Evaluating Chronic Pain in Autosomal Dominant Polycystic Kidney Disease using a Patient-Centred Approach to Data Collection and Synthesis: A National Prospective Observational Study (EASE-PKD)

RESEARCH QUESTION What is the extent and characteristics of ADPKD pain? BACKGROUND Autosomal Dominant Polycystic Kidney Disease (ADPKD) affects 60000 people in the UK and is characterised by the emergence and expansion of numerous kidney cysts. Progression results in enlarged painful kidneys, hypertension and kidney failure. Chronic pain affects >60% of patients and is associated with reduced quality of life and a considerable healthcare and economic burden. Despite the scale of the problem, there has been little research into ADPKD pain and its treatment. The mechanisms are inadequately understood, and the observation that pain is not closely correlated with kidney size and may have an onset during the teenage years before kidney enlargement occurs indicates that ADPKD pain is unlikely to be purely mechanical. Whilst there is a widely recognised need for interventional trials for APDKD pain, rational trial design and judicious selection of candidate interventions requires comprehensive characterisation of ADPKD pain. EASE-PKD was initiated by the PKD Charity in partnership with the Patient Led Research Hub and will harness a bespoke patient facing data system to allow detailed phenotypic characterisation and assessment of ADPKD pain using a standardised ADPKD Pain Assessment Tool (APAT). Through linkage with routinely collected data sources, EASE-PKD will minimise the requirement for and cost of manual data entry by utilising direct and contemporaneous participant data entry through web- or smartphone-based systems to enter pain data. AIMS AND OBJECTIVES EASE-PKD will catalogue the nature, patterns and characteristics of pain in ADPKD in a patient-led, co-produced detailed prospective observational study. Primary Objective To describe the features and patterns of pain in ADPKD Secondary Objectives To determine phenotypic associations with ADPKD pain To determine the impact of pain on health-related quality of life To determine the acceptability and utility of the APAT To evaluate healthcare resource use attributed to ADPKD pain management To build a robust patient-facing database for patients and healthcare professionals METHODS EASE-PKD is a patient-led, multi-centre, prospective, longitudinal observational cohort study of chronic pain in ADPKD. We will enroll 600 ADPKD patients aged 16 years older over an 18-month period, by (online) self-registration, direct contact with the local research/medical team, or enrolment by their physician. Patients will be invited through PatientView or the Registry of Rare Kidney Diseases (RaDaR). Participants will complete a detailed pain assessment questionnaire incorporating components of validated pain assessment tools at baseline and 3-monthly for 12 months. TIMELINES Recruitment will commence in April 2020 and continue for 18 months. The final participant follow-up is expected in October 2022. A final report is anticipated in March 2023. IMPACT AND DISSEMINATION EASE-PKD will provide a detailed assessment of ADPKD pain, generate mechanistic insights, and facilitate delivery of interventional ADPKD pain studies. It will create a legacy of an interactive patient portal allowing personalised symptom tracking and permitting the conduct of user-led streamlined interventional trials. Study findings will be disseminated though conventional academic publication and directly presented via the EASE-PKD portal and the PKD Charity website and social media groups.