Long-term safety and efficacy of eculizumab in generalized myasthenia gravis
Por:
Muppidi, S, Utsugisawa, K, Benatar, M, Murai, H, Barohn, RJ, Illa, I, Jacob, S, Vissing, J, Burns, TM, Kissel, JT, Nowak, RJ, Andersen, H, Casasnovas, C, de Bleecker, JL, Vu, TH, Mantegazza, R, O'Brien, FL, Wang, JJ, Fujita, KP, Howard, JF, Kaya, A, Khursigara, G, Armstrong, R, Diab, D, Capocelli, K, Lane, C, Sanders, V, Gandolfo, R, REGAIN Study Grp
Publicada:
1 jul 2019
Resumen:
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
Filiaciones:
Muppidi, S:
Stanford Univ, Sch Med, Dept Neurol & Neurosci, Stanford, CA 94305 USA
Utsugisawa, K:
Hanamaki Gen Hosp, Dept Neurol, Hanamaki, Japan
Benatar, M:
Univ Miami, Miller Sch Med, Dept Neurol, Miami, FL 33136 USA
Murai, H:
Int Univ Hlth & Welf, Dept Neurol, Narita, Japan
Barohn, RJ:
Univ Kansas, Med Ctr, Dept Neurol, Kansas City, KS 66103 USA
Illa, I:
Autonomous Univ Barcelona, Hosp St Pau, Dept Neurol, Barcelona, Spain
CIBERER, Barcelona, Spain
Jacob, S:
Univ Hosp Birmingham, Queen Elizabeth Neurosci Ctr, Birmingham, W Midlands, England
Univ Hosp Birmingham, Wellcome Trust Clin Res Facil, Birmingham, W Midlands, England
Vissing, J:
Univ Copenhagen, Dept Neurol, Rigshosp, Copenhagen, Denmark
Burns, TM:
Univ Virginia Hlth Syst, Dept Neurol, Charlottesville, VA USA
Kissel, JT:
Ohio State Univ, Dept Neurol, Columbus, OH 43210 USA
Nowak, RJ:
Yale Univ, Dept Neurol, Yale Sch Med, New Haven, CT USA
Andersen, H:
Aarhus Univ Hosp, Dept Neurol, Aarhus, Denmark
Casasnovas, C:
CIBERER, Barcelona, Spain
Bellvitge Univ Hosp, Dept Neurol, Barcelona, Spain
de Bleecker, JL:
Ghent Univ Hosp, Neuromuscular Reference Ctr, Ghent, Belgium
Vu, TH:
Univ S Florida, Dept Neurol, Tampa, FL USA
Mantegazza, R:
Fdn Carlo Besta Neurol Inst, IRCSS, Milan, Italy
O'Brien, FL:
Alexion Pharmaceut, Boston, MA USA
Wang, JJ:
Alexion Pharmaceut, Boston, MA USA
Fujita, KP:
Alexion Pharmaceut, Boston, MA USA
Howard, JF:
Univ N Carolina, Dept Neurol, Chapel Hill, NC 27515 USA
Kaya, A:
Alexion Pharmaceut, Boston, MA USA
Khursigara, G:
Alexion Pharmaceut, Boston, MA USA
Armstrong, R:
Alexion Pharmaceut, Boston, MA USA
Diab, D:
Alexion Pharmaceut, Boston, MA USA
Capocelli, K:
Alexion Pharmaceut, Boston, MA USA
Lane, C:
Alexion Pharmaceut, Boston, MA USA
Sanders, V:
Oxford PharmaGenesis, Oxford, England
Gandolfo, R:
Oxford PharmaGenesis, Oxford, England
Green Published, Hybrid Gold
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