Safety of thalidomide and bevacizumab in patients with hereditary hemorrhagic telangiectasia

[Background] Hereditary hemorrhagic telangiectasia (HHT) is a multisystemic inherited vascular dysplasia that leads to nosebleeds and visceral arteriovenous malformations (AVMs). Anti-angiogenic drugs thalidomide and bevacizumab have been increasingly used off-label with variable results. The HHT wo...

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Autores: Buscarini, Elisabetta, Botella, Luisa María, Geisthoff, Urban, Kjeldsen, Anette D., Mager, Hans J., Pagella, Fabio, Suppressa, Patrizia, Zarrabeitia, Roberto, Dupuis-Girod, Sophie, Shovlin, Claire L.
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2019
País:España
Institución:Consejo Superior de Investigaciones Científicas (CSIC)
Repositorio:DIGITAL.CSIC. Repositorio Institucional del CSIC
OAI Identifier:oai:digital.csic.es:10261/175650
Acceso en línea:http://hdl.handle.net/10261/175650
Access Level:acceso abierto
Palabra clave:Hereditary hemorrhagic telangiectasia
Bevacizumab
Thalidomide
Adverse events
Bleeding
Arteriovenous malformation
Epistaxis
Cardiac failure
Nosebleeds
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spelling Safety of thalidomide and bevacizumab in patients with hereditary hemorrhagic telangiectasiaBuscarini, ElisabettaBotella, Luisa MaríaGeisthoff, UrbanKjeldsen, Anette D.Mager, Hans J.Pagella, FabioSuppressa, PatriziaZarrabeitia, RobertoDupuis-Girod, SophieShovlin, Claire L.Hereditary hemorrhagic telangiectasiaBevacizumabThalidomideAdverse eventsBleedingArteriovenous malformationEpistaxisCardiac failureNosebleeds[Background] Hereditary hemorrhagic telangiectasia (HHT) is a multisystemic inherited vascular dysplasia that leads to nosebleeds and visceral arteriovenous malformations (AVMs). Anti-angiogenic drugs thalidomide and bevacizumab have been increasingly used off-label with variable results. The HHT working group within the ERN for Rare Multisystemic Vascular Diseases (VASCERN), developed a questionnaire-based retrospective capture of adverse events (AEs) classified using the Common Terminology Criteria for Adverse Events.[Results] Sixty-nine HHT patients received bevacizumab, 37 (50.6%) for high output cardiac failure/hepatic AVMs, and 32 (49.4%) for bleeding; the 69 patients received bevacizumab for a mean of 11 months for a total of 63.8 person/years treatment. 67 received thalidomide, all for epistaxis and/or gastrointestinal bleeding; they received thalidomide for a mean of 13.4 months/patient for a total of 75 person/years treatment. AEs were reported in 58 patients, 33 with bevacizumab, 37 with thalidomide. 32 grade 1–3 AEs related to bevacizumab were reported with an average incidence rate of 50 per 100 person-years. 34 grade 1–3 AEs related to thalidomide were reported with an average incidence rate of 45.3 per 100 person-years. Bevacizumab AEs were more common in females (27 AEs in 46 women) than males (6 in 23, p < 0.001). Thalidomide AEs occurred at more similar rates in males (25 AEs in 41 men, 60.9%) and females (12 in 26 (46.2%), but were more common in ENG patients (17 in 17) than in ACVRL1 (14 in 34, p < 0.0001). For bevacizumab, the most common reports were of joint pains (7/69, 10%), headache (3/69, 4.4%) and proteinuria (2/69, 3%), and for thalidomide, peripheral neuropathy (12/67, 18%); drowsiness (8/67, 12%); and dizziness (6/67, 9%). Fatal adverse events were more common in males (p = 0.009), and in patients with ENG pathogenic variants (p = 0.012). One fatal AE was possibly related to bevacizumab (average incidence rate: 1.5 per 100 person-years); 3 fatal AEs were possibly related to thalidomide (average incidence rate: 4 per 100 person-years).[Conclusions] With potential increase in use of Bevacizumab and Thalidomide in HHT patients, data presented support appropriate weighing of the toxicities which can arise in HHT settings and the practice recommendations for their prevention and management.Peer reviewedPeer reviewedBioMed CentralConsejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72]2019201920192019info:eu-repo/semantics/articlehttp://purl.org/coar/resource_type/c_6501Publisher's versionPublisher's versioninfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/publishedVersionhttp://hdl.handle.net/10261/175650reponame:DIGITAL.CSIC. Repositorio Institucional del CSICinstname:Consejo Superior de Investigaciones Científicas (CSIC)Ingléshttps://doi.org/10.1186/s13023-018-0982-4SíSíinfo:eu-repo/semantics/openAccessoai:digital.csic.es:10261/1756502026-05-22T06:33:51Z
