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 a Elsa MONDOU. Randomized Double-Blind Placebo-Controlled Trial of the Corticosteroid-Sparing Effects of Immunoglobulin in Myasthenia Gravis. Neurology. Philadelphia: LIPPINCOTT WILLIAMS & WILKINS, 2023, roč. 100, č. 7, s. "E671"-"E682", 12 s. ISSN 0028-3878. Dostupné z: https://dx.doi.org/10.1212/WNL.0000000000201501.

Základní údaje

• Originální název: Randomized Double-Blind Placebo-Controlled Trial of the Corticosteroid-Sparing Effects of Immunoglobulin in Myasthenia Gravis
• Autoři: BRIL, Vera (garant), Andrzej SZCZUDLIK, Antanas VAITKUS, Csilla ROZSA, Anna KOSTERA-PRUSZCZYK, Petr HON, Josef BEDNAŘÍK (203 Česká republika, domácí), 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 a Elsa MONDOU.
• Vydání: Neurology, Philadelphia, LIPPINCOTT WILLIAMS & WILKINS, 2023, 0028-3878.

Další údaje

• Originální jazyk: angličtina
• Typ výsledku: Článek v odborném periodiku
• Obor: 30210 Clinical neurology
• Stát vydavatele: Spojené státy
• Utajení: není předmětem státního či obchodního tajemství
• WWW: URL
• Impakt faktor: Impact factor: 9.900 v roce 2022
• Kód RIV: RIV/00216224:14110/23:00131012
• Organizační jednotka: Lékařská fakulta
• Doi: http://dx.doi.org/10.1212/WNL.0000000000201501
• UT WoS: 000983098800016
• Klíčová slova anglicky: Myasthenia Gravis; Immunoglobulin; Corticosteroid-Sparing Effects
• Štítky: 14110221, rivok
• Příznaky: Mezinárodní význam, Recenzováno

Anotace

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.