Evidence map›Paper›PMID 30767274›Full record

ArticleMuscle & nerve2019

Long-term safety and efficacy of eculizumab in generalized myasthenia gravis.

Srikanth Muppidi, Kimiaki Utsugisawa, Michael Benatar, Hiroyuki Murai, Richard J Barohn, Isabel Illa, Saiju Jacob, John Vissing, Ted M Burns, John T Kissel and 11 more

2 registry-linked trialsOpen access · hybridAbstract read
In one paragraph

Article in Muscle & nerve, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 137 papers, 6 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
137citing papers in PubMed, 6 pooled it
18.8field-weighted citation impact, top 1% 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

137 citing papers in PubMed, 6 syntheses or guidelines pooled it, 243 citations in OpenAlex.

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  11. Eculizumab monotherapy for NMOSD: Data from PREVENT and its open-label extension.Multiple sclerosis (Houndmills, Basingstoke, England) · 2022
    Trial
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  14. Consistent improvement with eculizumab across muscle groups in myasthenia gravis.Annals of clinical and translational neurology · 2020
    Trial
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  17. Eculizumab improves fatigue in refractory generalized myasthenia gravis.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2019
    Trial
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77 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

21 authors at 18 institutions in 7 countries.

Srikanth MuppidiDepartment of Neurology and Neurosciences, Stanford University School of Medicine, Stanford, California, USA.
Kimiaki UtsugisawaDepartment of Neurology, Hanamaki General Hospital, Hanamaki, Japan.
Michael BenatarDepartment of Neurology, University of Miami Miller School of Medicine, Miami, Florida, USA.
Hiroyuki MuraiDepartment of Neurology, International University of Health and Welfare, Narita, Japan.
Richard J BarohnDepartment of Neurology, University of Kansas Medical Center, Kansas City, Kansas, USA.
Isabel IllaNeurology Department, Hospital Sant Pau, Autonomous University of Barcelona, Barcelona, Spain.
Saiju JacobQueen Elizabeth Neuroscience Centre and Wellcome Trust Clinical Research Facility, University Hospital Birmingham, Birmingham, United Kingdom.
John VissingDepartment of Neurology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark.
Ted M BurnsDepartment of Neurology, University of Virginia Health System, Charlottesville, Virginia, USA.
John T KisselDepartment of Neurology, The Ohio State University, Columbus, Ohio, USA.
Richard J NowakDepartment of Neurology, Yale School of Medicine, Yale University, New Haven, Connecticut, USA.
Henning AndersenDepartment of Neurology, Aarhus University Hospital, Aarhus, Denmark.
Carlos CasasnovasCentro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Spain.
Jan L de BleeckerNeuromuscular Reference Centre, Ghent University Hospital, Ghent, Belgium.
Tuan H VuDepartment of Neurology, University of South Florida, Tampa, Florida, USA.
Renato MantegazzaFoundation of the Carlo Besta Neurological Institute, IRCSS, Milan, Italy.
Fanny L O'BrienAlexion Pharmaceuticals, Boston, Massachusetts, USA.
Jing Jing WangAlexion Pharmaceuticals, Boston, Massachusetts, USA.
Kenji P FujitaAlexion Pharmaceuticals, Boston, Massachusetts, USA.
James F HowardDepartment of Neurology, University of North Carolina, Chapel Hill, North Carolina, USA.ORCID 0000-0002-7136-8617
Regain Study Group
Alexion Pharmaceuticals (United States) · USAarhus University Hospital · DKBellvitge University Hospital · ESFondazione IRCCS Istituto Neurologico Carlo Besta · ITGhent University Hospital · BEInternational University of Health and Welfare · JPNational Hospital Organization Hanamaki Hospital · JPQueen Elizabeth Hospital Birmingham · GBStanford University · USThe Ohio State University · USUniversitat Autònoma de Barcelona · ESUniversity of Copenhagen · DKUniversity of Kansas Medical Center · USUniversity of Miami · USUniversity of North Carolina at Chapel Hill · USUniversity of South Florida · USUniversity of Virginia Health System · USYale University · US

Funding

Yale Clinical and Translational Science Award (U Component)UL1TR001863 · NCATS · YALE UNIVERSITY · PI John H. Krystal, LUCILA OHNO-MACHADO · 2016 to 2026
$102.9M
NCATS NIH HHS UL1 TR001863
6 · The paper itself

Abstract

introductionEculizumab is effective and well tolerated in patients with antiacetylcholine receptor antibody-positive refractory generalized myasthenia gravis (gMG; REGAIN; NCT01997229). We report an interim analysis of an open-label extension of REGAIN, evaluating eculizumab's long-term safety and efficacy.

methodsEculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients.

resultsThe safety profile of eculizumab was consistent with REGAIN; no cases of meningococcal infection were reported during the interim analysis period. Myasthenia gravis exacerbation rate was reduced by 75% from the year before REGAIN (P < 0.0001). Improvements with eculizumab in activities of daily living, muscle strength, functional ability, and quality of life in REGAIN were maintained through 3 years; 56% of patients achieved minimal manifestations or pharmacological remission. Patients who had received placebo during REGAIN experienced rapid and sustained improvements during open-label eculizumab (P < 0.0001). DISCUSSION: These findings provide evidence for the long-term safety and sustained efficacy of eculizumab for refractory gMG. Muscle Nerve 2019.

Indexed as

Activities of Daily LivingAdultAngioedemaAntibodies, Monoclonal, HumanizedAspergillosisComplement Inactivating AgentsDisease ProgressionFemaleHeart DiseasesHumansInjection Site ReactionLongitudinal StudiesMaleMeningococcal InfectionsMeningococcal VaccinesMiddle AgedAntibodies, Monoclonal, HumanizedComplement Inactivating AgentseculizumabMeningococcal VaccineseculizumabMG-ADLMGCMG-QOL15myasthenia gravisQMG

Identifiers

PMID30767274
PMCPMC6619057
OpenAlexW2912901461

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.