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

(2019) MUSCLE & NERVE. 60(1). p.14-24
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Abstract
Introduction: Eculizumab 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. Methods: Eculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients. Results: The 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
Keywords
eculizumab, MG-ADL, MGC, MG-QOL15, myasthenia gravis, QMG, COMPLEMENT INHIBITOR ECULIZUMAB, ACETYLCHOLINE-RECEPTOR, DOUBLE-BLIND, ANTIBODY, THERAPY

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MLA
Muppidi, Srikanth et al. “Long-term Safety and Efficacy of Eculizumab in Generalized Myasthenia Gravis.” MUSCLE & NERVE 60.1 (2019): 14–24. Print.
APA
Muppidi, Srikanth, Utsugisawa, K., Benatar, M., Murai, H., Barohn, R. J., Illa, I., Jacob, S., et al. (2019). Long-term safety and efficacy of eculizumab in generalized myasthenia gravis. MUSCLE & NERVE, 60(1), 14–24.
Chicago author-date
Muppidi, Srikanth, Kimiaki Utsugisawa, Michael Benatar, Hiroyuki Murai, Richard J Barohn, Isabel Illa, Saiju Jacob, et al. 2019. “Long-term Safety and Efficacy of Eculizumab in Generalized Myasthenia Gravis.” Muscle & Nerve 60 (1): 14–24.
Chicago author-date (all authors)
Muppidi, Srikanth, Kimiaki Utsugisawa, Michael Benatar, Hiroyuki Murai, Richard J Barohn, Isabel Illa, Saiju Jacob, John Vissing, Ted M Burns, John T Kissel, Richard J Nowak, Henning Andersen, Carlos Casasnovas, Jan De Bleecker, Tuan H Vu, Renato Mantegazza, Fanny L O’Brien, Jing Jing Wang, Kenji P Fujita, and James F Howard Jr. 2019. “Long-term Safety and Efficacy of Eculizumab in Generalized Myasthenia Gravis.” Muscle & Nerve 60 (1): 14–24.
Vancouver
1.
Muppidi S, Utsugisawa K, Benatar M, Murai H, Barohn RJ, Illa I, et al. Long-term safety and efficacy of eculizumab in generalized myasthenia gravis. MUSCLE & NERVE. 2019;60(1):14–24.
IEEE
[1]
S. Muppidi et al., “Long-term safety and efficacy of eculizumab in generalized myasthenia gravis,” MUSCLE & NERVE, vol. 60, no. 1, pp. 14–24, 2019.
@article{8618356,
  abstract     = {Introduction: Eculizumab 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.
Methods: Eculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients.
Results: The 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},
  author       = {Muppidi, Srikanth and Utsugisawa, Kimiaki and Benatar, Michael and Murai, Hiroyuki and Barohn, Richard J and Illa, Isabel and Jacob, Saiju and Vissing, John and Burns, Ted M and Kissel, John T and Nowak, Richard J and Andersen, Henning and Casasnovas, Carlos and De Bleecker, Jan and Vu, Tuan H and Mantegazza, Renato and O'Brien, Fanny L and Wang, Jing Jing and Fujita, Kenji P and Howard, James F, Jr.},
  issn         = {0148-639x},
  journal      = {MUSCLE & NERVE},
  keywords     = {eculizumab,MG-ADL,MGC,MG-QOL15,myasthenia gravis,QMG,COMPLEMENT INHIBITOR ECULIZUMAB,ACETYLCHOLINE-RECEPTOR,DOUBLE-BLIND,ANTIBODY,THERAPY},
  language     = {eng},
  number       = {1},
  pages        = {14--24},
  title        = {Long-term safety and efficacy of eculizumab in generalized myasthenia gravis},
  url          = {http://dx.doi.org/10.1002/mus.26447},
  volume       = {60},
  year         = {2019},
}

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