Long-term disability trajectories in primary progressivs MS patients - a latent class growth analysis.

A. Signori; G. Izquierdo; A. Lugaresi; R. Hupperts; F. Grand'Maison; P. Sola; D. Horakova; E. Havrdova; A. Prat; M. Girard; P. Duquette; C. Boz; P. Grammond; M. Terzi; B. Singhal; R. Alroughani; T. Petersen; C. Ramo; C. Oreja-Guevara; D. Spitaleri; V. Shaygannejad; H. Butzkueven; T. Kalincik; V. Jokubaitis; M. Slee; R. F. Bolaños; J. L. Sanchez-Menoyo; E. Pucci; F. Granella; J. Lechner-Scott; Gerardo Iuliano; S. Hughes; R. Bergamaschi; B. Taylor; F. Verheul; M. E. Rio; M. P. Amato; S. A. Sajedi; N. Majdinasab; V. Van Pesch; M. P. Sormani; M. Trojano; MSBase Study Group

doi:10.1177/1352458516663068

Long-term disability trajectories in primary progressivs MS patients - a latent class growth analysis.

A. Signori, G. Izquierdo, A. Lugaresi, R. Hupperts, F. Grand'Maison, P. Sola, D. Horakova, E. Havrdova, A. Prat, M. Girard, P. Duquette, C. Boz, P. Grammond, M. Terzi, B. Singhal, R. Alroughani, T. Petersen, C. Ramo, C. Oreja-Guevara, D. SpitaleriV. Shaygannejad, H. Butzkueven, T. Kalincik, V. Jokubaitis, M. Slee, R. F. Bolaños, J. L. Sanchez-Menoyo, E. Pucci, F. Granella, J. Lechner-Scott, Gerardo Iuliano, S. Hughes, R. Bergamaschi, B. Taylor, F. Verheul, M. E. Rio, M. P. Amato, S. A. Sajedi, N. Majdinasab, V. Van Pesch, M. P. Sormani, M. Trojano, MSBase Study Group

Research output: Contribution to journal › Meeting Abstract › peer-review

Abstract

Background: Over the last decades several natural history studies on primary progressive MS (PPMS) patients were reported from international registries. In this population a consistent heterogeneity was observed in the rate of disability accumulation, as time to reach the milestone of EDSS 6 ranged between 7 and 14 years1-3 from onset.

Objectives: To identify subgroups of PPMS patients with similar longitudinal trajectories of EDSS over time.

Methods: All PPMS patients collected within MSBase international registry, who had their first EDSS assessment within 5 years from onset were included in the analysis. Longitudinal EDSS scores were modelled 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 (latent classes) showing similar characteristics.

Results: A total of 853 PPMS (51.7% females) from 24 countries with a mean age at onset of 42.4 yrs (SD: 10.8 yrs), a median baseline EDSS of 4 (IQR: 2.5-5.5) and 2.4 yrs of disease duration (SD: 1.5 yrs) were included. LCMM detected 3 different subgroups of patients with, respectively, a mild (n=143 ;16.8%), a moderate (n=378; 44.3%) and a severe (n=332; 38.9%) disability trajectory. Median time to EDSS 4 was 14, 5 and 3.7 years, for the 3 groups respectively; the probability to reach EDSS 6 at 10 years was 0%, 46.5% and 83.1% respectively. Increasing the severity of the disability time course was related to a decreased frequency of patients with at least one relapse during follow-up from 47.6% to 36.5% (p=0.033). Using this modelling approach it is possible to predict the future disease course of a subject with PPMS using
early EDSS assessments: by using only 1 year of EDSS monitoring 73% of patients are correctly classified in their disability trajectory group (mild, moderate or severe); after 3 years this proportion is 87% and after 5 years it is 92%.

Conclusions: Using long term observations and a LCMM modelling approach it is possible to build a dynamic model, to predict the future disability trajectory of a new patient with PPMS. In the design of future clinical trials in PPMS, with time to reach disability milestones as the primary endpoint, the existence of heterogeneous classes of patients should be considered.

