Efficacy and safety of maintenance intravenous immunoglobulin
in generalized myasthenia gravis patients with acetylcholine
receptor antibodies: A multicenter, double-blind,
placebo-controlled trial

J 2025

Efficacy and safety of maintenance intravenous immunoglobulin in generalized myasthenia gravis patients with acetylcholine receptor antibodies: A multicenter, double-blind, placebo-controlled trial

BRIL, Vera; Tomasz BERKOWICZ; Andrzej SZCZUDLIK; Michael W NICOLLE; Josef BEDNAŘÍK et al.

Základní údaje

Originální název

Efficacy and safety of maintenance intravenous immunoglobulin in generalized myasthenia gravis patients with acetylcholine receptor antibodies: A multicenter, double-blind, placebo-controlled trial

Autoři

Vydání

MUSCLE & NERVE, HOBOKEN, WILEY, 2025, 0148-639X

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: 3.100 v roce 2024

Označené pro přenos do RIV

Ano

Kód RIV

RIV/00216224:14110/25:00140708

Organizační jednotka

Lékařská fakulta

Klíčová slova anglicky

autoimmune disease; intravenous immunoglobulin; myasthenia gravis; neuromuscular disease

Štítky

14110221, rivok

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 28. 2. 2025 10:07, Mgr. Tereza Miškechová

Anotace

V originále

Introduction/AimsProspective, randomized, controlled trials of intravenous immunoglobulin (IVIG) maintenance therapy in myasthenia gravis (MG) are lacking. In this trial, we evaluated the safety and efficacy of caprylate/chromatography-purified IVIG; (IGIV-C) in patients with generalized MG undergoing standard care.MethodsSixty-two patients enrolled in this phase 2, multicenter, international, randomized trial (1:1 IGIV-C [2 g/kg loading dose; 1 g/kg every 3 weeks through week 21] or placebo). Efficacy was assessed by changes in Quantitative MG (QMG) score at week 24 versus baseline (primary endpoint) and percentage of patients with clinical improvement in QMG, MG Composite (MGC), and MG-Activities of Daily Living (MG-ADL) scores (secondary endpoints). Safety assessments reported all adverse events (AEs).ResultsThe change in QMG at 24 weeks was -5.1 for IGIV-C and -3.1 for placebo (p = .187). Seventy percent of patients in the IGIV-C group had improvement in MG-ADL (>= 2-point decrease) versus 40.6% in the placebo group (p = .025). Patients showing clinical improvement in QMG and MGC (>= 3-point decrease) were 70.0% for IGIV-C versus 59.4% for placebo (p = .442) and 60.0% for IGIV-C versus 53.1% for placebo (p = .610). IGIV-C was well tolerated; serious AEs were similar between arms. Three of four MG exacerbations requiring hospitalizations occurred in the IGIV-C arm with one death.DiscussionSeveral efficacy parameters showed numerical results greater than those seen in the placebo group. This was a small study and may have been underpowered to see significant differences. Additional studies may be warranted to fully determine the efficacy of IVIG maintenance therapy in MG.

Citovat

BRIL, Vera; Tomasz BERKOWICZ; Andrzej SZCZUDLIK; Michael W NICOLLE; Josef BEDNAŘÍK; Petr HON; Antanas VAITKUS; Tuan VU; Csilla ROZSA; Tim MAGNUS; Gyula PANCZEL; Toomas TOOMSOO; Mamatha PASNOOR; Tahseen MOZAFFAR; Miriam FREIMER; Ulrike REUNER; Laszlo VECSEI; Nizar SOUAYAH; Todd LEVINE; Robert M PASCUZZI; Marinos C DALAKAS; Michael RIVNER; Rhonda GRIFFIN; Montse Querolt COLL a Elsa MONDOU. Efficacy and safety of maintenance intravenous immunoglobulin in generalized myasthenia gravis patients with acetylcholine receptor antibodies: A multicenter, double-blind, placebo-controlled trial. MUSCLE & NERVE. HOBOKEN: WILEY, 2025, roč. 71, č. 1, s. 43-54. ISSN 0148-639X. Dostupné z: https://doi.org/10.1002/mus.28289.

