TY - JOUR
T1 - Establishing a Core Outcome Set for Autosomal Dominant Polycystic Kidney Disease
T2 - Report of the Standardized Outcomes in Nephrology–Polycystic Kidney Disease (SONG-PKD) Consensus Workshop
AU - SONG-PKD Workshop Investigators
AU - Cho, Yeoungjee
AU - Tong, Allison
AU - Craig, Jonathan C.
AU - Mustafa, Reem A.
AU - Chapman, Arlene
AU - Perrone, Ronald D.
AU - Ahn, Curie
AU - Fowler, Kevin
AU - Torres, Vicente
AU - Gansevoort, Ron T.
AU - Ong, Albert C.M.
AU - Coolican, Helen
AU - Tze-Wah Kao, Juliana
AU - Harris, Tess
AU - Gutman, Talia
AU - Shen, Jenny I.
AU - Viecelli, Andrea K.
AU - Johnson, David W.
AU - Au, Eric
AU - El-Damanawi, Ragada
AU - Logeman, Charlotte
AU - Ju, Angela
AU - Manera, Karine E.
AU - Chonchol, Michel
AU - Odland, Dwight
AU - Baron, David
AU - Pei, York
AU - Sautenet, Benedicte
AU - Rastogi, Anjay
AU - Sharma, Ankit
AU - Rangan, Gopala
AU - Levin, Adeera
AU - Yu, Alan
AU - Thompson, Aliza
AU - Baumgart, Amanda
AU - Bernier-Jean, Amelie
AU - Kelly, Amy
AU - Mallett, Andrew
AU - Wang, Angela
AU - Rastog, Anjay
AU - Nadeau-Fredette, Annie Claire
AU - Teixeira-Pinto, Armando
AU - Kelly, Ayano
AU - Gillespie, Barbara
AU - Canaud, Bernard
AU - Manns, Braden
AU - Hemmelgarn, Brenda
AU - Hanson, Camilla
AU - Hogan, Marie
AU - Harris, Peter
N1 - Publisher Copyright:
© 2020 National Kidney Foundation, Inc.
PY - 2021/2
Y1 - 2021/2
N2 - The omission of outcomes that are of relevance to patients, clinicians, and regulators across trials in autosomal dominant polycystic kidney disease (ADPKD) limits shared decision making. The Standardized Outcomes in Nephrology–Polycystic Kidney Disease (SONG-PKD) Initiative convened an international consensus workshop on October 25, 2018, to discuss the identification and implementation of a potential core outcome set for all ADPKD trials. This article summarizes the discussion from the workshops and the SONG-PKD core outcome set. Key stakeholders including 11 patients/caregivers and 47 health professionals (nephrologists, policy makers, industry, and researchers) attended the workshop. Four themes emerged: “Relevance of trajectory and impact of kidney function” included concerns about a patient's prognosis and uncertainty of when they may need to commence kidney replacement therapy and the lack of an early prognostic marker to inform long-term decisions; “Discerning and defining pain specific to ADPKD” highlighted the challenges in determining the origin of pain, adapting to the chronicity and repeated episodes of pain, the need to place emphasis on pain management, and to have a validated measure for pain; “Highlighting ADPKD consequences” encompassed cyst-related complications and reflected patient's knowledge because of family history and the hereditary nature of ADPKD; and “Risk for life-threatening but rare consequences” such as cerebral aneurysm meant considering both frequency and severity of the outcome. Kidney function, mortality, cardiovascular disease, and pain were established as the core outcomes for ADPKD.
AB - The omission of outcomes that are of relevance to patients, clinicians, and regulators across trials in autosomal dominant polycystic kidney disease (ADPKD) limits shared decision making. The Standardized Outcomes in Nephrology–Polycystic Kidney Disease (SONG-PKD) Initiative convened an international consensus workshop on October 25, 2018, to discuss the identification and implementation of a potential core outcome set for all ADPKD trials. This article summarizes the discussion from the workshops and the SONG-PKD core outcome set. Key stakeholders including 11 patients/caregivers and 47 health professionals (nephrologists, policy makers, industry, and researchers) attended the workshop. Four themes emerged: “Relevance of trajectory and impact of kidney function” included concerns about a patient's prognosis and uncertainty of when they may need to commence kidney replacement therapy and the lack of an early prognostic marker to inform long-term decisions; “Discerning and defining pain specific to ADPKD” highlighted the challenges in determining the origin of pain, adapting to the chronicity and repeated episodes of pain, the need to place emphasis on pain management, and to have a validated measure for pain; “Highlighting ADPKD consequences” encompassed cyst-related complications and reflected patient's knowledge because of family history and the hereditary nature of ADPKD; and “Risk for life-threatening but rare consequences” such as cerebral aneurysm meant considering both frequency and severity of the outcome. Kidney function, mortality, cardiovascular disease, and pain were established as the core outcomes for ADPKD.
KW - Polycystic kidney disease (PKD)
KW - cardiovascular disease
KW - consensus conference
KW - core outcome set
KW - kidney function
KW - mortality
KW - outcomes trials
KW - pain
KW - patient-important outcomes
KW - patient/caregiver perspective
KW - research priorities
KW - standardized outcomes
KW - study design
KW - thematic synthesis
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U2 - 10.1053/j.ajkd.2020.05.024
DO - 10.1053/j.ajkd.2020.05.024
M3 - Article
C2 - 32771648
AN - SCOPUS:85091862574
SN - 0272-6386
VL - 77
SP - 255
EP - 263
JO - American Journal of Kidney Diseases
JF - American Journal of Kidney Diseases
IS - 2
ER -