Prevalence of disability improvement as a potential outcome for multiple sclerosis trials.
Disability improvement
Expanded Disability Status Scale
clinical trials
long-term
outcome
prevalence
Journal
Multiple sclerosis (Houndmills, Basingstoke, England)
ISSN: 1477-0970
Titre abrégé: Mult Scler
Pays: England
ID NLM: 9509185
Informations de publication
Date de publication:
04 2021
04 2021
Historique:
pubmed:
27
6
2020
medline:
25
9
2021
entrez:
27
6
2020
Statut:
ppublish
Résumé
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. To propose a simple estimator for assessing and comparing the prevalence of improvement over time between groups. 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. 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 ( 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.
Sections du résumé
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 (
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.
Identifiants
pubmed: 32589486
doi: 10.1177/1352458520936236
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM