Cladribine treatment in pediatric-onset multiple sclerosis: real-world clinical outcomes and safety insights
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By
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Esther Ganelin-Cohen
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Yair Wexler
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Sraya Kraus
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Ayal Rozenberg
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May 1, 2026
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Clinical Scorecard: Clinical Outcomes and Safety of Cladribine in Children with Pediatric-Onset Multiple Sclerosis: Insights from Real-World Data
At a Glance
| Category | Detail |
|---|
| Condition | Pediatric-Onset Multiple Sclerosis (POMS) |
| Key Mechanisms | Cladribine acts as an immune reconstitution therapy targeting lymphocyte depletion while preserving innate immune capabilities. |
| Target Population | Children and adolescents diagnosed with POMS aged ≤18 years at disease onset. |
| Care Setting | Specialized pediatric neuroimmunology centers. |
Key Highlights
- Cladribine showed a positive safety profile with no serious adverse events reported.
- Discontinuation rates were significantly lower in the cladribine group (9.4%) compared to conventional DMTs (52.6%).
- NEDA rates were similar between cladribine and conventional DMT cohorts at Year 1.
- Cladribine's unique dosing schedule may enhance adherence among pediatric patients.
- The study emphasizes the need for real-world data in evaluating treatment options for POMS.
Guideline-Based Recommendations
Diagnosis
- Diagnosis should follow the 2017 McDonald criteria for multiple sclerosis.
Management
- Cladribine can be considered as an off-label treatment option for POMS.
Monitoring & Follow-up
- Annual MRI assessments are recommended for monitoring disease progression.
Risks
- Previous exposure to high-potency biological treatments should be avoided.
Patient & Prescribing Data
Pediatric patients with a minimum of two years of uninterrupted treatment.
Cladribine's dosing schedule consists of 8–10 treatment days annually across two years, promoting better adherence.
Clinical Best Practices
- Utilize weight-adjusted dosing of cladribine for pediatric patients.
- Ensure comprehensive clinical and laboratory records for all patients.
- Monitor for treatment adherence and potential side effects regularly.
References
