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The Impact of Highly Effective Treatment in Pediatric-Onset Multiple Sclerosis: A Case Series

Introduction: Pediatric-onset multiple sclerosis (POMS) is characterized by high inflammatory disease activity. Our aim was to describe the treatment sequencing and report the impact highly effective disease-modifying treatment (HET) had on disease activity. Materials and Methods: Five consecutive p...

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Autores principales: Immovilli, Paolo, De Mitri, Paola, Bazzurri, Veronica, Vollaro, Stefano, Morelli, Nicola, Biasucci, Giacomo, Magnifico, Fabiola, Marchesi, Elena, Lombardelli, Maria Lara, Gelati, Lorenza, Guidetti, Donata
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9688929/
https://www.ncbi.nlm.nih.gov/pubmed/36360426
http://dx.doi.org/10.3390/children9111698
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author Immovilli, Paolo
De Mitri, Paola
Bazzurri, Veronica
Vollaro, Stefano
Morelli, Nicola
Biasucci, Giacomo
Magnifico, Fabiola
Marchesi, Elena
Lombardelli, Maria Lara
Gelati, Lorenza
Guidetti, Donata
author_facet Immovilli, Paolo
De Mitri, Paola
Bazzurri, Veronica
Vollaro, Stefano
Morelli, Nicola
Biasucci, Giacomo
Magnifico, Fabiola
Marchesi, Elena
Lombardelli, Maria Lara
Gelati, Lorenza
Guidetti, Donata
author_sort Immovilli, Paolo
collection PubMed
description Introduction: Pediatric-onset multiple sclerosis (POMS) is characterized by high inflammatory disease activity. Our aim was to describe the treatment sequencing and report the impact highly effective disease-modifying treatment (HET) had on disease activity. Materials and Methods: Five consecutive patients with POMS were administered HET following lower efficacy drug or as initial therapy. Data on treatment sequencing, relapses and MRIs were collected during the follow-up. Results: Our patients had an average age of 13.8 years (range 9–17) at diagnosis and 13.4 years (range 9–16) at disease onset, and 2/5 (40%) POMS were female. The pre-treatment average annualized relapse rate was 1.6 (range 0.8–2.8), and the average follow-up length was 5 years (range 3–7). A total of 2/5 (40%) patients were stable on HET at initial therapy, and 3/5 (60%) required an escalation to more aggressive treatment, even if two of them had been put on HET as initial treatment. Four out of five patients (80%) had No Evidence of Disease Activity-3 status (NEDA-3) at an average follow-up of 3 years (range 2–5). Conclusion: It has been observed that in a recent time period all the cases had prompt diagnosis, early HET or escalation to HET with a good outcome in 80% of the cases.
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spelling pubmed-96889292022-11-25 The Impact of Highly Effective Treatment in Pediatric-Onset Multiple Sclerosis: A Case Series Immovilli, Paolo De Mitri, Paola Bazzurri, Veronica Vollaro, Stefano Morelli, Nicola Biasucci, Giacomo Magnifico, Fabiola Marchesi, Elena Lombardelli, Maria Lara Gelati, Lorenza Guidetti, Donata Children (Basel) Article Introduction: Pediatric-onset multiple sclerosis (POMS) is characterized by high inflammatory disease activity. Our aim was to describe the treatment sequencing and report the impact highly effective disease-modifying treatment (HET) had on disease activity. Materials and Methods: Five consecutive patients with POMS were administered HET following lower efficacy drug or as initial therapy. Data on treatment sequencing, relapses and MRIs were collected during the follow-up. Results: Our patients had an average age of 13.8 years (range 9–17) at diagnosis and 13.4 years (range 9–16) at disease onset, and 2/5 (40%) POMS were female. The pre-treatment average annualized relapse rate was 1.6 (range 0.8–2.8), and the average follow-up length was 5 years (range 3–7). A total of 2/5 (40%) patients were stable on HET at initial therapy, and 3/5 (60%) required an escalation to more aggressive treatment, even if two of them had been put on HET as initial treatment. Four out of five patients (80%) had No Evidence of Disease Activity-3 status (NEDA-3) at an average follow-up of 3 years (range 2–5). Conclusion: It has been observed that in a recent time period all the cases had prompt diagnosis, early HET or escalation to HET with a good outcome in 80% of the cases. MDPI 2022-11-05 /pmc/articles/PMC9688929/ /pubmed/36360426 http://dx.doi.org/10.3390/children9111698 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Article
Immovilli, Paolo
De Mitri, Paola
Bazzurri, Veronica
Vollaro, Stefano
Morelli, Nicola
Biasucci, Giacomo
Magnifico, Fabiola
Marchesi, Elena
Lombardelli, Maria Lara
Gelati, Lorenza
Guidetti, Donata
The Impact of Highly Effective Treatment in Pediatric-Onset Multiple Sclerosis: A Case Series
title The Impact of Highly Effective Treatment in Pediatric-Onset Multiple Sclerosis: A Case Series
title_full The Impact of Highly Effective Treatment in Pediatric-Onset Multiple Sclerosis: A Case Series
title_fullStr The Impact of Highly Effective Treatment in Pediatric-Onset Multiple Sclerosis: A Case Series
title_full_unstemmed The Impact of Highly Effective Treatment in Pediatric-Onset Multiple Sclerosis: A Case Series
title_short The Impact of Highly Effective Treatment in Pediatric-Onset Multiple Sclerosis: A Case Series
title_sort impact of highly effective treatment in pediatric-onset multiple sclerosis: a case series
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9688929/
https://www.ncbi.nlm.nih.gov/pubmed/36360426
http://dx.doi.org/10.3390/children9111698
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