Pediatric multiple sclerosis (PedMS) is a rare disease with a more severe prognosis compared to adult-onset MS. It remains a challenging condition to treat because of the highly inflammatory nature of the disease, the prominent cognitive issues, and the limited knowledge about the efficacy and safety of current available disease-modifying therapies. Over the past decade, there has been a dramatic increase in the number of drugs licensed for adult-onset MS and several of them, although not tested in PedMS, are currently being used off-label in this population. To date, interferon-beta and glatiramer acetate are the most commonly used first-line treatments in children, although the efficacy and safety of these drugs have only been studied in observational cohorts and in unblinded randomized controlled trials. For children with breakthrough disease, escalation to higher efficacious second-line therapies, such as natalizumab, fingolimod, dimethyl fumarate, mitoxantrone, cyclophosphamide, rituximab, and daclizumab may be considered. Large observational studies showed natalizumab is an effective treatment with safety and efficacy comparable to those in adult populations. The safety, efficacy, and tolerability of the other second-line treatments in PedMS have been reported only in small size retrospective case series. Large phase III studies are underway which will provide important information regarding the efficacy and safety of fingolimod, teriflunomide, and dimethyl fumarate in PedMS. Symptomatic treatments for fatigue, spasticity, depression, bladder and bowel dysfunction, and neuropathic pain should be considered in PedMS, especially when these symptoms impact the quality of life. Further work is needed to ensure that new trials best address treatment outcomes tailored to PedMS

Use of Disease-Modifying Therapies in Pediatric MS

Marta Simone
;
2016

Abstract

Pediatric multiple sclerosis (PedMS) is a rare disease with a more severe prognosis compared to adult-onset MS. It remains a challenging condition to treat because of the highly inflammatory nature of the disease, the prominent cognitive issues, and the limited knowledge about the efficacy and safety of current available disease-modifying therapies. Over the past decade, there has been a dramatic increase in the number of drugs licensed for adult-onset MS and several of them, although not tested in PedMS, are currently being used off-label in this population. To date, interferon-beta and glatiramer acetate are the most commonly used first-line treatments in children, although the efficacy and safety of these drugs have only been studied in observational cohorts and in unblinded randomized controlled trials. For children with breakthrough disease, escalation to higher efficacious second-line therapies, such as natalizumab, fingolimod, dimethyl fumarate, mitoxantrone, cyclophosphamide, rituximab, and daclizumab may be considered. Large observational studies showed natalizumab is an effective treatment with safety and efficacy comparable to those in adult populations. The safety, efficacy, and tolerability of the other second-line treatments in PedMS have been reported only in small size retrospective case series. Large phase III studies are underway which will provide important information regarding the efficacy and safety of fingolimod, teriflunomide, and dimethyl fumarate in PedMS. Symptomatic treatments for fatigue, spasticity, depression, bladder and bowel dysfunction, and neuropathic pain should be considered in PedMS, especially when these symptoms impact the quality of life. Further work is needed to ensure that new trials best address treatment outcomes tailored to PedMS
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Utilizza questo identificativo per citare o creare un link a questo documento: http://hdl.handle.net/11586/250484
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