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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Main Authors: Paolo Immovilli, Paola De Mitri, Veronica Bazzurri, Stefano Vollaro, Nicola Morelli, Giacomo Biasucci, Fabiola Magnifico, Elena Marchesi, Maria Lara Lombardelli, Lorenza Gelati, Donata Guidetti
Format: Article
Language:English
Published: MDPI AG 2022-11-01
Series:Children
Subjects:
Online Access:https://www.mdpi.com/2227-9067/9/11/1698
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author Paolo Immovilli
Paola De Mitri
Veronica Bazzurri
Stefano Vollaro
Nicola Morelli
Giacomo Biasucci
Fabiola Magnifico
Elena Marchesi
Maria Lara Lombardelli
Lorenza Gelati
Donata Guidetti
author_facet Paolo Immovilli
Paola De Mitri
Veronica Bazzurri
Stefano Vollaro
Nicola Morelli
Giacomo Biasucci
Fabiola Magnifico
Elena Marchesi
Maria Lara Lombardelli
Lorenza Gelati
Donata Guidetti
author_sort Paolo Immovilli
collection DOAJ
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 doaj.art-dcf513d86dd84b3088eda2a093b37e962023-11-24T04:12:23ZengMDPI AGChildren2227-90672022-11-01911169810.3390/children9111698The Impact of Highly Effective Treatment in Pediatric-Onset Multiple Sclerosis: A Case SeriesPaolo Immovilli0Paola De Mitri1Veronica Bazzurri2Stefano Vollaro3Nicola Morelli4Giacomo Biasucci5Fabiola Magnifico6Elena Marchesi7Maria Lara Lombardelli8Lorenza Gelati9Donata Guidetti10The Neurology Unit, Emergency Department, Guglielmo da Saliceto Hospital, Via Taverna 39, 29121 Piacenza, ItalyThe Neurology Unit, Emergency Department, Guglielmo da Saliceto Hospital, Via Taverna 39, 29121 Piacenza, ItalyThe Neurology Unit, Emergency Department, Guglielmo da Saliceto Hospital, Via Taverna 39, 29121 Piacenza, ItalyThe Neurology Unit, Emergency Department, Guglielmo da Saliceto Hospital, Via Taverna 39, 29121 Piacenza, ItalyThe Neurology Unit, Emergency Department, Guglielmo da Saliceto Hospital, Via Taverna 39, 29121 Piacenza, ItalyThe Pediatric Unit, Maternal and Child Department, Guglielmo da Saliceto Hospital, Via Taverna 39, 29121 Piacenza, ItalyThe Neurology Unit, Emergency Department, Guglielmo da Saliceto Hospital, Via Taverna 39, 29121 Piacenza, ItalyThe Neurology Unit, Emergency Department, Guglielmo da Saliceto Hospital, Via Taverna 39, 29121 Piacenza, ItalyThe Neurology Unit, Emergency Department, Guglielmo da Saliceto Hospital, Via Taverna 39, 29121 Piacenza, ItalyThe Neurology Unit, Emergency Department, Guglielmo da Saliceto Hospital, Via Taverna 39, 29121 Piacenza, ItalyThe Neurology Unit, Emergency Department, Guglielmo da Saliceto Hospital, Via Taverna 39, 29121 Piacenza, ItalyIntroduction: 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.https://www.mdpi.com/2227-9067/9/11/1698pediatric onset multiple sclerosisPOMShighly effective treatmentsdisease-modifying drugsDMD
spellingShingle Paolo Immovilli
Paola De Mitri
Veronica Bazzurri
Stefano Vollaro
Nicola Morelli
Giacomo Biasucci
Fabiola Magnifico
Elena Marchesi
Maria Lara Lombardelli
Lorenza Gelati
Donata Guidetti
The Impact of Highly Effective Treatment in Pediatric-Onset Multiple Sclerosis: A Case Series
Children
pediatric onset multiple sclerosis
POMS
highly effective treatments
disease-modifying drugs
DMD
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 pediatric onset multiple sclerosis
POMS
highly effective treatments
disease-modifying drugs
DMD
url https://www.mdpi.com/2227-9067/9/11/1698
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