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

Madeline Pe; Lien Dorme; Corneel Coens; Ethan Basch; Melanie Calvert; Alicyn Campbell; Charles Cleeland; Kim Cocks; Laurence Collette; Linda Dirven; Amylou C Dueck; Nancy Devlin; Hans-Henning Flechtner; Carolyn Gotay; Ingolf Griebsch; Mogens Grønvold; Madeleine King; Michael Koller; Daniel C Malone; Francesca Martinelli; Sandra A Mitchell; Jammbe Z Musoro; Kathy Oliver; Elisabeth Piault-Louis; Martine Piccart; Francisco L Pimentel; Chantal Quinten; Jaap C Reijneveld; Jeff Sloan; Galina Velikova; Andrew Bottomley

doi:10.1016/S1470-2045(18)30418-2

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

Madeline Pe, Lien Dorme, Corneel Coens, Ethan Basch, Melanie Calvert, Alicyn Campbell, Charles Cleeland, Kim Cocks, Laurence Collette, Linda Dirven, Amylou C Dueck, Nancy Devlin, Hans-Henning Flechtner, Carolyn Gotay, Ingolf Griebsch, Mogens Grønvold, Madeleine King, Michael Koller, Daniel C Malone, Francesca MartinelliSandra A Mitchell, Jammbe Z Musoro, Kathy Oliver, Elisabeth Piault-Louis, Martine Piccart, Francisco L Pimentel, Chantal Quinten, Jaap C Reijneveld, Jeff Sloan, Galina Velikova, Andrew Bottomley

Section of Health Services Research

24 Citations (Scopus)

Abstract

Although patient-reported outcomes (PROs), such as health-related quality of life, are important endpoints in randomised controlled trials (RCTs), there is little consensus about the analysis, interpretation, and reporting of these data. We did a systematic review to assess the variability, quality, and standards of PRO data analyses in advanced breast cancer RCTs. We searched PubMed for English language articles published in peer-reviewed journals between Jan 1, 2001, and Oct 30, 2017. Eligible articles were those that reported PRO results from RCTs of adult patients with advanced breast cancer receiving anti-cancer treatments with reported sample sizes of at least 50 patients-66 RCTs met the selection criteria. Only eight (12%) RCTs reported a specific PRO research hypothesis. Heterogeneity in the statistical methods used to assess PRO data was observed, with a mixture of longitudinal and cross-sectional techniques. Not all articles addressed the problem of multiple testing. Fewer than half of RCTs (28 [42%]) reported the clinical significance of their findings. 48 (73%) did not report how missing data were handled. Our systematic review shows a need to improve standards in the analysis, interpretation, and reporting of PRO data in cancer RCTs. Lack of standardisation 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 Consortium was set up to address this need and develop recommendations on the analysis of PRO data in RCTs.

Original language	English
Journal	Lancet Oncology
Volume	19
Issue number	9
Pages (from-to)	e459-e469
Number of pages	11
ISSN	1470-2045
DOIs	https://doi.org/10.1016/S1470-2045(18)30418-2
Publication status	Published - Sept 2018

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Pe, M., Dorme, L., Coens, C., Basch, E., Calvert, M., Campbell, A., Cleeland, C., Cocks, K., Collette, L., Dirven, L., Dueck, A. C., Devlin, N., Flechtner, H.-H., Gotay, C., Griebsch, I., Grønvold, M., King, M., Koller, M., Malone, D. C., ... Bottomley, A. (2018). Statistical analysis of patient-reported outcome data in randomised controlled trials of locally advanced and metastatic breast cancer: a systematic review. Lancet Oncology, 19(9), e459-e469. https://doi.org/10.1016/S1470-2045(18)30418-2

Pe, M, Dorme, L, Coens, C, Basch, E, Calvert, M, Campbell, A, Cleeland, C, Cocks, K, Collette, L, Dirven, L, Dueck, AC, Devlin, N, Flechtner, H-H, Gotay, C, Griebsch, I, Grønvold, M, King, M, Koller, M, Malone, DC, Martinelli, F, Mitchell, SA, Musoro, JZ, Oliver, K, Piault-Louis, E, Piccart, M, Pimentel, FL, Quinten, C, Reijneveld, JC, Sloan, J, Velikova, G & Bottomley, A 2018, 'Statistical analysis of patient-reported outcome data in randomised controlled trials of locally advanced and metastatic breast cancer: a systematic review', Lancet Oncology, vol. 19, no. 9, pp. e459-e469. https://doi.org/10.1016/S1470-2045(18)30418-2

