.1%/year [3.0 to 7.3], p = 0.001). Empagliflozin caused an acute 1-month eGFR dip (-6.2 vs +0.1 mL/min/1.73 m², p = 0.01), with no significant difference in chronic 1-to-12-month eGFR slope (-0.77 ± 1.44 vs -2.87 ± 1.96 mL/min/1.73 m²/year, p = 0.4) or in aortic pulse wave velocity, plasma copeptin, urine KIM-1, or ADPKD Impact Score.
Question for your next visit
What does recent research show about SGLT2 inhibitors in ADPKD, and which kidney-protective treatments are proven for my situation?