Timing and comparative effectiveness of high-efficacy disease-modifying therapies in childhood-onset multiple sclerosis
SYNOPSIS AND RESULTS
About 2–10% of all multiple sclerosis (MS) patients experience the onset of their disease under the age of 18. In patients with childhood-onset MS (COMS), the clinical course is highly inflammatory with frequent exacerbations (relapses), especially during the initial years following symptoms onset. The active inflammatory nature of COMS is countered with disease-modifying therapies, which have been shown to reduce relapse rate and worsening of disability (Chitnis et al. Neurology 2016; Kornek et al. JAMA Neurol 2013). The current treatment paradigm for patients with COMS is ‘treatment escalation’. Almost half of the patients require escalation to more effective therapies (Yeh EA et al. Archives of Neurology 2011), which often comes too late when significant damage has already taken place. Although early commencement of high-efficacy therapy is associated with reduced long-term disability in adult-onset MS (He et al. Lancet Neurol 2020), appropriate timing of high-efficacy treatment in COMS has not yet been studied. We hypothesise that children who commence high-efficacy DMTs at the early active phase of the disease have lower risk of disability accumulation over the long-term.
Fondazione Italiana Sclerosi Multipla – FISM – Ente del Terzo Settore/ETS e, in forma abbreviata, FISM ETS. Iscrizione al RUNTS Rep. N° 89695 - Fondazione con Riconoscimento di Personalità Giuridica - C.F. 95051730109
Timing and comparative effectiveness of high-efficacy disease-modifying therapies in childhood-onset multiple sclerosis
About 2–10% of all multiple sclerosis (MS) patients experience the onset of their disease under the age of 18. In patients with childhood-onset MS (COMS), the clinical course is highly inflammatory with frequent exacerbations (relapses), especially during the initial years following symptoms onset. The active inflammatory nature of COMS is countered with disease-modifying therapies, which have been shown to reduce relapse rate and worsening of disability (Chitnis et al. Neurology 2016; Kornek et al. JAMA Neurol 2013). The current treatment paradigm for patients with COMS is ‘treatment escalation’. Almost half of the patients require escalation to more effective therapies (Yeh EA et al. Archives of Neurology 2011), which often comes too late when significant damage has already taken place. Although early commencement of high-efficacy therapy is associated with reduced long-term disability in adult-onset MS (He et al. Lancet Neurol 2020), appropriate timing of high-efficacy treatment in COMS has not yet been studied. We hypothesise that children who commence high-efficacy DMTs at the early active phase of the disease have lower risk of disability accumulation over the long-term.
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