An update review of new therapies in sickle cell disease: the prospects for drug combinations

Detalhes bibliográficos
Autor(a) principal: Lugthart, Sanne
Data de Publicação: 2024
Outros Autores: Ginete, Catarina, Kuona, Patience, Brito, Miguel, Inusa, Baba Psalm
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)
Texto Completo: http://hdl.handle.net/10400.21/17124
Resumo: S Lugthart received funding to attend international conferences; Pfizer (2023), Vertex (2023), GBT (2022), and Novartis (2021). Consultancy work: Sanius Health, UK (2023). The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
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spelling An update review of new therapies in sickle cell disease: the prospects for drug combinationsSickle cell diseaseCombination therapyCrizanlizumabEtavopivatGene therapyHaematopoietic stem cell transplantationHydroxyureaL-GlutamineMitapivatVaso-occlusive crisesVoxelotorAnemiaFetal hemoglobinS Lugthart received funding to attend international conferences; Pfizer (2023), Vertex (2023), GBT (2022), and Novartis (2021). Consultancy work: Sanius Health, UK (2023). The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.Introduction: Sickle cell disease (SCD) is an inherited disorder characterized by polymerization of deoxygenated hemoglobin and microvascular obstruction. Generalized, it affects millions of people over 85% from low-and-middle-income countries. The cardinal feature is generalized pain referred to as vaso-occlusive crises (VOC), multi-organ damage, and premature death.). SCD is the most prevalent inherited reduced life-threatening disorder in the world and over 85% of the world's 400,000 annual births occur in low-and-middle-income countries. In the UK with about 250 annual births (1:200 live births, and over 14,00 living with the disorder. For decades span. Since 1998 Hydroxyurea remained the only disease-modifying therapy until the FDA approved L-glutamine (2017), Crizanlizumab and Voxelotor (2019), and gene therapies (Exa-cel and Lovo-cel, 2023). Areas covered: A literature review, we discuss established and new treatments. We provide an in-depth review of key clinical trials from 2013-2023. However, for pragmatic purposes we have approached this review in line with the different mechanisms of action, considering the possible options for the search performed in Pubmed Central using the search terms [sickle cell disease] or [sickle cell anemia] and the known treatments, i.e. Hydroxycarbamide/Hydroxyurea, L-Glutamine, Voxelotor, Crizanlizumab, Mitapivat, Etopivat, gene therapy, hematopoietic stem cell transplantation, and combination therapy. Clinical trials performed in the last 10 years (November 2013 - November 2023) were selected. Expert opinion: In our opinion section, we recommend the consideration of combination therapies for specific complications such as VOCs, pain, and renal impairment as well as personalized medicine based on disease phenotype and patient patience characteristics. Following the wake of the recent approval of gene therapy for SCD, the more curative option is now a reality, the challenge is addressing how to address issues such as access, affordability, and shared decision-making with families.Taylor & FrancisRCIPLLugthart, SanneGinete, CatarinaKuona, PatienceBrito, MiguelInusa, Baba Psalm2024-022024-02-01T00:00:00Z2026-02-19T00:00:00Zinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/10400.21/17124engLugthart S, Ginete C, Kuona P, Brito M, Inusa BP. An update review of new therapies in sickle cell disease: the prospects for drug combinations. Expert Opin Pharmacother. 2024 Feb 12. [Online ahead of print.]10.1080/14656566.2024.2317336info:eu-repo/semantics/embargoedAccessreponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãoinstacron:RCAAP2024-02-21T02:17:26Zoai:repositorio.ipl.pt:10400.21/17124Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireopendoar:71602024-03-20T02:39:23.211991Repositório 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 An update review of new therapies in sickle cell disease: the prospects for drug combinations
title An update review of new therapies in sickle cell disease: the prospects for drug combinations
spellingShingle An update review of new therapies in sickle cell disease: the prospects for drug combinations
Lugthart, Sanne
Sickle cell disease
Combination therapy
Crizanlizumab
Etavopivat
Gene therapy
Haematopoietic stem cell transplantation
Hydroxyurea
L-Glutamine
Mitapivat
Vaso-occlusive crises
Voxelotor
Anemia
Fetal hemoglobin
title_short An update review of new therapies in sickle cell disease: the prospects for drug combinations
title_full An update review of new therapies in sickle cell disease: the prospects for drug combinations
title_fullStr An update review of new therapies in sickle cell disease: the prospects for drug combinations
title_full_unstemmed An update review of new therapies in sickle cell disease: the prospects for drug combinations
title_sort An update review of new therapies in sickle cell disease: the prospects for drug combinations
author Lugthart, Sanne
author_facet Lugthart, Sanne
Ginete, Catarina
Kuona, Patience
Brito, Miguel
Inusa, Baba Psalm
author_role author
author2 Ginete, Catarina
Kuona, Patience
Brito, Miguel
Inusa, Baba Psalm
author2_role author
author
author
author
dc.contributor.none.fl_str_mv RCIPL
dc.contributor.author.fl_str_mv Lugthart, Sanne
Ginete, Catarina
Kuona, Patience
Brito, Miguel
Inusa, Baba Psalm
dc.subject.por.fl_str_mv Sickle cell disease
Combination therapy
Crizanlizumab
Etavopivat
Gene therapy
Haematopoietic stem cell transplantation
Hydroxyurea
L-Glutamine
Mitapivat
Vaso-occlusive crises
Voxelotor
Anemia
Fetal hemoglobin
topic Sickle cell disease
Combination therapy
Crizanlizumab
Etavopivat
Gene therapy
Haematopoietic stem cell transplantation
Hydroxyurea
L-Glutamine
Mitapivat
Vaso-occlusive crises
Voxelotor
Anemia
Fetal hemoglobin
description S Lugthart received funding to attend international conferences; Pfizer (2023), Vertex (2023), GBT (2022), and Novartis (2021). Consultancy work: Sanius Health, UK (2023). The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
publishDate 2024
dc.date.none.fl_str_mv 2024-02
2024-02-01T00:00:00Z
2026-02-19T00:00:00Z
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/10400.21/17124
url http://hdl.handle.net/10400.21/17124
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv Lugthart S, Ginete C, Kuona P, Brito M, Inusa BP. An update review of new therapies in sickle cell disease: the prospects for drug combinations. Expert Opin Pharmacother. 2024 Feb 12. [Online ahead of print.]
10.1080/14656566.2024.2317336
dc.rights.driver.fl_str_mv info:eu-repo/semantics/embargoedAccess
eu_rights_str_mv embargoedAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Taylor & Francis
publisher.none.fl_str_mv Taylor & Francis
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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