Evidence map›Paper›PMID 30905021›Full record

Trial reportQuality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation2019

Eculizumab improves fatigue in refractory generalized myasthenia gravis.

Henning Andersen, Renato Mantegazza, Jing Jing Wang, Fanny O'Brien, Kaushik Patra, James F Howard, REGAIN Study Group

Erratum issued 2 registry-linked trialsOpen access · hybridAbstract readClinical Trial, Phase IIIRandomized Controlled Trial
In one paragraph

Trial report in Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation, 2019. 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 2 registered trials, which are not on this map. Cited by 33 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
33citing papers in PubMed, 1 pooled it
4.4field-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.

NCT01997229 phase3completednot on this map

A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Eculizumab in Subjects With Refractory Generalized Myasthenia Gravis (gMG)

TypeinterventionalSponsorAlexion Pharmaceuticals, Inc.Ran2013 to 2016Enrolled125ConditionsRefractory Generalized Myasthenia GravisArmsEculizumab, Placebo
NCT02301624 phase3completednot on this map

A Phase III, Open-label Extension Trial of ECU-MG-301 to Evaluate the Safety and Efficacy of Eculizumab in Subjects With Refractory Generalized Myasthenia Gravis (gMG)

TypeinterventionalSponsorAlexion Pharmaceuticals, Inc.Ran2014 to 2019Enrolled117ConditionsRefractory Generalized Myasthenia GravisArmsEculizumab, Placebo
3 · Its place in the literature

Who cites it

33 citing papers in PubMed, 1 synthesis or guideline pooled it, 54 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Trial
  4. Review
  5. Article
  6. Observational
  7. Review
  8. Review
  9. Article
  10. New and Emerging Biological Therapies for Myasthenia Gravis: A Focussed Review for Clinical Decision-Making.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2025
    Review
  11. Review
  12. Article
  13. Eculizumab in thymoma-associated myasthenia gravis: a real-world cohort study.Therapeutic advances in neurological disorders · 2024
    Article
  14. Article
  15. Article
  16. Review
  17. Ravulizumab in Myasthenia Gravis: A Review of the Current Evidence.Neuropsychiatric disease and treatment · 2023
    Review
  18. Long-term effects of anti-Frontiers in neurology · 2023
    Article
  19. Review
  20. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

7 authors at 4 institutions in 3 countries.

Henning AndersenDepartment of Neurology, Aarhus University Hospital, Indgang J, Plan 5, Krydspunkt 504, Palle Juul-Jensens Boulevard, 8200, Aarhus N, Denmark. hennande@rm.dk.ORCID http://orcid.org/0000-0002-9082-2694
Renato MantegazzaFoundation of the Carlo Besta Neurological Institute, IRCCS, Milan, Italy.ORCID http://orcid.org/0000-0002-9810-5737
Jing Jing WangFormerly of Alexion Pharmaceuticals, Inc., New Haven, CT, USA.
Fanny O'BrienAlexion Pharmaceuticals, Inc., New Haven, CT, USA.
Kaushik PatraAlexion Pharmaceuticals, Inc., New Haven, CT, USA.
James F HowardDepartment of Neurology, UNC School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.ORCID http://orcid.org/0000-0002-7136-8617
REGAIN Study Group
Fondazione IRCCS Istituto Neurologico Carlo Besta · ITAarhus University Hospital · DKUniversity of North Carolina at Chapel Hill · USAlexion Pharmaceuticals (United States) · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purposeTo evaluate the effect of eculizumab on perceived fatigue in patients with anti-acetylcholine receptor antibody-positive, refractory, generalized myasthenia gravis (MG) using the Quality of Life in Neurological Disorders (Neuro-QOL) Fatigue subscale, and to evaluate correlations between improvements in Neuro-QOL Fatigue and other clinical endpoints.

methodsNeuro-QOL Fatigue, MG Activities of Daily Living (MG-ADL), Quantitative MG (QMG), and the 15-item MG Quality of Life (MG-QOL15) scales were administered during the phase 3, randomized, placebo-controlled REGAIN study (eculizumab, n = 62; placebo, n = 63) and subsequent open-label extension (OLE). Data were analyzed using repeated-measures models. Correlations between changes in Neuro-QOL Fatigue and in MG-ADL, QMG, and MG-QOL15 scores were determined at REGAIN week 26.

resultsAt REGAIN week 26, eculizumab-treated patients showed significantly greater improvements in Neuro-QOL Fatigue scores than placebo-treated patients (consistent with improvements in MG-ADL, QMG, and MG-QOL15 scores previously reported in REGAIN). Improvements with eculizumab were sustained through OLE week 52. Correlations between Neuro-QOL Fatigue and MG-QOL15, MG-ADL, and QMG scores were strong for eculizumab-treated patients at REGAIN week 26, and strong, moderate, and weak, respectively, for placebo-treated patients.

conclusionsCompared with placebo, eculizumab was associated with improvements in perceived fatigue that strongly correlated with improvements in MG-specific outcome measures. Trial ID Registration: NCT01997229, NCT02301624.

Indexed as

Activities of Daily LivingQuality of LifeAntibodies, Monoclonal, HumanizedFatigueFemaleHumansMaleMiddle AgedMyasthenia GravisOutcome Assessment, Health CarePlacebosReceptors, CholinergicAntibodies, Monoclonal, HumanizedeculizumabPlacebosReceptors, CholinergicComplementEculizumabFatigueMyasthenia gravisNeuro-QOL FatigueQuality of lifeTerminal complement inhibition

Identifiers

PMID30905021
PMCPMC6620379
OpenAlexW2940643432

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.