@article{2480507,
   author = {Bril, Vera and Berkowicz, Tomasz and Szczudlik, Andrzej and Nicolle, Michael W and Bednařík, Josef and Hon, Petr and Vaitkus, Antanas and Vu, Tuan and Rozsa, Csilla and Magnus, Tim and Panczel, Gyula and Toomsoo, Toomas and Pasnoor, Mamatha and Mozaffar, Tahseen and Freimer, Miriam and Reuner, Ulrike and Vecsei, Laszlo and Souayah, Nizar and Levine, Todd and Pascuzzi, Robert M and Dalakas, Marinos C and Rivner, Michael and Griffin, Rhonda and Coll, Montse Querolt and Mondou, Elsa},
   article_location = {HOBOKEN},
   article_number = {1},
   doi = {https://doi.org/10.1002/mus.28289},
   keywords = {autoimmune disease; intravenous immunoglobulin; myasthenia gravis; neuromuscular disease},
   language = {eng},
   issn = {0148-639X},
   journal = {MUSCLE & NERVE},
   title = {Efficacy and safety of maintenance intravenous immunoglobulin in generalized myasthenia gravis patients with acetylcholine receptor antibodies: A multicenter, double-blind, placebo-controlled trial},
   url = {https://onlinelibrary.wiley.com/doi/10.1002/mus.28289},
   volume = {71},
   year = {2025}
}

TY  - JOUR
ID  - 2480507
AU  - Bril, Vera - Berkowicz, Tomasz - Szczudlik, Andrzej - Nicolle, Michael W - Bednařík, Josef - Hon, Petr - Vaitkus, Antanas - Vu, Tuan - Rozsa, Csilla - Magnus, Tim - Panczel, Gyula - Toomsoo, Toomas - Pasnoor, Mamatha - Mozaffar, Tahseen - Freimer, Miriam - Reuner, Ulrike - Vecsei, Laszlo - Souayah, Nizar - Levine, Todd - Pascuzzi, Robert M - Dalakas, Marinos C - Rivner, Michael - Griffin, Rhonda - Coll, Montse Querolt - Mondou, Elsa
PY  - 2025
TI  - Efficacy and safety of maintenance intravenous immunoglobulin in generalized myasthenia gravis patients with acetylcholine receptor antibodies: A multicenter, double-blind, placebo-controlled trial
JF  - MUSCLE & NERVE
VL  - 71
IS  - 1
SP  - 43-54
EP  - 43-54
PB  - WILEY
SN  - 0148639X
KW  - autoimmune disease
KW  - intravenous immunoglobulin
KW  - myasthenia gravis
KW  - neuromuscular disease
UR  - https://onlinelibrary.wiley.com/doi/10.1002/mus.28289
N2  - Introduction/AimsProspective, randomized, controlled trials of intravenous immunoglobulin (IVIG) maintenance therapy in myasthenia gravis (MG) are lacking. In this trial, we evaluated the safety and efficacy of caprylate/chromatography-purified IVIG; (IGIV-C) in patients with generalized MG undergoing standard care.MethodsSixty-two patients enrolled in this phase 2, multicenter, international, randomized trial (1:1 IGIV-C [2 g/kg loading dose; 1 g/kg every 3 weeks through week 21] or placebo). Efficacy was assessed by changes in Quantitative MG (QMG) score at week 24 versus baseline (primary endpoint) and percentage of patients with clinical improvement in QMG, MG Composite (MGC), and MG-Activities of Daily Living (MG-ADL) scores (secondary endpoints). Safety assessments reported all adverse events (AEs).ResultsThe change in QMG at 24 weeks was -5.1 for IGIV-C and -3.1 for placebo (p = .187). Seventy percent of patients in the IGIV-C group had improvement in MG-ADL (>= 2-point decrease) versus 40.6% in the placebo group (p = .025). Patients showing clinical improvement in QMG and MGC (>= 3-point decrease) were 70.0% for IGIV-C versus 59.4% for placebo (p = .442) and 60.0% for IGIV-C versus 53.1% for placebo (p = .610). IGIV-C was well tolerated; serious AEs were similar between arms. Three of four MG exacerbations requiring hospitalizations occurred in the IGIV-C arm with one death.DiscussionSeveral efficacy parameters showed numerical results greater than those seen in the placebo group. This was a small study and may have been underpowered to see significant differences. Additional studies may be warranted to fully determine the efficacy of IVIG maintenance therapy in MG.
ER  -

BRIL, Vera; Tomasz BERKOWICZ; Andrzej SZCZUDLIK; Michael W NICOLLE; Josef BEDNAŘÍK; Petr HON; Antanas VAITKUS; Tuan VU; Csilla ROZSA; Tim MAGNUS; Gyula PANCZEL; Toomas TOOMSOO; Mamatha PASNOOR; Tahseen MOZAFFAR; Miriam FREIMER; Ulrike REUNER; Laszlo VECSEI; Nizar SOUAYAH; Todd LEVINE; Robert M PASCUZZI; Marinos C DALAKAS; Michael RIVNER; Rhonda GRIFFIN; Montse Querolt COLL a Elsa MONDOU. Efficacy and safety of maintenance intravenous immunoglobulin in generalized myasthenia gravis patients with acetylcholine receptor antibodies: A multicenter, double-blind, placebo-controlled trial. \textit{MUSCLE \&{} NERVE}. HOBOKEN: WILEY, 2025, roč.~71, č.~1, s.~43-54. ISSN~0148-639X. Dostupné z: https://doi.org/10.1002/mus.28289.

Přehled o publikaci