Long-term follow-up of pediatric MS patients starting treatment with injectable first-line agents

A multicentre, Italian, retrospective, observational study

iMED Registry and Gruppo di Studio SM-Società Italiana di Neurologia

Research output: Contribution to journalArticle

Abstract

BACKGROUND: Few data are available on very long-term follow-up of pediatric multiple sclerosis (MS) patients treated with disease modifying treatments (DMTs).

OBJECTIVES: To present a long-term follow-up of a cohort of Pediatric-MS patients starting injectable first-line agents.

METHODS: Data regarding treatments, annualized relapse rate (ARR), Expanded Disability Status Scale (EDSS) score, and serious adverse event were collected. Baseline characteristics were tested in multivariate analysis to identify predictors of disease evolution.

RESULTS: In total, 97 patients were followed for 12.5 ± 3.3 years. They started therapy at 13.9 ± 2.1 years, 88 with interferons and 9 with copaxone. During the whole follow-up, 82 patients changed therapy, switching to immunosuppressors/second-line treatment in 58% of cases. Compared to pre-treatment phase, the ARR was significantly reduced during the first treatment (from 3.2 ± 2.6 to 0.7 ± 1.5, p < 0.001), and it remained low during the whole follow-up (0.3 ± 0.2, p < 0.001). At last observation, 40% had disability worsening, but EDSS score remained <4 in 89%. One patient died at age of 23 years due to MS. One case of natalizumab-related progressive multifocal encephalopathy (PML) was recorded. Starting therapy before 12 years of age resulted in a better course of disease in multivariate analysis.

CONCLUSION: Pediatric-MS patients benefited from interferons/copaxone, but the majority had to switch to more powerful drugs. Starting therapy before 12 years of age could lead to a more favorable outcome.

Original languageEnglish
Pages (from-to)1352458518754364
JournalMultiple Sclerosis
DOIs
Publication statusE-pub ahead of print - Jan 1 2018

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Multiple Sclerosis
Observational Studies
Retrospective Studies
Pediatrics
Injections
Therapeutics
Interferons
Multivariate Analysis
Recurrence
Brain Diseases
Observation
Pharmaceutical Preparations

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Long-term follow-up of pediatric MS patients starting treatment with injectable first-line agents : A multicentre, Italian, retrospective, observational study. / iMED Registry and Gruppo di Studio SM-Società Italiana di Neurologia.

In: Multiple Sclerosis, 01.01.2018, p. 1352458518754364.

Research output: Contribution to journalArticle

@article{67bff2c4810b4d4eb445e9e82f888681,
title = "Long-term follow-up of pediatric MS patients starting treatment with injectable first-line agents: A multicentre, Italian, retrospective, observational study",
abstract = "BACKGROUND: Few data are available on very long-term follow-up of pediatric multiple sclerosis (MS) patients treated with disease modifying treatments (DMTs).OBJECTIVES: To present a long-term follow-up of a cohort of Pediatric-MS patients starting injectable first-line agents.METHODS: Data regarding treatments, annualized relapse rate (ARR), Expanded Disability Status Scale (EDSS) score, and serious adverse event were collected. Baseline characteristics were tested in multivariate analysis to identify predictors of disease evolution.RESULTS: In total, 97 patients were followed for 12.5 ± 3.3 years. They started therapy at 13.9 ± 2.1 years, 88 with interferons and 9 with copaxone. During the whole follow-up, 82 patients changed therapy, switching to immunosuppressors/second-line treatment in 58{\%} of cases. Compared to pre-treatment phase, the ARR was significantly reduced during the first treatment (from 3.2 ± 2.6 to 0.7 ± 1.5, p < 0.001), and it remained low during the whole follow-up (0.3 ± 0.2, p < 0.001). At last observation, 40{\%} had disability worsening, but EDSS score remained <4 in 89{\%}. One patient died at age of 23 years due to MS. One case of natalizumab-related progressive multifocal encephalopathy (PML) was recorded. Starting therapy before 12 years of age resulted in a better course of disease in multivariate analysis.CONCLUSION: Pediatric-MS patients benefited from interferons/copaxone, but the majority had to switch to more powerful drugs. Starting therapy before 12 years of age could lead to a more favorable outcome.",
author = "{iMED Registry and Gruppo di Studio SM-Societ{\`a} Italiana di Neurologia} and Damiano Baroncini and Mauro Zaffaroni and Lucia Moiola and Lorena Lorefice and Giuseppe Fenu and Pietro Iaffaldano and Marta Simone and Fulvia Fanelli and Francesco Patti and Emanuele D'Amico and Marco Capobianco and Antonio Bertolotto and Paolo Gallo and Monica Margoni and Silvia Miante and Nicoletta Milani and Amato, {Maria Pia} and Isabella Righini and Paolo Bellantonio and Cinzia Scandellari and Gianfranco Costantino and Elio Scarpini and Roberto Bergamaschi and Giulia Mallucci and Giancarlo Comi and Angelo Ghezzi",
year = "2018",
month = "1",
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journal = "Multiple Sclerosis",
issn = "1352-4585",
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TY - JOUR

