Developing core outcome sets for clinical trials: issues to consider

Williamson, Paula; Altman, Douglas G.; Blazeby, Jane; Clarke, Mike; Devane, Declan; Gargon, Elizabeth; Tugwell, Peter

doi:10.1186/1745-6215-13-132

Cited by 1,297 publications

(1,581 citation statements)

References 23 publications

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“…Most studies included in this review were at high risk of bias30 31, 51 52. Although one‐quarter of studies used a prospective database, none of these published their protocols or defined their core clinical outcome sets52 53, or reported study size calculations for the surgical groups54.…”

Section: Discussionmentioning

confidence: 99%

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Systematic review of the impact of breast‐conserving surgery on cancer outcomes of multiple ipsilateral breast cancers

Winters¹,

Horsnell

Elvers

et al. 2018

BJS Open

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BackgroundThe clinical effectiveness of treating ipsilateral multifocal (MF) and multicentric (MC) breast cancers using breast‐conserving surgery (BCS) compared with the standard of mastectomy is uncertain. Inconsistencies relate to definitions, incidence, staging and intertumoral heterogeneity. The primary aim of this systematic review was to compare clinical outcomes after BCS versus mastectomy for MF and MC cancers, collectively defined as multiple ipsilateral breast cancers (MIBC).MethodsComprehensive electronic searches were undertaken to identify complete papers published in English between May 1988 and July 2015, primarily comparing clinical outcomes of BCS and mastectomy for MIBC. All study designs were included, and studies were appraised critically using the Newcastle–Ottawa Scale. The characteristics and results of identified studies were summarized.ResultsTwenty‐four retrospective studies were included in the review: 17 comparative studies and seven case series. They included 3537 women with MIBC undergoing BCS; breast cancers were defined as MF in 2677 women, MC in 292, and reported as MIBC in 568. Six studies evaluated MIBC treated by BCS or mastectomy, with locoregional recurrence (LRR) rates of 2–23 per cent after BCS at median follow‐up of 59·5 (i.q.r. 56–81) months. BCS and mastectomy showed apparently equivalent rates of LRR (risk ratio 0·94, 95 per cent c.i. 0·65 to 1·36). Thirteen studies compared BCS in women with MIBC versus those with unifocal cancers, reporting LRR rates of 2–40 per cent after BCS at a median follow‐up of 64 (i.q.r. 57–73) months. One high‐quality study reported 10‐year actuarial LRR rates of 5·5 per cent for BCS in 300 women versus 6·5 per cent for mastectomy among 887 women.ConclusionThe available studies were mainly of moderate quality, historical and underpowered, with limited follow‐up and biased case selection favouring BCS rather than mastectomy for low‐risk patients. The evidence was inconclusive, weakening support for the St Gallen consensus and supporting a future randomized trial.

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Section: Discussionmentioning

confidence: 99%

“…Although one‐quarter of studies used a prospective database, none of these published their protocols or defined their core clinical outcome sets52 53, or reported study size calculations for the surgical groups54. Other markers of study quality were lacking, such as ethical approvals and conflicts of interest30 31, 51.…”

Section: Discussionmentioning

confidence: 99%

Systematic review of the impact of breast‐conserving surgery on cancer outcomes of multiple ipsilateral breast cancers

Winters¹,

Horsnell

Elvers

et al. 2018

BJS Open

View full text Add to dashboard Cite

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“…Guidelines for Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) [27] and recent publications on the development of a Core Outcome Set (COS) [28] were applied.…”

Section: Methodsmentioning

confidence: 99%

Measuring outcomes in adult spinal deformity surgery: a systematic review to identify current strengths, weaknesses and gaps in patient-reported outcome measures

et al. 2017

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Purpose Adult spinal deformity (ASD) causes severe disability, reduces overall quality of life, and results in a substantial societal burden of disease. As healthcare is becoming more value based, and to facilitate global benchmarking, it is critical to identify and standardize patient-reported outcome measures (PROMs). This study aims to identify the current strengths, weaknesses, and gaps in PROMs used for ASD. Methods Studies were included following a systematic search in multiple bibliographic databases between 2000 and 2015. PROMs were extracted and linked to the outcome domains of WHO's International Classification of Functioning and Health (ICF) framework. Subsequently, the clinimetric quality of identified PROMs was evaluated. Results The literature search identified 144 papers that met the inclusion criteria, and nine frequently used PROMs were identified. These covered 29 ICF outcome domains, which could be grouped into three of the four main ICF chapters: body function (n = 7), activity and participation (n = 19), environmental factors (n = 3), and body structure (n = 0). A low quantity (n = 3) of papers was identified that studied the clinimetric quality of PROMs. The Scoliosis Research Society (SRS)-22 has the highest level of clinimetric quality for ASD. Conclusions Outcome domains related to mobility and pain were well represented. We identified a gap in current outcome measures regarding neurological and pulmonary function. In addition, no outcome domains were measured in the ICF chapter body structure. These results will serve as a foundation for the process of seeking international consensus on a standard set of outcome domains, accompanied PROMs and contributing factors to be used in future clinical trials and spine registries.

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“…The development of a core outcome set for a specific clinical condition has also been suggested as a way of addressing heterogeneity in outcome measures. These sets are recommended as the minimum outcomes to be measured in clinical trials of the condition [21]. However, outcomes need not necessarily be restricted to those in the core outcome set.…”

Section: Assessing the Clinical Relevance Of Functional Rating Scalesmentioning

confidence: 99%

The Spectrum of Functional Rating Scales in Neurology Clinical Trials

Narayanaswami

2017

Neurotherapeutics

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The selection of an appropriate outcome measure is crucial to the success of a clinical trial, in order to obtain accurate results, which, in turn, influence patient care and future research. Outcomes that can be directly measured are mortality/survival. More frequently, neurology clinical trials evaluate outcomes that cannot be directly measured, such as disability, cognitive function, or change in symptoms of the condition under study. These complex outcomes are abstract ideas or latent constructs and are measured using rating scales. Functional rating scales typically assess the ability of patients to perform tasks and roles for everyday life. Rating scales should be valid (measure what they are supposed to measure), reliable (provide similar results if administered under the same conditions), and responsive (able to detect clinically important changes over time). The clinical relevance of rating scales depends on their ability to detect a minimal clinically important difference, and should be distinguished from statistical significance. Most rating scales are ordinal scales and have limitations. Modern psychometric methods of Rasch analysis and item response theory, termed latent trait theory, are increasingly being utilized to convert ordinal data to interval measurements, both to validate existing scales and to develop new scales. Patient-reported outcomes are being increasingly used in clinical trials and have a role in clinical quality assessment. The PROMIS and NeuroQoL databases are excellent resources for rigorously developed and validated patientreported outcomes.

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Developing core outcome sets for clinical trials: issues to consider

Cited by 1,297 publications

References 23 publications

Systematic review of the impact of breast‐conserving surgery on cancer outcomes of multiple ipsilateral breast cancers

Systematic review of the impact of breast‐conserving surgery on cancer outcomes of multiple ipsilateral breast cancers

Measuring outcomes in adult spinal deformity surgery: a systematic review to identify current strengths, weaknesses and gaps in patient-reported outcome measures

The Spectrum of Functional Rating Scales in Neurology Clinical Trials

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