Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complex

Renal angiomyolipomas (AML) are usual manifestations of tuberous sclerosis complex (TSC) that may cause aneurism-related haemorrhages and renal impairment. Everolimus has emerged as an alternative to surgery/embolization. We provide further insight into everolimus safety and efficacy for TSC-related...

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Autores: Robles, Nicolás Roberto|||0000-0003-4984-6691, Peces, Ramón, Gómez-Ferrer Lozano, Álvaro, Villacampa, Felipe, Álvarez-Ossorio, Jose Luis, Morote Robles, Juan|||0000-0002-2168-323X, Herrera-Imbroda, Bernardo, Nieto, Javier, Carballido, Joaquín, Anido, Urbano|||0000-0003-0376-5642, Valero, Marian, Meseguer, Cristina, Torra Balcells, Roser|||0000-0001-8714-2332
Tipo de recurso: artículo
Fecha de publicación:2016
País:España
Institución:Universitat Autònoma de Barcelona
Repositorio:Dipòsit Digital de Documents de la UAB
Idioma:inglés
OAI Identifier:oai:ddd.uab.cat:254237
Acceso en línea:https://ddd.uab.cat/record/254237
https://dx.doi.org/urn:doi:10.1186/s13023-016-0517-9
Access Level:acceso abierto
Palabra clave:Angiomyolipoma
Everolimus
Safety
Tuberous sclerosis complex
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spelling Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complexa Spanish expanded access trialRobles, Nicolás Roberto|||0000-0003-4984-6691Peces, RamónGómez-Ferrer Lozano, ÁlvaroVillacampa, FelipeÁlvarez-Ossorio, Jose LuisMorote Robles, Juan|||0000-0002-2168-323XHerrera-Imbroda, BernardoNieto, JavierCarballido, JoaquínAnido, Urbano|||0000-0003-0376-5642Valero, MarianMeseguer, CristinaTorra Balcells, Roser|||0000-0001-8714-2332AngiomyolipomaEverolimusSafetyTuberous sclerosis complexRenal angiomyolipomas (AML) are usual manifestations of tuberous sclerosis complex (TSC) that may cause aneurism-related haemorrhages and renal impairment. Everolimus has emerged as an alternative to surgery/embolization. We provide further insight into everolimus safety and efficacy for TSC-related AML. This was a Spanish expanded access trial including patients aged ≥18 years with TSC-related AML. They received 10 mg everolimus once daily until AML progression, unacceptable toxicity, death/withdrawal, commercialisation for TSC-related AML, or 1 year after first patient enrolment. The primary outcome was dose-limiting safety according to grade 3/4 adverse events, serious adverse events, or adverse events leading to treatment modification. Secondary outcomes included overall safety and efficacy. Nineteen patients were enrolled and received everolimus for a median of 6.6 (5.3-10.9) months. Eleven (57.9 %) remained on 10 mg/day throughout the study and eight (42.1 %) required treatment modifications due to adverse events; none permanently discontinued treatment. Adverse events were overall grade 1/2 and most frequently included aphthous stomatitis/mucosal inflammation, hypercholesterolaemia/hypertriglyceridaemia, urinary tract infection, hypertension, dermatitis acneiform, and insomnia. Four (21.1 %) patients experienced grade 3 adverse events, none was grade 4, and only one (5.3 %) was serious (pneumonia). AML volume was reduced ≥30 % in 11 (57.9 %) patients and ≥50 % in 9 (47.4 %); none progressed. Right and left kidney sizes decreased in 16 and 14 patients, respectively. These findings support the benefit of everolimus for renal AML due to a manageable safety profile accompanied by reduced AML and kidney volumes. EudraCT number ; date of registration 22 Nov 2012.Universitat Autònoma de Barcelona 22016-01-0120162016-01-01Articlehttp://purl.org/coar/resource_type/c_6501VoRhttp://purl.org/coar/version/c_970fb48d4fbd8a85info:eu-repo/semantics/articleapplication/pdfhttps://ddd.uab.cat/record/254237https://dx.doi.org/urn:doi:10.1186/s13023-016-0517-9reponame:Dipòsit Digital de Documents de la UABinstname:Universitat Autònoma de BarcelonaInglésengopen accesshttp://purl.org/coar/access_right/c_abf2Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, la comunicació pública de l'obra i la creació d'obres derivades, fins i tot amb finalitats comercials, sempre i quan es reconegui l'autoria de l'obra original.https://creativecommons.org/licenses/by/4.0/info:eu-repo/semantics/openAccessoai:ddd.uab.cat:2542372026-06-06T12:50:31Z
