Review of Pharmacological Strategies with Repurposed Drugs for Hereditary Hemorrhagic Telangiectasia Related Bleeding

© 2020 by the authors.

Detalles Bibliográficos
Autores: Albiñana, Virginia, Cuesta, Ángel M., Rojas-P, Isabel de, Gallardo-Vara, Eunate, Recio-Poveda, Lucía, Bernabéu, Carmelo, Botella, Luisa María
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2020
País:España
Institución:Consejo Superior de Investigaciones Científicas (CSIC)
Repositorio:DIGITAL.CSIC. Repositorio Institucional del CSIC
OAI Identifier:oai:digital.csic.es:10261/215680
Acceso en línea:http://hdl.handle.net/10261/215680
Access Level:acceso abierto
Palabra clave:HHT
ALK1
Endoglin
Raloxifene
Bazedoxifene
Tranexamic acid
Propranolol
FK506
Etamsylate
N-acetylcysteine
id ES_d41c774f0987cf0ccd873e8a024188a2
oai_identifier_str oai:digital.csic.es:10261/215680
network_acronym_str ES
network_name_str España
repository_id_str
spelling Review of Pharmacological Strategies with Repurposed Drugs for Hereditary Hemorrhagic Telangiectasia Related BleedingAlbiñana, VirginiaCuesta, Ángel M.Rojas-P, Isabel deGallardo-Vara, EunateRecio-Poveda, LucíaBernabéu, CarmeloBotella, Luisa MaríaHHTALK1EndoglinRaloxifeneBazedoxifeneTranexamic acidPropranololFK506EtamsylateN-acetylcysteine© 2020 by the authors.The diagnosis of hereditary hemorrhagic telangiectasia (HHT) is based on the Curaçao criteria: epistaxis, telangiectases, arteriovenous malformations in internal organs, and family history. Genetically speaking, more than 90% of HHT patients show mutations in ENG or ACVRL1/ALK1 genes, both belonging to the TGF-β/BMP9 signaling pathway. Despite clear knowledge of the symptoms and genes of the disease, we still lack a definite cure for HHT, having just palliative measures and pharmacological trials. Among the former, two strategies are: intervention at “ground zero” to minimize by iron and blood transfusions in order to counteract anemia. Among the later, along the last 15 years, three different strategies have been tested: (1) To favor coagulation with antifibrinolytic agents (tranexamic acid); (2) to increase transcription of ENG and ALK1 with specific estrogen-receptor modulators (bazedoxifene or raloxifene), antioxidants (N-acetylcysteine, resveratrol), or immunosuppressants (tacrolimus); and (3) to impair the abnormal angiogenic process with antibodies (bevacizumab) or blocking drugs like etamsylate, and propranolol. This manuscript reviews the main strategies and sums up the clinical trials developed with drugs alleviating HHT.This research was funded by Ministry of Economy and Competitivity, grant number SAF2017-83351-R to L.M.B.Peer reviewedMultidisciplinary Digital Publishing InstituteConsejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72]2020202020202020info:eu-repo/semantics/articlehttp://purl.org/coar/resource_type/c_6501Publisher's versioninfo:eu-repo/semantics/publishedVersionhttp://hdl.handle.net/10261/215680reponame:DIGITAL.CSIC. Repositorio Institucional del CSICinstname:Consejo Superior de Investigaciones Científicas (CSIC)Inglés#PLACEHOLDER_PARENT_METADATA_VALUE##PLACEHOLDER_PARENT_METADATA_VALUE#info:eu-repo/grantAgreement/AEI/Plan Estatal de Investigación Científica y Técnica y de Innovación 2017-2020/SAF2017-83351-RSAF2017-83351-R/AEI/10.13039/501100011033https://doi.org/10.3390/jcm9061766Síinfo:eu-repo/semantics/openAccessoai:digital.csic.es:10261/2156802026-05-22T06:33:51Z
