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SAGE Publications, Therapeutic Advances in Neurological Disorders, (13), p. 175628642091178, 2020

DOI: 10.1177/1756286420911784



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

Journal article published in 2020 by Kathy de Koning, Luiz Augusto da Silva, Anneke van der Kooi, Marianne de Visser, Ezgi Yilmaz, Lindsay Zilliox, Valerie Young, Saiju Jacob ORCID, Fabio Barroso, Juliet Saba, Jan De Bleecker, Guy Van den Abeele, Katrien De Mey, Alzira Alves de Siqueira Carvalho, Miguel Wilken and other authors.
This paper is made freely available by the publisher.
This paper is made freely available by the publisher.

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Background: In the phase III eculizumab for refractory generalized myasthenia gravis REGAIN study [ identifier: NCT01997229] and its open-label extension (OLE) [ 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 [ identifier: NCT01997229]; REGAIN open-label extension [ identifier: NCT02301624].