International standards for the analysis of quality-of-life and patient-reported outcome endpoints in cancer randomised controlled trials: recommendations of the SISAQOL Consortium.


Journal

The Lancet. Oncology
ISSN: 1474-5488
Titre abrégé: Lancet Oncol
Pays: England
ID NLM: 100957246

Informations de publication

Date de publication:
02 2020
Historique:
received: 21 10 2019
revised: 12 11 2019
accepted: 18 11 2019
entrez: 3 2 2020
pubmed: 3 2 2020
medline: 17 7 2020
Statut: ppublish

Résumé

Patient-reported outcomes (PROs), such as symptoms, function, and other health-related quality-of-life aspects, are increasingly evaluated in cancer randomised controlled trials (RCTs) to provide information about treatment risks, benefits, and tolerability. However, expert opinion and critical review of the literature showed no consensus on optimal methods of PRO analysis in cancer RCTs, hindering interpretation of results. The Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium was formed to establish PRO analysis recommendations. Four issues were prioritised: developing a taxonomy of research objectives that can be matched with appropriate statistical methods, identifying appropriate statistical methods for PRO analysis, standardising statistical terminology related to missing data, and determining appropriate ways to manage missing data. This Policy Review presents recommendations for PRO analysis developed through critical literature reviews and a structured collaborative process with diverse international stakeholders, which provides a foundation for endorsement; ongoing developments of these recommendations are also discussed.

Identifiants

pubmed: 32007209
pii: S1470-2045(19)30790-9
doi: 10.1016/S1470-2045(19)30790-9
pii:
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

e83-e96

Informations de copyright

Copyright © 2020 Elsevier Ltd. All rights reserved.

Auteurs

Corneel Coens (C)

European Organisation for Research and Treatment of Cancer, Brussels, Belgium.

Madeline Pe (M)

European Organisation for Research and Treatment of Cancer, Brussels, Belgium. Electronic address: madeline.pe@eortc.org.

Amylou C Dueck (AC)

Alliance Statistics and Data Center, Mayo Clinic, Scottsdale, AZ, USA.

Jeff Sloan (J)

Alliance Statistics and Data Center, Mayo Clinic, Rochester, MN, USA.

Ethan Basch (E)

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

Melanie Calvert (M)

Centre for Patient Reported Outcomes Research, Institute of Applied Health Research and National Institute for Health Research Birmingham Biomedical Research Centre, University of Birmingham, Birmingham, UK.

Alicyn Campbell (A)

Patient Relevant Evidence, San Francisco, CA, USA.

Charles Cleeland (C)

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

Kim Cocks (K)

Adelphi Values, Bollington, Cheshire, UK.

Laurence Collette (L)

European Organisation for Research and Treatment of Cancer, Brussels, Belgium.

Nancy Devlin (N)

Centre for Health Policy, School of Population and Global Health, University of Melbourne, Melbourne, VIC, Australia.

Lien Dorme (L)

European Organisation for Research and Treatment of Cancer, Brussels, Belgium.

Hans-Henning Flechtner (HH)

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

Carolyn Gotay (C)

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

Ingolf Griebsch (I)

Boehringer Ingelheim International GmbH, Ingelheim, Germany.

Mogens Groenvold (M)

Department of Public Health, Bispebjerg Hospital and University of Copenhagen, Copenhagen, Denmark.

Madeleine King (M)

School of Psychology, University of Sydney, Sydney, NSW, Australia.

Paul G Kluetz (PG)

Office of Hematology and Oncology Products, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA.

Michael Koller (M)

Center for Clinical Studies, University Hospital Regensburg, Regensburg, Germany.

Daniel C Malone (DC)

College of Pharmacy, University of Arizona, Tucson, AZ, USA.

Francesca Martinelli (F)

European Organisation for Research and Treatment of Cancer, Brussels, Belgium.

Sandra A Mitchell (SA)

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

Jammbe Z Musoro (JZ)

European Organisation for Research and Treatment of Cancer, Brussels, Belgium.

Daniel O'Connor (D)

Medicines and Healthcare products Regulatory Agency, London, UK.

Kathy Oliver (K)

International Brain Tumour Alliance, Surrey, UK.

Elisabeth Piault-Louis (E)

Genentech, Roche Group, San Francisco, CA, USA.

Martine Piccart (M)

Institut Jules Bordet, Université Libre de Bruxelles, Brussels, Belgium.

Chantal Quinten (C)

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

Jaap C Reijneveld (JC)

Department of Neurology and Brain Tumor Center, VU University Medical Center, Amsterdam, Netherlands.

Christoph Schürmann (C)

Institute for Quality and Efficiency in Health Care, Cologne, Germany.

Ashley Wilder Smith (AW)

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

Katherine M Soltys (KM)

Health Canada, Ottawa, ON, Canada.

Martin J B Taphoorn (MJB)

Department of Neurology, Leiden University Medical Center, Leiden; Department of Neurology, Haaglanden Medical Center, The Hague, Netherlands.

Galina Velikova (G)

Leeds Institute of Cancer and Pathology, University of Leeds, St James's Hospital, Leeds, UK; International Society for Quality of Life Research, Milwaukee, WI, USA.

Andrew Bottomley (A)

European Organisation for Research and Treatment of Cancer, Brussels, Belgium.

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