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Exploring treatment strategies for paroxysmal nocturnal hemoglobinuria: an overview of registered clinical trials

2024-05-16Journal article Restricted access
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dc.contributor.authorPeixoto, Vanda
dc.contributor.authorPrudêncio, Cristina
dc.contributor.authorVieira, Mónica
dc.date.accessioned2024-07-04T08:25:54Z
dc.date.available2024-07-04T08:25:54Z
dc.date.issued2024-05-16
dc.description.abstractParoxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disease in which blood cells lack anchored proteins that regulate the complement system. The erythrocytes are then destroyed because of uncontrolled complement activity, leading to intravascular hemolysis (IVH) and a high risk of thrombosis outcome. A huge alteration in the treatment of the disease was the development of terminal complement inhibitors, with the achievement of IVH blockade, reduction or abolishment of red blood cell (RBC) transfusions, and thromboembolic events prevention. However, patients treated with these inhibitors can still present extravascular hemolysis (EVH) caused by C3 activation and residual IVH or clinically relevant levels of breakthrough hemolysis (BTH). Proximal complement inhibitors turned out to be the key to the solution of this problem by targeting components of the proximal complement pathway, avoiding intra and extravascular hemolysis. FDA approved eculizumab, ravulizumab (terminal inhibitors), pegcetacoplan, iptacopan, and danicopan (proximal inhibitors) as a treatment for PNH so far. Various clinical trials are underway to find the most effective method to treat patients with PNH. This review aimed to summarize 71 registered clinical trials in the ClinicalTrials.gov database with the various treatment drugs, possible mechanisms, and novel findings related to PNH treatment.pt_PT
dc.description.versioninfo:eu-repo/semantics/publishedVersionpt_PT
dc.identifier.citationPeixoto, V. P., Prudêncio, C., & Vieira, M. (2024). Exploring treatment strategies for paroxysmal nocturnal hemoglobinuria: An overview of registered clinical trials. Current Medical Research and Opinion, 40(6), 935–945. https://doi.org/10.1080/03007995.2024.2354533pt_PT
dc.identifier.doi10.1080/03007995.2024.2354533pt_PT
dc.identifier.eissn1473-4877
dc.identifier.issn0300-7995
dc.identifier.urihttp://hdl.handle.net/10400.22/25733
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.publisherTaylor & Francispt_PT
dc.relation.publisherversionhttps://www.tandfonline.com/doi/full/10.1080/03007995.2024.2354533pt_PT
dc.subjectParoxysmal nocturnal hemoglobinuriapt_PT
dc.subjectClinical trialspt_PT
dc.subjectEculizumabpt_PT
dc.subjectRavulizumabpt_PT
dc.subjectPegcetacoplanpt_PT
dc.subjectIptacopanpt_PT
dc.subjectDanicopanpt_PT
dc.titleExploring treatment strategies for paroxysmal nocturnal hemoglobinuria: an overview of registered clinical trialspt_PT
dc.typejournal article
dspace.entity.typePublication
oaire.citation.endPage945pt_PT
oaire.citation.startPage935pt_PT
oaire.citation.titleCurrent Medical Research and Opinionpt_PT
oaire.citation.volume40 (6)pt_PT
person.familyNamePeixoto
person.familyNamePrudêncio
person.familyNameAlmeida Vieira
person.givenNameVanda
person.givenNameCristina
person.givenNameMónica Andreia
person.identifier1200571
person.identifier.ciencia-idC81E-F4EE-FADE
person.identifier.ciencia-idA01E-9178-9B48
person.identifier.orcid0000-0003-4883-3186
person.identifier.orcid0000-0002-9920-936X
person.identifier.orcid0000-0002-8687-4811
person.identifier.scopus-author-id6508057930
rcaap.rightsclosedAccesspt_PT
rcaap.typearticlept_PT
relation.isAuthorOfPublicationaf5cb379-3640-49bc-9d0a-43dd212821a1
relation.isAuthorOfPublication881a8ad5-ab13-4e49-89f4-08ca61cc81e3
relation.isAuthorOfPublication861e9c68-4ecc-4be1-a794-852343368e9a
relation.isAuthorOfPublication.latestForDiscovery861e9c68-4ecc-4be1-a794-852343368e9a

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