@article{187d84e470ae44daa6e7c40040ba0df5,
title = "A Phase 3 Multicenter, Prospective, Open-Label Efficacy and Safety Study of Immune Globulin (Human) 10\% Caprylate/Chromatography Purified in Patients with Myasthenia Gravis Exacerbations",
abstract = "Background: Myasthenia gravis (MG) is an autoimmune disorder affecting neuromuscular transmission. Exacerbations may involve increasing bulbar weakness and/or sudden respiratory failure, both of which can be critically disabling. Management of MG exacerbations includes plasma exchange and intravenous immunoglobulin (IVIG); they are equally effective, but patients experience fewer side effects with IVIG. The objective of this study was to assess the efficacy and safety of immune globulin caprylate/chromatography purified (IGIV-C) in subjects with MG exacerbations. Methods: This prospective, open-label, non-controlled 28-day clinical trial was conducted in adults with MG Foundation of America class IVb or V status. Subjects received IGIV-C 2 g/kg over 2 consecutive days (1 g/kg/day) and were assessed for efficacy/safety on Days 7, 14, 21, and 28. The primary efficacy endpoint was the change from Baseline in quantitative MG (QMG) score to Day 14. Secondary endpoints of clinical response, Baseline to Day 14, included at least a 3-point decrease in QMG and MG Composite and a 2-point decrease in MG-activities of daily living (MG-ADL). Results: Forty-nine subjects enrolled. The change in QMG score at Day 14 was significant (p < 0.001) in the Evaluable (-6.4, n = 43) and Safety (-6.7, n = 49) populations. Among evaluable subjects, Day 14 response rates were 77, 86, and 88\% for QMG, MG Composite, and MG-ADL, respectively. IGIV-C showed good tolerability with no serious adverse events. Conclusions: The results of this study show that IGIV-C was effective, safe, and well tolerated in the treatment of MG exacerbations.",
keywords = "Exacerbations, Immune globulin caprylate/ chromatography purified, IV immune globulin, Myasthenia gravis",
author = "Guntis Karelis and Rodica Balasa and \{De Bleecker\}, \{Jan L.\} and Tima Stuchevskaya and Andres Villa and \{Van Damme\}, Philip and Emmeline Lagrange and Heckmann, \{Jeannine M.\} and Michael Nicolle and Crisandra Vilciu and Vera Bril and Elsa Mondou and Rhonda Griffin and Junliang Chen and Waleska Henriquez and Beatriz Garcia and Sandra Camprubi and Jaume Ayguasanosa",
note = "Funding Information: aDepartment of Infectology and Dermatology, Rīga Stradiņ{\v s} University, Rīga, Latvia; bSpitalul Clinic Jude{\c t}ean de Urgen{\c t}{\u a} T{\^a}rgu Mure{\c s}, Clinica Neurologie I, T{\^a}rgu Mure{\c s}, Romania; cDepartment of Neurology and Neuromuscular Reference Center, Ghent University Hospital, Ghent, Belgium; dCity Hospital No. 2, Department of Neurology (Neuromuscular Center), Saint Petersburg, Russia; eHospital General de Agudos Dr. J. M. Ramos Mejia Urquiza 609, CABA, Buenos Aires, Argentina; fDepartment of Neurology, University Hospitals Leuven, Department of Neurosciences, KU Leuven and Center for Brain and Disease Research, VIB, Leuven, Belgium; gService de Neurologie, Centre Hospitalier Universitaire de Grenoble, Grenoble, France; hDivision of Neurology, Department of Medicine, University of Cape Town, New Groote Schuur Hospital, Cape Town, South Africa; iDepartment of Clinical Neurological Sciences, Western University, London, ON, Canada; jDepartment of Neurology, Institutul Clinic Fundeni, Bucharest, Romania; kEllen and Martin Prosserman Centre for Neuromuscular Diseases, Division of Neurology, Department of Medicine, University Health Network, University of Toronto, Toronto, ON, Canada; lInstitute for Research and Medical Consultations, Imam Abdulrahman Bin Faisal University, Dammam, Saudi Arabia; mGrifols Bioscience Research Group, Research Triangle Park, NC, USA; nGrifols Bioscience Research Group, Sant Cugat del Vall{\`e}s, Spain Funding Information: Bleeker reports receiving financial compensation from Grifols for the conduct of this study; P.V.D. reports non-financial support from CSL Behring, Pfizer, and Alexion Pharmaceuticals outside the submitted work; J.M.H. reports non-financial support from University of Cape Town during the conduct of the study; V.B. reports receiving grants from the University Health Network during the conduct of the study, grants and personal fees from CSL Behring, UCB, Octapharma, Takeda (Shire, Baxalta), Alnylam, personal fees from Akcea and Pfizer outside the submitted work; E.M. reports being an employee of Grifols Therapeutics LLC; R.G. reports being an employee of Grifols Therapeutics LLC; J.C. reports being an employee of Grifols Therapeutics LLC; W.H. reports being an employee of Grifols Therapeutics LLC; Garcia reports being an employee of Grifols Therapeutics LLC; S.C. reports being an employee of Grifols Therapeutics LLC; J.A. reports being an employee of Grifols Therapeutics LLC. Publisher Copyright: {\textcopyright} 2019 S. Karger AG, Basel. Copyright: All rights reserved.",
year = "2019",
month = nov,
day = "1",
doi = "10.1159/000502818",
language = "English",
volume = "81",
pages = "223--230",
journal = "European Neurology",
issn = "0014-3022",
publisher = "S. Karger AG",
number = "5-6",
}