Abstract
Abstract: Background and Objectives: Myasthenia gravis (MG) is an autoimmune disease characterized by dysfunction at the neuromuscular junction. Treatment frequently includes corticosteroids (CS) and intravenous immunoglobulin (IVIG). This study was conducted to determine if immune globulin (human), 10% caprylate/chromatography purified (IGIV-C) could facilitate CS dose reduction in CS-dependent MG patients.
Methods: In this randomized, double-blind, placebo-controlled trial, CS-dependent MG patients (MGFA class II-Iva; AChR+) received a loading dose of 2 g/kg IGIV-C over 2 days (maximum 80 g/day) or placebo at week 0 (baseline). Maintenance doses (1 g/kg IGIV-C or placebo) were administered every three weeks through week 36. Tapering of CS was initiated at week 9 and continued through week 36 unless the patient worsened (QMG score ≥ 4 points from baseline). CS doses were increased (based on current CS dose) in patients who worsened. Patients were withdrawn if worsening failed to improve within 6 weeks or if a second CS increase was required. The primary efficacy endpoint (at week 39) was a ≥ 50% reduction in CS dose. Secondary and safety endpoints were assessed throughout the study and follow-up (weeks 42 and 45). The study results and full protocol are available at: ://clinicaltrials.gov/ct2/show/NCT02473965.
Results: The primary endpoint (≥ 50% reduction in CS dose) showed no significant difference between the IGIV-C treatment (60.0% of patients) and placebo (63.3%). There were no significant differences for secondary endpoints. Safety data indicated that IGIV-C was well-tolerated.
Discussion: In this study, IGIV-C was not more effective than placebo in reducing daily CS dose. These results suggest that effects of IGIV-C and CS are not synergistic and may be mechanistically different.
Trial Registration Information: The trial was registered on clinicaltrialsregister.eu (EudraCT #: 2013-005099-17) and clinicaltrials.gov (identifier NCT02473965).
Classification of Evidence: This study provides Class II evidence that IVIG infusions in adult patients with MG do not increase the percentage of patients achieving a ≥ 50% reduction in corticosteroid dose compared to placebo.
- Received January 31, 2022.
- Accepted in final form September 16, 2022.
- Copyright © 2022 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology.
This is an open access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License 4.0 (CC BY-NC-ND), which permits downloading and sharing the work provided it is properly cited. The work cannot be changed in any way or used commercially without permission from the journal.
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