TY - JOUR
T1 - Long-term disability trajectories in primary progressive MS patients
T2 - A latent class growth analysis
AU - Signori, Alessio
AU - Izquierdo, Guillermo
AU - Lugaresi, Alessandra
AU - Hupperts, Raymond
AU - Grand’Maison, Francois
AU - Sola, Patrizia
AU - Horakova, Dana
AU - Havrdova, Eva
AU - Prat, Alexandre
AU - Girard, Marc
AU - Duquette, Pierre
AU - Boz, Cavit
AU - Grammond, Pierre
AU - Terzi, Murat
AU - Singhal, Bhim
AU - Alroughani, Raed
AU - Petersen, Thor
AU - Ramo, Cristina
AU - Oreja-Guevara, Celia
AU - Spitaleri, Daniele
AU - Shaygannejad, Vahid
AU - Butzkueven, Helmut
AU - Kalincik, Tomas
AU - Jokubaitis, Vilija
AU - Slee, Mark
AU - Fernandez Bolaños, Ricardo
AU - Sanchez-Menoyo, Jose Luis
AU - Pucci, Eugenio
AU - Granella, Franco
AU - Lechner-Scott, Jeannette
AU - Iuliano, Gerardo
AU - Hughes, Stella
AU - Bergamaschi, Roberto
AU - Taylor, Bruce
AU - Verheul, Freek
AU - Edite Rio, Maria
AU - Amato, Maria Pia
AU - Sajedi, Seyed Aidin
AU - Majdinasab, Nastaran
AU - Van Pesch, Vincent
AU - Sormani, Maria Pia
AU - Trojano, Maria
PY - 2018/4/1
Y1 - 2018/4/1
N2 - 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.
AB - 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.
KW - clinical trials
KW - disability
KW - heterogeneity
KW - long-term
KW - Primary progressive multiple sclerosis
KW - trajectories
UR - http://www.scopus.com/inward/record.url?scp=85032873925&partnerID=8YFLogxK
U2 - 10.1177/1352458517703800
DO - 10.1177/1352458517703800
M3 - Article
AN - SCOPUS:85032873925
VL - 24
SP - 642
EP - 652
JO - Multiple Sclerosis
JF - Multiple Sclerosis
SN - 1352-4585
IS - 5
ER -