VALIDATING A CORE OUTCOME MEASURE FOR PAIN IN PATIENTS WITH AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE.
ROSANNA CAZZOLLI1,2, ANGELA JU1,2, PATRIZIA NATALE2,3,4, EVA BURNETTE5, NIEK F. CASTELEIJN6, ARLENE CHAPMAN7, SARAH EASTTY5, RON T. GANSEVOORT8, TESS HARRIS9, MARIE HOGAN10, SHIGEO HORIE11, BERTRAND KNEBELMANN12, RICHARD LEE13, REEM A. MUSTAFA14, RONALD D. PERRONE15, RICHARD SANDFORD16, ARMANDO TEIXEIRA-PINTO1,2, ALLISON JAURE1,2, YEOUNGJEE CHO17,18,19
1Centre for Kidney Research, The Children’s Hospital at Westmead, Westmead, New South Wales, Australia
2Sydney School of Public Health, The University of Sydney, Sydney, New South Wales, Australia
3Department of Precision and Regenerative Medicine and Ionian Area (DIMEPRE-J), University of Bari, Aldo Moro, Bari, Italy
4Nephrology, Dialysis and Transplantation Unit, Department of Medical and Surgical Sciences, University of Foggia, Foggia, Italy
5United Kingdom
6Department of Urology, University Medical Centre Groningen, University of Groningen, The Netherlands
7Department of Nephrology, The University of Chicago, United States
8Department of Nephrology, University Medical Centre Groningen, University of Groningen, The Netherlands
9Polycystic Kidney Disease International, London, United Kingdom
10Division of Nephrology & Hypertension, Department of Internal Medicine Mayo Clinic, United States
11Department of Urology and Department of Advanced Informatics for Genetic Diseases, Juntendo University Graduate School of Medicine, Japan
12 Université de Paris; APHP; Hôpital Universitaire Necker, Service de Nephrologie, Paris, France
13Australia,
14Department of Internal Medicine, Division of Nephrology and Hypertension, University of Kansas Medical Centre, United States
15Medicine, Nephrology, Clinical and Translational Research Center, Tufts Medical Center and Tufts University School of Medicine, United States
16Academic Department of Medical Genetics, University of Cambridge, United Kingdom
17Australasian Kidney Trials Network, University of Queensland, Brisbane, Queensland, Australia
Abstract
Background: Pain is a common symptom affecting more than 60% of adults with autosomal dominant polycystic kidney disease (ADPKD). Although pain has been identified as a critically important outcome in people with ADPKD, it is infrequently and inconsistently reported in clinical trials. The Standardized Outcomes in Nephrology-PKD (SONG-PKD) Pain measure was developed based on a systematic review of pain measures used in trials involving patients with ADPKD and with the involvement of patients, caregivers and health professionals.
Aim: To provide preliminary evidence for the suitability of the SONG-PKD Pain measure to be used in all clinical trials involving patients with ADPKD.
Methods: Questionnaires were administered online from August 2022 to November 2022. At baseline, participants completed a demographic questionnaire, brief medical history, the SONG-PKD Pain measure and the short form brief pain inventory (BPI-SF). The pain measures were re-administered after 2 days to assess test-retest reliability. Internal consistency was evaluated by calculating Cronbach’s alpha. Test-retest reliability and convergent validity with the BPI-SF were assessed using Spearman’s Rho.
Results: 316 adult ADPKD patients from 21 countries participated. The SONG-PKD Pain measure demonstrated high internal consistency (Cronbach’s alpha = 0.94, 95% confidence intervals, 0.93 to 0.95) and test-retest reliability (Spearman’s rho = 0.92, 95%CI 0.90 to 0.94). There was high correlation (Spearman’s rho = 0.84, 95%CI 0.80 to 0.87) with the BPI-SF.
Conclusion: The SONG-PKD Pain instrument is a simple measure that has demonstrated high internal consistency and test-retest reliability in patients with ADPKD. A standardised measure of pain to be reported for all ADPKD trials will make it easier to compare the effect of interventions for pain across trials and improve patient treatment outcomes.