SUN-370 TOLVAPTAN USE IN YOUNG ADULTS WITH RAPIDLY PROGRESSING AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE

Abstract

Regulatory approval of tolvaptan as a treatment for autosomal dominant polycystic kidney disease (ADPKD) in adults with evidence of rapidly progressing disease has changed the management of this condition. Given that young adults with ADPKD are likely to have early-stage disease and that questions on the screening, assessment, and treatment of these patients remain debated, a manuscript on tolvaptan use in the 18−24-year age group is needed. The phase 3 Tolvaptan Efficacy and Safety in Management of Autosomal Dominant Polycystic Kidney Disease and Its Outcomes (TEMPO 3:4) clinical trial evaluated tolvaptan in a large population (N=1,445) of subjects aged 18−50 years over a 3-year period. However, it did not specifically assess the use of tolvaptan in adolescents and young adults (AYAs) with ADPKD. A post hoc analysis of the TEMPO 3:4 trials was performed for patients 18-24 years old. The inclusion criteria were a diagnosis of ADPKD by Ravine criteria, and estimated creatinine clearance ≥60 mL/min (by Cockcroft-Gault) or rapidly progressive kidney growth (total volume ≥750 mL) by MRI at randomization. The primary outcome was the annual rate of change in total kidney volume (TKV). A total of 51 patients in the 18-24 group were analyzed. Out of the 51 patients, 29 were subjected to tolvaptan treatment while 22 were given placebo. The tolvaptan group had a lower mean % TKV growth per year compared to the placebo group (3.9% vs. 6.5%, p=0.0491). Tolvaptan, with appropriate patient selection and management, can provide effective and acceptably safe treatment in AYAs with ADPKD.