Statistical analysis of patient-reported outcome data in randomised controlled trials of locally advanced and metastatic breast cancer: a systematic review

Research output: Contribution to journalArticle

Standard

Statistical analysis of patient-reported outcome data in randomised controlled trials of locally advanced and metastatic breast cancer : a systematic review. / Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium (SISAQOL); Pe, Madeline; Calvert, Melanie.

In: The Lancet Oncology, Vol. 19, No. 9, 09.2018, p. e459-e469.

Research output: Contribution to journalArticle

Harvard

Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium (SISAQOL), Pe, M & Calvert, M 2018, 'Statistical analysis of patient-reported outcome data in randomised controlled trials of locally advanced and metastatic breast cancer: a systematic review', The Lancet Oncology, vol. 19, no. 9, pp. e459-e469. https://doi.org/10.1016/S1470-2045(18)30418-2

APA

Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium (SISAQOL), Pe, M., & Calvert, M. (2018). Statistical analysis of patient-reported outcome data in randomised controlled trials of locally advanced and metastatic breast cancer: a systematic review. The Lancet Oncology, 19(9), e459-e469. https://doi.org/10.1016/S1470-2045(18)30418-2

Vancouver

Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium (SISAQOL), Pe M, Calvert M. Statistical analysis of patient-reported outcome data in randomised controlled trials of locally advanced and metastatic breast cancer: a systematic review. The Lancet Oncology. 2018 Sep;19(9):e459-e469. https://doi.org/10.1016/S1470-2045(18)30418-2

Author

Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium (SISAQOL) ; Pe, Madeline ; Calvert, Melanie. / Statistical analysis of patient-reported outcome data in randomised controlled trials of locally advanced and metastatic breast cancer : a systematic review. In: The Lancet Oncology. 2018 ; Vol. 19, No. 9. pp. e459-e469.

Bibtex

@article{058e3b454e6c47c4bb1f1d83108d5306,
title = "Statistical analysis of patient-reported outcome data in randomised controlled trials of locally advanced and metastatic breast cancer: a systematic review",
abstract = "Although patient reported outcomes (PROs) such as health-related quality of life (HRQOL) are important endpoints in randomized controlled trials (RCTs), there is little consensus about analysis, interpretation and reporting of these data. A systematic review was conducted to assess variability, quality, and standards of PRO data analyses in advanced breast cancer RCTs. We searched through PubMed for English language articles published in peer-reviewed journals between January 2001 and October 2017. Eligible articles reported PRO results from RCTs involving adult advanced breast cancer patients receiving anti-cancer treatments with reported sample sizes of at least 50 patients. Sixty-six RCTs met the selection criteria. A small number of RCTs reported a specific PRO research hypothesis (8/66, 12{\%}). There was heterogeneity in the statistical methods used to assess PRO data, with a mixture of longitudinal and cross-sectional techniques. Not all articles addressed the problem of inflated type I error resulting from multiple testing. Fewer than half of RCTs reported the clinical significance of their findings (28/66, 42{\%}). The majority of trials did not report how missing data was handled (48/66, 73{\%}). Our review demonstrates a need to improve standards in analysis, interpretation and reporting of PRO data in cancer RCTs. Lack of standardization makes it difficult to draw robust conclusions and compare findings across trials. The Setting International Standards in the Analyzing Patient-Reported Outcomes and Quality of Life Data (SISAQOL) Consortium was set up to address this need and develop recommendations on the analysis of PRO data in RCTs.",
keywords = "health-related quality of life, advanced breast cancer, systematic review, randomized controlled trials, statistical methodology, patient reported outcomes",
author = "{Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium (SISAQOL)} and Madeline Pe and Lien Dorme and Corneel Coens and Ethan Basch and Melanie Calvert and Alicyn Campbell and Charles Cleeland and Kim Cocks and Laurence Collette and Linda Dirven and Dueck, {Amylou C} and Nancy Devlin and Hans-Henning Flechtner and Carolyn Gotay and Ingolf Griebsch and Mogens Groenvold and Madeleine King and Michael Koller and Malone, {Daniel C} and Francesca Martinelli and Mitchell, {Sandra A} and Jammbe Musoro and Kathy Oliver and Elisabeth Piault-Louis and Martine Piccart and Pimentel, {Francisco L} and Chantal Quinten and Reijneveld, {Jaap C} and Jeff Sloan and Galina Velikova and Andrew Bottomley",
year = "2018",
month = "9",
doi = "10.1016/S1470-2045(18)30418-2",
language = "English",
volume = "19",
pages = "e459--e469",
journal = "The Lancet Oncology",
issn = "1470-2045",
publisher = "Elsevier",
number = "9",

