Newer Treatment Approaches in Pediatric-Onset Multiple Sclerosis

Macaron, Gabrielle; Feng, Jenny; Moodley, Manikum; Rensel, Mary

doi:10.1007/s11940-019-0592-z

Cited by 26 publications

(20 citation statements)

References 113 publications

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“…In our cohort, no difference was observed in time from onset to therapy start between age groups; however, this effect might be masked by the small sample size in the early-onset group. Taken together, these observations highlight the generally more conservative treatment approach in childhood chronic neurological autoimmune disorders, as seen in pediatric-onset MS, 15 and suggest that early high-efficacy therapy should be considered in the young with NMOSD due to high risk of SRVL. Another potential explanation might be the vulnerability of the optic nerve to AQP-4 IgG-related inflammation, astrocyte and oligodendrocyte injury, demyelination, and axonal degeneration in children compared to adults.…”

Section: Discussionmentioning

confidence: 73%

Early age of onset predicts severity of visual impairment in patients with neuromyelitis optica spectrum disorder

et al. 2021

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Background: Severe residual visual loss (SRVL) is frequent in neuromyelitis optica spectrum disorders (NMOSD). Identifying higher-risk patients at onset is important to prevent disability accumulation. Objective: To determine predictors of SRVL in a large NMOSD cohort. Methods: Patient characteristics at last visual acuity (VA) evaluation were retrospectively collected. VA was scored 0: better than 20/40, 1: 20/40–20/99, 2: 20/100–20/200, and 3: worse than 20/200. SRVL was defined as a combined score (VA worst + best eye) ⩾ 4. Descriptive statistics were used to compare groups and logistic regression to evaluate predictors of VA. Results: 106 patients (mean age at disease onset (AO): 35.8 ± 16.5 years) were included. Patients with SRVL had earlier AO (mean: 26.7 vs 38.0 years) compared to non-SRVL group ( p = 0.005). Patients with AO < 21 years were more likely to have SRVL, be blind, present with binocular optic neuritis, have recurrent optic neuritis, and receive oral therapy first-line than those with AO ⩾ 21. After adjusting for race, sex, and disease duration, the odds of SRVL were 4.68 times higher in patients < 21 at disease onset (95% CI: 1.53–14.34, p = 0.007). Conclusion: Early AO predicts SRVL in NMOSD, independent of disease duration. High-efficacy therapies should be considered for first-line treatment in this group.

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Section: Discussionmentioning

confidence: 73%

Early age of onset predicts severity of visual impairment in patients with neuromyelitis optica spectrum disorder

et al. 2021

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“…Our clinical validity and population impact results (Table 4, Figure 2), modeled for natalizumab with an adverse event frequency of 1.3% (maximal risk reported by the manufacturer, see Methods), show that at least a quarter of PML cases could be prevented. Natalizumab is a highly effective treatment for many MS patients and some would benefit from its use as a first line therapy, such as those with aggressive or early onset forms of the disease (21,(83)(84)(85). Furthermore, an MRI surveillance study suggests that therapy duration is potentially not a very effective risk stratification factor (8).…”

Section: Discussionmentioning

confidence: 99%

Germline Genetic Risk Variants for Progressive Multifocal Leukoencephalopathy

Eis

Bruno²,

Richmond³

et al. 2020

Front. Neurol.

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“…Additionally, there is no standard definition of treatment failure across treatment centers, and with this, no guidelines for the transition of medications. The IPMSSG has proposed definitions for breakthrough disease, including an increase or no reduction in relapse rate, development of new T2 or contrast-enhancing lesions on MRI, or two or greater clinical or MRI relapses within 12 months [67]. Some children achieve a state of NEDA on first-line medications, but some require a transition of medications due to the breakthrough of disease, while other patients may change medications due to poor tolerance or non-compliance.…”

Section: Treatment Optionsmentioning

confidence: 99%

Current Advances in Pediatric Onset Multiple Sclerosis

et al. 2020

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Multiple sclerosis (MS) is an autoimmune inflammatory disease affecting the central nervous system leading to demyelination. MS in the pediatric population is rare, but has been shown to lead to significant disability over the duration of the disease. As we have learned more about pediatric MS, there has been a development of improved diagnostic criteria leading to earlier diagnosis, earlier initiation of disease-modifying therapies (DMT), and an increasing number of DMT used in the treatment of pediatric MS. Over time, treatment with DMT has trended towards the initiation of higher efficacy treatment at time of diagnosis to help prevent further disease progression and accrual of disability over time, and there is evidence in current literature that supports this change in treatment patterns. In this review, we discuss the current knowledge in diagnosis, treatment, and clinical outcomes in pediatric MS.

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Newer Treatment Approaches in Pediatric-Onset Multiple Sclerosis

Cited by 26 publications

References 113 publications

Early age of onset predicts severity of visual impairment in patients with neuromyelitis optica spectrum disorder

Early age of onset predicts severity of visual impairment in patients with neuromyelitis optica spectrum disorder

Germline Genetic Risk Variants for Progressive Multifocal Leukoencephalopathy

Current Advances in Pediatric Onset Multiple Sclerosis

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