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

Authors	BRIL Vera SZCZUDLIK Andrzej VAITKUS Antanas ROZSA Csilla KOSTERA-PRUSZCZYK Anna HON Petr BEDNAŘÍK Josef TYBLOVA Michaela KOEHLER Wolfgang TOOMSOO Toomas NOWAK Richard J MOZAFFAR Tahseen FREIMER Miriam L NICOLLE Michael W MAGNUS Tim PULLEY Michael T RIVNER Michael DIMACHKIE Mazen M DISTAD B Jane PASCUZZI Robert M BABIAR Donna LIN Jiang MONTSE Querolt Coll GRIFFIN Rhonda MONDOU Elsa
Year of publication	2023
Type	Article in Periodical
Magazine / Source	Neurology
MU Faculty or unit	Faculty of Medicine
Citation
Web	https://n.neurology.org/content/100/7/e671
Doi	http://dx.doi.org/10.1212/WNL.0000000000201501
Keywords	Myasthenia Gravis; Immunoglobulin; Corticosteroid-Sparing Effects
Description	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 Patients were withdrawn if worsening failed to improve within 6 weeks or if a second CS increase was required.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.