Alessio Signori1, Giacomo Boffa2, Francesca Bovis1, Alice Mariottini3, Annamaria Repice4, Matilde Inglese2, Maria Pia Amato5, Gianluigi Mancardi6, Luca Massacesi3, Riccardo Saccardi7, Maria Pia Sormani8. 1. Department of Health Sciences, University of Genova, Italy. 2. Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health and Center of Excellence for Biomedical Research (CEBR), University of Genova, Genova, Italy/IRCCS Ospedale Policlinico San Martino, Genova, Italy. 3. Department of Neurology 2, Careggi University Hospital, Florence, Italy/Department of Neurosciences, University of Florence, Florence, Italy. 4. Department of Neurology 2, Careggi University Hospital, Florence, Italy. 5. Department of Neurosciences, University of Florence, Florence, Italy/IRCCS Fondazione Don Carlo Gnocchi, Florence, Italy. 6. Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health and Center of Excellence for Biomedical Research (CEBR), University of Genova, Genova, Italy. 7. Department of Cell Therapy and Transfusional Medicine, Careggi University Hospital, Florence, Italy. 8. Department of Health Sciences, University of Genova, Italy/IRCCS Ospedale Policlinico San Martino, Genova, Italy.
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.
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.
Authors: Marcus W Koch; Jop P Mostert; Jerry S Wolinsky; Fred D Lublin; Bernard Uitdehaag; Gary R Cutter Journal: Neurology Date: 2021-08-25 Impact factor: 11.800
Authors: Samuel F Hunter; Rany A Aburashed; Raed Alroughani; Andrew Chan; Dominique Dive; Sara Eichau; Daniel Kantor; Ho Jin Kim; Jan Lycke; Richard A L Macdonell; Carlo Pozzilli; Thomas Scott; Basil Sharrack; Heinz Wiendl; Luke Chung; Nadia Daizadeh; Darren P Baker; Patrick Vermersch Journal: Neurol Ther Date: 2021-06-24