Prevalence of disability improvement as a potential outcome for multiple sclerosis trials

Alessio Signori, Giacomo Boffa, Francesca Bovis, Alice Mariottini, Annamaria Repice, Matilde Inglese, Maria Pia Amato, Gianluigi Mancardi, Luca Massacesi, Riccardo Saccardi, Maria Pia Sormani

Research output: Contribution to journalArticle

Abstract

Background: The concept of improvement of disability recently emerged as a new target in multiple sclerosis (MS) studies since the approval of new potent drugs and for testing drugs for neuroprotection and repair. Objective: To propose a simple estimator for assessing and comparing the prevalence of improvement over time between groups. Methods: The prevalence of a transient condition takes into account the incidence and the duration of such condition. We propose here the application of a modified Kaplan–Meier estimator to evaluate and compare between groups the prevalence of improvement over time in a cohort of 121 patients treated with autologous hematopoietic stem cell transplantation. Results: The prevalence of improvement after 5 years from transplant was 50.3% (95%CI: [38.0–63.0]) in relapsing–remitting patients and 6.5% (95%CI: [0–17.8]) in secondary-progressive patients (p < 0.001). Such a difference wouldn’t be evident considering the traditional cumulative probability of improvement at 5 years (55.5% in relapsing–remitting vs 33.4% in secondary-progressive patients, p = 0.10). Conclusion: This study shows the relevance of a new estimator of prevalence of improvement in MS. This estimator gives simple information on whether a drug can induce a durable improvement in disability and can be considered a potential outcome for trials assessing drugs for neuroprotection or repair.

Original languageEnglish
JournalMultiple Sclerosis Journal
DOIs
Publication statusAccepted/In press - Jan 1 2020

Keywords

  • clinical trials
  • Disability improvement
  • Expanded Disability Status Scale
  • long-term
  • outcome
  • prevalence

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

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