Evidence map›Paper›PMID 33229455›Full record

Trial reportNeurology2021

Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension.

Renato Mantegazza, Gil I Wolfe, Srikanth Muppidi, Heinz Wiendl, Kenji P Fujita, Fanny L O'Brien, Heather D E Booth, James F Howard, REGAIN Study Group

2 registry-linked trialsOpen access · hybridAbstract readClinical Trial, Phase IIIRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Neurology, 2021. 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 40 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
40citing papers in PubMed, 2 pooled it
3.7field-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

40 citing papers in PubMed, 2 syntheses or guidelines pooled it, 76 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Trial
  4. Article
  5. Article
  6. Review
  7. Article
  8. Article
  9. Review
  10. Eculizumab in refractory myasthenia gravis: a real-world single-center experience.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2025
    Article
  11. Article
  12. Therapeutic challenges and unmet needs in the management of myasthenia gravis: an Italian expert opinion.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2024
    Review
  13. Review
  14. Article
  15. Review
  16. Article
  17. Article
  18. Eculizumab for myasthenic exacerbation during treatment with immune-checkpoint inhibitors.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2024
    Article
  19. An angel or a devil? Current view on the role of CD8Journal of translational medicine · 2024
    Review
  20. Review
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 1 institution in 3 countries.

Renato MantegazzaFrom the Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.), Milan, Italy; Department of Neurology (G.I.W.), Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, State University of New York; Department of Neurology and Neurological Sciences (S.M.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (H.W.), University of Münster, Germany; Alnylam Pharmaceuticals (K.P.F.), Cambridge, MA; Alexion Pharmaceuticals (F.L.O.), Boston, MA; Oxford PharmaGenesis (H.D.E.B.), UK; and Department of Neurology (J.F.H.), University of North Carolina, Chapel Hill. K.P.F. was formerly affiliated with Alexion Pharmaceuticals, Boston, MA.ORCID http://orcid.org/0000-0002-9810-5737
Gil I WolfeFrom the Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.), Milan, Italy; Department of Neurology (G.I.W.), Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, State University of New York; Department of Neurology and Neurological Sciences (S.M.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (H.W.), University of Münster, Germany; Alnylam Pharmaceuticals (K.P.F.), Cambridge, MA; Alexion Pharmaceuticals (F.L.O.), Boston, MA; Oxford PharmaGenesis (H.D.E.B.), UK; and Department of Neurology (J.F.H.), University of North Carolina, Chapel Hill. K.P.F. was formerly affiliated with Alexion Pharmaceuticals, Boston, MA.ORCID http://orcid.org/0000-0003-1471-9617
Srikanth MuppidiFrom the Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.), Milan, Italy; Department of Neurology (G.I.W.), Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, State University of New York; Department of Neurology and Neurological Sciences (S.M.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (H.W.), University of Münster, Germany; Alnylam Pharmaceuticals (K.P.F.), Cambridge, MA; Alexion Pharmaceuticals (F.L.O.), Boston, MA; Oxford PharmaGenesis (H.D.E.B.), UK; and Department of Neurology (J.F.H.), University of North Carolina, Chapel Hill. K.P.F. was formerly affiliated with Alexion Pharmaceuticals, Boston, MA.ORCID http://orcid.org/0000-0002-2773-2506
Heinz WiendlFrom the Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.), Milan, Italy; Department of Neurology (G.I.W.), Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, State University of New York; Department of Neurology and Neurological Sciences (S.M.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (H.W.), University of Münster, Germany; Alnylam Pharmaceuticals (K.P.F.), Cambridge, MA; Alexion Pharmaceuticals (F.L.O.), Boston, MA; Oxford PharmaGenesis (H.D.E.B.), UK; and Department of Neurology (J.F.H.), University of North Carolina, Chapel Hill. K.P.F. was formerly affiliated with Alexion Pharmaceuticals, Boston, MA.ORCID http://orcid.org/0000-0003-4310-3432
Kenji P FujitaFrom the Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.), Milan, Italy; Department of Neurology (G.I.W.), Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, State University of New York; Department of Neurology and Neurological Sciences (S.M.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (H.W.), University of Münster, Germany; Alnylam Pharmaceuticals (K.P.F.), Cambridge, MA; Alexion Pharmaceuticals (F.L.O.), Boston, MA; Oxford PharmaGenesis (H.D.E.B.), UK; and Department of Neurology (J.F.H.), University of North Carolina, Chapel Hill. K.P.F. was formerly affiliated with Alexion Pharmaceuticals, Boston, MA.ORCID http://orcid.org/0000-0003-4320-7557
Fanny L O'BrienFrom the Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.), Milan, Italy; Department of Neurology (G.I.W.), Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, State University of New York; Department of Neurology and Neurological Sciences (S.M.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (H.W.), University of Münster, Germany; Alnylam Pharmaceuticals (K.P.F.), Cambridge, MA; Alexion Pharmaceuticals (F.L.O.), Boston, MA; Oxford PharmaGenesis (H.D.E.B.), UK; and Department of Neurology (J.F.H.), University of North Carolina, Chapel Hill. K.P.F. was formerly affiliated with Alexion Pharmaceuticals, Boston, MA.ORCID http://orcid.org/0000-0002-5840-4089
Heather D E BoothFrom the Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.), Milan, Italy; Department of Neurology (G.I.W.), Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, State University of New York; Department of Neurology and Neurological Sciences (S.M.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (H.W.), University of Münster, Germany; Alnylam Pharmaceuticals (K.P.F.), Cambridge, MA; Alexion Pharmaceuticals (F.L.O.), Boston, MA; Oxford PharmaGenesis (H.D.E.B.), UK; and Department of Neurology (J.F.H.), University of North Carolina, Chapel Hill. K.P.F. was formerly affiliated with Alexion Pharmaceuticals, Boston, MA.ORCID http://orcid.org/0000-0003-2915-5578
James F HowardFrom the Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.), Milan, Italy; Department of Neurology (G.I.W.), Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, State University of New York; Department of Neurology and Neurological Sciences (S.M.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (H.W.), University of Münster, Germany; Alnylam Pharmaceuticals (K.P.F.), Cambridge, MA; Alexion Pharmaceuticals (F.L.O.), Boston, MA; Oxford PharmaGenesis (H.D.E.B.), UK; and Department of Neurology (J.F.H.), University of North Carolina, Chapel Hill. K.P.F. was formerly affiliated with Alexion Pharmaceuticals, Boston, MA. howardj@neurology.unc.edu.ORCID http://orcid.org/0000-0002-7136-8617
REGAIN Study Group
Alnylam Pharmaceuticals (United States) · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveTo evaluate whether eculizumab helps patients with anti-acetylcholine receptor-positive (AChR+) refractory generalized myasthenia gravis (gMG) achieve the Myasthenia Gravis Foundation of America (MGFA) post-intervention status of minimal manifestations (MM), we assessed patients' status throughout REGAIN (Safety and Efficacy of Eculizumab in AChR+ Refractory Generalized Myasthenia Gravis) and its open-label extension.

