Journal
MULTIPLE SCLEROSIS AND RELATED DISORDERS
Volume 24, Issue -, Pages 20-27Publisher
ELSEVIER SCI LTD
DOI: 10.1016/j.msard.2018.05.010
Keywords
Progressive multiple sclerosis; Neurofilaments; Cerebro-spinal fluid; Cladribine; Disease activity
Categories
Funding
- Multiple Sclerosis of Great Britain & Northern Ireland [69/2017]
- MND association
- ECTRIMS Clinical Training fellowship 2016/17
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Background: Evidence suggests people with non-relapsing deteriorating (progressive) multiple sclerosis (pwPMS) may benefit from disease-modifying immune therapy (DMT). However, only one such treatment (ocrelizumab) has been licensed and is highly restricted to pwPMS suffering from the primary progressive phenotype. The difficulties assessing treatment outcome in pwPMS is one important reason for the lack of respective DMT. The concentration of neurofilaments in the cerebrospinal fluid (CSF) provides a biomarker of neuro-axonal damage, and both neurofilament light (NfL) and heavy chain (NfH) levels have been used as outcome indices and to guide treatment choices. Methods: We report on two pwPMS, who were treated with subcutaneous cladribine undergoing CSF NfL testing, alongside MRI and clinical follow-up, before and after treatment. Results: Cladribine treatment was well tolerated without any side effects. CSF NfL after treatment revealed significant reduction (by 73% and 80%, respectively) corroborating the MRI detectable drop in disease activity. Disability mildly progressed in one, and remained stable in the other pwPMS. Conclusions: pwPMS with detectable disease activity (MRI, elevated NfL) should be considered for DMT. NfL appears to be a sensitive index of treatment effect in pwPMS, and may be a useful outcome in clinical trials targeting this patient group. Over and above its licensed indication (relapsing MS), cladribine may be an effective treatment option for pwPMS.
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