Evidence map›Paper›PMID 37166675›Full record

ArticleNeurology and therapy2023

Measuring Overall Severity of Myasthenia Gravis (MG): Evidence for the Added Value of the MG Symptoms PRO.

Antoine Regnault, Thomas Morel, Christine de la Loge, Flora Mazerolle, Henry J Kaminski, Ali A Habib

Erratum issued Registry-linked trialOpen access · goldAbstract read
In one paragraph

Article in Neurology and therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to trial NCT03052751 (A Multicenter, Randomized, Investigator- and Subject-Blind, Placebo-Controlled, Treatment Sequence Study Evaluating the Safety, Tolerability, and Efficacy of UCB7665 in Subjects With Moderate to Severe Myasthenia Gravis), which is not on this map. Cited by 18 papers.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed
4.3field-weighted citation impact, top 5% 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.

NCT03052751 phase2completednot on this map

A Multicenter, Randomized, Investigator- and Subject-Blind, Placebo-Controlled, Treatment Sequence Study Evaluating the Safety, Tolerability, and Efficacy of UCB7665 in Subjects With Moderate to Severe Myasthenia Gravis

TypeinterventionalSponsorUCB Biopharma S.P.R.L.Ran2017 to 2018Enrolled43ConditionsMyasthenia GravisArmsUCB7665, Placebo
3 · Its place in the literature

Who cites it

18 citing papers in PubMed, 21 citations in OpenAlex.

  1. Trial
  2. Trial
  3. Trial
  4. Muscles (Basel, Switzerland) · 2026
    Review
  5. Italian recommendations for the diagnosis and treatment of myasthenia gravis.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2026
    Article
  6. Article
  7. Article
  8. Article
  9. Review
  10. Article
  11. Review
  12. Article
  13. Myasthenia gravis: the future is here.The Journal of clinical investigation · 2024
    Review
  14. Article
  15. Article
  16. Article
  17. Article
  18. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

6 authors at 3 institutions in 2 countries.

Antoine RegnaultModus Outcomes, A Division of THREAD, Lyon, France.ORCID http://orcid.org/0000-0001-8234-2896
Thomas MorelUCB Pharma SRL, Allée de la Recherche, 60, 1070, Brussels, Belgium. Thomas.Morel@ucb.com.ORCID http://orcid.org/0000-0002-0690-5359
Christine de la LogePCOM Analytics, Avallon, France.ORCID http://orcid.org/0000-0002-5896-5927
Flora MazerolleModus Outcomes, A Division of THREAD, Lyon, France.ORCID http://orcid.org/0000-0002-3854-632X
Henry J KaminskiDepartment of Neurology and Rehabilitation Medicine, George Washington University, Washington, DC, USA.ORCID http://orcid.org/0000-0002-8195-0141
Ali A HabibDepartment of Neurology, University of California, Irvine, CA, USA.ORCID http://orcid.org/0000-0003-3432-9747
George Washington University · USUCB Pharma (Belgium) · BEUniversity of California, Irvine · US

Funding

Rare Disease Network for Myasthenia GravisU54NS115054 · NINDS · GEORGE WASHINGTON UNIVERSITY · PI HENRY J KAMINSKI · 2019 to 2026
$11.9M
NINDS NIH HHS U54 NS115054
6 · The paper itself

Abstract

introductionAccurate measurement of myasthenia gravis (MG) severity is required for appropriate clinical monitoring of patients with MG and assessment of the benefit of new treatments in clinical trials. Our objective was to explore how MG severity can be measured and to determine how the newly developed MG Symptoms Patient-Reported Outcome (PRO) instrument complements the available measures of MG severity.

methodsThe conceptual coverage of the Quantitative MG (QMG), MG Composite (MGC), MG-Activities of Daily Living (MG-ADL), and MG Symptoms PRO was scrutinized against core symptoms of MG: muscle weakness in three muscle groups (ocular, bulbar, and respiratory), muscle weakness fatigability, and physical fatigue. Post hoc analyses of the MG0002 study, a Phase 2a clinical trial of rozanolixizumab in adults with moderate to severe generalized MG, included correlation and Rasch model analyses.

resultsThe qualitative appraisal highlighted that only the MG Symptoms PRO captured physical fatigue. Data from 541 assessments (43 unique patients) were used for the analyses. Correlations ranged between 0.56 and 0.74 for the MG-ADL, QMG, MGC, and MG Symptoms PRO Muscle Weakness Fatigability score, and between 0.20 and 0.71 for the MG Symptoms PRO scores focusing on independent muscle groups. Analyses with the Rasch model estimated a meaningful continuum of severity of MG, including all items, except ocular muscles, from the four instruments. The QMG and MG Symptoms PRO had the broadest coverage of the MG severity continuum. Muscle fatigability and physical fatigue were more characteristic of low severity while bulbar weakness indicated more severe MG.

conclusionThe severity of MG can be reflected in a meaningful continuum underpinned by the MG-specific outcome measures. Only ocular muscle manifestations were shown to reflect a possibly different facet of MG severity. With its modular nature and comprehensive content, the MG Symptoms PRO provides complementary information to the outcome measures widely used in MG.

trial registrationClinicalTrials.gov identifier: NCT03052751.

Indexed as

Clinician-reported outcomesMyasthenia gravisOutcome measurePatient-reported outcomesSymptoms

Identifiers

PMID37166675
PMCPMC10444722
OpenAlexW4376131314

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

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.