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Treatment persistence in paediatric-onset multiple sclerosis: A Swedish nationwide registry study
Fredrik Sandesjö1, Kyla A McKay2, Katharina Fink3
1Neuropediatric Unit, Astrid Lindgren Children's Hospital, Karolinska University Hospital, Stockholm, Sweden; Department of Women's and Children's Health, Karolinska Institutet, Stockholm, Sweden.
Insights
Rituximab showed the highest treatment persistence in paediatric-onset multiple sclerosis (PoMS), suggesting B-cell depletion is a strong first-line therapy. This study compared various disease-modifying therapies (DMTs) for effectiveness and tolerability in young MS patients.
Area of Science:
- Neurology
- Immunology
- Pharmacology
Background:
- Comparative studies on highly effective therapies for paediatric-onset multiple sclerosis (PoMS) are limited.
- Understanding treatment persistence is crucial for assessing effectiveness and tolerability in PoMS.
Purpose of the Study:
- To compare treatment persistence across various disease-modifying therapies (DMTs) in individuals with PoMS.
- To evaluate the effectiveness and tolerability of different DMTs as a proxy for treatment persistence.
Main Methods:
- A nationwide cohort study utilizing the Swedish MS Registry.
- Inclusion of individuals with MS onset before age 18, from 2000 to 2024.
- Analysis of treatment persistence using Kaplan-Meier and Cox models, adjusted for covariates.
Main Results:
- Rituximab demonstrated the highest treatment persistence, never dropping below 50%.
- Compared to injectables, rituximab, natalizumab, fingolimod, and dimethyl fumarate showed significantly lower discontinuation rates.
- Median persistence varied, with rituximab showing the longest duration, while injectables had the shortest.
Conclusions:
- Rituximab exhibits superior treatment persistence in PoMS patients.
- B-cell depletion therapy, exemplified by rituximab, is supported as a potential first-line treatment option for PoMS.
Background:
Comparative studies in paediatric-onset multiple sclerosis (PoMS) that also include highly effective monoclonal antibody therapies are rare.
Objective:
To compare treatment persistence, as a proxy of effectiveness and tolerability, across disease-modifying therapies (DMTs).
Methods:
Nationwide cohort study using Swedish MS Registry data on individuals with MS onset before age 18 from 2000 to 2024. Treatment persistence was analysed using Kaplan-Meier and Cox models, adjusted for demographics and treatment epoch.
Results:
We included 383 individuals (69.2% female; median onset age 15.8 and 17.2 years at first DMT start), observed for a median 10.1 years (interquartile range (IQR): 5.5-15.5), yielding 934 treatment episodes: rituximab (272), injectables (263), natalizumab (248), fingolimod (86) and dimethyl fumarate (65). Median age at any treatment initiation, regardless of treatment sequence, was 18.9 years, with a median Expanded Disability Status Scale (EDSS) score of 1.5 (IQR: 0.0-2.0). Treatment persistence never dropped below 50% for rituximab, while median persistence was 35.6 months for natalizumab (95% confidence interval (CI): 28.8-44.9), 34.7 for dimethyl fumarate (95% CI: 21.7-56.9), 32.0 for fingolimod (95% CI: 21.1-48.1) and 17.6 for injectables (95% CI: 14.4-20.8). Compared to injectables, adjusted hazard ratios (HRs) for discontinuation were significantly lower for rituximab (0.09; 95% CI: 0.07-0.12), natalizumab (0.39; 95% CI: 0.31-0.48), fingolimod (0.41; 95% CI: 0.30-0.55) and dimethyl fumarate (0.49; 95% CI: 0.36-0.68).
Conclusion:
Rituximab displayed the highest treatment persistence, supporting B-cell depletion as a first-line option in PoMS.
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