Publication

Range and Variability of Outcomes Reported in Randomized Trials Conducted in Patients With Polycystic Kidney Disease: A Systematic Review

SONG-PKD Initiative, Sautenet, B., Cho, Y., Gutman, T., Rangan, G., Ong, A., Chapman, A. B., Ahn, C., Coolican, H., Tze-Wah Kao, J., Fowler, K., Gansevoort, R. T., Geneste, C., Perrone, R. D., Harris, T., Torres, V. E., Pei, Y., Craig, J. C. & Tong, A., Aug-2020, In : American Journal of Kidney Diseases. 76, 2, p. 213-223 11 p.

Research output: Contribution to journalReview articleAcademicpeer-review

APA

SONG-PKD Initiative, Sautenet, B., Cho, Y., Gutman, T., Rangan, G., Ong, A., Chapman, A. B., Ahn, C., Coolican, H., Tze-Wah Kao, J., Fowler, K., Gansevoort, R. T., Geneste, C., Perrone, R. D., Harris, T., Torres, V. E., Pei, Y., Craig, J. C., & Tong, A. (2020). Range and Variability of Outcomes Reported in Randomized Trials Conducted in Patients With Polycystic Kidney Disease: A Systematic Review. American Journal of Kidney Diseases, 76(2), 213-223. https://doi.org/10.1053/j.ajkd.2019.12.003

Author

SONG-PKD Initiative ; Sautenet, Bénédicte ; Cho, Yeoungjee ; Gutman, Talia ; Rangan, Gopala ; Ong, Albert ; Chapman, Arlene B ; Ahn, Curie ; Coolican, Helen ; Tze-Wah Kao, Juliana ; Fowler, Kevin ; Gansevoort, Ron T ; Geneste, Claire ; Perrone, Ronald D ; Harris, Tess ; Torres, Vicente E ; Pei, York ; Craig, Jonathan C ; Tong, Allison. / Range and Variability of Outcomes Reported in Randomized Trials Conducted in Patients With Polycystic Kidney Disease : A Systematic Review. In: American Journal of Kidney Diseases. 2020 ; Vol. 76, No. 2. pp. 213-223.

Harvard

SONG-PKD Initiative, Sautenet, B, Cho, Y, Gutman, T, Rangan, G, Ong, A, Chapman, AB, Ahn, C, Coolican, H, Tze-Wah Kao, J, Fowler, K, Gansevoort, RT, Geneste, C, Perrone, RD, Harris, T, Torres, VE, Pei, Y, Craig, JC & Tong, A 2020, 'Range and Variability of Outcomes Reported in Randomized Trials Conducted in Patients With Polycystic Kidney Disease: A Systematic Review', American Journal of Kidney Diseases, vol. 76, no. 2, pp. 213-223. https://doi.org/10.1053/j.ajkd.2019.12.003

Standard

Range and Variability of Outcomes Reported in Randomized Trials Conducted in Patients With Polycystic Kidney Disease : A Systematic Review. / SONG-PKD Initiative; Sautenet, Bénédicte; Cho, Yeoungjee; Gutman, Talia; Rangan, Gopala; Ong, Albert; Chapman, Arlene B; Ahn, Curie; Coolican, Helen; Tze-Wah Kao, Juliana; Fowler, Kevin; Gansevoort, Ron T; Geneste, Claire; Perrone, Ronald D; Harris, Tess; Torres, Vicente E; Pei, York; Craig, Jonathan C; Tong, Allison.

In: American Journal of Kidney Diseases, Vol. 76, No. 2, 08.2020, p. 213-223.

Research output: Contribution to journalReview articleAcademicpeer-review

Vancouver

SONG-PKD Initiative, Sautenet B, Cho Y, Gutman T, Rangan G, Ong A et al. Range and Variability of Outcomes Reported in Randomized Trials Conducted in Patients With Polycystic Kidney Disease: A Systematic Review. American Journal of Kidney Diseases. 2020 Aug;76(2):213-223. https://doi.org/10.1053/j.ajkd.2019.12.003


