1. Long-term disability trajectories in primary progressive MS patients: A latent class growth analysis
- Author
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Raed Alroughani, Maria Pia Amato, Patrizia Sola, Francois Grand'Maison, Eva Havrdova, Pierre Grammond, Ricardo Fernandez Bolanos, Murat Terzi, Bhim Singhal, Tomas Kalincik, Alessandra Lugaresi, Marc Girard, Jeannette Lechner-Scott, Dana Horakova, Freek Verheul, Alexandre Prat, Thor Petersen, Vahid Shaygannejad, Raymond Hupperts, Vilija Jokubaitis, Guillermo Izquierdo, Daniele Spitaleri, Jose Luis Sanchez-Menoyo, Maria Trojano, Franco Granella, Helmut Butzkueven, Mark Slee, Seyed Aidin Sajedi, Cavit Boz, Maria Pia Sormani, Pierre Duquette, Cristina Ramo, Eugenio Pucci, Alessio Signori, Celia Oreja-Guevara, Roberto Bergamaschi, Nastaran Majdinasab, Vincent Van Pesch, Bruce V. Taylor, Stella Hughes, Gerardo Iuliano, Maria Edite Rio, Ondokuz Mayıs Üniversitesi, Signori, Alessio, Izquierdo, Guillermo, Lugaresi, Alessandra, Hupperts, Raymond, Grand'Maison, Francoi, Sola, Patrizia, Horakova, Dana, Havrdova, Eva, Prat, Alexandre, Girard, Marc, Duquette, Pierre, Boz, Cavit, Grammond, Pierre, Terzi, Murat, Singhal, Bhim, Alroughani, Raed, Petersen, Thor, Ramo, Cristina, Oreja-Guevara, Celia, Spitaleri, Daniele, Shaygannejad, Vahid, Butzkueven, Helmut, Kalincik, Toma, Jokubaitis, Vilija, Slee, Mark, Fernandez Bolaños, Ricardo, Sanchez-Menoyo, Jose Lui, Pucci, Eugenio, Granella, Franco, Lechner-Scott, Jeannette, Iuliano, Gerardo, Hughes, Stella, Bergamaschi, Roberto, Taylor, Bruce, Verheul, Freek, Edite Rio, Maria, Amato, Maria Pia, Sajedi, Seyed Aidin, Majdinasab, Nastaran, Van Pesch, Vincent, Sormani, Maria Pia, and Trojano, Maria
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0301 basic medicine ,Male ,Pediatrics ,Delayed Diagnosis ,Placebo-controlled study ,Disease ,Disability Evaluation ,Primary progressive multiple sclerosis ,0302 clinical medicine ,Longitudinal Studies ,Prospective Studies ,Registries ,Prospective cohort study ,Age Factors ,clinical trial ,Middle Aged ,Multiple Sclerosis, Chronic Progressive ,Prognosis ,Latent class model ,Natural history ,Neurology ,Latent Class Analysis ,Disease Progression ,Female ,Adult ,medicine.medical_specialty ,Primary Progressive Multiple Sclerosis ,03 medical and health sciences ,Primary progressive multiple sclerosis, clinical trials, disability, heterogeneity, long-term ,trajectories ,Sex Factors ,medicine ,Humans ,Disabled Persons ,long-term ,clinical trials ,business.industry ,Multiple sclerosis ,Bayes Theorem ,Long term disability ,medicine.disease ,Primary progressive multiple sclerosi ,030104 developmental biology ,disability ,Physical therapy ,Neurology (clinical) ,heterogeneity ,business ,030217 neurology & neurosurgery ,Follow-Up Studies - Abstract
Signori, Alessio/0000-0001-6289-9144; Sajedi, Seyed Aidin/0000-0002-6704-9787; Jokubaitis, Vilija G./0000-0002-3942-4340; Shaygannejad, Vahid/0000-0002-6226-0161; Oreja-Guevara, Celia/0000-0002-9221-5716; amato, Maria Pia/0000-0003-3325-3760; Lugaresi, Alessandra/0000-0003-2902-5589; Majdinasab, Nastaran/0000-0001-5163-7210; Kalincik, Tomas/0000-0003-3778-1376; Butzkueven, Helmut/0000-0003-3940-8727; van Pesch, Vincent/0000-0003-2885-9004; pucci, eugenio/0000-0001-7606-7330; Petersen, Thor/0000-0001-5633-2600; Prat, Alexandre/0000-0001-6188-0580; Sanchez Menoyo, Jose Luis/0000-0003-2634-8294; Slee, Mark/0000-0003-4323-2453; SORMANI, MARIA PIA/0000-0001-6892-104X; Trojano, Maria/0000-0002-6329-8946 WOS: 000432098100013 PubMed: 28382837 Background: Several natural history studies on primary progressive multiple sclerosis (PPMS) patients detected a consistent heterogeneity in the rate of disability accumulation. Objectives: To identify subgroups of PPMS patients with similar longitudinal trajectories of Expanded Disability Status Scale (EDSS) over time. Methods: All PPMS patients collected within the MSBase registry, who had their first EDSS assessment within 5 years from onset, were included in the analysis. Longitudinal EDSS scores were modeled by a latent class mixed model (LCMM), using a nonlinear function of time from onset. LCMM is an advanced statistical approach that models heterogeneity between patients by classifying them into unobserved groups showing similar characteristics. Results: A total of 853 PPMS (51.7% females) from 24 countries with a mean age at onset of 42.4 years (standard deviation (SD): 10.8 years), a median baseline EDSS of 4 (interquartile range (IQR): 2.5-5.5), and 2.4 years of disease duration (SD: 1.5 years) were included. LCMM detected three different subgroups of patients with a mild (n = 143; 16.8%), moderate (n = 378; 44.3%), or severe (n = 332; 38.9%) disability trajectory. The probability of reaching EDSS 6 at 10 years was 0%, 46.4%, and 81.9% respectively. Conclusion: Applying an LCMM modeling approach to long-term EDSS data, it is possible to identify groups of PPMS patients with different prognosis. MSBase Foundation; Merck SeronoMerck SeronoMerck & Company; BiogenBiogen; Novartis Pharma; Bayer ScheringBayer AG; Sanofi-AventisSanofi-Aventis; BioCSL The author(s) disclosed receipt of the following financial support for the research, authorship, and/or publication of this article: This study was funded by the MSBase Foundation. The MSBase Foundation is a not-for-profit organization that receives support from Merck Serono, Biogen, Novartis Pharma, Bayer Schering, Sanofi-Aventis and BioCSL. The study was conducted separately and apart from the guidance of the sponsors.
- Published
- 2017