dc.title.none.fl_str_mv Review of Pharmacological Strategies with Repurposed Drugs for Hereditary Hemorrhagic Telangiectasia Related Bleeding
title Review of Pharmacological Strategies with Repurposed Drugs for Hereditary Hemorrhagic Telangiectasia Related Bleeding
spellingShingle Review of Pharmacological Strategies with Repurposed Drugs for Hereditary Hemorrhagic Telangiectasia Related Bleeding
Albiñana, Virginia
HHT
ALK1
Endoglin
Raloxifene
Bazedoxifene
Tranexamic acid
Propranolol
FK506
Etamsylate
N-acetylcysteine
title_short Review of Pharmacological Strategies with Repurposed Drugs for Hereditary Hemorrhagic Telangiectasia Related Bleeding
title_full Review of Pharmacological Strategies with Repurposed Drugs for Hereditary Hemorrhagic Telangiectasia Related Bleeding
title_fullStr Review of Pharmacological Strategies with Repurposed Drugs for Hereditary Hemorrhagic Telangiectasia Related Bleeding
title_full_unstemmed Review of Pharmacological Strategies with Repurposed Drugs for Hereditary Hemorrhagic Telangiectasia Related Bleeding
title_sort Review of Pharmacological Strategies with Repurposed Drugs for Hereditary Hemorrhagic Telangiectasia Related Bleeding
dc.creator.none.fl_str_mv Albiñana, Virginia
Cuesta, Ángel M.
Rojas-P, Isabel de
Gallardo-Vara, Eunate
Recio-Poveda, Lucía
Bernabéu, Carmelo
Botella, Luisa María
author Albiñana, Virginia
author_facet Albiñana, Virginia
Cuesta, Ángel M.
Rojas-P, Isabel de
Gallardo-Vara, Eunate
Recio-Poveda, Lucía
Bernabéu, Carmelo
Botella, Luisa María
author_role author
author2 Cuesta, Ángel M.
Rojas-P, Isabel de
Gallardo-Vara, Eunate
Recio-Poveda, Lucía
Bernabéu, Carmelo
Botella, Luisa María
author2_role author
author
author
author
author
author
dc.contributor.none.fl_str_mv Consejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72]
dc.subject.none.fl_str_mv HHT
ALK1
Endoglin
Raloxifene
Bazedoxifene
Tranexamic acid
Propranolol
FK506
Etamsylate
N-acetylcysteine
topic HHT
ALK1
Endoglin
Raloxifene
Bazedoxifene
Tranexamic acid
Propranolol
FK506
Etamsylate
N-acetylcysteine
description © 2020 by the authors.
publishDate 2020
dc.date.none.fl_str_mv 2020
2020
2020
2020
dc.type.none.fl_str_mv info:eu-repo/semantics/article
http://purl.org/coar/resource_type/c_6501
Publisher's version
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv http://hdl.handle.net/10261/215680
url http://hdl.handle.net/10261/215680
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv #PLACEHOLDER_PARENT_METADATA_VALUE#
#PLACEHOLDER_PARENT_METADATA_VALUE#
info:eu-repo/grantAgreement/AEI/Plan Estatal de Investigación Científica y Técnica y de Innovación 2017-2020/SAF2017-83351-R
SAF2017-83351-R/AEI/10.13039/501100011033
https://doi.org/10.3390/jcm9061766
Sí
dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.publisher.none.fl_str_mv Multidisciplinary Digital Publishing Institute
publisher.none.fl_str_mv Multidisciplinary Digital Publishing Institute
dc.source.none.fl_str_mv reponame:DIGITAL.CSIC. Repositorio Institucional del CSIC
instname:Consejo Superior de Investigaciones Científicas (CSIC)
instname_str Consejo Superior de Investigaciones Científicas (CSIC)
reponame_str DIGITAL.CSIC. Repositorio Institucional del CSIC
collection DIGITAL.CSIC. Repositorio Institucional del CSIC
repository.name.fl_str_mv
repository.mail.fl_str_mv
_version_ 1869420518294159360
score 15,81155

Similares en LA Referencia