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Characterization of a cohort of Angolan children with sickle cell anemia treated with hydroxyurea

2024-01-04Artigo científico Acesso aberto
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dc.contributor.authorSantos, Brígida
dc.contributor.authorGinete, Catarina
dc.contributor.authorGonçalves, Elisângela
dc.contributor.authorDelgadinho, Mariana
dc.contributor.authorMiranda, Armandina
dc.contributor.authorFaustino, Paula
dc.contributor.authorArez, Ana Paula
dc.contributor.authorBrito, Miguel
dc.date.accessioned2024-01-16T12:17:32Z
dc.date.available2024-01-16T12:17:32Z
dc.date.issued2024-01-04
dc.description.abstractBackground: Sickle Cell Anemia (SCA) is a monogenic disease, although its severity and response to treatment are very heterogeneous. Objectives: This study aims to characterize a cohort of Angolan children with SCA and evaluate their response to hydroxyurea (HU) treatment and the potential side effects and toxicity. Methods: The study enrolled 215 patients between 3 and 12 years old before and after the administration of HU, at a fix dose of 20 mg/kg/day for 12 months. Results: A total of 157 patients started HU medication and 141 of them completed the 12-month treatment. After initiating HU treatment, the frequency of clinical events decreased (transfusions 53.4 %, hospitalizations 47.1 %). The response to HU medication varied among patients, with some experiencing an increase in fetal hemoglobin (HbF) of <5 %. The mean increase in HbF was 11.9 %, ranging from 1.8 % to 31 %. Responders to HU treatment were 57 %, inadequate responders 38.7 % and non-adherent 4.2 %. No clinical side effects related to HU were reported. Hematological toxicities were transient and reversible. Children naïve to HU and with lower HbF reported higher number of hospitalizations caused by malaria infection. During HU treatment, the frequency of malaria episodes did not appear to be affected by HbF levels. Conclusions: the present study provided a valuable contribution to the understanding of the clinical and laboratory profiles of Angolan children with SCA. These findings support the evidence that the implementation of prophylactic measures and treatment with HU is associated with increased survival in children with SCA.pt_PT
dc.description.sponsorshipThis research was funded by Fundação para a Ciência e Tecnologia Ministério da Ciência, Tecnologia e Ensino Superior (FCT-MCTES) and Aga Khan Foundation (FCT/MCTES/Aga Khan, project n◦ 330842553), by FCT/MCTES funding to H&TRC (UIDB/05608/2020, UIDP/05608/2020) and to GHTM IHMT NOVA (UIBD/04413/2020) and LA-REAL –LA/P/0117/2020.pt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationBlood Cells Mol Dis. 2024 Mar:105:102822. doi: 10.1016/j.bcmd.2023.102822. Epub 2024 Jan 4pt_PT
dc.identifier.doi10.1016/j.bcmd.2023.102822pt_PT
dc.identifier.pmid38215581
dc.identifier.urihttp://hdl.handle.net/10400.18/8905
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherElsevierpt_PT
dc.relationHealth and Technology Research Center
dc.relationHealth and Technology Research Center
dc.relationGlobal Health and Tropical Medicine
dc.relationTranslation and Innovation towards Global Health
dc.relation.publisherversionhttps://www.sciencedirect.com/science/article/pii/S1079979623000992?via%3Dihubpt_PT
dc.rights.urihttp://creativecommons.org/licenses/by/4.0/pt_PT
dc.subjectSickle Cell Anemiapt_PT
dc.subjectFetal Hemoglobinpt_PT
dc.subjectHydroxyuerapt_PT
dc.subjectMalariapt_PT
dc.subjectAngolapt_PT
dc.subjectHemoglobinopatias
dc.subjectDrepanocitose
dc.subjectAnemia
dc.subjectDoenças Genéticas
dc.subjectPatologias do Glóbulo Vermelhopt_PT
dc.subjectGenética Humana
dc.subjectPALOP
dc.titleCharacterization of a cohort of Angolan children with sickle cell anemia treated with hydroxyureapt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.awardTitleHealth and Technology Research Center
oaire.awardTitleHealth and Technology Research Center
oaire.awardTitleGlobal Health and Tropical Medicine
oaire.awardTitleTranslation and Innovation towards Global Health
oaire.awardURIinfo:eu-repo/grantAgreement/FCT/6817 - DCRRNI ID/UIDB%2F05608%2F2020/PT
oaire.awardURIinfo:eu-repo/grantAgreement/FCT/6817 - DCRRNI ID/UIDP%2F05608%2F2020/PT
oaire.awardURIinfo:eu-repo/grantAgreement/FCT/6817 - DCRRNI ID/UIDB%2F04413%2F2020/PT
oaire.awardURIinfo:eu-repo/grantAgreement/FCT/6817 - DCRRNI ID/LA%2FP%2F0117%2F2020/PT
oaire.citation.startPage102822pt_PT
oaire.citation.titleBlood Cells, Molecules and Diseasespt_PT
oaire.citation.volume105pt_PT
oaire.fundingStream6817 - DCRRNI ID
oaire.fundingStream6817 - DCRRNI ID
oaire.fundingStream6817 - DCRRNI ID
oaire.fundingStream6817 - DCRRNI ID
project.funder.identifierhttp://doi.org/10.13039/501100001871
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project.funder.identifierhttp://doi.org/10.13039/501100001871
project.funder.identifierhttp://doi.org/10.13039/501100001871
project.funder.nameFundação para a Ciência e a Tecnologia
project.funder.nameFundação para a Ciência e a Tecnologia
project.funder.nameFundação para a Ciência e a Tecnologia
project.funder.nameFundação para a Ciência e a Tecnologia
rcaap.embargofctAcesso de acordo com a política editorial da revista.pt_PT
rcaap.rightsopenAccesspt_PT
rcaap.typearticlept_PT
relation.isProjectOfPublication875a9c35-558d-4af9-abf0-254eadf9819d
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