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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, are important endpoints in randomised controlled trials (RCTs), there is little consensus about the analysis, interpretation, and reporting of these data. We did a systematic review to assess the variability, quality, and standards of PRO data analyses in advanced breast cancer RCTs. We searched PubMed for English language articles published in peer-reviewed journals between Jan 1, 2001, and Oct 30, 2017. Eligible articles were those that reported PRO results from RCTs of adult patients with advanced breast cancer receiving anti-cancer treatments with reported sample sizes of at least 50 patients-66 RCTs met the selection criteria. Only eight (12%) RCTs reported a specific PRO research hypothesis. Heterogeneity in the statistical methods used to assess PRO data was observed, with a mixture of longitudinal and cross-sectional techniques. Not all articles addressed the problem of multiple testing. Fewer than half of RCTs (28 [42%]) reported the clinical significance of their findings. 48 (73%) did not report how missing data were handled. Our systematic review shows a need to improve standards in the analysis, interpretation, and reporting of PRO data in cancer RCTs. Lack of standardisation 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 Consortium was set up to address this need and develop recommendations on the analysis of PRO data in RCTs.",

author = "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 Gr{\o}nvold and Madeleine King and Michael Koller and Malone, {Daniel C} and Francesca Martinelli and Mitchell, {Sandra A} and Musoro, {Jammbe Z} 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 = sep,

doi = "10.1016/S1470-2045(18)30418-2",

language = "English",

volume = "19",

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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 - 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 - Grønvold, Mogens

AU - King, Madeleine

AU - Koller, Michael

AU - Malone, Daniel C

AU - Martinelli, Francesca

AU - Mitchell, Sandra A

AU - Musoro, Jammbe Z

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, are important endpoints in randomised controlled trials (RCTs), there is little consensus about the analysis, interpretation, and reporting of these data. We did a systematic review to assess the variability, quality, and standards of PRO data analyses in advanced breast cancer RCTs. We searched PubMed for English language articles published in peer-reviewed journals between Jan 1, 2001, and Oct 30, 2017. Eligible articles were those that reported PRO results from RCTs of adult patients with advanced breast cancer receiving anti-cancer treatments with reported sample sizes of at least 50 patients-66 RCTs met the selection criteria. Only eight (12%) RCTs reported a specific PRO research hypothesis. Heterogeneity in the statistical methods used to assess PRO data was observed, with a mixture of longitudinal and cross-sectional techniques. Not all articles addressed the problem of multiple testing. Fewer than half of RCTs (28 [42%]) reported the clinical significance of their findings. 48 (73%) did not report how missing data were handled. Our systematic review shows a need to improve standards in the analysis, interpretation, and reporting of PRO data in cancer RCTs. Lack of standardisation 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 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, are important endpoints in randomised controlled trials (RCTs), there is little consensus about the analysis, interpretation, and reporting of these data. We did a systematic review to assess the variability, quality, and standards of PRO data analyses in advanced breast cancer RCTs. We searched PubMed for English language articles published in peer-reviewed journals between Jan 1, 2001, and Oct 30, 2017. Eligible articles were those that reported PRO results from RCTs of adult patients with advanced breast cancer receiving anti-cancer treatments with reported sample sizes of at least 50 patients-66 RCTs met the selection criteria. Only eight (12%) RCTs reported a specific PRO research hypothesis. Heterogeneity in the statistical methods used to assess PRO data was observed, with a mixture of longitudinal and cross-sectional techniques. Not all articles addressed the problem of multiple testing. Fewer than half of RCTs (28 [42%]) reported the clinical significance of their findings. 48 (73%) did not report how missing data were handled. Our systematic review shows a need to improve standards in the analysis, interpretation, and reporting of PRO data in cancer RCTs. Lack of standardisation 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 Consortium was set up to address this need and develop recommendations on the analysis of PRO data in RCTs.

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

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

M3 - Review

C2 - 30191850

SN - 1470-2045

VL - 19

SP - e459-e469

JO - Lancet Oncology

JF - Lancet Oncology

IS - 9

ER -

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

Abstract

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