BibTeX

@article{a6dcc131c9f140a8ae4f1dde4830c96c,
title = "Range and Variability of Outcomes Reported in Randomized Trials Conducted in Patients With Polycystic Kidney Disease: A Systematic Review",
abstract = "Rationale & Objective: Trials in autosomal dominant polycystic kidney disease (ADPKD) have increased, but their impact on decision making has been limited. Because heterogeneity in reported outcomes may be responsible, we assessed their range and variability in ADPKD trials.Study Design: Systematic review.Setting & Study Population: Adult participants in clinical trials in ADPKD.Selection Criteria for Studies: We included trials that studied adults and were published in English. For trials that enrolled patients without ADPKD, only those enrolling >= 50% of participants with ADPKD were included.Data Extraction: We extracted information on all discrete outcome measures, grouped them into 97 domains, and classified them into clinical, surrogate, and patient-reported categories. For each category, we choose the 3 most frequently reported domains and performed a detailed analysis of outcome measures.Analytical Approach: Frequencies and characteristics of outcome measures were described.Results: Among 68 trials, 1,413 different outcome measures were reported. 97 domains were identified; 41 (42%) were surrogate, 30 (31%) were clinical, and 26 (27%) were patient reported. The 3 most frequently reported domains were in the surrogate category: kidney function (54; 79% of trials; using 46 measures), kidney and cyst volumes (43; 63% of trials; 52 measures), and blood pressure (27; 40% of trials, 30 measures); in the clinical category: infection (10; 15%; 21 measures), cardiovascular events (9; 13%; 6 measures), and kidney failure requiring kidney replacement therapy (8; 12%; 5 measures); and in the patient-reported category: pain related to ADPKD (16; 24%; 26 measures), pain for other reasons (11; 16%; 11 measures), and diarrhea/constipation/gas (10; 15%; 9 measures).Limitations: Outcome measures were assessed for only the top 3 domains in each category.Conclusions: The outcomes in ADPKD trials are broad in scope and highly variable. Surrogate outcomes were most frequently reported. Patient-reported outcomes were uncommon. A consensus-based set of core outcomes meaningful to patients and clinicians is needed for future ADPKD trials.",
keywords = "QUALITY-OF-LIFE, CLINICAL-TRIALS, PROGRESSION, DECLINE, VOLUME, CKD",
author = "{SONG-PKD Initiative} and B{\'e}n{\'e}dicte Sautenet and Yeoungjee Cho and Talia Gutman and Gopala Rangan and Albert Ong and Chapman, {Arlene B} and Curie Ahn and Helen Coolican and {Tze-Wah Kao}, Juliana and Kevin Fowler and Gansevoort, {Ron T} and Claire Geneste and Perrone, {Ronald D} and Tess Harris and Torres, {Vicente E} and York Pei and Craig, {Jonathan C} and Allison Tong",
note = "Copyright {\textcopyright} 2019 National Kidney Foundation, Inc. Published by Elsevier Inc. All rights reserved.",
year = "2020",
month = aug,
doi = "10.1053/j.ajkd.2019.12.003",
language = "English",
volume = "76",
pages = "213--223",
journal = "American Journal of Kidney Diseases",
issn = "0272-6386",
publisher = "W B SAUNDERS CO-ELSEVIER INC",
number = "2",

}

RIS

TY - JOUR

T1 - Range and Variability of Outcomes Reported in Randomized Trials Conducted in Patients With Polycystic Kidney Disease

T2 - A Systematic Review

AU - SONG-PKD Initiative

AU - Sautenet, Bénédicte

AU - Cho, Yeoungjee

AU - Gutman, Talia

AU - Rangan, Gopala

AU - Ong, Albert

AU - Chapman, Arlene B

AU - Ahn, Curie

AU - Coolican, Helen

AU - Tze-Wah Kao, Juliana

AU - Fowler, Kevin

AU - Gansevoort, Ron T

AU - Geneste, Claire

AU - Perrone, Ronald D

AU - Harris, Tess

AU - Torres, Vicente E

AU - Pei, York

AU - Craig, Jonathan C

AU - Tong, Allison

N1 - Copyright © 2019 National Kidney Foundation, Inc. Published by Elsevier Inc. All rights reserved.

