Evidence map›Paper›PMID 30537955›Full record

ReviewHealth and quality of life outcomes2018

Distribution- and anchor-based methods to determine the minimally important difference on patient-reported outcome questionnaires in oncology: a structured review.

Ahmad Ousmen, Célia Touraine, Nina Deliu, Francesco Cottone, Franck Bonnetain, Fabio Efficace, Anne Brédart, Caroline Mollevi, Amélie Anota

Registry-linked trialOpen access · goldAbstract readReview
In one paragraph

Review in Health and quality of life outcomes, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06630650 (A Single Center Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes), which is not on this map. Cited by 66 papers, 7 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
66citing papers in PubMed, 7 pooled it
3.9field-weighted citation impact, top 4% 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.

NCT06630650 recruitingnot on this mapstarted 2025, after this paper: background citation

A Single Center Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes

TypeobservationalSponsorNational Institute of Neurological Disorders and Stroke (NINDS)Ran2025 to 2044Enrolled75ConditionsMyasthenic Syndromes, Congenital
3 · Its place in the literature

Who cites it

66 citing papers in PubMed, 7 syntheses or guidelines pooled it, 154 citations in OpenAlex.

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  11. A minimal clinically important difference for the sleep inertia visual analog scale in idiopathic hypersomnia.Journal of clinical sleep medicine : JCSM : official publication of the American Academy of Sleep Medicine · 2025
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  18. Unraveling assumptions about clinical relevance in patient-reported outcome data : A qualitative study on perspectives of different interest groups.Supportive care in cancer : official journal of the Multinational Association of Supportive Care in Cancer · 2026
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  19. Calculating the minimally important difference for the FACT-E from esophageal cancer surgery to recovery using distribution-based methods.Diseases of the esophagus : official journal of the International Society for Diseases of the Esophagus · 2025
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6 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

9 authors at 4 institutions in 2 countries.

Ahmad OusmenMethodology and Quality of Life in Oncology Unit (INSERM UMR 1098), University Hospital of Besançon, Besançon, France. ousmen.ahmad@hotmail.com.
Célia TouraineMontpellier Cancer Institute (ICM) - Val d'Aurelle, University of Montpellier, Montpellier, France.
Nina DeliuItalian Group for Adult Hematologic Diseases (GIMEMA), Data Center and Health Outcomes Research Unit, Rome, Italy.
Francesco CottoneItalian Group for Adult Hematologic Diseases (GIMEMA), Data Center and Health Outcomes Research Unit, Rome, Italy.
Franck BonnetainMethodology and Quality of Life in Oncology Unit (INSERM UMR 1098), University Hospital of Besançon, Besançon, France.
Fabio EfficaceItalian Group for Adult Hematologic Diseases (GIMEMA), Data Center and Health Outcomes Research Unit, Rome, Italy.
Anne BrédartInstitut Curie, Supportive Care Department, Psycho-Oncology Unit, Paris, France.
Caroline MolleviMontpellier Cancer Institute (ICM) - Val d'Aurelle, University of Montpellier, Montpellier, France.
Amélie AnotaMethodology and Quality of Life in Oncology Unit (INSERM UMR 1098), University Hospital of Besançon, Besançon, France.
Inserm · FRFondazione Gimema Onlus · ITCEA Valduc · FRUniversité Paris Cité · FR

Funding

Institut National Du Cancer INCA_10846
6 · The paper itself

Abstract

backgroundInterpretation of differences or changes in patient-reported outcome scores should not only consider statistical significance, but also clinical relevance. Accordingly, accurate determination of the minimally important difference (MID) is crucial to assess the effectiveness of health care interventions, as well as for sample size calculation. Several methods have been proposed to determine the MID. Our aim was to review the statistical methods used to determine MID in patient-reported outcome (PRO) questionnaires in cancer patients, focusing on the distribution- and anchor-based approaches and to present the variability of criteria used as well as possible limitations.

methodsWe performed a systematic search using PubMed. We searched for all cancer studies related to MID determination on a PRO questionnaire. Two reviewers independently screened titles and abstracts to identify relevant articles. Data were extracted from eligible articles using a predefined data collection form. Discrepancies were resolved by discussion and the involvement of a third reviewer.

resultsSixty-three articles were identified, of which 46 were retained for final analysis. Both distribution- and anchor-based approaches were used to assess the MID in 37 studies (80.4%). Different time points were used to apply the distribution-based method and the most frequently reported distribution was the 0.5 standard deviation at baseline. A change in a PRO external scale (N = 13, 30.2%) and performance status (N = 15, 34.9%) were the most frequently used anchors. The stability of the MID over time was rarely investigated and only 28.2% of studies used at least 3 assessment timepoints. The robustness of anchor-based MID was questionable in 37.2% of the studies where the minimal number of patients by anchor category was less than 20.

conclusionEfforts are needed to improve the quality of the methodology used for MID determination in PRO questionnaires used in oncology. In particular, increased attention to the sample size should be paid to guarantee reliable results. This could increase the use of these specific thresholds in future studies.

Indexed as

Patient Reported Outcome MeasuresHumansMedical OncologyNeoplasmsPsychometricsQuality of LifeAnchor-based approachDistribution-based approachMinimally important differencePatient-reported outcomes

Identifiers

PMID30537955
PMCPMC6288886
OpenAlexW2905297638

What OpenQuestion holds

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LicenceCC BY
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Registered trials

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.