Evidence map›Paper›PMID 32859657›Full record

ArticleBMJ open2020

Analysis of change in patient-reported outcome measures with floor and ceiling effects using the multilevel Tobit model: a simulation study and an example from a National Joint Register using body mass index and the Oxford Hip Score.

Adrian Sayers, Michael R Whitehouse, Andrew Judge, Alex J MacGregor, Ashley W Blom, Yoav Ben-Shlomo

Open access · goldAbstract read
In one paragraph

Article in BMJ open, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
0.8field-weighted citation impact, top 27% 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

6 citing papers in PubMed, 15 citations in OpenAlex.

  1. Review
  2. Observational
  3. Observational
  4. Article
  5. Observational
  6. Article
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

6 authors at 4 institutions in 1 country.

Adrian SayersMusculoskeletal Research Unit, Bristol Medical School, 1st Floor Learning & Research Building, Southmead Hospital, University of Bristol, Bristol, BS10 5NB, UK adrian.sayers@bristol.ac.uk.ORCID 0000-0001-7452-5043
Michael R WhitehouseMusculoskeletal Research Unit, Bristol Medical School, 1st Floor Learning & Research Building, Southmead Hospital, University of Bristol, Bristol, BS10 5NB, UK.ORCID 0000-0003-2436-9024
Andrew JudgeMusculoskeletal Research Unit, Bristol Medical School, 1st Floor Learning & Research Building, Southmead Hospital, University of Bristol, Bristol, BS10 5NB, UK.
Alex J MacGregorNorwich Medical School, University of East Anglia, Norwich, UK.
Ashley W BlomNational Institute for Health Research Bristol Biomedical Research Centre, University of Bristol, Bristol, UK.
Yoav Ben-ShlomoPopulation Health Sciences, Bristol Medical School, Canynge Hall, 39 Whatley Road, University of Bristol, Bristol, BS8 2PS, UK.
Southmead Hospital · GBNational Institute for Health Research · GBUniversity of Bristol · GBUniversity of East Anglia · GB

Funding

Medical Research Council MR/L01226X/1
6 · The paper itself

Abstract

objectivesThis study has three objectives. (1) Investigate the association between body mass index (BMI) and the efficacy of primary hip replacement using a patient-reported outcome measure (PROMs) with a measurement floor and ceiling, (2) Explore the performance of different estimation methods to estimate change in PROMs score following surgery using a simulation study and real word data where data has measurement floors and ceilings and (3) Lastly, develop guidance for practising researchers on the analysis of PROMs in the presence of floor and ceiling effects.

designSimulation study and prospective national medical device register.

settingNational Register of Joint Replacement and Medical Devices.

methodsUsing a Monte Carlo simulation study and data from a national joint replacement register (162 513 patients with pre- and post-surgery PROMs), we investigate simple approaches for the analysis of outcomes with floor and ceiling effects that are measured at two occasions: linear and Tobit regression (baseline adjusted analysis of covariance, change-score analysis, post-score analysis) in addition to linear and multilevel Tobit models. PRIMARY OUTCOME: The primary outcome of interest is change in PROMs from pre-surgery to 6 months post-surgery.

resultsAnalysis of data with floor and ceiling effects with models that fail to account for these features induce substantial bias. Single-level Tobit models only correct for floor or ceiling effects when the exposure of interest is not associated with the baseline score. In observational data scenarios, only multilevel Tobit models are capable of providing unbiased inferences.

conclusionsInferences from pre- post-studies that fail to account for floor and ceiling effects may induce spurious associations with substantial risk of bias. Multilevel Tobit models indicate the efficacy of total hip replacement is independent of BMI. Restricting access to total hip replacement based on a patients BMI can not be supported by the data.

Indexed as

Arthroplasty, Replacement, HipPatient Reported Outcome MeasuresBody Mass IndexHumansProspective StudiesTreatment Outcomearthroplastychange scoresepidemiologic methodslongitudinal studiesmulti-level tobit modelpatient reported outcome measures

Identifiers

PMID32859657
PMCPMC7454239
OpenAlexW3037214112

What OpenQuestion holds

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LicenceCC BY
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Registered trials

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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.