Randomized study of adjunctive belimumab in participants with generalized myasthenia gravis

作者：K. Hewett, Donald B. Sanders, Richard Grove, Christine L. Broderick, Todd Rudo, Ashlyn Bassiri, Marina Zvartau‐Hind, Vera Bril, On behalf of the BEL115123 Study Group, BEL115123 Study Group, Laurence Adams, Sankar Bandyopadhyay, Said R. Beydoun, Felix Bischof, Mazen M. Dimachkie, Miriam Freimer, Maurizio Inghilleri, Henry J. Kaminski, Renato Mantegazza, Tahseen Mozaffar, Michael Nicolle, Khema R. Sharma, Zaeemi Siddiqi, Ericka Simpson, Florian Then Bergh, Tuan Vu, Radwa Aly, Carlo Antozzi, Richard J. Barohn, Derrick Blackmore, Silvia Bonanno, Angela Campanella, Tiyonnoh Cash, Bakri Elsheikh, Vittorio Frasca, Namita Goyal, Brittany Harvey, Eugene C. Lai, Lorenzo Maggi, Brian Minton, Verónica Puertas‐Martín, Emanuela Onesti, Mamatha Pasnoor, Gulmohor Roy, Sheetal Shroff, Jason R. Thonhoff · 发表于：Neurology · 年份：2018 · DOI：10.1212/wnl.0000000000005323 · 被引用次数：130 · 研究领域：Myasthenia Gravis and Thymoma、Peripheral Neuropathies and Disorders、Adrenal Hormones and Disorders

OBJECTIVE: To investigate the efficacy and safety of belimumab, a fully human immunoglobulin G1λ monoclonal antibody against B-lymphocyte stimulator, in participants with generalized myasthenia gravis (MG) who remained symptomatic despite standard of care (SoC) therapy. METHODS: Eligible participants with MG were randomized 1:1 to receive IV belimumab 10 mg/kg or placebo in this phase II, placebo-controlled, multicenter, double-blind study (NCT01480596; BEL115123). Participants received SoC therapies throughout the 24-week treatment phase and 12-week follow-up period. The primary efficacy endpoint was mean change from baseline in the Quantitative Myasthenia Gravis (QMG) scale at week 24; safety assessments included the frequency and severity of adverse events (AEs) and serious AEs. RESULTS: = 0.256). There were no statistically significant differences between treatment groups for secondary endpoints, including the MG Composite and MG-Activity of Daily Living scores. Acetylcholine receptor antibody levels decreased over time in both treatment groups. No unexpected AEs were identified and occurrence was similar in the belimumab (78%) and placebo (91%) groups. One participant receiving placebo died (severe sepsis) during the treatment phase. CONCLUSIONS: The primary endpoint was not met for belimumab in participants with generalized MG receiving SoC. There was no significant difference in mean change in the QMG score at week 24 for belimumab vs placebo. The safety profile of bel...