}

RIS

TY - JOUR

T1 - Statistical analysis of patient-reported outcome data in randomised controlled trials of locally advanced and metastatic breast cancer

T2 - a systematic review

AU - Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium (SISAQOL)

AU - Pe, Madeline

AU - Dorme, Lien

AU - Coens, Corneel

AU - Basch, Ethan

AU - Calvert, Melanie

AU - Campbell, Alicyn

AU - Cleeland, Charles

AU - Cocks, Kim

AU - Collette, Laurence

AU - Dirven, Linda

AU - Dueck, Amylou C

AU - Devlin, Nancy

AU - Flechtner, Hans-Henning

AU - Gotay, Carolyn

AU - Griebsch, Ingolf

AU - Groenvold, Mogens

AU - King, Madeleine

AU - Koller, Michael

AU - Malone, Daniel C

AU - Martinelli, Francesca

AU - Mitchell, Sandra A

AU - Musoro, Jammbe

AU - Oliver, Kathy

AU - Piault-Louis, Elisabeth

AU - Piccart, Martine

AU - Pimentel, Francisco L

AU - Quinten, Chantal

AU - Reijneveld, Jaap C

AU - Sloan, Jeff

AU - Velikova, Galina

AU - Bottomley, Andrew

PY - 2018/9

Y1 - 2018/9

N2 - Although patient reported outcomes (PROs) such as health-related quality of life (HRQOL) are important endpoints in randomized controlled trials (RCTs), there is little consensus about analysis, interpretation and reporting of these data. A systematic review was conducted to assess variability, quality, and standards of PRO data analyses in advanced breast cancer RCTs. We searched through PubMed for English language articles published in peer-reviewed journals between January 2001 and October 2017. Eligible articles reported PRO results from RCTs involving adult advanced breast cancer patients receiving anti-cancer treatments with reported sample sizes of at least 50 patients. Sixty-six RCTs met the selection criteria. A small number of RCTs reported a specific PRO research hypothesis (8/66, 12%). There was heterogeneity in the statistical methods used to assess PRO data, with a mixture of longitudinal and cross-sectional techniques. Not all articles addressed the problem of inflated type I error resulting from multiple testing. Fewer than half of RCTs reported the clinical significance of their findings (28/66, 42%). The majority of trials did not report how missing data was handled (48/66, 73%). Our review demonstrates a need to improve standards in analysis, interpretation and reporting of PRO data in cancer RCTs. Lack of standardization makes it difficult to draw robust conclusions and compare findings across trials. The Setting International Standards in the Analyzing Patient-Reported Outcomes and Quality of Life Data (SISAQOL) Consortium was set up to address this need and develop recommendations on the analysis of PRO data in RCTs.

AB - Although patient reported outcomes (PROs) such as health-related quality of life (HRQOL) are important endpoints in randomized controlled trials (RCTs), there is little consensus about analysis, interpretation and reporting of these data. A systematic review was conducted to assess variability, quality, and standards of PRO data analyses in advanced breast cancer RCTs. We searched through PubMed for English language articles published in peer-reviewed journals between January 2001 and October 2017. Eligible articles reported PRO results from RCTs involving adult advanced breast cancer patients receiving anti-cancer treatments with reported sample sizes of at least 50 patients. Sixty-six RCTs met the selection criteria. A small number of RCTs reported a specific PRO research hypothesis (8/66, 12%). There was heterogeneity in the statistical methods used to assess PRO data, with a mixture of longitudinal and cross-sectional techniques. Not all articles addressed the problem of inflated type I error resulting from multiple testing. Fewer than half of RCTs reported the clinical significance of their findings (28/66, 42%). The majority of trials did not report how missing data was handled (48/66, 73%). Our review demonstrates a need to improve standards in analysis, interpretation and reporting of PRO data in cancer RCTs. Lack of standardization makes it difficult to draw robust conclusions and compare findings across trials. The Setting International Standards in the Analyzing Patient-Reported Outcomes and Quality of Life Data (SISAQOL) Consortium was set up to address this need and develop recommendations on the analysis of PRO data in RCTs.

KW - health-related quality of life

KW - advanced breast cancer

KW - systematic review

KW - randomized controlled trials

KW - statistical methodology

KW - patient reported outcomes

U2 - 10.1016/S1470-2045(18)30418-2

DO - 10.1016/S1470-2045(18)30418-2

M3 - Article

VL - 19

SP - e459-e469

JO - The Lancet Oncology

JF - The Lancet Oncology

SN - 1470-2045

IS - 9

ER -