Novel therapies to address unmet needs in ITP
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- dc.contributor.author Mingot-Castellano, M. Eva
- dc.contributor.author Bastida, José María
- dc.contributor.author Caballero-Navarro, Gonzalo
- dc.contributor.author Entrena Ureña, Laura
- dc.contributor.author González-López, Tomás José
- dc.contributor.author González Porras, José Ramón
- dc.contributor.author Butta, Nora
- dc.contributor.author Canaro, Mariana
- dc.contributor.author Jiménez-Bárcenas, Reyes
- dc.contributor.author Gómez Del Castillo Solano, María Del Carmen
- dc.contributor.author Sánchez González, Blanca
- dc.contributor.author Pascual-Izquierdo, Cristina
- dc.contributor.author GEPTI
- dc.date.accessioned 2023-02-10T07:30:57Z
- dc.date.available 2023-02-10T07:30:57Z
- dc.date.issued 2022
- dc.description.abstract Primary immune thrombocytopenia (ITP) is an autoimmune disorder that causes low platelet counts and subsequent bleeding risk. Although current corticosteroid-based ITP therapies are able to improve platelet counts, up to 70% of subjects with an ITP diagnosis do not achieve a sustained clinical response in the absence of treatment, thus requiring a second-line therapy option as well as additional care to prevent bleeding. Less than 40% of patients treated with thrombopoietin analogs, 60% of those treated with splenectomy, and 20% or fewer of those treated with rituximab or fostamatinib reach sustained remission in the absence of treatment. Therefore, optimizing therapeutic options for ITP management is mandatory. The pathophysiology of ITP is complex and involves several mechanisms that are apparently unrelated. These include the clearance of autoantibody-coated platelets by splenic macrophages or by the complement system, hepatic desialylated platelet destruction, and the inhibition of platelet production from megakaryocytes. The number of pathways involved may challenge treatment, but, at the same time, offer the possibility of unveiling a variety of new targets as the knowledge of the involved mechanisms progresses. The aim of this work, after revising the limitations of the current treatments, is to perform a thorough review of the mechanisms of action, pharmacokinetics/pharmacodynamics, efficacy, safety, and development stage of the novel ITP therapies under investigation. Hopefully, several of the options included herein may allow us to personalize ITP management according to the needs of each patient in the near future.
- dc.format.mimetype application/pdf
- dc.identifier.citation Mingot-Castellano ME, Bastida JM, Caballero-Navarro G, Entrena Ureña L, González-López TJ, González-Porras JR, Butta N, Canaro M, Jiménez-Bárcenas R, Gómez Del Castillo Solano MDC, Sánchez-González B, Pascual-Izquierdo C, on behalf of the GEPTI. Novel therapies to address unmet needs in ITP. Pharmaceuticals (Basel). 2022 Jun 23;15(7):779. DOI: 10.3390/ph15070779
- dc.identifier.doi http://dx.doi.org/10.3390/ph15070779
- dc.identifier.issn 1424-8247
- dc.identifier.uri http://hdl.handle.net/10230/55713
- dc.language.iso eng
- dc.publisher MDPI
- dc.relation.ispartof Pharmaceuticals (Basel). 2022 Jun 23;15(7):779
- dc.rights © 2022 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
- dc.rights.accessRights info:eu-repo/semantics/openAccess
- dc.rights.uri https://creativecommons.org/licenses/by/4.0/
- dc.subject.keyword Autoantibodies
- dc.subject.keyword Immune thrombocytopenia
- dc.subject.keyword Platelets
- dc.subject.keyword Targeted therapies
- dc.subject.keyword Thrombopoietin
- dc.title Novel therapies to address unmet needs in ITP
- dc.type info:eu-repo/semantics/article
- dc.type.version info:eu-repo/semantics/publishedVersion