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The Impact of Highly Effective Treatment in Pediatric-Onset Multiple Sclerosis: A Case Series
Paolo Immovilli1, Paola De Mitri1, Veronica Bazzurri1
1The Neurology Unit, Emergency Department, Guglielmo da Saliceto Hospital, Via Taverna 39, 29121 Piacenza, Italy.
Insights
Highly effective disease-modifying treatment (HET) in pediatric-onset multiple sclerosis (POMS) showed positive outcomes. Early HET or escalation led to 80% of patients achieving No Evidence of Disease Activity-3 status (NEDA-3).
Area of Science:
- Pediatric Neurology
- Neuroimmunology
- Multiple Sclerosis Research
Background:
- Pediatric-onset multiple sclerosis (POMS) is a condition marked by significant inflammatory activity.
- Understanding treatment pathways and the effectiveness of advanced therapies is crucial for managing POMS.
Purpose of the Study:
- To analyze treatment sequencing in pediatric-onset multiple sclerosis (POMS).
- To evaluate the impact of highly effective disease-modifying treatment (HET) on disease activity in POMS.
Main Methods:
- Retrospective analysis of five consecutive pediatric-onset multiple sclerosis (POMS) patients.
- Patients received highly effective disease-modifying treatment (HET) either initially or after less effective therapies.
- Data collected included treatment history, relapse rates, and MRI findings over a 5-year follow-up period.
Main Results:
- Patients diagnosed at an average age of 13.8 years, with a pre-treatment annualized relapse rate of 1.6.
- 40% of patients were stable on HET as initial therapy; 60% required treatment escalation.
- 80% of patients achieved No Evidence of Disease Activity-3 status (NEDA-3) within 3 years of follow-up.
Conclusions:
- Prompt diagnosis and early initiation or escalation to highly effective disease-modifying treatment (HET) are associated with favorable outcomes in pediatric-onset multiple sclerosis (POMS).
- A significant majority (80%) of patients achieved NEDA-3, indicating successful disease control.
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.
