Affiliations 

  • 1 Department of Neurology, Research Institute and Hospital, National Cancer Center, 323 Ilsan-ro, Ilsandong-gu, Goyang 10408, South Korea. Electronic address: hojinkim@ncc.re.kr
  • 2 Department of Neurology, Tohoku University Graduate School of Medicine, 1-1 Seiryomachi, Aobaku, Sendai, Miyagi 980-8574, Japan; Division of Neurology, Tohoku Medical and Pharmaceutical University, 1-12-1 Fukumuro, Miyaginoku, Sendai, Miyagi 983-8512, Japan. Electronic address: nakashima@tohoku-mpu.ac.jp
  • 3 Department of Neurology, Kuala Lumpur Hospital, Jalan Pahang, Kuala Lumpur 50560, Malaysia
  • 4 Cheng Hsin General Hospital, 45 Zhenxing Street, Beitou District, Taipei, Taiwan 112; School of Medicine, National Yang-Ming University, 155, Section 2, Linong St, Beitou District, Taipei, Taiwan 112
  • 5 Alexion Pharmaceuticals, 121 Seaport Boulevard, Boston, MA 02210, United States. Electronic address: shulian.shang@alexion.com
  • 6 Alexion Pharmaceuticals, 121 Seaport Boulevard, Boston, MA 02210, United States. Electronic address: Larisa.Miller@alexion.com
  • 7 Alexion Pharmaceuticals, 121 Seaport Boulevard, Boston, MA 02210, United States. Electronic address: Marcus.Yountz@alexion.com
  • 8 Mayo Clinic Arizona, 13400 East Shea Boulevard, Scottsdale, AZ 85259, United States. Electronic address: wingerchuk.dean@mayo.edu
  • 9 Mayo Clinic, 200 First Street SW, Rochester, MN 55905, United States. Electronic address: Pittock.Sean@mayo.edu
  • 10 Department of Neurology, Johns Hopkins University, 1800 Orleans Street, Baltimore, MD 21287, United States. Electronic address: mlevy11@mgh.harvard.edu
  • 11 Technical University of Munich, School of Medicine, Klinikum rechts der Isar, Neuro-Kopf-Zentrum, Ismaninger Str. 22, 81675 Munich, Germany. Electronic address: achim.berthele@tum.de
  • 12 Department of Neurology, First Pavlov State Medical University of St. Petersburg, St. 6/8, Lva Tolstogo str., 197022 Petersburg, Russia. Electronic address: ntotolyan@mail.ru
  • 13 Nuffield Department of Clinical Neurosciences, West Wing, John Radcliffe Hospital, University of Oxford, Headley Way, Oxford OX3 9DU, United Kingdom. Electronic address: jacqueline.palace@ndcn.ox.ac.uk
  • 14 Brain and Mind Centre, University of Sydney, 94, Mallett Street, Camperdown, Sydney, NSW 2050, Australia; Royal Prince Alfred Hospital, University of Sydney, Sydney, NSW, Australia. Electronic address: michael@sydneyneurology.com.au
  • 15 Department of Neurology, Tohoku University Graduate School of Medicine, 1-1 Seiryomachi, Aobaku, Sendai, Miyagi 980-8574, Japan; Department of Multiple Sclerosis Therapeutics, Fukushima Medical University School of Medicine, 1 Hikariga-oka, Fukushima City, Fukushima 960-1295, Japan; Multiple Sclerosis and Neuromyelitis Optica Center, Southern TOHOKU Research Institute for Neuroscience (STRINS), Koriyama, 7-115, Yatsuyamada, Koriyama, Fukushima 963-8563, Japan. Electronic address: fujikazu@med.tohoku.ac.jp
Mult Scler Relat Disord, 2021 May;50:102849.
PMID: 33676197 DOI: 10.1016/j.msard.2021.102849

Abstract

Background Eculizumab, a terminal complement inhibitor, significantly reduced the risk of relapse compared with placebo in patients with anti-aquaporin-4 immunoglobulin G-positive (AQP4+) neuromyelitis optica spectrum disorder (NMOSD) in the PREVENT trial. We report efficacy and safety analyses in Asian patients in PREVENT and its open-label extension (OLE). Methods PREVENT was a double-blind, randomized, phase 3 trial. Patients with AQP4+ NMOSD were randomly assigned (2:1) to receive intravenous eculizumab (maintenance dose, 1200 mg/2 weeks) or placebo. Patients who completed PREVENT could receive eculizumab in an OLE. Analyses were performed in a prespecified subgroup of Asian patients. Results Of 143 patients enrolled, 52 (36.4%) were included in the Asian subgroup (eculizumab, n = 37; placebo, n = 15); 45 Asian patients received eculizumab in the OLE. Most Asian patients (86.5%) received concomitant immunosuppressive therapy. During PREVENT, one adjudicated relapse occurred in patients receiving eculizumab and six occurred in patients receiving placebo in the Asian subgroup (hazard ratio, 0.05; 95% confidence interval: 0.01-0.35; p = 0.0002). An estimated 95.2% of Asian patients remained relapse-free after 144 weeks of eculizumab treatment. Upper respiratory tract infections, headache, and nasopharyngitis were the most common adverse events with eculizumab in the Asian subgroup. Conclusion Eculizumab reduces the risk of relapse in Asian patients with AQP4+ NMOSD, with a benefit-risk profile similar to the overall PREVENT population. The benefits of eculizumab were maintained during long-term therapy. Clinical trial registration ClinicalTrials.gov identifiers: NCT01892345 (PREVENT); NCT02003144 (open-label extension).

* Title and MeSH Headings from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.