Repository logo
 
Publication

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

2024-02Journal article Embargoed access
Simple item page
dc.contributor.authorLugthart, Sanne
dc.contributor.authorGinete, Catarina
dc.contributor.authorKuona, Patience
dc.contributor.authorBrito, Miguel
dc.contributor.authorInusa, Baba Psalm
dc.date.accessioned2024-02-19T11:06:54Z
dc.date.embargo2026-02-19
dc.date.issued2024-02
dc.descriptionS 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.pt_PT
dc.description.abstractIntroduction: 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.pt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationLugthart 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;25(2):157-70.pt_PT
dc.identifier.doi10.1080/14656566.2024.2317336pt_PT
dc.identifier.urihttp://hdl.handle.net/10400.21/17124
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherTaylor & Francispt_PT
dc.relation.publisherversionhttps://www.tandfonline.com/doi/full/10.1080/14656566.2024.2317336pt_PT
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/pt_PT
dc.subjectSickle cell diseasept_PT
dc.subjectCombination therapypt_PT
dc.subjectCrizanlizumabpt_PT
dc.subjectEtavopivatpt_PT
dc.subjectGene therapypt_PT
dc.subjectHaematopoietic stem cell transplantationpt_PT
dc.subjectHydroxyureapt_PT
dc.subjectL-Glutaminept_PT
dc.subjectMitapivatpt_PT
dc.subjectVaso-occlusive crisespt_PT
dc.subjectVoxelotorpt_PT
dc.subjectAnemiapt_PT
dc.subjectFetal hemoglobinpt_PT
dc.titleAn update review of new therapies in sickle cell disease: the prospects for drug combinationspt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.citation.endPage170pt_PT
oaire.citation.issue2pt_PT
oaire.citation.startPage157pt_PT
oaire.citation.titleExpert Opinion on Pharmacotherapypt_PT
oaire.citation.volume25pt_PT
rcaap.rightsembargoedAccesspt_PT
rcaap.typearticlept_PT

Files

Original bundle
Now showing 1 - 1 of 1
No Thumbnail Available
Name:
An update review of new therapies in sickle cell disease the prospects for drug.pdf
Size:
1 MB
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