Pediatric Multiple Sclerosis in Portugal: A Multicentre Study

Correia, Ana Sofia; Augusto, Luís; Meireles, Joana; Pinto, Joana; Sousa, Ana Paula

doi:10.20344/amp.6346

Cited by 7 publications

(15 citation statements)

References 16 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…Furthermore, studies show that in follow-up MRI scans, POMS patients tend to have more new T2 WI and Gd+ lesions. [8][9][10] In our study, we did not find a tendency or statistically significant differences between groups for these variables, which can be due to the fact that late-onset POMS tends to be more similar to the MRI phenotype of AOMS compared to early-onset POMS (age under 11 years old). We also found statistically significant differences between groups in the number of T2 WI diffuse lesions with poorly defined borders, both at diagnosis and one year after DMT, with POMS patients having fewer of these lesions compared to AOMS patients.…”

Section: Discussioncontrasting

confidence: 69%

Inflammatory Activity and Treatment Response in Pediatric Compared to Adult Multiple Sclerosis: A Pilot, Retrospective and Observational Study of the First Year After Diagnosis

et al. 2021

Self Cite

View full text Add to dashboard Cite

Introduction: Pediatric-onset multiple sclerosis may contrast with adult-onset multiple sclerosis, in terms of disease activity. We aimed to determine differentiating features between pediatric-onset multiple sclerosis and adult-onset multiple sclerosis, at diagnosis and after one year under disease modifying therapies, and analyse the attainment of the status of “No Evidence of Disease Activity” between groups.Material and Methods: We analyzed demographical, laboratory, clinical and imaging features of patients with relapsing-remitting multiple sclerosis diagnosed at our center, according to the McDonald’s 2010 criteria, with ≥ 1 year under disease modifying therapies and with available magnetic resonance imaging scans at diagnosis and one year after disease modifying therapies initiation. Patients were paired according to gender and disease modifying therapies in use. “No Evidence of Disease Activity” status was assessed, and differences were studied.Results: Fifteen pediatric-onset multiple sclerosis (aged ≥ 8 and < 18 years) and 15 adult-onset multiple sclerosis (≥ 18 and < 55 years) patients were recruited. We found a statistically significant difference in the number of T2 weighted image diffuse lesions/with poorly defined borders (p = 0.015). The mean expanded disability status scale score after one year under disease modifying therapies was lower in the pediatric-onset multiple sclerosis group (1.6 ± 0.8) compared to the adult-onset multiple sclerosis group (2.3 ± 0.8; p = 0.032). Nevertheless, no differences were found regarding the percentage of cases achieving “No Evidence of Disease Activity” in either group.Discussion: Although there is an empirical impression about the difference in inflammatory activity between pediatric-onset multiple sclerosis and adult-onset multiple sclerosis, it was not possible to corroborate it in our study. Nevertheless, this was an exploratory and retrospective analysis of a small sample of patients, identifying variables in which such differences appear to be most important.Conclusion: Extensive studies of children, adolescents and adults with multiple sclerosis will be needed to categorize the clinical and radiological differences that allow the identification of drug response biomarkers in the early stages of the disease.

show abstract

Section: Discussioncontrasting

confidence: 69%

Inflammatory Activity and Treatment Response in Pediatric Compared to Adult Multiple Sclerosis: A Pilot, Retrospective and Observational Study of the First Year After Diagnosis

et al. 2021

Self Cite

View full text Add to dashboard Cite

show abstract

“…The mean age at diagnosis and female predominance were in accordance with previous studies, particularly reflecting the reality of the pediatric population in Portugal. 5 Our patients had a higher prevalence of a positive family history for MS (20.0% vs 6%-13.5% in international studies and 2% in another Portuguese study), 5 which may reflect regional differences yet to be explored (our center is the reference hospital for the whole central region of Portugal, with a population of 2 216 569 million, according to the latest data published by the National Statistical Institute). 13 Portugal is a sunny country and the analysis of a very young population was an opportunity to explore the possible relationship between the month of birth, as an indirect variable related to neonatal vitamin D levels, and the later diagnosis of POMS.…”

Section: Discussionmentioning

confidence: 79%

“…Oligoclonal bands were also an important part of the diagnostic workup and were present in the majority of cases (85.7%), although in lower numbers than was reported in adult-onset MS (98%). 5 It is important to mention that in the last few years we have been working on raising awareness of POMS in the pediatric departments of smaller hospitals in our region in order to achieve the early diagnosis of the disease and recognition of the clinical manifestations that may correspond to first relapses. In fact, we have observed a much earlier referral of these children recently, which naturally translates into an increasingly early diagnosis.…”

Section: Discussionmentioning

confidence: 99%

“…Pediatric-onset multiple sclerosis (POMS) has been increasingly recognized as having some distinctive features from adult-onset multiple sclerosis (MS), with a more inflammatory component and higher relapse rates, but slower progression. [1][2][3][4][5] Diagnosis in childhood is currently based on the International Pediatric Multiple Sclerosis Study Group (IPMSSG) criteria, 6 which incorporate the McDonald criteria, 7 but can be challenging due to several clinical entities having similar findings. The 2017 revised Mc-Donald criteria 7 have proven to be equally applicable to POMS and MS in adults, providing a simplified means to reach the diagnosis at onset and over time.…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Pediatric Multiple Sclerosis Before the PARADIGMS Study: Nine Years of Experience in a Portuguese Tertiary Center