dc.title.none.fl_str_mv Safety of thalidomide and bevacizumab in patients with hereditary hemorrhagic telangiectasia
title Safety of thalidomide and bevacizumab in patients with hereditary hemorrhagic telangiectasia
spellingShingle Safety of thalidomide and bevacizumab in patients with hereditary hemorrhagic telangiectasia
Buscarini, Elisabetta
Hereditary hemorrhagic telangiectasia
Bevacizumab
Thalidomide
Adverse events
Bleeding
Arteriovenous malformation
Epistaxis
Cardiac failure
Nosebleeds
title_short Safety of thalidomide and bevacizumab in patients with hereditary hemorrhagic telangiectasia
title_full Safety of thalidomide and bevacizumab in patients with hereditary hemorrhagic telangiectasia
title_fullStr Safety of thalidomide and bevacizumab in patients with hereditary hemorrhagic telangiectasia
title_full_unstemmed Safety of thalidomide and bevacizumab in patients with hereditary hemorrhagic telangiectasia
title_sort Safety of thalidomide and bevacizumab in patients with hereditary hemorrhagic telangiectasia
dc.creator.none.fl_str_mv Buscarini, Elisabetta
Botella, Luisa María
Geisthoff, Urban
Kjeldsen, Anette D.
Mager, Hans J.
Pagella, Fabio
Suppressa, Patrizia
Zarrabeitia, Roberto
Dupuis-Girod, Sophie
Shovlin, Claire L.
author Buscarini, Elisabetta
author_facet Buscarini, Elisabetta
Botella, Luisa María
Geisthoff, Urban
Kjeldsen, Anette D.
Mager, Hans J.
Pagella, Fabio
Suppressa, Patrizia
Zarrabeitia, Roberto
Dupuis-Girod, Sophie
Shovlin, Claire L.
author_role author
author2 Botella, Luisa María
Geisthoff, Urban
Kjeldsen, Anette D.
Mager, Hans J.
Pagella, Fabio
Suppressa, Patrizia
Zarrabeitia, Roberto
Dupuis-Girod, Sophie
Shovlin, Claire L.
author2_role author
author
author
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 Hereditary hemorrhagic telangiectasia
Bevacizumab
Thalidomide
Adverse events
Bleeding
Arteriovenous malformation
Epistaxis
Cardiac failure
Nosebleeds
topic Hereditary hemorrhagic telangiectasia
Bevacizumab
Thalidomide
Adverse events
Bleeding
Arteriovenous malformation
Epistaxis
Cardiac failure
Nosebleeds
description [Background] Hereditary hemorrhagic telangiectasia (HHT) is a multisystemic inherited vascular dysplasia that leads to nosebleeds and visceral arteriovenous malformations (AVMs). Anti-angiogenic drugs thalidomide and bevacizumab have been increasingly used off-label with variable results. The HHT working group within the ERN for Rare Multisystemic Vascular Diseases (VASCERN), developed a questionnaire-based retrospective capture of adverse events (AEs) classified using the Common Terminology Criteria for Adverse Events.
publishDate 2019
dc.date.none.fl_str_mv 2019
2019
2019
2019
dc.type.none.fl_str_mv info:eu-repo/semantics/article
http://purl.org/coar/resource_type/c_6501
Publisher's version
Publisher's version
info:eu-repo/semantics/publishedVersion
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv http://hdl.handle.net/10261/175650
url http://hdl.handle.net/10261/175650
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv https://doi.org/10.1186/s13023-018-0982-4
Sí
Sí
dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.publisher.none.fl_str_mv BioMed Central
publisher.none.fl_str_mv BioMed Central
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
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score 15,812429

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