ECTRIMS eLearning

Disease-modifying therapy of paediatric onset multiple sclerosis. A nationwide Danish cohort study
Author(s): ,
J.L. Erdal
Affiliations:
Department of Neurology, Rigshospitalet, The Danish Multiple Sclerosis Registry
,
T. Iskov Kopp
Affiliations:
Department of Neurology, Rigshospitalet, The Danish Multiple Sclerosis Registry; Department of Neurology, Rigshospitalet, University of Copenhagen, Danish Multiple Sclerosis Center, Copenhagen
,
M. Blinkenberg
Affiliations:
Department of Neurology, Rigshospitalet, University of Copenhagen, Danish Multiple Sclerosis Center, Copenhagen
,
T. Petersen
Affiliations:
Department of Neurology, Aarhus University Hospital, Aarhus
,
M. Ravnborg
Affiliations:
Odense University Hospital, University of Southern Denmark, Odense, Denmark
,
T. Chalmer
Affiliations:
Department of Neurology, Rigshospitalet, The Danish Multiple Sclerosis Registry; Department of Neurology, Rigshospitalet, University of Copenhagen, Danish Multiple Sclerosis Center, Copenhagen
,
P. Soelberg Sørensen
Affiliations:
Department of Neurology, Rigshospitalet, University of Copenhagen, Danish Multiple Sclerosis Center, Copenhagen
M. Magyari
Affiliations:
Department of Neurology, Rigshospitalet, The Danish Multiple Sclerosis Registry; Department of Neurology, Rigshospitalet, University of Copenhagen, Danish Multiple Sclerosis Center, Copenhagen
ECTRIMS Learn. Magyari M. 10/10/18; 229439; EP1602
Dr. Melinda Magyari
Dr. Melinda Magyari
Contributions
Abstract

Abstract: EP1602

Type: Poster Sessions

Abstract Category: Therapy - Immunomodulation/Immunosuppression

Introduction: Paediatric onset multiple sclerosis (POMS) is rarer than adult-onset disease, and can represent challenges in the diagnosis and treatment.
Aims: The aim of the study was to describe the cohort of persons with disease onset before the age of 18 and to characterize the treatment of POMS in Denmark since 1996, when disease-modifying therapy (DMT) became available, until data were sourced in February 2018.
Methods: The complete cohort of 134 POMS patients treated with a DMT before the age of 18 years was extracted from the nationwide Danish Multiple Sclerosis Registry and followed until the age of 25 years. Baseline characteristics were tested in a multivariate regression analysis to predict annualized relapse rate (ARR); and in a Cox proportional hazard model to predict time to confirmed Expanded Disability Status Scale (EDSS) score worsening and improvement.
Results: Persons with POMS started their first DMT at 16.0±1.8 years and were followed for 6.4±2.9 years. In total, 100 patients (75%) switched to another DMT during follow-up, of whom 55 escalated to a second line therapy. Only 29 (22%) persons with POMS continued on their first DMT, with mean treatment duration of 71.2 months (range 7.4-198.6).
ARR was reduced from 1.8±1.1 prior to start of first DMT to 0.27±0.3 during the whole follow-up period. Males had a significantly lower ARR during follow-up compared to females (0.12 (95% confidence interval (CI): 0.073-0.19) vs. 0.37 (95% CI: 0.29-0.46), p=< .0001). Disease duration at treatment start of < 2 years was associated with lower ARR compared to ≥ years (0.16 (95% CI: 0.12-0.21) vs. 0.28 (95% CI: 0.19-0.40), p=0.0063). Relapse-free survival on first DMT was significantly lower among females than males (p=0.020). Risk of confirmed EDSS worsening among patients with an EDSS baseline ≥2 was decreased with 60% compared to patients with an EDSS baseline of < 2 (HR=0.40, 95% CI: 0.20-0.79). The chance of EDSS improvement was nearly 2-fold increased among females compared to males (HR=1.87 (95%CI: 0.94-3.74)), and an EDSS baseline ≥ 2 was associated with a 3-fold hazard of improvement (HR=3.14 (95% CI: 1.72-5.73)).
Conclusions: Predictors of disease activity on DMT were sex, disease duration and EDSS at treatment start.
Disclosure: Julie Laub Erdal: Nothing to disclose.
Tine Iskov Kopp: Nothing to disclose.
Morten Blinkenberg: has served on scientific advisory boards for Genzyme, Roche, Biogen Idec, Merck Serono, Novartis and Teva; has received speaker honoraria from Genzyme, Biogen Idec, Merck Serono, Bayer Schering, Novartis, Teva and Roche; has received consulting honoraria from the Danish Multiple Sclerosis Society, Biogen, Teva, Biogen Idec, and Merck Serono; has received funding for travel from Genzyme, Roche, Biogen Idec and Merck Serono.
Thor Petersen: Thor Petersen has received research grant support from Biogen, Merck, Novartis, Sanofi, Alexion, Roche and Genzyme.
Mads Ravnborg: Nothing to disclose.
Thor Chalmer: has received support for congress participation from Merck, Novartis, Biogen and Roche.
Per Soelberg Sørensen: Sorensen PS. has received personal compensation for serving on advisory boards for Biogen, Merck, Novartis, Teva, MedDay Pharmaceuticals and GSK; on steering committees or independent data monitoring boards in trials sponsored by Merck, Teva, GSK, and Novartis; and has received speaker honoraria from Biogen, Merck Serono, Teva, Sanofi-Aventis, Genzyme, and Novartis.
Melinda Magyari: has served on scientific advisory board for Biogen, Sanofi, Teva, Roche, Novartis, Merck, has received honoraria for lecturing from Biogen, Merck, Novartis, Sanofi, Genzyme, has received support for congress participation from Biogen, Genzyme, Teva, Roche.