methodsPatients who completed the REGAIN randomized controlled trial and continued into the open-label extension were included in this tertiary endpoint analysis. Patients were assessed for the MGFA post-intervention status of improved, unchanged, worse, MM, and pharmacologic remission at defined time points during REGAIN and through week 130 of the open-label study.

resultsA total of 117 patients completed REGAIN and continued into the open-label study (eculizumab/eculizumab: 56; placebo/eculizumab: 61). At week 26 of REGAIN, more eculizumab-treated patients than placebo-treated patients achieved a status of improved (60.7% vs 41.7%) or MM (25.0% vs 13.3%; common OR: 2.3; 95% CI: 1.1-4.5). After 130 weeks of eculizumab treatment, 88.0% of patients achieved improved status and 57.3% of patients achieved MM status. The safety profile of eculizumab was consistent with its known profile and no new safety signals were detected.

conclusionEculizumab led to rapid and sustained achievement of MM in patients with AChR+ refractory gMG. These findings support the use of eculizumab in this previously difficult-to-treat patient population. CLINICALTRIALSGOV IDENTIFIER: REGAIN, NCT01997229; REGAIN open-label extension, NCT02301624. CLASSIFICATION OF EVIDENCE: This study provides Class II evidence that, after 26 weeks of eculizumab treatment, 25.0% of adults with AChR+ refractory gMG achieved MM, compared with 13.3% who received placebo.

Indexed as

AdultAntibodies, Monoclonal, HumanizedComplement Inactivating AgentsFemaleHumansMaleMiddle AgedMyasthenia GravisTreatment OutcomeAntibodies, Monoclonal, HumanizedComplement Inactivating Agentseculizumab

Identifiers

PMID33229455
PMCPMC7905790
OpenAlexW3106936335

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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.