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

Publication ,  Journal Article
Mantegazza, R; Wolfe, GI; Muppidi, S; Wiendl, H; Fujita, KP; O'Brien, FL; Booth, HDE; Howard, JF; REGAIN Study Group,
Published in: Neurology
January 26, 2021

OBJECTIVE: To 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. METHODS: Patients 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. RESULTS: A 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. CONCLUSION: Eculizumab 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.

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Published In

Neurology

DOI

EISSN

1526-632X

Publication Date

January 26, 2021

Volume

96

Issue

4

Start / End Page

e610 / e618

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Neurology & Neurosurgery
  • Myasthenia Gravis
  • Middle Aged
  • Male
  • Humans
  • Female
  • Complement Inactivating Agents
  • Antibodies, Monoclonal, Humanized
  • Adult
 

Citation

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Mantegazza, R., Wolfe, G. I., Muppidi, S., Wiendl, H., Fujita, K. P., O’Brien, F. L., … REGAIN Study Group, . (2021). Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension. Neurology, 96(4), e610–e618. https://doi.org/10.1212/WNL.0000000000011207
Mantegazza, Renato, Gil I. Wolfe, Srikanth Muppidi, Heinz Wiendl, Kenji P. Fujita, Fanny L. O’Brien, Heather D. E. Booth, James F. Howard, and James F. REGAIN Study Group. “Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension.Neurology 96, no. 4 (January 26, 2021): e610–18. https://doi.org/10.1212/WNL.0000000000011207.
Mantegazza R, Wolfe GI, Muppidi S, Wiendl H, Fujita KP, O’Brien FL, et al. Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension. Neurology. 2021 Jan 26;96(4):e610–8.
Mantegazza, Renato, et al. “Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension.Neurology, vol. 96, no. 4, Jan. 2021, pp. e610–18. Pubmed, doi:10.1212/WNL.0000000000011207.
Mantegazza R, Wolfe GI, Muppidi S, Wiendl H, Fujita KP, O’Brien FL, Booth HDE, Howard JF, REGAIN Study Group. Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension. Neurology. 2021 Jan 26;96(4):e610–e618.

Published In

Neurology

DOI

EISSN

1526-632X

Publication Date

January 26, 2021

Volume

96

Issue

4

Start / End Page

e610 / e618

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Neurology & Neurosurgery
  • Myasthenia Gravis
  • Middle Aged
  • Male
  • Humans
  • Female
  • Complement Inactivating Agents
  • Antibodies, Monoclonal, Humanized
  • Adult