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

2024-03Journal article Open access
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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.accessioned2024-01-12T12:15:13Z
dc.date.available2024-01-12T12:15:13Z
dc.date.issued2024-03
dc.descriptionThis 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 (UID/04413/2020) and LA-REAL – LA/P/0117/2020.pt_PT
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 fixed 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 a 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.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationSantos B, Ginete C, Gonçalves E, Delgadinho M, Miranda A, Brito M, et al. Characterization of a cohort of Angolan children with sickle cell anemia treated with hydroxyurea. Blood Cells, Mol Dis. 2024;105:102822.pt_PT
dc.identifier.doi10.1016/j.bcmd.2023.102822pt_PT
dc.identifier.urihttp://hdl.handle.net/10400.21/16872
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherElsevierpt_PT
dc.relationFCT/MCTES/Aga Khan, project n◦ 330842553pt_PT
dc.relation.publisherversionhttps://www.sciencedirect.com/science/article/pii/S1079979623000992pt_PT
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/pt_PT
dc.subjectSickle cell anemiapt_PT
dc.subjectFetal hemoglobinpt_PT
dc.subjectHydroxyureapt_PT
dc.subjectMalariapt_PT
dc.subjectAngolapt_PT
dc.subjectFCT/MCTES/Aga Khan, project n◦ 330842553pt_PT
dc.subjectFCT_UIDB/05608/2020pt_PT
dc.subjectFCT_UIDP/05608/2020pt_PT
dc.titleCharacterization of a cohort of Angolan children with sickle cell anemia treated with hydroxyureapt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.citation.startPage102822pt_PT
oaire.citation.titleBlood Cells, Molecules, and Diseasespt_PT
oaire.citation.volume105pt_PT
person.familyNameHonrado Ginete
person.familyNameNeves Delgadinho
person.familyNameBrito
person.givenNameAna Catarina
person.givenNameMariana Isabel
person.givenNameMiguel
person.identifierCAJ-5082-2022
person.identifier.ciencia-id8715-F62E-1E0F
person.identifier.ciencia-id231E-02E3-D9A9
person.identifier.ciencia-id231F-F341-7E93
person.identifier.orcid0000-0002-2334-782X
person.identifier.orcid0000-0003-0586-9154
person.identifier.orcid0000-0001-6394-658X
person.identifier.ridA-7970-2016
person.identifier.scopus-author-id35224551000
rcaap.rightsopenAccesspt_PT
rcaap.typearticlept_PT
relation.isAuthorOfPublicationdfb2fbba-17ff-42fb-905a-fcfc8f326e1c
relation.isAuthorOfPublicationca55aab6-9a58-4f79-ab79-20513414099f
relation.isAuthorOfPublication4252d8e0-800c-4d67-8b13-0b711d860669
relation.isAuthorOfPublication.latestForDiscoverydfb2fbba-17ff-42fb-905a-fcfc8f326e1c

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