To assess the real-world safety and clinical outcomes of cladribine tablets compared to conventional disease-modifying therapies (DMTs) in children and adolescents with pediatric-onset multiple sclerosis (POMS).
Key Findings:
Mean age at onset was 15.1 years for cladribine and 13.8 years for controls.
NEDA rates were similar between groups at Year 1.
Relapse rates showed significant variation over time (p = 0.028).
Discontinuation rates were lower in the cladribine group (9.4% vs. 52.6%).
No serious adverse events reported.
Interpretation:
Cladribine demonstrated a favorable safety profile and adherence, with clinical outcomes comparable to conventional DMTs, suggesting its potential as an off-label treatment option for pediatric patients.
Limitations:
Retrospective design may introduce bias.
Small sample size limits generalizability.
Lack of long-term follow-up data.
Conclusion:
Cladribine is a viable off-label treatment for POMS, showing good safety and adherence with outcomes similar to conventional therapies.
