A 35-year-old man with autosomal dominant polycystic kidney disease has eGFR 48 mL/min. Total kidney volume is 1200 mL on MRI. He has rapidly declining eGFR (6 mL/min/year). According to the TEMPO 3:4 trial, which medication is approved to slow disease progression?
- A Sirolimus (mTOR inhibitor)
- B Octreotide (somatostatin analogue)
- C Mycophenolate mofetil
- D Tolvaptan (vasopressin V2 receptor antagonist) ✓
Explanation
Tolvaptan is the only FDA-approved therapy for ADPKD. The TEMPO 3:4 trial showed tolvaptan slows total kidney volume growth and eGFR decline in patients with rapidly progressive ADPKD (CKD stage 1-3, TKV >750 mL, rapid progression). It inhibits cyst fluid secretion by blocking vasopressin V2 receptors. mTOR inhibitors and octreotide have not shown consistent benefit in clinical trials.
Reference: Brenner and Rector's The Kidney, 11th ed.
High-yield for: NEET PGINI-CETNExTFMGEUSMLEPLABMRCP
Written and medically reviewed by the StethoPrep medical team.