Guntis Karelis1, Rodica Balasa2, Jan L De Bleecker3, Tima Stuchevskaya4, Andres Villa5, Philip Van Damme6, Emmeline Lagrange7, Jeannine M Heckmann8, Michael Nicolle9, Crisandra Vilciu10, Vera Bril11,12, Elsa Mondou13, Rhonda Griffin13, Junliang Chen13, Waleska Henriquez13, Beatriz Garcia14, Sandra Camprubi14, Jaume Ayguasanosa14. 1. Department of Infectology and Dermatology, Rīga Stradiņš University, Rīga, Latvia, guntis.karelis@gmail.com. 2. Spitalul Clinic Judeţean de Urgenţă Târgu Mureş, Clinica Neurologie I, Târgu Mureş, Romania. 3. Department of Neurology and Neuromuscular Reference Center, Ghent University Hospital, Ghent, Belgium. 4. City Hospital No. 2, Department of Neurology (Neuromuscular Center), Saint Petersburg, Russian Federation. 5. Hospital General de Agudos Dr. J. M. Ramos Mejia Urquiza 609, CABA, Buenos Aires, Argentina. 6. Department of Neurology, University Hospitals Leuven, Department of Neurosciences, KU Leuven and Center for Brain and Disease Research, VIB, Leuven, Belgium. 7. Service de Neurologie, Centre Hospitalier Universitaire de Grenoble, Grenoble, France. 8. Division of Neurology, Department of Medicine, University of Cape Town, New Groote Schuur Hospital, Cape Town, South Africa. 9. Department of Clinical Neurological Sciences, Western University, London, Ontario, Canada. 10. Department of Neurology, Institutul Clinic Fundeni, Bucharest, Romania. 11. Ellen and Martin Prosserman Centre for Neuromuscular Diseases, Division of Neurology, Department of Medicine, University Health Network, University of Toronto, Toronto, Ontario, Canada. 12. Institute for Research and Medical Consultations, Imam Abdulrahman Bin Faisal University, Dammam, Saudi Arabia. 13. Grifols Bioscience Research Group, Research Triangle Park, North Carolina, USA. 14. Grifols Bioscience Research Group, Sant Cugat del Vallès, Spain.
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.
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.