Evidence map›Paper›PMID 35797589›Full record

SynthesisSleep2022

Evaluation of psychometric properties of patient-reported outcome measures frequently used in narcolepsy randomized controlled trials: a systematic review.

Aaron Schokman, Yu Sun Bin, Diana Naehrig, Janet M Y Cheung, Kristina Kairaitis, Nick Glozier

Open access · hybridAbstract readSystematic Review
PubMed Full text at PMC Publisher
In one paragraph

Synthesis in Sleep, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
0.9field-weighted citation impact, top 28% 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

5 citing papers in PubMed, 8 citations in OpenAlex.

  1. Trial
  2. Trial
  3. Trial
  4. Review
  5. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

6 authors at 1 institution in 1 country.

Aaron SchokmanFaculty of Medicine and Health, University of Sydney, Camperdown, NSW, Australia.ORCID 0000-0003-0419-9347
Yu Sun BinSleep Theme at Charles Perkins Centre, University of Sydney, Camperdown, NSW, Australia.ORCID 0000-0002-4954-2658
Diana NaehrigFaculty of Medicine and Health, University of Sydney, Camperdown, NSW, Australia.ORCID 0000-0002-1162-4092
Janet M Y CheungFaculty of Medicine and Health, University of Sydney, Camperdown, NSW, Australia.ORCID 0000-0001-6341-5594
Kristina KairaitisDepartment of Respiratory and Sleep Medicine, University of Sydney at Westmead Hospital, Westmead, NSW, Australia.ORCID 0000-0002-8808-3183
Nick GlozierFaculty of Medicine and Health, University of Sydney, Camperdown, NSW, Australia.ORCID 0000-0002-0476-9146
The University of Sydney · AU

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

STUDY

objectivesTo systematically determine subjective and objective outcome measures used to measure the efficacy of narcolepsy interventions in randomized controlled trials (RCTs) in adults and children and assess psychometric properties of patient-reported outcome measures (PROMs) used.

methodsWe searched bibliographical databases and clinical trial registries for narcolepsy RCTs and extracted objective and subjective outcome measures. If PROMs were used, we searched for psychometric studies conducted in a narcolepsy population using bibliographical databases and appraised using Consensus-based Standards for the Selection of Health Measurement Instruments (COSMIN) guidelines.

resultsIn total, 80 different outcome measures were used across 100 RCTs. Epworth Sleepiness Scale (ESS) (n = 49) and Maintenance of Wakefulness Test (n = 47) were the most frequently used outcome measures. We found 19 validation studies of 10 PROMs in narcolepsy populations. There was limited evidence for validity or responsiveness of the ESS; yet sufficient reliability (pooled ICC: 0.81-0.87). Narcolepsy Severity Scale (NSS) had sufficient reliability (pooled ICC: 0.71-0.92) and both adult and pediatric versions had sufficient discriminant validity (treated/untreated). Content validity was only evaluated in pediatric populations for ESS-CHAD and NSS-P and rated inconclusive. Quality of evidence of the psychometric studies for all scales ranged from very low to low.

conclusionsAlthough recognized by regulatory bodies and widely used as primary outcome measures in trials, there is surprisingly little evidence for the validity, reliability, and responsiveness of PROMs frequently used to assess treatment efficacy in narcolepsy. The field needs to establish patient-centered minimal clinically important differences for the PROMs used in these trials.

Indexed as

NarcolepsyAdultChildHumansPatient Reported Outcome MeasuresPsychometricsQuality of LifeRandomized Controlled Trials as TopicReproducibility of ResultsWakefulnesscataplexycosminnarcolepsypatient-reported outcome measurespsychometric propertiesrandomized controlled trialsreliabilityresponsivenesssystematic reviewvalidity

Identifiers

PMID35797589
PMCPMC9548672
OpenAlexW4284881886

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.