T1 - Long-term follow-up of pediatric MS patients starting treatment with injectable first-line agents

T2 - A multicentre, Italian, retrospective, observational study

AU - iMED Registry and Gruppo di Studio SM-Società Italiana di Neurologia

AU - Baroncini, Damiano

AU - Zaffaroni, Mauro

AU - Moiola, Lucia

AU - Lorefice, Lorena

AU - Fenu, Giuseppe

AU - Iaffaldano, Pietro

AU - Simone, Marta

AU - Fanelli, Fulvia

AU - Patti, Francesco

AU - D'Amico, Emanuele

AU - Capobianco, Marco

AU - Bertolotto, Antonio

AU - Gallo, Paolo

AU - Margoni, Monica

AU - Miante, Silvia

AU - Milani, Nicoletta

AU - Amato, Maria Pia

AU - Righini, Isabella

AU - Bellantonio, Paolo

AU - Scandellari, Cinzia

AU - Costantino, Gianfranco

AU - Scarpini, Elio

AU - Bergamaschi, Roberto

AU - Mallucci, Giulia

AU - Comi, Giancarlo

AU - Ghezzi, Angelo

PY - 2018/1/1

Y1 - 2018/1/1

N2 - BACKGROUND: Few data are available on very long-term follow-up of pediatric multiple sclerosis (MS) patients treated with disease modifying treatments (DMTs).OBJECTIVES: To present a long-term follow-up of a cohort of Pediatric-MS patients starting injectable first-line agents.METHODS: Data regarding treatments, annualized relapse rate (ARR), Expanded Disability Status Scale (EDSS) score, and serious adverse event were collected. Baseline characteristics were tested in multivariate analysis to identify predictors of disease evolution.RESULTS: In total, 97 patients were followed for 12.5 ± 3.3 years. They started therapy at 13.9 ± 2.1 years, 88 with interferons and 9 with copaxone. During the whole follow-up, 82 patients changed therapy, switching to immunosuppressors/second-line treatment in 58% of cases. Compared to pre-treatment phase, the ARR was significantly reduced during the first treatment (from 3.2 ± 2.6 to 0.7 ± 1.5, p < 0.001), and it remained low during the whole follow-up (0.3 ± 0.2, p < 0.001). At last observation, 40% had disability worsening, but EDSS score remained <4 in 89%. One patient died at age of 23 years due to MS. One case of natalizumab-related progressive multifocal encephalopathy (PML) was recorded. Starting therapy before 12 years of age resulted in a better course of disease in multivariate analysis.CONCLUSION: Pediatric-MS patients benefited from interferons/copaxone, but the majority had to switch to more powerful drugs. Starting therapy before 12 years of age could lead to a more favorable outcome.

AB - BACKGROUND: Few data are available on very long-term follow-up of pediatric multiple sclerosis (MS) patients treated with disease modifying treatments (DMTs).OBJECTIVES: To present a long-term follow-up of a cohort of Pediatric-MS patients starting injectable first-line agents.METHODS: Data regarding treatments, annualized relapse rate (ARR), Expanded Disability Status Scale (EDSS) score, and serious adverse event were collected. Baseline characteristics were tested in multivariate analysis to identify predictors of disease evolution.RESULTS: In total, 97 patients were followed for 12.5 ± 3.3 years. They started therapy at 13.9 ± 2.1 years, 88 with interferons and 9 with copaxone. During the whole follow-up, 82 patients changed therapy, switching to immunosuppressors/second-line treatment in 58% of cases. Compared to pre-treatment phase, the ARR was significantly reduced during the first treatment (from 3.2 ± 2.6 to 0.7 ± 1.5, p < 0.001), and it remained low during the whole follow-up (0.3 ± 0.2, p < 0.001). At last observation, 40% had disability worsening, but EDSS score remained <4 in 89%. One patient died at age of 23 years due to MS. One case of natalizumab-related progressive multifocal encephalopathy (PML) was recorded. Starting therapy before 12 years of age resulted in a better course of disease in multivariate analysis.CONCLUSION: Pediatric-MS patients benefited from interferons/copaxone, but the majority had to switch to more powerful drugs. Starting therapy before 12 years of age could lead to a more favorable outcome.

U2 - 10.1177/1352458518754364

DO - 10.1177/1352458518754364

M3 - Article

SP - 1352458518754364

JO - Multiple Sclerosis

JF - Multiple Sclerosis

SN - 1352-4585

ER -