Repository logo
 
Publication

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

2023-02Journal article Open access
Simple item page
dc.contributor.authorTonin, Fernanda
dc.contributor.authorGinete, Catarina
dc.contributor.authorFernandez-Llimos, Fernando
dc.contributor.authorFerreira, Joana
dc.contributor.authorDelgadinho, Mariana
dc.contributor.authorBrito, Miguel
dc.date.accessioned2023-02-10T18:58:56Z
dc.date.available2023-02-10T18:58:56Z
dc.date.issued2023-02
dc.descriptionFCT_UIDB/05608/2020. FCT_UIDP/05608/2020.pt_PT
dc.description.abstractIntroduction: Sickle cell disease (SCD), an inherited haemoglobinopathy, has an important impact on morbidity and mortality, especially in paediatrics. Previous systematic reviews are limited to adult patients or focused only on a few therapies. We aim to synthesise the evidence on the efficacy and safety of pharmacological interventions for managing SCD in children and adolescents. Methods and analysis: This systematic review protocol is available at Open Science Framework (doi:10.17605/OSF.IO/CWAE9). We will follow international recommendations on the conduction and report of systematic reviews and meta-analyses. Searches will be conducted in PubMed, Scopus, and Web of Science (no language nor time restrictions) (first pilot searches performed in May 2022). We will include randomised controlled trials comparing the effects of disease-modifying agents in patients with SCD under 18 years old. Outcomes of interest will include: vaso-occlusive crisis, haemoglobin levels, chest syndrome, stroke, overall survival, and adverse events. We will provide a narrative synthesis of the findings, and whenever possible, results will be pooled by means of pairwise or Bayesian network meta-analyses with the surface under the cumulative ranking curve analyses. Different statistical methods and models will be tested. Dichotomous outcomes will be reported as OR, risk ratio, or HR, while continuous data will be reported as standard mean differences, both with 95% CI/credibility interval. The methodological quality of the trials will be evaluated using the Risk of Bias 2.0 tool, and the certainty of the evidence will be assessed with the Grading of Recommendations Assessment, Development, and Evaluation approach. Ethics and dissemination: This study refers to a systematic review, so no ethics approval is necessary. We intend to publish our findings in an international, peer-reviewed journal. Data will also be presented to peers at scientific events. Additionally, the results obtained in this study may contribute towards the update of therapeutic guidelines and the development of health policies for SCD.pt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationTonin FS, Ginete C, Fernandez-Llimos F, Ferreira J, Delgadinho M, Brito M. Efficacy and safety of pharmacological interventions for managing sickle cell disease in children and adolescents: protocol for a systematic review with network meta-analysis. BMJ Open. 2023;13(2):e064872.pt_PT
dc.identifier.doi10.1136/bmjopen-2022-064872pt_PT
dc.identifier.urihttp://hdl.handle.net/10400.21/15549
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherBMJpt_PT
dc.relation.publisherversionhttps://bmjopen.bmj.com/content/13/2/e064872pt_PT
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/pt_PT
dc.subjectPharmacologypt_PT
dc.subjectSickle cell diseasept_PT
dc.subjectChildrenpt_PT
dc.subjectAdolescentpt_PT
dc.subjectProtocolpt_PT
dc.subjectSystematic reviewpt_PT
dc.subjectMeta-analysispt_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 in children and adolescents: protocol for a systematic review with network meta-analysispt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.citation.issue2pt_PT
oaire.citation.issue2pt_PT
oaire.citation.startPagee064872pt_PT
oaire.citation.titleBMJ Openpt_PT
oaire.citation.volume13pt_PT
person.familyNameTonin
person.familyNameHonrado Ginete
person.familyNameFernandez-Llimos
person.familyNameCardoso Ferreira
person.familyNameNeves Delgadinho
person.familyNameBrito
person.givenNameFernanda
person.givenNameAna Catarina
person.givenNameFernando
person.givenNameJoana
person.givenNameMariana Isabel
person.givenNameMiguel
person.identifierB-8931-2008
person.identifierA-8277-2012
person.identifierCAJ-5082-2022
person.identifier.ciencia-idD01C-C700-9411
person.identifier.ciencia-id8715-F62E-1E0F
person.identifier.ciencia-idF716-B3AD-341A
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.orcid0000-0002-8529-9595
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.rightsopenAccesspt_PT
rcaap.typearticlept_PT
relation.isAuthorOfPublication61ded30e-ecec-4b3e-b953-2293e080ebdd
relation.isAuthorOfPublicationdfb2fbba-17ff-42fb-905a-fcfc8f326e1c
relation.isAuthorOfPublicationffddaa12-a4fa-4aeb-b84a-0c36015a7d9c
relation.isAuthorOfPublication1ed1a1fb-b34e-4926-900c-41fec6736491
relation.isAuthorOfPublicationca55aab6-9a58-4f79-ab79-20513414099f
relation.isAuthorOfPublication4252d8e0-800c-4d67-8b13-0b711d860669
relation.isAuthorOfPublication.latestForDiscoveryca55aab6-9a58-4f79-ab79-20513414099f

Files

Original bundle
Now showing 1 - 1 of 1
No Thumbnail Available
Name:
Efficacy and safety of pharmacological interventions for managing sickle cell disease in children and adolescents_protocol for a systematic review with network meta-analysis.pdf
Size:
423.75 KB
Format:
Adobe Portable Document Format
Download
License bundle
Now showing 1 - 1 of 1
No Thumbnail Available
Name:
license.txt
Size:
1.71 KB
Format:
Item-specific license agreed upon to submission
Description:
Download

Collections

ESTeSL - Artigos