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Eculizumab monotherapy for NMOSD: Data from PREVENT and its open-label extension

Eculizumab monotherapy for NMOSD: Data from PREVENT and its open-label extension

Journal: Multiple Sclerosis Journal; September 9, 2021

Author(s): Sean J Pittock, Kazuo Fujihara, Jacqueline Palace, Achim Berthele, Ho Jin Kim, Celia Oreja-Guevara, Ichiro Nakashima, Michael Levy, Shulian Shang, Marcus Yountz, Larisa Miller, Róisín Armstrong, Dean M Wingerchuk; PREVENT Study Group

Potential long-term benefits of eculizumab alone in NMOSD treatment

In the PREVENT clinical trial for eculizumab, a recently approved therapy for NMOSD, most patients received eculizumab along with immunosuppressant therapy. This study assessed the effects of eculizumab alone (monotherapy) on patients from the PREVENT study and its open-label extension (the phase of a trial where participants are aware what drug they are being administered). Of the PREVENT trial participants, 33 who were receiving eculizumab monotherapy were monitored for a median duration of 2.9 years (the duration ranging from 14 weeks to 5.2 years). All patients receiving eculizumab monotherapy remained relapse-free at week 96, versus 40% of those receiving placebo alone. At 192 weeks of eculizumab monotherapy, 96% of patients were relapse-free. During the PREVENT study, 95% of patients receiving eculizumab monotherapy showed no worsening in disability and greater quality of life improvements versus placebo alone. Since this study involved only 33 patients who were followed for a wide range of durations, these findings only point to the potential long-term benefits of eculizumab monotherapy. Additional studies are needed to validate these findings and how long the potential benefits last.

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