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Treatments of paediatric multiple sclerosis: Efficacy and tolerance in a longitudinal follow-up study
Anne-Charlotte Saponaro1, Thomas Tully2, Elisabeth Maillart3
1Paediatric Neurology Unit, Children's Medicine Department, Children's Hospital, University Hospital of Nancy, France.
Insights
Newer disease-modifying treatments (DMTs) demonstrated superior efficacy over interferon beta-1a in reducing relapses and new T2 lesions in pediatric multiple sclerosis patients. Natalizumab showed the most significant effectiveness in this real-world study.
Area of Science:
- Pediatric Neurology
- Neuroimmunology
- Multiple Sclerosis Therapeutics
Background:
- Interferon beta-1a has been a standard treatment for multiple sclerosis (MS).
- There is a need to evaluate newer disease-modifying treatments (DMTs) in pediatric MS populations.
- Real-world data is crucial for understanding treatment effectiveness beyond clinical trials.
Purpose of the Study:
- To compare the efficacy and safety of newer/second-line DMTs against interferon beta-1a in pediatric relapsing MS.
- To assess the impact of different DMTs on annualized relapse rate (ARR) and MRI lesion development.
- To identify which newer DMTs offer the greatest benefit in this patient group.
Main Methods:
- Observational retrospective study of the French KIDBIOSEP cohort.
- Included patients under 18 years old diagnosed with relapsing MS between 2008-2019.
- Primary outcome: annualized relapse rate (ARR); Secondary outcomes: new T2 and gadolinium-enhanced MRI lesions.
Main Results:
- Newer DMTs significantly reduced ARR compared to interferon beta-1a.
- Fingolimod, dimethyl-fumarate, and natalizumab showed significant ARR reductions.
- Newer DMTs were more effective than interferon in reducing new T2 lesions; natalizumab also reduced gadolinium-enhanced lesions.
- Natalizumab demonstrated the most pronounced efficacy in reducing both relapses and new lesions.
Conclusions:
- Newer DMTs exhibit superior efficacy compared to interferon beta-1a for pediatric MS in real-world settings.
- These treatments effectively reduce relapse rates and new T2 lesions.
- Natalizumab appears to be the most effective DMT among those studied, with a favorable safety profile.
Aim:
To compare the efficacy and safety of newer and/or second-line disease-modifying treatments (DMTs) with interferon beta-1a.
Method:
This observational retrospective study included patients younger than 18 years old in the French KIDBIOSEP cohort who had a diagnosis of relapsing multiple sclerosis between 2008 and 2019 and received at least one DMT. Primary outcome was the annualized relapse rate (ARR). Secondary outcomes were the risk of new T2 or gadolinium-enhanced lesions on brain MRI.
Results:
Among 78 patients enrolled, 50 were exposed to interferon and 76 to newer DMTs. Mean ARR went from 1.65 during pre-treatment period to 0.45 with interferon (p < 0.001). Newer DMTs reduced ARR compared to interferon: fingolimod 0.27 (p = 0.013), teriflunomide 0.25 (p = 0.225), dimethyl-fumarate 0.14 (p = 0.045), natalizumab 0.03 (p = 0.007). Risk of new lesions on MRI was reduced with interferon compared to pre-treatment period; it decreased even more with newer DMTs for T2 lesions. Regarding risk of new gadolinium-enhanced lesions, the added value of new treatments compared to interferon was less obvious, except for natalizumab (p = 0.031).
Conclusion:
In this real-world setting, newer DMTs showed better efficacy than interferon beta-1a on ARR and risk of new T2 lesions, with a good safety profile. Natalizumab tend to emerge as the most effective treatment.
