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Consistent improvement with eculizumab across muscle groups in myasthenia gravis
- Source :
- Annals of Clinical and Translational Neurology, Annals of Clinical and Translational Neurology, Vol 7, Iss 8, Pp 1327-1339 (2020), ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY, Annals of clinical and translational neurology, 7(8), 1327-1339. John Wiley and Sons Ltd
- Publication Year :
- 2020
-
Abstract
- OBJECTIVE: To assess whether eculizumab, a terminal complement inhibitor, improves patient- and physician-reported outcomes (evaluated using the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale, respectively) in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis across four domains, representing ocular, bulbar, respiratory, and limb/gross motor muscle groups. METHODS: Patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis were randomized 1:1 to receive either placebo or eculizumab during the REGAIN study (NCT01997229). Patients who completed REGAIN were eligible to continue into the open-label extension trial (NCT02301624) for up to 4 years. The four domain scores of each of the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale recorded throughout REGAIN and through 130 weeks of the open-label extension were analyzed. RESULTS: Of the 125 patients who participated in REGAIN, 117 enrolled in the open-label extension; 61 had received placebo and 56 had received eculizumab during REGAIN. Patients experienced rapid improvements in total scores and all four domain scores of both the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale with eculizumab treatment. These improvements were sustained through 130 weeks of the open-label extension. INTERPRETATION: Eculizumab treatment elicits rapid and sustained improvements in muscle strength across ocular, bulbar, respiratory, and limb/gross motor muscle groups and in associated daily activities in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis. ispartof: ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY vol:7 issue:8 pages:1327-1339 ispartof: location:United States status: published
- Subjects :
- 0301 basic medicine
Malalties neuromusculars
Activities of daily living
Autoimmune diseases
Severity of Illness Index
Complement inhibitor
0302 clinical medicine
CYCLOPHOSPHAMIDE
Activities of Daily Living
Outcome Assessment, Health Care
Medicine and Health Sciences
Research Articles
Malalties autoimmunitàries
General Neuroscience
Eculizumab
myasthenia
Neuromuscular diseases
Life Sciences & Biomedicine
medicine.drug
RC321-571
Research Article
Adult
medicine.medical_specialty
Cyclophosphamide
Gross motor skill
Clinical Neurology
Neurosciences. Biological psychiatry. Neuropsychiatry
Placebo
Antibodies, Monoclonal, Humanized
ACETYLCHOLINE-RECEPTOR
03 medical and health sciences
Refractory
Double-Blind Method
Internal medicine
Myasthenia Gravis
medicine
Humans
Muscle Strength
Patient Reported Outcome Measures
RC346-429
Muscle, Skeletal
Science & Technology
business.industry
Neurosciences
medicine.disease
Myasthenia gravis
030104 developmental biology
Complement Inactivating Agents
ANTIBODY
Monoclonal antibodies
Neurosciences & Neurology
Neurology (clinical)
Neurology. Diseases of the nervous system
business
Anticossos monoclonals
030217 neurology & neurosurgery
Subjects
Details
- Language :
- English
- ISSN :
- 23289503
- Database :
- OpenAIRE
- Journal :
- Annals of Clinical and Translational Neurology, Annals of Clinical and Translational Neurology, Vol 7, Iss 8, Pp 1327-1339 (2020), ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY, Annals of clinical and translational neurology, 7(8), 1327-1339. John Wiley and Sons Ltd
- Accession number :
- edsair.doi.dedup.....303532dfeb1abc03cdcc192053d4d9fc