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Signori, Alessio; Boffa, Giacomo; Bovis, Francesca; Mariottini, Alice; Repice, Annamaria; Inglese, Matilde; Amato, Maria Pia; Mancardi, Gianluigi; Massacesi, Luca; Saccardi, Riccardo; Sormani, Maria Pia
Multiple sclerosis, 04/2021, Letnik: 27, Številka: 5Journal Article
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.
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JCR | SNIP | JCR | SNIP | JCR | SNIP | JCR | SNIP |
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in: SICRIS
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