Fernandes¹

2021

Sinapse

View full text Add to dashboard Cite

Background: Multiple sclerosis (MS) is an inflammatory and demyelinating disease of the central nervous system that may have a pediatric onset. This study aims to provide a characterization of a pediatric-onset MS (POMS) population followed at our center in the 9-year period prior to the PARADIGMS study, which resulted in the first formal approval of a drug to treat POMS.Material and Methods: We performed a retrospective, observational and unicentric study. We included data from the records of patients with an MS diagnosis confirmed before the age of 18, according to the McDonald 2010 diagnostic criteria, from 1st January 2010 onwards.Results: In a group of 32 patients, 30 (73.3% female) fulfilled the inclusion criteria, with a mean age at diagnosis of 15.5±2.2 years and median value of Expanded Disability Status Scale (EDSS) score at diagnosis of 1.5. All the cases had relapsing-remitting MS and in 43.3% optic nerve involvement was the first clinical manifestation. At diagnosis, magnetic resonance imaging showed gadolinium-enhancing lesions in 50% of the cases and the study of cerebrospinal fluid revealed oligoclonal bands in 85.7%. Interferon beta-1a was the most frequent first treatment option. In a mean follow-up of 4.1±2.5 years, the treatment was changed in 67.9% of cases. On the last visit, the median EDSS score was 1.5. Conclusion:Our results are in line with contemporaneous research. As well as contributing an important descriptive analysis of this population's characteristics, it also provides a window of opportunity for further prospective analyses regarding potential treatment paradigm changes. ResumoIntrodução: A esclerose múltipla (EM) é uma doença inflamatória e desmielinizante do sistema nervoso central que pode ter início em idade pediátrica. Este estudo tem como objetivo a caracterização de uma população de doentes com EM de início em idade pediátrica (POMS) acompanhada no nosso centro no período de 9 anos, antes da publicação do estudo PARADIGMS, de que resultou a aprovação formal de um primeiro fármaco para tratamento da POMS.Material e Métodos: Estudo retrospectivo, observacional e unicêntrico, incluindo dados das crianças e adolescentes com diagnóstico de EM confirmado antes dos 18 anos de idade, a partir da data de 1 de Janeiro de 2010, de acordo com os critérios de McDonald 2010.

show abstract

“…These guidelines are based on the efficacy and safety data from observational studies or small retrospective case reports and series, and none of the currently recommended DMT therapies have completed randomized controlled trials in a pediatric population. IFN-β and glatiramer acetate have been the most frequently used agents for the treatment of POMS [20][21][22]. The results of observational studies suggest that treatment with IFN-β and glatiramer acetate in POMS is associated with a reduction in the relapse rate and stabilization of Expanded Disability Status Scale (EDSS) [23,24].…”

Section: Introductionmentioning

confidence: 99%

Cost-effectiveness of fingolimod versus interferon-β1a for the treatment of pediatric-onset multiple sclerosis in Canada

Nakhai-Pour

Vudumula

Khurana

et al. 2020

Journal of Medical Economics

View full text Add to dashboard Cite

Aims: To evaluate the cost effectiveness of fingolimod versus interferon (IFN)-β1a at a dose of 30 μg per week for the treatment of relapsing pediatric-onset multiple sclerosis (POMS) in Canada. Material and methods: A discrete time Markov model was developed to compare fingolimod with IFN β-1a over a time horizon of two years representing patients followed up to mean age of 18 years from a Canadian health care system perspective. Twenty-one health states based on the Expanded Disability Status Scale (EDSS) were considered: EDSS 0-9 for relapsing multiple sclerosis (MS), EDSS 0-9 for secondary progressive MS, and "Death." Relative treatment efficacy for fingolimod vs. IFN-β1a was estimated from the PARADIGMS study. Costs and resource use were obtained from published literature and Canadian sources. Utilities were estimated by mapping the Pediatric Quality of Life inventory data onto the Child Health Utility Index-9 Dimension using a published mapping algorithm. Future costs and benefits were discounted at 1.5% per annum. Results: Compared with IFN β-1a, fingolimod led to an increase in qualityadjusted life years (QALYs) (0.125) with incremental costs (Canadian dollars [CAD] 2,977) and to an incremental cost-effectiveness ratio (ICER) of CAD 23,886/QALY over a time horizon of two years representing patients followed up to mean age of 18 years. The monetary benefits of fingolimod treatment versus IFN β-1a at a willingness-to-pay (WTP) threshold of CAD 50,000 per QALY gained were higher than the costs. One-way sensitivity analysis and probabilistic sensitivity analysis (PSA) both confirmed the robustness of the results. Limitations: The main limitations of this analysis primarily stem from the limited data availability in POMS. Conclusions: Fingolimod is cost effective compared with IFN β-1a for the treatment of POMS over a time horizon of two years representing patients followed up to a mean age of 18 years in Canada.

show abstract

Pediatric Multiple Sclerosis in Portugal: A Multicentre Study

Cited by 7 publications

References 16 publications

Inflammatory Activity and Treatment Response in Pediatric Compared to Adult Multiple Sclerosis: A Pilot, Retrospective and Observational Study of the First Year After Diagnosis

Inflammatory Activity and Treatment Response in Pediatric Compared to Adult Multiple Sclerosis: A Pilot, Retrospective and Observational Study of the First Year After Diagnosis

Pediatric Multiple Sclerosis Before the PARADIGMS Study: Nine Years of Experience in a Portuguese Tertiary Center

Cost-effectiveness of fingolimod versus interferon-β1a for the treatment of pediatric-onset multiple sclerosis in Canada

Contact Info

Product

Resources

About