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

2023-06Journal article Embargoed access
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dc.contributor.authorTonin, Fernanda
dc.contributor.authorGinete, Catarina
dc.contributor.authorFerreira, Joana
dc.contributor.authorDelgadinho, Mariana
dc.contributor.authorSantos, Brígida
dc.contributor.authorFernandez‐Llimos, Fernando
dc.contributor.authorBrito, Miguel
dc.date.accessioned2023-04-20T16:40:24Z
dc.date.embargo2025-04-20
dc.date.issued2023-06
dc.descriptionThis work was partially supported by FCT_UIDB/05608/2020 and FCT_UIDP/05608/2020.pt_PT
dc.description.abstractThis 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.pt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationTonin 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.pt_PT
dc.identifier.doi10.1002/pbc.30294pt_PT
dc.identifier.urihttp://hdl.handle.net/10400.21/15924
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherWileypt_PT
dc.relation.publisherversionhttps://onlinelibrary.wiley.com/doi/10.1002/pbc.30294pt_PT
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/pt_PT
dc.subjectAdolescentspt_PT
dc.subjectChildrenpt_PT
dc.subjectDisease-modifying agentspt_PT
dc.subjectSickle cell diseasept_PT
dc.subjectMeta-analysispt_PT
dc.subjectSystematic reviewpt_PT
dc.subjectFCT_UIDB/05608/2020pt_PT
dc.subjectFCT_UIDP/05608/2020pt_PT
dc.titleEfficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: systematic review with network meta‐analysispt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.citation.issue6pt_PT
oaire.citation.startPagee30294pt_PT
oaire.citation.titlePediatric Blood & Cancerpt_PT
oaire.citation.volume70pt_PT
person.familyNameTonin
person.familyNameHonrado Ginete
person.familyNameCardoso Ferreira
person.familyNameNeves Delgadinho
person.familyNameBrito
person.givenNameFernanda
person.givenNameAna Catarina
person.givenNameJoana
person.givenNameMariana Isabel
person.givenNameMiguel
person.identifierA-8277-2012
person.identifierCAJ-5082-2022
person.identifier.ciencia-idD01C-C700-9411
person.identifier.ciencia-id8715-F62E-1E0F
person.identifier.ciencia-idA910-13EB-21D1
person.identifier.ciencia-id231E-02E3-D9A9
person.identifier.ciencia-id231F-F341-7E93
person.identifier.orcid0000-0003-4262-8608
person.identifier.orcid0000-0002-2334-782X
person.identifier.orcid0000-0002-9438-4185
person.identifier.orcid0000-0003-0586-9154
person.identifier.orcid0000-0001-6394-658X
person.identifier.ridO-2050-2017
person.identifier.ridA-7970-2016
person.identifier.scopus-author-id56085115800
person.identifier.scopus-author-id7403252327
person.identifier.scopus-author-id35224551000
rcaap.rightsembargoedAccesspt_PT
rcaap.typearticlept_PT
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