FP064ASSESSING THE LONG TERM OUTCOMES OF AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE (ADPKD) USING THE ADPKD OUTCOMES MODEL: A UK CASE STUDY

Introduction and Aims: The ADPKD Outcomes Model was developed to predict renal progression to end-stage renal disease (ESRD) in ADPKD based on measurable/observable baseline patient characteristics. The objective of this study was to: • Explore the full ADPKD pathway, from baseline to death, by combining the ADPKD Outcomes Model with a country- specific ESRD module • Estimate the long-term outcomes in a population similar to that enrolled in the TEMPO 3:4 trial. Methods: The model uses patient-level data from the TEMPO 3:4 placebo group to simulate renal progression, with decline in renal function as the principal measure of progression. At the onset of ESRD, modelled patients enter an ESRD module adapted from existing UK NICE guidance to include management prior to renal replacement therapy (RRT) initiation and conservative care. Mortality risk pre-ESRD was conservatively assumed to match the UK population, with ESRD mortality based on UK data for patients receiving RRT. The model was initialised with a patient cohort mimicking the baseline profile from the TEMPO 3:4 trial: age 38.7±7.1 years, female 48.4%, eGFR 81.61±21.6 ml/min/1.73m2, TKV 1692±905 ml. TEMPO 3:4 targeted ADPKD patients with early disease projected to have rapid cyst growth and accelerated outcomes, i.e. at increased risk of rapid progression. Accordingly, a range of results is presented based on increasing/decreasing baseline eGFR and TKV by one standard deviation. Uncertainty was tested using probabilistic sensitivity analysis.