Randomized Double-Blind Placebo-Controlled Trial of the Corticosteroid-Sparing Effects of Immunoglobulin in Myasthenia Gravis

BRIL, Vera, Andrzej SZCZUDLIK, Antanas VAITKUS, Csilla ROZSA, Anna KOSTERA-PRUSZCZYK, Petr HON, Josef BEDNAŘÍK, Michaela TYBLOVA, Wolfgang KOEHLER, Toomas TOOMSOO, Richard J NOWAK, Tahseen MOZAFFAR, Miriam L FREIMER, Michael W NICOLLE, Tim MAGNUS, Michael T PULLEY, Michael RIVNER, Mazen M DIMACHKIE, B Jane DISTAD, Robert M PASCUZZI, Donna BABIAR, Jiang LIN, Querolt Coll MONTSE, Rhonda GRIFFIN and Elsa MONDOU. Randomized Double-Blind Placebo-Controlled Trial of the Corticosteroid-Sparing Effects of Immunoglobulin in Myasthenia Gravis. Neurology. Philadelphia: LIPPINCOTT WILLIAMS & WILKINS, 2023, vol. 100, No 7, p. "E671"-"E682", 12 pp. ISSN 0028-3878. Available from: https://dx.doi.org/10.1212/WNL.0000000000201501.

Basic information

• Original name: Randomized Double-Blind Placebo-Controlled Trial of the Corticosteroid-Sparing Effects of Immunoglobulin in Myasthenia Gravis
• Authors: BRIL, Vera (guarantor), Andrzej SZCZUDLIK, Antanas VAITKUS, Csilla ROZSA, Anna KOSTERA-PRUSZCZYK, Petr HON, Josef BEDNAŘÍK (203 Czech Republic, belonging to the institution), Michaela TYBLOVA, Wolfgang KOEHLER, Toomas TOOMSOO, Richard J NOWAK, Tahseen MOZAFFAR, Miriam L FREIMER, Michael W NICOLLE, Tim MAGNUS, Michael T PULLEY, Michael RIVNER, Mazen M DIMACHKIE, B Jane DISTAD, Robert M PASCUZZI, Donna BABIAR, Jiang LIN, Querolt Coll MONTSE, Rhonda GRIFFIN and Elsa MONDOU.
• Edition: Neurology, Philadelphia, LIPPINCOTT WILLIAMS & WILKINS, 2023, 0028-3878.

Other information

• Original language: English
• Type of outcome: Article in a journal
• Field of Study: 30210 Clinical neurology
• Country of publisher: United States of America
• Confidentiality degree: is not subject to a state or trade secret
• WWW: URL
• Impact factor: Impact factor: 9.900 in 2022
• RIV identification code: RIV/00216224:14110/23:00131012
• Organization unit: Faculty of Medicine
• Doi: http://dx.doi.org/10.1212/WNL.0000000000201501
• UT WoS: 000983098800016
• Keywords in English: Myasthenia Gravis; Immunoglobulin; Corticosteroid-Sparing Effects
• Tags: 14110221, rivok
• Tags: International impact, Reviewed

Abstract

Background and ObjectivesMyasthenia gravis (MG) is an autoimmune disease characterized by dysfunction at the neuromuscular junction. Treatment frequently includes corticosteroids (CSs) and IV immunoglobulin (IVIG). This study was conducted to determine whether immune globulin (human), 10% caprylate/chromatography purified (IGIV-C) could facilitate CS dose reduction in CS dependent patients with MG.MethodsIn this randomized double-blind placebo-controlled trial, CS-dependent patients with MG (Myasthenia Gravis Foundation of America Class II-Iva; AChR+) received a loading dose of 2 g/kg IGIV-C over 2 days (maximum 80 g/d) or placebo at week 0 (baseline). Maintenance doses (1 g/kg IGIV-C or placebo) were administered every 3 weeks through week 36. Tapering of CS was initiated at week 9 and continued through week 36 unless the patient worsened (quantitative MG score .4 points from baseline). CS doses were increased (based on the 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 end point (at week 39) was a .50% reduction in CS dose. Secondary and safety end points 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. ResultsThe primary end point (.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 end points. Safety data indicated that IGIV-C was well tolerated.DiscussionIn this study, IGIV-C was not more effective than placebo in reducing daily CS dose. These results suggest that the effects of IGIV-C and CS are not synergistic and maybe mechanistically different.