Statistical analysis of patient-reported outcome data in randomised controlled trials of locally advanced and metastatic breast cancer: a systematic review - 01/09/18

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

Madeline Pe, PhD ^a,^⁎ , Lien Dorme, MSc ^a, Corneel Coens, MSc ^a, Ethan Basch, MD ^b, Melanie Calvert, ProfPhD ^c, Alicyn Campbell, MPh ^d, Charles Cleeland, ProfPhD ^e, Kim Cocks, PhD ^f, Laurence Collette, PhD ^a, Linda Dirven, PhD ^g, Amylou C Dueck, PhD ^h, Nancy Devlin, PhD ⁱ, Hans-Henning Flechtner, ProfMD ^j, Carolyn Gotay, ProfPhD ^k, Ingolf Griebsch, PhD ^l, Mogens Groenvold, ProfMD ^m, Madeleine King, ProfPhD ⁿ, Michael Koller, PhD ^o, Daniel C Malone, ProfPhD ^p, Francesca Martinelli, MSc ^a, Sandra A Mitchell, PhD ^q, Jammbe Z Musoro, PhD ^a, Kathy Oliver, BA ^r, Elisabeth Piault-Louis, PharmD ^d, Martine Piccart, MD ^s, Francisco L Pimentel, MD ^t,^u, Chantal Quinten, MSc ^v, Jaap C Reijneveld, MD ^w, Jeff Sloan, PhD ^x, Galina Velikova, ProfPhD ^y, Andrew Bottomley, PhD ^a
for the
Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium (SISAQOL)

^a European Organisation for Research and Treatment of Cancer, Brussels, Belgium

^b Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, NC, USA

^c Centre for Patient Reported Outcomes Research, Institute of Applied Health Research, College of Medical and Dental Sciences, University of Birmingham, Birmingham, UK

^d Genentech, a member of the Roche Group, San Francisco, CA, USA

^e Department of Symptom Research, The University of Texas MD Anderson Cancer Center, Houston, TX, USA

^f Adelphi Values, Bollington, Cheshire, UK

^g Leiden University Medical Center and Haaglanden Medical Center, Leiden and The Hague, Netherlands

^h Alliance Statistics and Data Center, Mayo Clinic, Scottsdale, AZ, USA

ⁱ Office of Health Economics, London, UK

^j Clinic for Child and Adolescent Psychiatry and Psychotherapy, University of Magdeburg, Magdeburg, Germany

^k School of Population and Public Health, University of British Columbia, Vancouver, BC, Canada

^l Boehringer Ingelheim International GmBH, Ingelheim, Germany

^m Department of Public Health and Bispebjerg Hospital, University of Copenhagen, Copenhagen, Denmark

ⁿ School of Psychology and Sydney Medical School, University of Sydney, Sydney, NSW, Australia

^o Center for Clinical Studies, University Hospital Regensburg, Regensburg, Germany

^p College of Pharmacy, University of Arizona, Tucson, AZ, USA

^q Outcomes Research Branch, Healthcare Delivery Research Program, Division of Cancer Control and Population Sciences, National Cancer Institute, Bethesda, MD, USA

^r International Brain Tumour Alliance, Surrey, UK

^s Institut Jules Bordet, Université Libre de Bruxelles, Brussels, Belgium

^t Blueclinical Phase I, Porto, Portugal

^u Centro de Estudos e Investigação em Saúde da Universidade de Coimbra, Coimbra, Portugal

^v European Centre for Disease Prevention and Control, Surveillance and Response Support Unit, Epidemiological Methods Section, Stockholm, Sweden

^w VU University Medical Center, Department of Neurology & Brain Tumor Center, Amsterdam, Netherlands

^x Alliance Statistics and Data Center, Mayo Clinic, Rochester, MN, USA

^y Leeds Institute of Cancer and Pathology, University of Leeds, St James’s Hospital, Leeds, UK

^* Correspondence to: Dr Madeline Pe, Quality of Life Department, European Organisation for Research and Treatment of Cancer, 1200 Brussels, Belgium Quality of Life Department European Organisation for Research and Treatment of Cancer Brussels 1200 Belgium

Summary

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.

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Plan

Introduction

Data collection

Search strategy and selection criteria

Evaluation process

Findings

Descriptive statistics

Reporting of research objectives

Statistical analysis and clinical relevance

Baseline assessment

Amount and handling of missing data

Discussion

Conclusion

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Vol 19 - N° 9

P. e459-e469 - septembre 2018 Retour au numéro

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Statistical analysis of patient-reported outcome data in randomised controlled trials of locally advanced and metastatic breast cancer: a systematic review - 01/09/18

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