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. 2022 Nov 5;9(11):1698.
doi: 10.3390/children9111698.

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

Paolo Immovilli  1 , Paola De Mitri  1 , Veronica Bazzurri  1 , Stefano Vollaro  1 , Nicola Morelli  1 , Giacomo Biasucci  2 , Fabiola Magnifico  1 , Elena Marchesi  1 , Maria Lara Lombardelli  1 , Lorenza Gelati  1 , Donata Guidetti  1
Affiliations

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

Paolo Immovilli et al. Children (Basel). .

Abstract

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.

Keywords: DMD; POMS; disease-modifying drugs; highly effective treatments; pediatric onset multiple sclerosis.

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Conflict of interest statement

Paolo Immovilli received honoraria for consulting or public speaking from Biogen, Roche, Merck, TEVA, Sanofi, Novartis. Stefano Vollaro received honoraria for advising or speaking from Novartis, Roche and Merck. Veronica Bazzurri received travel expense reimbursements from Sanofi Genzyme, Biogen and Roche. Paola De Mitri, Elena Marchesi, Fabiola Magnifico, Nicola Morelli, Giacomo Biasucci, Maria Lara Lombardelli, Lorenza Gelati and Donata Guidetti have no conflict of interest to declare.

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