Original language	English
Article number	154
Pages (from-to)	44-46
Number of pages	3
Journal	Multiple Sclerosis
Volume	22
Issue number	Supp: 3
DOIs	https://doi.org/10.1177/1352458516663068
Publication status	Published - Sept 2016

Keywords

Multiple Sclerosis
primary progressive MS (PPMS)
Trajectories

Access to Document

10.1177/1352458516663068Licence: In Copyright

Cite this

Signori, A., Izquierdo, G., Lugaresi, A., Hupperts, R., Grand'Maison, F., Sola, P., Horakova, D., Havrdova, E., Prat, A., Girard, M., Duquette, P., Boz, C., Grammond, P., Terzi, M., Singhal, B., Alroughani, R., Petersen, T., Ramo, C., Oreja-Guevara, C., ... MSBase Study Group (2016). Long-term disability trajectories in primary progressivs MS patients - a latent class growth analysis. Multiple Sclerosis, 22(Supp: 3), 44-46. Article 154. https://doi.org/10.1177/1352458516663068

@article{fdba4f42cd524fb99694324703f3a50c,

title = "Long-term disability trajectories in primary progressivs MS patients - a latent class growth analysis.",

abstract = "Background: Over the last decades several natural history studies on primary progressive MS (PPMS) patients were reported from international registries. In this population a consistent heterogeneity was observed in the rate of disability accumulation, as time to reach the milestone of EDSS 6 ranged between 7 and 14 years1-3 from onset.Objectives: To identify subgroups of PPMS patients with similar longitudinal trajectories of EDSS over time.Methods: All PPMS patients collected within MSBase international registry, who had their first EDSS assessment within 5 years from onset were included in the analysis. Longitudinal EDSS scores were modelled 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 (latent classes) showing similar characteristics.Results: A total of 853 PPMS (51.7% females) from 24 countries with a mean age at onset of 42.4 yrs (SD: 10.8 yrs), a median baseline EDSS of 4 (IQR: 2.5-5.5) and 2.4 yrs of disease duration (SD: 1.5 yrs) were included. LCMM detected 3 different subgroups of patients with, respectively, a mild (n=143 ;16.8%), a moderate (n=378; 44.3%) and a severe (n=332; 38.9%) disability trajectory. Median time to EDSS 4 was 14, 5 and 3.7 years, for the 3 groups respectively; the probability to reach EDSS 6 at 10 years was 0%, 46.5% and 83.1% respectively. Increasing the severity of the disability time course was related to a decreased frequency of patients with at least one relapse during follow-up from 47.6% to 36.5% (p=0.033). Using this modelling approach it is possible to predict the future disease course of a subject with PPMS usingearly EDSS assessments: by using only 1 year of EDSS monitoring 73% of patients are correctly classified in their disability trajectory group (mild, moderate or severe); after 3 years this proportion is 87% and after 5 years it is 92%.Conclusions: Using long term observations and a LCMM modelling approach it is possible to build a dynamic model, to predict the future disability trajectory of a new patient with PPMS. In the design of future clinical trials in PPMS, with time to reach disability milestones as the primary endpoint, the existence of heterogeneous classes of patients should be considered.",

keywords = "Multiple Sclerosis, primary progressive MS (PPMS), Trajectories",

author = "A. Signori and G. Izquierdo and A. Lugaresi and R. Hupperts and F. Grand'Maison and P. Sola and D. Horakova and E. Havrdova and A. Prat and M. Girard and P. Duquette and C. Boz and P. Grammond and M. Terzi and B. Singhal and R. Alroughani and T. Petersen and C. Ramo and C. Oreja-Guevara and D. Spitaleri and V. Shaygannejad and H. Butzkueven and T. Kalincik and V. Jokubaitis and M. Slee and Bola{\~n}os, {R. F.} and Sanchez-Menoyo, {J. L.} and E. Pucci and F. Granella and J. Lechner-Scott and Gerardo Iuliano and S. Hughes and R. Bergamaschi and B. Taylor and F. Verheul and Rio, {M. E.} and Amato, {M. P.} and Sajedi, {S. A.} and N. Majdinasab and {Van Pesch}, V. and Sormani, {M. P.} and M. Trojano and {MSBase Study Group}",