PY - 2020/8

Y1 - 2020/8

N2 - Rationale & Objective: Trials in autosomal dominant polycystic kidney disease (ADPKD) have increased, but their impact on decision making has been limited. Because heterogeneity in reported outcomes may be responsible, we assessed their range and variability in ADPKD trials.Study Design: Systematic review.Setting & Study Population: Adult participants in clinical trials in ADPKD.Selection Criteria for Studies: We included trials that studied adults and were published in English. For trials that enrolled patients without ADPKD, only those enrolling >= 50% of participants with ADPKD were included.Data Extraction: We extracted information on all discrete outcome measures, grouped them into 97 domains, and classified them into clinical, surrogate, and patient-reported categories. For each category, we choose the 3 most frequently reported domains and performed a detailed analysis of outcome measures.Analytical Approach: Frequencies and characteristics of outcome measures were described.Results: Among 68 trials, 1,413 different outcome measures were reported. 97 domains were identified; 41 (42%) were surrogate, 30 (31%) were clinical, and 26 (27%) were patient reported. The 3 most frequently reported domains were in the surrogate category: kidney function (54; 79% of trials; using 46 measures), kidney and cyst volumes (43; 63% of trials; 52 measures), and blood pressure (27; 40% of trials, 30 measures); in the clinical category: infection (10; 15%; 21 measures), cardiovascular events (9; 13%; 6 measures), and kidney failure requiring kidney replacement therapy (8; 12%; 5 measures); and in the patient-reported category: pain related to ADPKD (16; 24%; 26 measures), pain for other reasons (11; 16%; 11 measures), and diarrhea/constipation/gas (10; 15%; 9 measures).Limitations: Outcome measures were assessed for only the top 3 domains in each category.Conclusions: The outcomes in ADPKD trials are broad in scope and highly variable. Surrogate outcomes were most frequently reported. Patient-reported outcomes were uncommon. A consensus-based set of core outcomes meaningful to patients and clinicians is needed for future ADPKD trials.

AB - Rationale & Objective: Trials in autosomal dominant polycystic kidney disease (ADPKD) have increased, but their impact on decision making has been limited. Because heterogeneity in reported outcomes may be responsible, we assessed their range and variability in ADPKD trials.Study Design: Systematic review.Setting & Study Population: Adult participants in clinical trials in ADPKD.Selection Criteria for Studies: We included trials that studied adults and were published in English. For trials that enrolled patients without ADPKD, only those enrolling >= 50% of participants with ADPKD were included.Data Extraction: We extracted information on all discrete outcome measures, grouped them into 97 domains, and classified them into clinical, surrogate, and patient-reported categories. For each category, we choose the 3 most frequently reported domains and performed a detailed analysis of outcome measures.Analytical Approach: Frequencies and characteristics of outcome measures were described.Results: Among 68 trials, 1,413 different outcome measures were reported. 97 domains were identified; 41 (42%) were surrogate, 30 (31%) were clinical, and 26 (27%) were patient reported. The 3 most frequently reported domains were in the surrogate category: kidney function (54; 79% of trials; using 46 measures), kidney and cyst volumes (43; 63% of trials; 52 measures), and blood pressure (27; 40% of trials, 30 measures); in the clinical category: infection (10; 15%; 21 measures), cardiovascular events (9; 13%; 6 measures), and kidney failure requiring kidney replacement therapy (8; 12%; 5 measures); and in the patient-reported category: pain related to ADPKD (16; 24%; 26 measures), pain for other reasons (11; 16%; 11 measures), and diarrhea/constipation/gas (10; 15%; 9 measures).Limitations: Outcome measures were assessed for only the top 3 domains in each category.Conclusions: The outcomes in ADPKD trials are broad in scope and highly variable. Surrogate outcomes were most frequently reported. Patient-reported outcomes were uncommon. A consensus-based set of core outcomes meaningful to patients and clinicians is needed for future ADPKD trials.

KW - QUALITY-OF-LIFE

KW - CLINICAL-TRIALS

KW - PROGRESSION

KW - DECLINE

KW - VOLUME

KW - CKD

U2 - 10.1053/j.ajkd.2019.12.003

DO - 10.1053/j.ajkd.2019.12.003

M3 - Review article

C2 - 32171640

VL - 76

SP - 213

EP - 223

JO - American Journal of Kidney Diseases

JF - American Journal of Kidney Diseases

SN - 0272-6386

IS - 2

ER -

ID: 127130515