Efficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: systematic review with network meta‐analysis

Detalhes bibliográficos
Autor(a) principal: Tonin, Fernanda
Data de Publicação: 2023
Outros Autores: Ginete, Catarina, Ferreira, Joana, Delgadinho, Mariana, Santos, Brígida, Fernandez‐Llimos, Fernando, Brito, Miguel
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
Texto Completo: http://hdl.handle.net/10400.21/15924
Resumo: This work was partially supported by FCT_UIDB/05608/2020 and FCT_UIDP/05608/2020.
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spelling Efficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: systematic review with network meta‐analysisAdolescentsChildrenDisease-modifying agentsSickle cell diseaseMeta-analysisSystematic reviewFCT_UIDB/05608/2020FCT_UIDP/05608/2020This work was partially supported by FCT_UIDB/05608/2020 and FCT_UIDP/05608/2020.This study aimed to synthesize the evidence on the effects of disease-modifying agents for managing sickle cell disease (SCD) in children and adolescents by means of a systematic review with network meta-analyses, the surface under the cumulative ranking curve (SUCRA) and stochastic multicriteria acceptability analyses (SMAA) (CRD42022328471). Eighteen randomized controlled trials (hydroxyurea [n = 7], l-arginine [n = 3], antiplatelets [n = 2], immunotherapy/monoclonal antibodies [n = 2], sulfates [n = 2], docosahexaenoic acid [n = 1], niprisan [n = 1]) were analyzed. SUCRA and SMAA demonstrated that hydroxyurea at higher doses (30 mg/kg/day) or at fixed doses (20 mg/kg/day) and immunotherapy/monoclonal antibodies are more effective for preventing vaso-occlusive crisis (i.e., lower probabilities of incidence of this event; 14, 25, and 30%, respectively), acute chest syndrome (probabilities ranging from 8 to 30%), and needing of transfusions (11-31%), while l-arginine (100-200 mg/kg) and placebo were more prone to these events. Therapies were overall considered safe; however, antiplatelets and sulfates may lead to more severe adverse events. Although the evidence was graded as insufficient and weak, hydroxyurea remains the standard of care for this population, especially if a maximum tolerated dose schedule is considered.WileyRCIPLTonin, FernandaGinete, CatarinaFerreira, JoanaDelgadinho, MarianaSantos, BrígidaFernandez‐Llimos, FernandoBrito, Miguel2023-062023-06-01T00:00:00Z2025-04-20T00:00:00Zinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/10400.21/15924engTonin FS, Ginete C, Ferreira J, Delgadinho M, Santos B, Fernandez-Llimos F, Brito M. Efficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: systematic review with network meta-analysis. Pediatr Blood Cancer. 2023;70(6):e30294.10.1002/pbc.30294info:eu-repo/semantics/embargoedAccessreponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãoinstacron:RCAAP2023-08-03T10:14:01Zoai:repositorio.ipl.pt:10400.21/15924Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireopendoar:71602024-03-19T20:23:30.683722Repositório 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 Efficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: systematic review with network meta‐analysis
title Efficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: systematic review with network meta‐analysis
spellingShingle Efficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: systematic review with network meta‐analysis
Tonin, Fernanda
Adolescents
Children
Disease-modifying agents
Sickle cell disease
Meta-analysis
Systematic review
FCT_UIDB/05608/2020
FCT_UIDP/05608/2020
title_short Efficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: systematic review with network meta‐analysis
title_full Efficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: systematic review with network meta‐analysis
title_fullStr Efficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: systematic review with network meta‐analysis
title_full_unstemmed Efficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: systematic review with network meta‐analysis
title_sort Efficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: systematic review with network meta‐analysis
author Tonin, Fernanda
author_facet Tonin, Fernanda
Ginete, Catarina
Ferreira, Joana
Delgadinho, Mariana
Santos, Brígida
Fernandez‐Llimos, Fernando
Brito, Miguel
author_role author
author2 Ginete, Catarina
Ferreira, Joana
Delgadinho, Mariana
Santos, Brígida
Fernandez‐Llimos, Fernando
Brito, Miguel
author2_role author
author
author
author
author
author
dc.contributor.none.fl_str_mv RCIPL
dc.contributor.author.fl_str_mv Tonin, Fernanda
Ginete, Catarina
Ferreira, Joana
Delgadinho, Mariana
Santos, Brígida
Fernandez‐Llimos, Fernando
Brito, Miguel
dc.subject.por.fl_str_mv Adolescents
Children
Disease-modifying agents
Sickle cell disease
Meta-analysis
Systematic review
FCT_UIDB/05608/2020
FCT_UIDP/05608/2020
topic Adolescents
Children
Disease-modifying agents
Sickle cell disease
Meta-analysis
Systematic review
FCT_UIDB/05608/2020
FCT_UIDP/05608/2020
description This work was partially supported by FCT_UIDB/05608/2020 and FCT_UIDP/05608/2020.
publishDate 2023
dc.date.none.fl_str_mv 2023-06
2023-06-01T00:00:00Z
2025-04-20T00:00:00Z
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/10400.21/15924
url http://hdl.handle.net/10400.21/15924
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv Tonin FS, Ginete C, Ferreira J, Delgadinho M, Santos B, Fernandez-Llimos F, Brito M. Efficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: systematic review with network meta-analysis. Pediatr Blood Cancer. 2023;70(6):e30294.
10.1002/pbc.30294
dc.rights.driver.fl_str_mv info:eu-repo/semantics/embargoedAccess
eu_rights_str_mv embargoedAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Wiley
publisher.none.fl_str_mv Wiley
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
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