year = "2016",

month = sep,

doi = "10.1177/1352458516663068",

language = "English",

volume = "22",

pages = "44--46",

journal = "Multiple Sclerosis",

issn = "1352-4585",

publisher = "SAGE Publications Ltd",

number = "Supp: 3",

}

Signori, A, Izquierdo, G, Lugaresi, A, Hupperts, R, Grand'Maison, F, Sola, P, Horakova, D, Havrdova, E, Prat, A, Girard, M, Duquette, P, Boz, C, Grammond, P, Terzi, M, Singhal, B, Alroughani, R, Petersen, T, Ramo, C, Oreja-Guevara, C, Spitaleri, D, Shaygannejad, V, Butzkueven, H, Kalincik, T, Jokubaitis, V, Slee, M, Bolaños, RF, Sanchez-Menoyo, JL, Pucci, E, Granella, F, Lechner-Scott, J, Iuliano, G, Hughes, S, Bergamaschi, R, Taylor, B, Verheul, F, Rio, ME, Amato, MP, Sajedi, SA, Majdinasab, N, Van Pesch, V, Sormani, MP, Trojano, M & MSBase Study Group 2016, 'Long-term disability trajectories in primary progressivs MS patients - a latent class growth analysis.', Multiple Sclerosis, vol. 22, no. Supp: 3, 154, pp. 44-46. https://doi.org/10.1177/1352458516663068

TY - JOUR

T1 - Long-term disability trajectories in primary progressivs MS patients - a latent class growth analysis.

AU - Signori, A.

AU - Izquierdo, G.

AU - Lugaresi, A.

AU - Hupperts, R.

AU - Grand'Maison, F.

AU - Sola, P.

AU - Horakova, D.

AU - Havrdova, E.

AU - Prat, A.

AU - Girard, M.

AU - Duquette, P.

AU - Boz, C.

AU - Grammond, P.

AU - Terzi, M.

AU - Singhal, B.

AU - Alroughani, R.

AU - Petersen, T.

AU - Ramo, C.

AU - Oreja-Guevara, C.

AU - Spitaleri, D.

AU - Shaygannejad, V.

AU - Butzkueven, H.

AU - Kalincik, T.

AU - Jokubaitis, V.

AU - Slee, M.

AU - Bolaños, R. F.

AU - Sanchez-Menoyo, J. L.

AU - Pucci, E.

AU - Granella, F.

AU - Lechner-Scott, J.

AU - Iuliano, Gerardo

AU - Hughes, S.

AU - Bergamaschi, R.

AU - Taylor, B.

AU - Verheul, F.

AU - Rio, M. E.

AU - Amato, M. P.

AU - Sajedi, S. A.

AU - Majdinasab, N.

AU - Van Pesch, V.

AU - Sormani, M. P.

AU - Trojano, M.

