Clinical Experience with Eculizumab in Treatment-Refractory Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis

被引:19
作者
Katyal, Nakul [1 ]
Narula, Naureen [2 ]
Govindarajan, Raghav [1 ]
机构
[1] Univ Missouri Hlth Care, Columbia, MO USA
[2] Staten Isl Univ Hosp, New York, NY USA
关键词
Myasthenia gravis; ACh receptors; neuromuscular junction; complement inactivating agents; activities of daily living; corticosteroids; exacerbations; diplopia; prednisone; immunoglobulin G; DOUBLE-BLIND; INTRAVENOUS IMMUNOGLOBULIN; SAFETY;
D O I
10.3233/JND-200584
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Background: Although established therapies are effective in most patients with generalized myasthenia gravis (gMG), some patients do not respond or they experience intolerable adverse events, highlighting the need for better tolerated, targeted therapies for treatment-refractory gMG. Objective: To describe real-world experience with eculizumab in patients with treatment-refractory acetylcholine receptor antibody-positive (AChR+) gMG. Methods: Retrospective chart review of 15 patients with treatment-refractory AChR+ gMG treated for 12 months with eculizumab (900 mg/week for 4 weeks then 1200 mg every 2 weeks). Outcome measures were Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores, number of exacerbations, single-breath count test (SBCT) score, medication changes, selected Quantitative Myasthenia Gravis (QMG) evaluations, and adverse events. Data collected at 3-monthly intervals for 12 months before and after eculizumab initiation were analyzed. Results: Clinically meaningful reductions in total MG-ADL scores were observed at 3 months following eculizumab initiation and maintained up to 12 months in all patients. After 12 months' eculizumab treatment, there was a significant reduction in the number of acute exacerbations; mean (SD) SBCT score improved from 28.13 (0.33) to 50.26 (2.86); all patients achieved a 'none' or 'mild' rating for QMG evaluations; all patients reduced their daily prednisone dose; and nine patients had discontinued pyridostigmine. At the end of treatment, intravenous immunoglobulin was discontinued in all six patients receiving this therapy at eculizumab initiation. Eculizumab was well tolerated. Conclusions: This real-world study demonstrated improvement in outcome measures and decreased concomitant drug requirement within 12 months of eculizumab initiation in patients with treatment-refractory AChR+ gMG.
引用
收藏
页码:287 / 294
页数:8
相关论文
共 23 条
  • [1] Alexion Europe, SOL EC SUMM PROD CHA SOL EC SUMM PROD CHA
  • [2] Alexion Pharmaceuticals Inc, 2019, SOLIRIS EC INJ PRESC SOLIRIS EC INJ PRESC
  • [3] Eculizumab improves fatigue in refractory generalized myasthenia gravis
    Andersen, Henning
    Mantegazza, Renato
    Wang, Jing Jing
    O'Brien, Fanny
    Patra, Kaushik
    Howard, James F., Jr.
    [J]. QUALITY OF LIFE RESEARCH, 2019, 28 (08) : 2247 - 2254
  • [4] [Anonymous], 2019, EC GEN REC SOLIRIS I EC GEN REC SOLIRIS I
  • [5] Barnett Carolina, 2012, J Clin Neuromuscul Dis, V13, P201, DOI 10.1097/CND.0b013e31824619d5
  • [6] Eculizumab: A Review in Generalized Myasthenia Gravis
    Dhillon, Sohita
    [J]. DRUGS, 2018, 78 (03) : 367 - 376
  • [7] Unsatisfactory outcomes in myasthenia gravis: influence by care providers
    Dunand, Murielle
    Botez, Stephan A.
    Borruat, Francois-Xavier
    Roux-Lombard, Pascale
    Spertini, Francois
    Kuntzer, Thierry
    [J]. JOURNAL OF NEUROLOGY, 2010, 257 (03) : 338 - 343
  • [8] CORRELATION OF SINGLE-BREATH COUNT TEST AND NECK FLEXOR MUSCLE STRENGTH WITH SPIROMETRY IN MYASTHENIA GRAVIS
    Elsheikh, Bakri
    Arnold, W. David
    Gharibshahi, Shahram
    Reynolds, Jerold
    Freimer, Miriam
    Kissel, John T.
    [J]. MUSCLE & NERVE, 2016, 53 (01) : 134 - 136
  • [9] Treatment of Myasthenia Gravis
    Farmakidis, Constantine
    Pasnoor, Mamatha
    Dimachkie, Mazen M.
    Barohn, Richard J.
    [J]. NEUROLOGIC CLINICS, 2018, 36 (02) : 311 - +
  • [10] Treatment of Myasthenia Gravis exacerbation with intravenous immunoglobulin - A randomized double-blind clinical trial
    Gajdos, P
    Tranchant, C
    Clair, B
    Bolgert, F
    Eymard, B
    Stojkovic, T
    Attarian, S
    Chevret, S
    [J]. ARCHIVES OF NEUROLOGY, 2005, 62 (11) : 1689 - 1693