Long-term safety and efficacy of eculizumab in generalized myasthenia gravis
Muppidi, Srikanth (Stanford University School of Medicine)
Utsugisawa, Kimiaki (Hanamaki General Hospital)
Benatar, Michael (University of Miami Miller School of Medicine)
Murai, Hiroyuki (International University of Health and Welfare)
Barohn, Richard J. (University of Kansas Medical Center)
Illa, Isabel (Hospital Universitari Vall d'Hebron)
Jacob, Saiju (Birmingham Women's and Children's Hospital (Regne Unit))
Vissing, John (University of Copenhagen)
Burns, Ted M. (University of Virginia Health System)
Kissel, John T. (The Ohio State University)
Nowak, Richard J. (Yale University)
Andersen, Henning (Aarhus University Hospital (Aarhus, Dinamarca))
Casasnovas, Carlos (Hospital Universitari de Bellvitge)
De Bleecker, Jan (Universitair Ziekenhuis Gent)
Vu, Tuan H. (University of South Florida)
Mantegazza, Renato (Fondazione IRCCS Istituto Neurologico Carlo Besta (R.M.) (Milà, Itàlia))
O'Brien, Fanny L. (Alexion Pharmaceuticals)
Wang, Jing Jing (Alexion Pharmaceuticals)
Fujita, Kenji P (Alexion Pharmaceuticals)
Howard, James F. (University of North Carolina)

Data:	2019
Resum:	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.
Drets:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original.
Llengua:	Anglès
Document:	Article ; recerca ; Versió publicada
Matèria:	Eculizumab ; MG-ADL ; MGC ; MG-QOL15 ; Myasthenia gravis ; QMG
Publicat a:	Muscle & nerve, Vol. 60 (march 2019) , p. 14-24, ISSN 1097-4598

DOI: 10.1002/mus.26447
PMID: 30767274

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