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

Detalhes bibliográficos
Autor(a) principal: Santos, Brígida
Data de Publicação: 2024
Outros Autores: Ginete, Catarina, Gonçalves, Elisângela, Delgadinho, Mariana, Miranda, Armandina, Faustino, Paula, Arez, Ana Paula, Brito, Miguel
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
Texto Completo: http://hdl.handle.net/10451/62521
Resumo: © 2024 The Authors. Published by Elsevier Inc. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/)
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spelling Characterization of a cohort of Angolan children with sickle cell anemia treated with hydroxyureaAngolaFetal hemoglobinHydroxyureaMalariaSickle cell anemia© 2024 The Authors. Published by Elsevier Inc. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/)Background: 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.This 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.ElsevierRepositório da Universidade de LisboaSantos, BrígidaGinete, CatarinaGonçalves, ElisângelaDelgadinho, MarianaMiranda, ArmandinaFaustino, PaulaArez, Ana PaulaBrito, Miguel2024-02-08T14:37:21Z20242024-01-01T00:00:00Zinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/10451/62521engBlood Cells Mol Dis. 2024 Mar:105:1028221079-979610.1016/j.bcmd.2023.1028221096-0961info:eu-repo/semantics/openAccessreponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãoinstacron:RCAAP2024-02-12T01:19:53Zoai:repositorio.ul.pt:10451/62521Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireopendoar:71602024-03-20T02:37:53.365566Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãofalse
dc.title.none.fl_str_mv Characterization of a cohort of Angolan children with sickle cell anemia treated with hydroxyurea
title Characterization of a cohort of Angolan children with sickle cell anemia treated with hydroxyurea
spellingShingle Characterization of a cohort of Angolan children with sickle cell anemia treated with hydroxyurea
Santos, Brígida
Angola
Fetal hemoglobin
Hydroxyurea
Malaria
Sickle cell anemia
title_short Characterization of a cohort of Angolan children with sickle cell anemia treated with hydroxyurea
title_full Characterization of a cohort of Angolan children with sickle cell anemia treated with hydroxyurea
title_fullStr Characterization of a cohort of Angolan children with sickle cell anemia treated with hydroxyurea
title_full_unstemmed Characterization of a cohort of Angolan children with sickle cell anemia treated with hydroxyurea
title_sort Characterization of a cohort of Angolan children with sickle cell anemia treated with hydroxyurea
author Santos, Brígida
author_facet Santos, Brígida
Ginete, Catarina
Gonçalves, Elisângela
Delgadinho, Mariana
Miranda, Armandina
Faustino, Paula
Arez, Ana Paula
Brito, Miguel
author_role author
author2 Ginete, Catarina
Gonçalves, Elisângela
Delgadinho, Mariana
Miranda, Armandina
Faustino, Paula
Arez, Ana Paula
Brito, Miguel
author2_role author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Repositório da Universidade de Lisboa
dc.contributor.author.fl_str_mv Santos, Brígida
Ginete, Catarina
Gonçalves, Elisângela
Delgadinho, Mariana
Miranda, Armandina
Faustino, Paula
Arez, Ana Paula
Brito, Miguel
dc.subject.por.fl_str_mv Angola
Fetal hemoglobin
Hydroxyurea
Malaria
Sickle cell anemia
topic Angola
Fetal hemoglobin
Hydroxyurea
Malaria
Sickle cell anemia
description © 2024 The Authors. Published by Elsevier Inc. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/)
publishDate 2024
dc.date.none.fl_str_mv 2024-02-08T14:37:21Z
2024
2024-01-01T00:00:00Z
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://hdl.handle.net/10451/62521
url http://hdl.handle.net/10451/62521
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv Blood Cells Mol Dis. 2024 Mar:105:102822
1079-9796
10.1016/j.bcmd.2023.102822
1096-0961
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Elsevier
publisher.none.fl_str_mv Elsevier
dc.source.none.fl_str_mv reponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
instacron:RCAAP
instname_str Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
instacron_str RCAAP
institution RCAAP
reponame_str Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
collection Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
repository.name.fl_str_mv Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação
repository.mail.fl_str_mv
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