Response to eculizumab in patients with myasthenia gravis recently treated with chronic IVIg: a subgroup analysis of REGAIN and its open-label extension study. (April 2020)

• Record Type: Journal Article
• Authors: Jacob, Saiju; Murai, Hiroyuki; Utsugisawa, Kimiaki; Nowak, Richard J.; Wiendl, Heinz; Fujita, Kenji P.; O'Brien, Fanny; Howard, James F.
• Other Names: Mazia Claudio Gabriel non-byline-author.; Wilken Miguel non-byline-author.; Barroso Fabio non-byline-author.; Saba Juliet non-byline-author.; De Bleecker Jan non-byline-author.; Van den Abeele Guy non-byline-author.; de Koning Kathy non-byline-author.; De Mey Katrien non-byline-author.; Carvalho Alzira Alves de Siqueira non-byline-author.; Brockhausen Igor Dias non-byline-author.; Feder David non-byline-author.; Ambrosio Daniel non-byline-author.; César Pamela non-byline-author.; Melo Ana Paula non-byline-author.; Ribeiro Renata Martins non-byline-author.; Rocha Rosana non-byline-author.; Rosa Bruno Bezerra non-byline-author.; Veiga Thabata non-byline-author.; da Silva Luiz Augusto non-byline-author.; Engel Murilo Santos non-byline-author.; Gonçalves Geraldo Jordana non-byline-author.; Nagane Yuriko non-byline-author.; Kamegamori Ikuko non-byline-author.; Tsuda Tomoko non-byline-author.; Fujii Yuko non-byline-author.; Futono Kazumi non-byline-author.; Ozawa Yukiko non-byline-author.; Mizugami Aya non-byline-author.; Saito Yuka non-byline-author.; van der Kooi Anneke non-byline-author.; de Visser Marianne non-byline-author.; Gibson Tamar non-byline-author.; Kim Seung Min non-byline-author.; Jeong JinWoo non-byline-author.; Jung Ha-Neul non-byline-author.; Kim Yool-hee non-byline-author.; Lee Hyung Seok non-byline-author.; Shin Ha Young non-byline-author.; Hwang Eun Bi non-byline-author.; Shin Miju non-byline-author.; Carbonell Josep Gamez non-byline-author.; Sune Pilar non-byline-author.; Figueras Maria Salvado non-byline-author.; Gili Gisela non-byline-author.; Mazuela Gonzalo non-byline-author.; Piehl Fredrik non-byline-author.; Hietala Albert non-byline-author.; Bjarbo Lena non-byline-author.; Erdem-Ozdamar Sevim non-byline-author.; Bekircan-Kurt Can Ebru non-byline-author.; Acar Nazire Pinar non-byline-author.; Yilmaz Ezgi non-byline-author.; Caliskan Yagmur non-byline-author.; Orsel Gulsah non-byline-author.; Amato Anthony non-byline-author.; Cochrane Thomas non-byline-author.; Salajegheh Mohammed non-byline-author.; Roe Kristen non-byline-author.; Amato Katherine non-byline-author.; Toska Shirli non-byline-author.; McKinnon Jonathan non-byline-author.; Haar Laura non-byline-author.; McKinnon Naya non-byline-author.; Alcon Karan non-byline-author.; McKenna Kaitlyn non-byline-author.; Sattar Nadia non-byline-author.; Daniels Kevin non-byline-author.; Jeffery Dennis non-byline-author.; Mozaffar Tahseen non-byline-author.; Cash Tiyonnoh non-byline-author.; Goyal Namita non-byline-author.; Roy Gulmohor non-byline-author.; Mathew Veena non-byline-author.; Maqsood Fatima non-byline-author.; Minton Brian non-byline-author.; Hafer-Macko Charlene non-byline-author.; Kwan Justin non-byline-author.; Zilliox Lindsay non-byline-author.; Callison Karen non-byline-author.; Young Valerie non-byline-author.; DiSanzo Beth non-byline-author.; Naunton Kerry non-byline-author.; Vu Tuan non-byline-author.; Katzin Lara non-byline-author.; McClain Terry non-byline-author.; Harvey Brittany non-byline-author.; Hart Adam non-byline-author.; Huynh Kristin non-byline-author.; Beydoun Said non-byline-author.; Chilingaryan Amaiak non-byline-author.; Doan Victor non-byline-author.; Droker Brian non-byline-author.; Gong Hui non-byline-author.; Karimi Sanaz non-byline-author.; Lin Frank non-byline-author.; McClain Terry non-byline-author.; Polaka Krishna non-byline-author.; Shah Akshay non-byline-author.; Tran Anh non-byline-author.; Akhter Salma non-byline-author.; Malekniazi Ali non-byline-author.; Tandan Rup non-byline-author.; Hehir Michael non-byline-author.; Waheed Waqar non-byline-author.; Lucy Shannon non-byline-author.; Bertorini Tulio non-byline-author.; Arnold Thomas non-byline-author.; Hendersen Kendrick non-byline-author.; Pillai Rekha non-byline-author.; Liu Ye non-byline-author.; Wheeler Lauren non-byline-author.; Hewlett Jasmine non-byline-author.; Vanderhook Mollie non-byline-author.;
• Is Part Of: Therapeutic advances in neurological disorders. Volume 13(2020)
• Publication Date: 2020-04
• Subjects: eculizumab -- intravenous immunoglobulin -- IVIg -- myasthenia gravis -- REGAIN; Nervous system -- Diseases -- Periodicals; Nervous system -- Degeneration -- Periodicals; Nervous system -- Diseases -- Treatment -- Periodicals; Nervous System Diseases -- therapy -- Periodicals; Neurodegenerative Diseases -- Periodicals; Système nerveux -- Maladies -- Périodiques; Système nerveux -- Dégénérescence -- Périodiques; Système nerveux; Système nerveux -- Maladies -- Traitement -- Périodiques; 616.805
• DOI: 10.1177/1756286420911784
• Languages: English
• ISSNs: 1756-2856

Abstract

Background: In the phase III eculizumab for refractory generalized myasthenia gravis REGAIN study [ClinicalTrials.gov identifier: NCT01997229] and its open-label extension (OLE) [ClinicalTrials.gov identifier: NCT02301624], patients with treatment-refractory antiacetylcholine receptor antibody-positive generalized myasthenia gravis had clinically meaningful improvements with eculizumab versus placebo. This subgroup analysis evaluated data from patients with a recent history of chronic intravenous immunoglobulin (IVIg) use before study entry. Methods: The subgroup comprised patients who had received IVIg at least four times in 1 year, with at least one IVIg treatment cycle during the 6 months before the first REGAIN study dose. Data from REGAIN and the OLE were analyzed. Response to eculizumab versus placebo was assessed using four validated, disease-specific measures. Incidences of exacerbations and safety endpoints were recorded. Results: The subgroup had similar patient and disease characteristics as the overall REGAIN population. Clinical assessments showed sustained eculizumab efficacy during REGAIN and the OLE over 18 months. Patients receiving placebo in REGAIN experienced rapid improvements in assessment scores when treated with eculizumab in the OLE. There was a lower rate of disease exacerbations with eculizumab than with placebo during REGAIN, and eculizumab was well tolerated. Conclusion: Eculizumab treatment, compared with placebo, results in meaningful clinical improvements and fewer disease exacerbations for patients who previously received chronic IVIg. Trial registration: REGAIN [ClinicalTrials.gov identifier: NCT01997229]; REGAIN open-label extension [ClinicalTrials.gov identifier: NCT02301624].