Preprint Article Version 1 Preserved in Portico This version is not peer-reviewed

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

Paolo Immovilli
* ORCID logo ,
Paola De Mitri
,
Veronica Bazzurri
,
Stefano Vollaro
,
Nicola Morelli
ORCID logo ,
Giacomo Biasucci
ORCID logo ,
Fabiola Magnifico
,
Elena Marchesi
,
Maria Lara Lombardelli
,
Lorenza Gelati
,
Donata Guidetti
Version 1 : Received: 1 September 2022 / Approved: 1 September 2022 / Online: 1 September 2022 (08:48:04 CEST)

A peer-reviewed article of this Preprint also exists.

Immovilli, P.; De Mitri, P.; Bazzurri, V.; Vollaro, S.; Morelli, N.; Biasucci, G.; Magnifico, F.; Marchesi, E.; Lombardelli, M.L.; Gelati, L.; Guidetti, D. The Impact of Highly Effective Treatment in Pediatric-Onset Multiple Sclerosis: A Case Series. Children 2022, 9, 1698. Immovilli, P.; De Mitri, P.; Bazzurri, V.; Vollaro, S.; Morelli, N.; Biasucci, G.; Magnifico, F.; Marchesi, E.; Lombardelli, M.L.; Gelati, L.; Guidetti, D. The Impact of Highly Effective Treatment in Pediatric-Onset Multiple Sclerosis: A Case Series. Children 2022, 9, 1698.

Abstract

Introduction: Pediatric-Onset Multiple Sclerosis (POMS) is characterized by high inflammatory disease activ-ity. 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: 2/5 consecutive POMS were administered HET as initial therapy after diagnosis. 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 di-agnosis and 13.4 years (range 9-16) at disease onset, 2/5 (40%) POMS were female. The pre-treatment aver-age 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, 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.

Keywords

pediatric onset multiple sclerosis; POMS; highly effective treatments; disease modifying drugs; DMD

Subject

Medicine and Pharmacology, Neuroscience and Neurology

Copyright: This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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