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.

Long-term disability trajectories in primary progressive MS patients: A latent class growth analysis / Signori, Alessio; Izquierdo, Guillermo; Lugaresi, Alessandra; Hupperts, Raymond; Grand'Maison, Francois; 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, Tomas; Jokubaitis, Vilija; Slee, Mark; Fernandez Bolaños, Ricardo; Sanchez Menoyo, Jose Luis; 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; Trojano, Maria. - In: MULTIPLE SCLEROSIS. - ISSN 1352-4585. - 24:5(2018), pp. 642-652. [10.1177/1352458517703800]

Long-term disability trajectories in primary progressive MS patients: A latent class growth analysis

GRANELLA, Franco;
2018-01-01

Abstract

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.
Long-term disability trajectories in primary progressive MS patients: A latent class growth analysis / Signori, Alessio; Izquierdo, Guillermo; Lugaresi, Alessandra; Hupperts, Raymond; Grand'Maison, Francois; 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, Tomas; Jokubaitis, Vilija; Slee, Mark; Fernandez Bolaños, Ricardo; Sanchez Menoyo, Jose Luis; 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; Trojano, Maria. - In: MULTIPLE SCLEROSIS. - ISSN 1352-4585. - 24:5(2018), pp. 642-652. [10.1177/1352458517703800]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11381/2827442
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