Long-term safety and efficacy of eculizumab in generalized myasthenia gravis.
Muscle Nerve
; 60(1): 14-24, 2019 07.
Article
en En
| MEDLINE
| ID: mdl-30767274
ABSTRACT
INTRODUCTION:
Eculizumab is effective and well tolerated in patients with antiacetylcholine receptor antibody-positive refractory generalized myasthenia gravis (gMG; REGAIN; NCT01997229). We report an interim analysis of an open-label extension of REGAIN, evaluating eculizumab's long-term safety and efficacy.METHODS:
Eculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients.RESULTS:
The safety profile of eculizumab was consistent with REGAIN; no cases of meningococcal infection were reported during the interim analysis period. Myasthenia gravis exacerbation rate was reduced by 75% from the year before REGAIN (P < 0.0001). Improvements with eculizumab in activities of daily living, muscle strength, functional ability, and quality of life in REGAIN were maintained through 3 years; 56% of patients achieved minimal manifestations or pharmacological remission. Patients who had received placebo during REGAIN experienced rapid and sustained improvements during open-label eculizumab (P < 0.0001).DISCUSSION:
These findings provide evidence for the long-term safety and sustained efficacy of eculizumab for refractory gMG. Muscle Nerve 2019.Palabras clave
Texto completo:
1
Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Inactivadores del Complemento
/
Anticuerpos Monoclonales Humanizados
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Miastenia Gravis
Tipo de estudio:
Clinical_trials
/
Etiology_studies
/
Observational_studies
/
Risk_factors_studies
Límite:
Adult
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Female
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Humans
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Male
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Middle aged
Idioma:
En
Revista:
Muscle Nerve
Año:
2019
Tipo del documento:
Article
País de afiliación:
Estados Unidos