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

J 2023

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 et. al.

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

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30210 Clinical neurology

Country of publisher

United States of America

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

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

Abstract

V originále

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.

Citovat

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.

@article{2292837,
   author = {Bril, Vera and Szczudlik, Andrzej and Vaitkus, Antanas and Rozsa, Csilla and KosteraandPruszczyk, Anna and Hon, Petr and Bednařík, Josef and Tyblova, Michaela and Koehler, Wolfgang and Toomsoo, Toomas and Nowak, Richard J and Mozaffar, Tahseen and Freimer, Miriam L and Nicolle, Michael W and Magnus, Tim and Pulley, Michael T and Rivner, Michael and Dimachkie, Mazen M and Distad, B Jane and Pascuzzi, Robert M and Babiar, Donna and Lin, Jiang and Montse, Querolt Coll and Griffin, Rhonda and Mondou, Elsa},
   article_location = {Philadelphia},
   article_number = {7},
   doi = {http://dx.doi.org/10.1212/WNL.0000000000201501},
   keywords = {Myasthenia Gravis; Immunoglobulin; Corticosteroid-Sparing Effects},
   language = {eng},
   issn = {0028-3878},
   journal = {Neurology},
   title = {Randomized Double-Blind Placebo-Controlled Trial of the Corticosteroid-Sparing Effects of Immunoglobulin in Myasthenia Gravis},
   url = {https://n.neurology.org/content/100/7/e671},
   volume = {100},
   year = {2023}
}

TY  - JOUR
ID  - 2292837
AU  - 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
PY  - 2023
TI  - Randomized Double-Blind Placebo-Controlled Trial of the Corticosteroid-Sparing Effects of Immunoglobulin in Myasthenia Gravis
JF  - Neurology
VL  - 100
IS  - 7
SP  - "E671"-"E682"
EP  - "E671"-"E682"
PB  - LIPPINCOTT WILLIAMS & WILKINS
SN  - 00283878
KW  - Myasthenia Gravis
KW  - Immunoglobulin
KW  - Corticosteroid-Sparing Effects
UR  - https://n.neurology.org/content/100/7/e671
N2  - 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.
ER  -

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. \textit{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.

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