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.

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

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30210 Clinical neurology

Stát vydavatele

Spojené státy

Utajení

není předmětem státního či obchodního tajemství

Odkazy

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

Změněno: 20. 6. 2023 14:31, Mgr. Tereza Miškechová

Anotace

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 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.

@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 a 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, roč.~100, č.~7, s.~''E671''-''E682'', 12 s. ISSN~0028-3878. Dostupné z: https://dx.doi.org/10.1212/WNL.0000000000201501.

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