A single relapse induces worsening of disability and health-related quality of life in patients with neuromyelitis optica spectrum disorder

Achim Berthele, Michael Levy, Dean M. Wingerchuk, Sean J. Pittock, Shulian Shang, Adrian Kielhorn, Minying Royston, Guido Sabatella, Jacqueline Palace

Research output: Contribution to journalArticlepeer-review

Abstract

Background: Cumulative damage from multiple relapses in neuromyelitis optica spectrum disorder (NMOSD) is associated with poor health-related quality of life (HRQoL) and long-term disability in patients positive for anti-aquaporin 4 antibodies (AQP4+). This study assessed the effect of an individual relapse on HRQoL and disability outcomes in AQP4+ NMOSD. Methods: Post hoc analyses of data pooled from the PREVENT study and its open-label extension, which evaluated the efficacy and safety of eculizumab in AQP4+ NMOSD, examined the effect of a single relapse on 3 disability and 4 HRQoL outcome measures. Assuming the effect of 1 relapse extends to multiple relapses, an extrapolation was done to assess the effect of 2 relapses on these outcomes. Results: In 27 patients (placebo: n = 20; eculizumab: n = 7) experiencing an independently adjudicated relapse, 1 relapse led to significantly worse disability (modified Rankin Scale and Expanded Disability Status Scale [EDSS]) and HRQoL (36-item Short-Form Health Survey mental and physical component summaries; European Quality of Life 5-Dimension questionnaire 3-Level visual analogue scale and utility index) scores. In 4 of 7 outcomes, clinically meaningful worsening was more likely for relapsing versus non-relapsing patients (n = 116). Extrapolating the effect of 2 relapses predicted that clinically meaningful worsening was more likely in 6 out of 7 outcomes, including EDSS, for patients experiencing multiple relapses versus patients experiencing no relapses. Conclusion: Findings from these clinical trial data demonstrate that a single NMOSD relapse can worsen disability and HRQoL, underscoring the role of relapse prevention in improving long-term outcomes in patients with AQP4+ NMOSD.

Original languageEnglish (US)
Article number1099376
JournalFrontiers in Neurology
Volume14
DOIs
StatePublished - 2023

Keywords

  • AQP4
  • eculizumab
  • HRQoL – health-related quality of life
  • neuromyelitis optica
  • relapse

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

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