TyPed study

Detalhes bibliográficos
Autor(a) principal: Palavra, Filipe
Data de Publicação: 2021
Outros Autores: Figueiroa, Sónia, Correia, Ana Sofia, Tapadinhas, Fernando, Cerqueira, João, Guerreiro, Rui Pedro, de Sá, João, Sá, Maria José, Almeida, Sofia, Mota, Patrícia, Sousa, Lívia
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
Texto Completo: http://hdl.handle.net/10362/147434
Resumo: Background: A significant proportion of pediatric-onset multiple sclerosis (POMS) patients do not respond to first-line disease-modifying therapies. Clinical trials showed that natalizumab is effective and safe in adults, but there are limited clinical trial data for children. Natalizumab is currently prescribed off-label for POMS. We aimed to characterize the effectiveness, safety and tolerability of natalizumab in all POMS cases treated in Portugal (from 2007 to 2018). Methods: Data from clinical records were retrospectively collected for all POMS cases treated with natalizumab in Portugal. Results: Twenty-one patients were included, 14 (67%) of which were female. The median age at POMS diagnosis was 13 years old. The median duration of treatment with natalizumab was 2 years and 3 months. Median Expanded Disability Status Scale score decreased from 1.5 to 1.0 after 24 months. The Annualized Relapse Rate decreased from 1.31 events/patient/year before treatment with natalizumab to 0 after 12 months of treatment and to 0.04 after 24 months. No gadolinium-enhancing lesions or new or enlarged T2 hyperintense lesions were observed in 8/8 patients (100%) after 12 months, and 4/5 (80%) after 24 months. There was one possible serious adverse event, which did not require dose adjustment. Five patients discontinued treatment due to positive anti-JCV (JC virus) antibody JC serostatus. Conclusion: Natalizumab may be an effective and safe disease-modifying therapy for POMS. Our results are in line with data published for the adult population, as well as with similar observational studies in pediatric populations in other regions.
id RCAP_41ea4409be9aefb31815289cb71c8a8e
oai_identifier_str oai:run.unl.pt:10362/147434
network_acronym_str RCAP
network_name_str Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
repository_id_str 7160
spelling TyPed studyNatalizumab for the treatment of pediatric-onset multiple sclerosis in PortugalDMTMultiple sclerosisNatalizumabPediatricPOMSPortugalNeurologyClinical NeurologyBackground: A significant proportion of pediatric-onset multiple sclerosis (POMS) patients do not respond to first-line disease-modifying therapies. Clinical trials showed that natalizumab is effective and safe in adults, but there are limited clinical trial data for children. Natalizumab is currently prescribed off-label for POMS. We aimed to characterize the effectiveness, safety and tolerability of natalizumab in all POMS cases treated in Portugal (from 2007 to 2018). Methods: Data from clinical records were retrospectively collected for all POMS cases treated with natalizumab in Portugal. Results: Twenty-one patients were included, 14 (67%) of which were female. The median age at POMS diagnosis was 13 years old. The median duration of treatment with natalizumab was 2 years and 3 months. Median Expanded Disability Status Scale score decreased from 1.5 to 1.0 after 24 months. The Annualized Relapse Rate decreased from 1.31 events/patient/year before treatment with natalizumab to 0 after 12 months of treatment and to 0.04 after 24 months. No gadolinium-enhancing lesions or new or enlarged T2 hyperintense lesions were observed in 8/8 patients (100%) after 12 months, and 4/5 (80%) after 24 months. There was one possible serious adverse event, which did not require dose adjustment. Five patients discontinued treatment due to positive anti-JCV (JC virus) antibody JC serostatus. Conclusion: Natalizumab may be an effective and safe disease-modifying therapy for POMS. Our results are in line with data published for the adult population, as well as with similar observational studies in pediatric populations in other regions.Centro de Estudos de Doenças Crónicas (CEDOC)NOVA Medical School|Faculdade de Ciências Médicas (NMS|FCM)RUNPalavra, FilipeFigueiroa, SóniaCorreia, Ana SofiaTapadinhas, FernandoCerqueira, JoãoGuerreiro, Rui Pedrode Sá, JoãoSá, Maria JoséAlmeida, SofiaMota, PatríciaSousa, Lívia2023-01-12T22:16:21Z2021-062021-06-01T00:00:00Zinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/10362/147434eng2211-0348PURE: 28703707https://doi.org/10.1016/j.msard.2021.102865info:eu-repo/semantics/openAccessreponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãoinstacron:RCAAP2024-03-11T05:28:32Zoai:run.unl.pt:10362/147434Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireopendoar:71602024-03-20T03:52:55.584152Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãofalse
dc.title.none.fl_str_mv TyPed study
Natalizumab for the treatment of pediatric-onset multiple sclerosis in Portugal
title TyPed study
spellingShingle TyPed study
Palavra, Filipe
DMT
Multiple sclerosis
Natalizumab
Pediatric
POMS
Portugal
Neurology
Clinical Neurology
title_short TyPed study
title_full TyPed study
title_fullStr TyPed study
title_full_unstemmed TyPed study
title_sort TyPed study
author Palavra, Filipe
author_facet Palavra, Filipe
Figueiroa, Sónia
Correia, Ana Sofia
Tapadinhas, Fernando
Cerqueira, João
Guerreiro, Rui Pedro
de Sá, João
Sá, Maria José
Almeida, Sofia
Mota, Patrícia
Sousa, Lívia
author_role author
author2 Figueiroa, Sónia
Correia, Ana Sofia
Tapadinhas, Fernando
Cerqueira, João
Guerreiro, Rui Pedro
de Sá, João
Sá, Maria José
Almeida, Sofia
Mota, Patrícia
Sousa, Lívia
author2_role author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Centro de Estudos de Doenças Crónicas (CEDOC)
NOVA Medical School|Faculdade de Ciências Médicas (NMS|FCM)
RUN
dc.contributor.author.fl_str_mv Palavra, Filipe
Figueiroa, Sónia
Correia, Ana Sofia
Tapadinhas, Fernando
Cerqueira, João
Guerreiro, Rui Pedro
de Sá, João
Sá, Maria José
Almeida, Sofia
Mota, Patrícia
Sousa, Lívia
dc.subject.por.fl_str_mv DMT
Multiple sclerosis
Natalizumab
Pediatric
POMS
Portugal
Neurology
Clinical Neurology
topic DMT
Multiple sclerosis
Natalizumab
Pediatric
POMS
Portugal
Neurology
Clinical Neurology
description Background: A significant proportion of pediatric-onset multiple sclerosis (POMS) patients do not respond to first-line disease-modifying therapies. Clinical trials showed that natalizumab is effective and safe in adults, but there are limited clinical trial data for children. Natalizumab is currently prescribed off-label for POMS. We aimed to characterize the effectiveness, safety and tolerability of natalizumab in all POMS cases treated in Portugal (from 2007 to 2018). Methods: Data from clinical records were retrospectively collected for all POMS cases treated with natalizumab in Portugal. Results: Twenty-one patients were included, 14 (67%) of which were female. The median age at POMS diagnosis was 13 years old. The median duration of treatment with natalizumab was 2 years and 3 months. Median Expanded Disability Status Scale score decreased from 1.5 to 1.0 after 24 months. The Annualized Relapse Rate decreased from 1.31 events/patient/year before treatment with natalizumab to 0 after 12 months of treatment and to 0.04 after 24 months. No gadolinium-enhancing lesions or new or enlarged T2 hyperintense lesions were observed in 8/8 patients (100%) after 12 months, and 4/5 (80%) after 24 months. There was one possible serious adverse event, which did not require dose adjustment. Five patients discontinued treatment due to positive anti-JCV (JC virus) antibody JC serostatus. Conclusion: Natalizumab may be an effective and safe disease-modifying therapy for POMS. Our results are in line with data published for the adult population, as well as with similar observational studies in pediatric populations in other regions.
publishDate 2021
dc.date.none.fl_str_mv 2021-06
2021-06-01T00:00:00Z
2023-01-12T22:16:21Z
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://hdl.handle.net/10362/147434
url http://hdl.handle.net/10362/147434
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv 2211-0348
PURE: 28703707
https://doi.org/10.1016/j.msard.2021.102865
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.source.none.fl_str_mv reponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
instacron:RCAAP
instname_str Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
instacron_str RCAAP
institution RCAAP
reponame_str Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
collection Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
repository.name.fl_str_mv Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
repository.mail.fl_str_mv
_version_ 1799138120781791232