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Comparative efficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: a systematic review with network meta-analyses

2022-12Conference object Open access
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dc.contributor.authorTonin, Fernanda
dc.contributor.authorGinete, Catarina
dc.contributor.authorFerreira, Joao
dc.contributor.authorDelgadinho, Mariana
dc.contributor.authorFernandez-Llimos, Fernando
dc.contributor.authorBrito, Miguel
dc.date.accessioned2023-03-02T11:13:36Z
dc.date.available2023-03-02T11:13:36Z
dc.date.issued2022-12
dc.description.abstractObjectives: Sickle cell disease (SCD), an inherited hemoglobinopathy that causes anemia, severe pain, and vaso-occlusive crisis (VOC), is currently recognized as a global public health concern, being the leading cause of pediatric stroke. Our aim was to synthesize the evidence on the efficacy and safety of interventions for managing SCD in this population. Methods: A systematic review with searches in PubMed, Scopus, and Web of Science was performed (April-2022). Randomized controlled trials comparing disease-modifying agents in SCD patients under 18 years old were included. For each outcome of interest, data were pooled by means of Bayesian network meta-analyses with the surface under the cumulative ranking curve analyses (SUCRA). Results were reported as odds ratio (OR) with 95% credibility intervals (CrI). Results: Seventeen trials (1982-2022) mostly from African countries (65%) and North America (53%), assessing the effect of different interventions’ regimens (hydroxyurea [n=6 trials], L-arginine [n=3], antiplatelets [n=2], immunotherapy/monoclonal antibodies [n=2], sulfates [n=2], docosahexaenoic acid [n=1], niprisan [n=1]) and placebo were included. No statistical differences among treatments were found for the main outcomes. SUCRA revealed that immunotherapy/monoclonal antibodies and hydroxyurea 20 mg/kg are potentially more effective against acute chest syndrome (83% and 76% probabilities, respectively), VOC (71% and 80%, respectively) and needing of transfusions (72% and 75%, respectively), while L-arginine (100-200 mg/kg) and placebo were more prone to these events. Although therapies were overall considered safe, antiplatelet and sulfates may lead to more discontinuations and severe adverse events (uncertainty evidence). Results were similar between age subgroups (<10 years vs. 10-19 years). Conclusions: The available evidence on the effect of drugs on managing SCD in children and adolescents is insufficient and weak. No clear definition for some outcomes exists. Hydroxyurea may remain the standard of care for this population, however, long-term well-designed, and well-reported trials comparing new immunotherapy/monoclonal antibodies should be performed.pt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationTonin FS, Ginete AC, Ferreira J, Delgadinho M, Fernandez-Llimos F, Brito M. Comparative efficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: a systematic review with network meta-analyses. In: ISPOR Europe 2022, Vienna (Austria), November 6-9, 2022. Value Health. 2022;25;12(Suppl):S46.pt_PT
dc.identifier.doi10.1016/j.jval.2022.09.224pt_PT
dc.identifier.urihttp://hdl.handle.net/10400.21/15744
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherElsevierpt_PT
dc.relation.publisherversionhttps://www.sciencedirect.com/science/article/pii/S1098301522024287pt_PT
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/pt_PT
dc.subjectSickle cell diseasept_PT
dc.subjectPharmacological interventionpt_PT
dc.subjectChildrenpt_PT
dc.subjectAdolescentpt_PT
dc.subjectSystematic reviewpt_PT
dc.subjectMeta-analysespt_PT
dc.titleComparative efficacy and safety of pharmacological interventions for managing sickle cell disease complications in children and adolescents: a systematic review with network meta-analysespt_PT
dc.typeconference object
dspace.entity.typePublication
oaire.citation.issue12pt_PT
oaire.citation.startPageS46pt_PT
oaire.citation.titleValue in Healthpt_PT
oaire.citation.volume25pt_PT
person.familyNameTonin
person.familyNameHonrado Ginete
person.familyNameAmaro Ferreira
person.familyNameNeves Delgadinho
person.familyNameBrito
person.givenNameFernanda
person.givenNameAna Catarina
person.givenNameJoao Carlos
person.givenNameMariana Isabel
person.givenNameMiguel
person.identifierCAJ-5082-2022
person.identifier.ciencia-idD01C-C700-9411
person.identifier.ciencia-id8715-F62E-1E0F
person.identifier.ciencia-id7E1E-BA67-7DC9
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.orcid0000-0002-6662-0806
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-id35224551000
rcaap.rightsopenAccesspt_PT
rcaap.typeconferenceObjectpt_PT
relation.isAuthorOfPublication61ded30e-ecec-4b3e-b953-2293e080ebdd
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