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Comprehensive Autopsy Program for Individuals with Multiple Sclerosis
Published on: July 19, 2019
Predictors of treatment outcome in patients with paediatric onset multiple sclerosis
Tine Iskov Kopp1, Morten Blinkenberg2, Thor Ameri Chalmer1
1The Danish Multiple Sclerosis Registry, Department of Neurology, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark/Danish Multiple Sclerosis Center, Department of Neurology, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
Insights
Disease-modifying therapies (DMTs) show comparable efficacy in pediatric multiple sclerosis (MS) patients to adults. Earlier DMT initiation in pediatric MS is linked to better outcomes, with disease duration and baseline disability predicting treatment response.
Area of Science:
- Pediatric Neurology
- Neuroimmunology
- Clinical Therapeutics
Background:
- Disease-modifying therapies (DMTs) are increasingly used in pediatric multiple sclerosis (MS).
- Most efficacy and safety trials for DMTs have focused on adult populations.
- Limited data exists on treatment outcomes specific to pediatric MS patients.
Purpose of the Study:
- To identify predictors of treatment outcomes in pediatric-onset MS patients receiving DMTs.
- To evaluate factors influencing relapse rates and disability progression in young MS patients.
Main Methods:
- Prospective cohort study utilizing the Danish Multiple Sclerosis Registry.
- Inclusion of 137 pediatric MS patients initiating DMT before age 18.
- Analysis of baseline predictors including disease duration and EDSS using regression models.
Main Results:
- Female sex and disease duration over two years predicted higher relapse rates.
- Disease duration and baseline EDSS predicted both disability worsening and improvement.
- No significant difference in outcomes was observed between early (<13 years) and later (≥13 years) MS onset.
Conclusions:
- DMT efficacy in pediatric MS is comparable to adult MS.
- Early initiation of DMT in pediatric MS is associated with a favorable prognosis.
- Predictors of treatment response in pediatric MS include disease duration and baseline disability.
Background:
Disease-modifying therapies (DMT) are increasingly used for children with multiple sclerosis (MS) even though most double-blinded randomized controlled trials evaluating efficacy, safety and dosing strategy of a specific drug have included adults.
Objective:
To investigate predictors of treatment outcomes in patients with paediatric onset MS treated with DMTs.
Methods:
Prospective cohort study from the nationwide Danish Multiple Sclerosis Registry including all patients with a MS diagnosis who initiated treatment with an approved DMT before the age of 18 (N = 137) and followed until their 25th birthday. Selected baseline predictors were tested in univariate and multivariate regression models.
Results:
Multivariate analyses showed that being female and having disease duration for 2 or more years prior to DMT initiation predicted a higher relapse rate. In addition, disease duration and baseline expanded disability status scale (EDSS) predicted both confirmed disability worsening and improvement. We found no difference in treatment outcome between children with MS onset before and after the age of 13 years.
Conclusions:
The efficacy of DMT in paediatric onset MS patients is comparable to that seen in adult onset MS patients. Earlier treatment start is associated with a beneficial prognosis in the paediatric cohort.
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