AU - MSBase Study Group

PY - 2016/9

Y1 - 2016/9

N2 - Background: Over the last decades several natural history studies on primary progressive MS (PPMS) patients were reported from international registries. In this population a consistent heterogeneity was observed in the rate of disability accumulation, as time to reach the milestone of EDSS 6 ranged between 7 and 14 years1-3 from onset.Objectives: To identify subgroups of PPMS patients with similar longitudinal trajectories of EDSS over time.Methods: All PPMS patients collected within MSBase international registry, who had their first EDSS assessment within 5 years from onset were included in the analysis. Longitudinal EDSS scores were modelled 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 (latent classes) showing similar characteristics.Results: A total of 853 PPMS (51.7% females) from 24 countries with a mean age at onset of 42.4 yrs (SD: 10.8 yrs), a median baseline EDSS of 4 (IQR: 2.5-5.5) and 2.4 yrs of disease duration (SD: 1.5 yrs) were included. LCMM detected 3 different subgroups of patients with, respectively, a mild (n=143 ;16.8%), a moderate (n=378; 44.3%) and a severe (n=332; 38.9%) disability trajectory. Median time to EDSS 4 was 14, 5 and 3.7 years, for the 3 groups respectively; the probability to reach EDSS 6 at 10 years was 0%, 46.5% and 83.1% respectively. Increasing the severity of the disability time course was related to a decreased frequency of patients with at least one relapse during follow-up from 47.6% to 36.5% (p=0.033). Using this modelling approach it is possible to predict the future disease course of a subject with PPMS usingearly EDSS assessments: by using only 1 year of EDSS monitoring 73% of patients are correctly classified in their disability trajectory group (mild, moderate or severe); after 3 years this proportion is 87% and after 5 years it is 92%.Conclusions: Using long term observations and a LCMM modelling approach it is possible to build a dynamic model, to predict the future disability trajectory of a new patient with PPMS. In the design of future clinical trials in PPMS, with time to reach disability milestones as the primary endpoint, the existence of heterogeneous classes of patients should be considered.

AB - Background: Over the last decades several natural history studies on primary progressive MS (PPMS) patients were reported from international registries. In this population a consistent heterogeneity was observed in the rate of disability accumulation, as time to reach the milestone of EDSS 6 ranged between 7 and 14 years1-3 from onset.Objectives: To identify subgroups of PPMS patients with similar longitudinal trajectories of EDSS over time.Methods: All PPMS patients collected within MSBase international registry, who had their first EDSS assessment within 5 years from onset were included in the analysis. Longitudinal EDSS scores were modelled 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 (latent classes) showing similar characteristics.Results: A total of 853 PPMS (51.7% females) from 24 countries with a mean age at onset of 42.4 yrs (SD: 10.8 yrs), a median baseline EDSS of 4 (IQR: 2.5-5.5) and 2.4 yrs of disease duration (SD: 1.5 yrs) were included. LCMM detected 3 different subgroups of patients with, respectively, a mild (n=143 ;16.8%), a moderate (n=378; 44.3%) and a severe (n=332; 38.9%) disability trajectory. Median time to EDSS 4 was 14, 5 and 3.7 years, for the 3 groups respectively; the probability to reach EDSS 6 at 10 years was 0%, 46.5% and 83.1% respectively. Increasing the severity of the disability time course was related to a decreased frequency of patients with at least one relapse during follow-up from 47.6% to 36.5% (p=0.033). Using this modelling approach it is possible to predict the future disease course of a subject with PPMS usingearly EDSS assessments: by using only 1 year of EDSS monitoring 73% of patients are correctly classified in their disability trajectory group (mild, moderate or severe); after 3 years this proportion is 87% and after 5 years it is 92%.Conclusions: Using long term observations and a LCMM modelling approach it is possible to build a dynamic model, to predict the future disability trajectory of a new patient with PPMS. In the design of future clinical trials in PPMS, with time to reach disability milestones as the primary endpoint, the existence of heterogeneous classes of patients should be considered.

KW - Multiple Sclerosis

KW - primary progressive MS (PPMS)

KW - Trajectories

U2 - 10.1177/1352458516663068

DO - 10.1177/1352458516663068

M3 - Meeting Abstract

SN - 1352-4585

VL - 22

SP - 44

EP - 46

JO - Multiple Sclerosis

JF - Multiple Sclerosis

IS - Supp: 3

M1 - 154

ER -

Long-term disability trajectories in primary progressivs MS patients - a latent class growth analysis.

Abstract

Keywords

Access to Document

Fingerprint

Cite this