Evidence map›Paper›PMID 31619428›Full record

SynthesisBMJ open2019

Partially randomised patient preference trials as an alternative design to randomised controlled trials: systematic review and meta-analyses.

Karin A Wasmann, Pieta Wijsman, Susan van Dieren, Willem Bemelman, Christianne Buskens

Open access · goldAbstract readComparative StudyMeta-AnalysisSystematic Review
PubMed Full text at PMC Publisher
In one paragraph

Synthesis in BMJ open, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 63 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
63citing papers in PubMed, 2 pooled it
16.2field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

63 citing papers in PubMed, 2 syntheses or guidelines pooled it, 104 citations in OpenAlex.

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3 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors at 2 institutions in 1 country.

Karin A WasmannDepartment of Surgery, Amsterdam UMC-Location AMC, Amsterdam, Netherlands k.a.wasmann@amsterdamumc.nl.ORCID 0000-0003-2295-238X
Pieta WijsmanDepartment of Internal Medicine, Spaarne Gasthuis, Haarlem, Netherlands.
Susan van DierenDepartment of Statistics and Epidemiology, Amsterdam UMC-Location AMC, Amsterdam, Netherlands.
Willem BemelmanDepartment of Surgery, Amsterdam UMC-Location AMC, Amsterdam, Netherlands.
Christianne BuskensDepartment of Surgery, Amsterdam UMC-Location AMC, Amsterdam, Netherlands.
Amsterdam University Medical Centers · NLSpaarne Gasthuis · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveRandomised controlled trials (RCT) are the gold standard to provide unbiased data. However, when patients have a treatment preference, randomisation may influence participation and outcomes (eg, external and internal validity). The aim of this study was to assess the influence of patients' preference in RCTs by analysing partially randomised patient preference trials (RPPT); an RCT and preference cohort combined.

designSystematic review and meta-analyses. DATA SOURCES: MEDLINE, Embase, PsycINFO and the Cochrane Library. ELIGIBILITY CRITERIA FOR SELECTING STUDIES: RPPTs published between January 2005 and October 2018 reporting on allocation of patients to randomised and preference cohorts were included. DATA EXTRACTION AND SYNTHESIS: Two independent reviewers extracted data. The main outcomes were the difference in external validity (participation and baseline characteristics) and internal validity (lost to follow-up, crossover and the primary outcome) between the randomised and the preference cohort within each RPPT, compared in a meta-regression using a Wald test. Risk of bias was not assessed, as no quality assessment for RPPTs has yet been developed.

resultsIn total, 117 of 3734 identified articles met screening criteria and 44 were eligible (24 873 patients). The participation rate in RPPTs was >95% in 14 trials (range: 48%-100%) and the randomisation refusal rate was >50% in 26 trials (range: 19%-99%). Higher education, female, older age, race and prior experience with one treatment arm were characteristics of patients declining randomisation. The lost to follow-up and cross-over rate were significantly higher in the randomised cohort compared with the preference cohort. Following the meta-analysis, the reported primary outcomes were comparable between both cohorts of the RPPTs, mean difference 0.093 (95% CI -0.178 to 0.364, p

conclusionsPatients' preference led to a substantial proportion of a specific patient group refusing randomisation, while it did not influence the primary outcome within an RPPT. Therefore, RPPTs could increase external validity without compromising the internal validity compared with RCTs. PROSPERO REGISTRATION NUMBER: CRD42019094438.

Indexed as

Patient PreferenceAge FactorsHumansLost to Follow-UpOutcome Assessment, Health CarePatient ParticipationRandomized Controlled Trials as TopicSex Factorscomprehensive cohort designexternal validityinternal validitypatients’ preferenceRandomised controlled trialsrandomised patient preference trials

Identifiers

PMID31619428
PMCPMC6797441
OpenAlexW2981158081

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.