Abstract: EP1602

Type: Poster Sessions

Abstract Category: Therapy - Immunomodulation/Immunosuppression

Introduction: Paediatric onset multiple sclerosis (POMS) is rarer than adult-onset disease, and can represent challenges in the diagnosis and treatment.
Aims: The aim of the study was to describe the cohort of persons with disease onset before the age of 18 and to characterize the treatment of POMS in Denmark since 1996, when disease-modifying therapy (DMT) became available, until data were sourced in February 2018.
Methods: The complete cohort of 134 POMS patients treated with a DMT before the age of 18 years was extracted from the nationwide Danish Multiple Sclerosis Registry and followed until the age of 25 years. Baseline characteristics were tested in a multivariate regression analysis to predict annualized relapse rate (ARR); and in a Cox proportional hazard model to predict time to confirmed Expanded Disability Status Scale (EDSS) score worsening and improvement.
Results: Persons with POMS started their first DMT at 16.0±1.8 years and were followed for 6.4±2.9 years. In total, 100 patients (75%) switched to another DMT during follow-up, of whom 55 escalated to a second line therapy. Only 29 (22%) persons with POMS continued on their first DMT, with mean treatment duration of 71.2 months (range 7.4-198.6).
ARR was reduced from 1.8±1.1 prior to start of first DMT to 0.27±0.3 during the whole follow-up period. Males had a significantly lower ARR during follow-up compared to females (0.12 (95% confidence interval (CI): 0.073-0.19) vs. 0.37 (95% CI: 0.29-0.46), p=< .0001). Disease duration at treatment start of < 2 years was associated with lower ARR compared to ≥ years (0.16 (95% CI: 0.12-0.21) vs. 0.28 (95% CI: 0.19-0.40), p=0.0063). Relapse-free survival on first DMT was significantly lower among females than males (p=0.020). Risk of confirmed EDSS worsening among patients with an EDSS baseline ≥2 was decreased with 60% compared to patients with an EDSS baseline of < 2 (HR=0.40, 95% CI: 0.20-0.79). The chance of EDSS improvement was nearly 2-fold increased among females compared to males (HR=1.87 (95%CI: 0.94-3.74)), and an EDSS baseline ≥ 2 was associated with a 3-fold hazard of improvement (HR=3.14 (95% CI: 1.72-5.73)).
Conclusions: Predictors of disease activity on DMT were sex, disease duration and EDSS at treatment start.
Disclosure: Julie Laub Erdal: Nothing to disclose.
Tine Iskov Kopp: Nothing to disclose.
Morten Blinkenberg: has served on scientific advisory boards for Genzyme, Roche, Biogen Idec, Merck Serono, Novartis and Teva; has received speaker honoraria from Genzyme, Biogen Idec, Merck Serono, Bayer Schering, Novartis, Teva and Roche; has received consulting honoraria from the Danish Multiple Sclerosis Society, Biogen, Teva, Biogen Idec, and Merck Serono; has received funding for travel from Genzyme, Roche, Biogen Idec and Merck Serono.
Thor Petersen: Thor Petersen has received research grant support from Biogen, Merck, Novartis, Sanofi, Alexion, Roche and Genzyme.
Mads Ravnborg: Nothing to disclose.
Thor Chalmer: has received support for congress participation from Merck, Novartis, Biogen and Roche.
Per Soelberg Sørensen: Sorensen PS. has received personal compensation for serving on advisory boards for Biogen, Merck, Novartis, Teva, MedDay Pharmaceuticals and GSK; on steering committees or independent data monitoring boards in trials sponsored by Merck, Teva, GSK, and Novartis; and has received speaker honoraria from Biogen, Merck Serono, Teva, Sanofi-Aventis, Genzyme, and Novartis.
Melinda Magyari: has served on scientific advisory board for Biogen, Sanofi, Teva, Roche, Novartis, Merck, has received honoraria for lecturing from Biogen, Merck, Novartis, Sanofi, Genzyme, has received support for congress participation from Biogen, Genzyme, Teva, Roche.

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