MON-329 RAPID-ADPKD (RETROSPECTIVE EPIDEMIOLOGIC STUDY OF ASIAN-PACIFIC PATIENTS WITH RAPID DISEASE PROGRESSION OF AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE): DESIGN AND METHODS

Abstract

Autosomal dominant polycystic kidney disease (ADPKD) patients reach end-stage renal disease (ESRD) in the 5th decade on average. For effective treatment and early intervention, identifying subgroups with rapid progression are important in ADPKD. However, there are no epidemiologic data regarding the clinical manifestations and disease progression of ADPKD patients from the Asia-pacific region. The RAPID-ADPKD is a multinational retrospective observational cohort study of ADPKD patients in the Asia-pacific area. This study was designed to identify the clinical characteristics of ADPKD patients with rapid progression, based on the change of renal function, kidney size and progression to ESRD. Seven hospitals from six regions (Australia, China, Hong Kong, South Korea, Taipei and Turkey) are participating in this study. ADPKD patients ≥18 years old, diagnosed by the unified ultrasound criteria and with estimated glomerular filtration rate (eGFR) ≥45 mL/min/1.73m2 at baseline will be included. The cohort will include patients with more than two records of eGFR, at least 24 months follow up data. Patients with severe heart failure, severe liver disease and/or other comorbidities that can effect renal function will be excluded. Demographic information, clinical characteristics, premorbid comorbidities, medications, eGFR, radiologic findings that can calculate height adjusted total kidney volume (htTKV), PKD-related complications and the PRO-PKD score will be collected using an electrical case report form. Rapid progression will defined as when any of following criteria are met: (i) an annual eGFR decline ≥5 mL/min/1.73m2 in 1 year and/or ≥2.5mL/min/1.73m2 per year over a period of 5 years; (ii) an increase in htTKV ≥ 5% per year from ≥3 radiologic images; (iii) Mayo classification 1C, 1D, or 1E or kidney length from ultrasonography of >16.5 cm’ (iv) PKD1 truncated mutation with early symptoms (PRO-PKD score >6). All other patients without any of the criteria are classified as slow progression. The clinical characteristics will be compared between patients with rapid progression and slow progression. In addition, the incidence rate, age of diagnosis, treatment complications between patients with rapid and slow progression will be analyzed. The clinical characteristic differences according to age groups will be analyzed as secondary analysis. The planned sample size of the cohort is 2,000 patients, and as October 8th 2018, data from 280 patients have been collected. RAPID-ADPKD is the first large-scale multinational retrospective observational study of ADPKD in Asia-Pacific region and will identify the clinical characteristics, risk factors for disease progression and patterns of complications in Asian populations with ADPKD.