dc.title.none.fl_str_mv Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complex
a Spanish expanded access trial
title Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complex
spellingShingle Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complex
Robles, Nicolás Roberto|||0000-0003-4984-6691
Angiomyolipoma
Everolimus
Safety
Tuberous sclerosis complex
title_short Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complex
title_full Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complex
title_fullStr Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complex
title_full_unstemmed Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complex
title_sort Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complex
dc.creator.none.fl_str_mv Robles, Nicolás Roberto|||0000-0003-4984-6691
Peces, Ramón
Gómez-Ferrer Lozano, Álvaro
Villacampa, Felipe
Álvarez-Ossorio, Jose Luis
Morote Robles, Juan|||0000-0002-2168-323X
Herrera-Imbroda, Bernardo
Nieto, Javier
Carballido, Joaquín
Anido, Urbano|||0000-0003-0376-5642
Valero, Marian
Meseguer, Cristina
Torra Balcells, Roser|||0000-0001-8714-2332
author Robles, Nicolás Roberto|||0000-0003-4984-6691
author_facet Robles, Nicolás Roberto|||0000-0003-4984-6691
Peces, Ramón
Gómez-Ferrer Lozano, Álvaro
Villacampa, Felipe
Álvarez-Ossorio, Jose Luis
Morote Robles, Juan|||0000-0002-2168-323X
Herrera-Imbroda, Bernardo
Nieto, Javier
Carballido, Joaquín
Anido, Urbano|||0000-0003-0376-5642
Valero, Marian
Meseguer, Cristina
Torra Balcells, Roser|||0000-0001-8714-2332
author_role author
author2 Peces, Ramón
Gómez-Ferrer Lozano, Álvaro
Villacampa, Felipe
Álvarez-Ossorio, Jose Luis
Morote Robles, Juan|||0000-0002-2168-323X
Herrera-Imbroda, Bernardo
Nieto, Javier
Carballido, Joaquín
Anido, Urbano|||0000-0003-0376-5642
Valero, Marian
Meseguer, Cristina
Torra Balcells, Roser|||0000-0001-8714-2332
author2_role author
author
author
author
author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Universitat Autònoma de Barcelona
dc.subject.none.fl_str_mv Angiomyolipoma
Everolimus
Safety
Tuberous sclerosis complex
topic Angiomyolipoma
Everolimus
Safety
Tuberous sclerosis complex
description Renal angiomyolipomas (AML) are usual manifestations of tuberous sclerosis complex (TSC) that may cause aneurism-related haemorrhages and renal impairment. Everolimus has emerged as an alternative to surgery/embolization. We provide further insight into everolimus safety and efficacy for TSC-related AML. This was a Spanish expanded access trial including patients aged ≥18 years with TSC-related AML. They received 10 mg everolimus once daily until AML progression, unacceptable toxicity, death/withdrawal, commercialisation for TSC-related AML, or 1 year after first patient enrolment. The primary outcome was dose-limiting safety according to grade 3/4 adverse events, serious adverse events, or adverse events leading to treatment modification. Secondary outcomes included overall safety and efficacy. Nineteen patients were enrolled and received everolimus for a median of 6.6 (5.3-10.9) months. Eleven (57.9 %) remained on 10 mg/day throughout the study and eight (42.1 %) required treatment modifications due to adverse events; none permanently discontinued treatment. Adverse events were overall grade 1/2 and most frequently included aphthous stomatitis/mucosal inflammation, hypercholesterolaemia/hypertriglyceridaemia, urinary tract infection, hypertension, dermatitis acneiform, and insomnia. Four (21.1 %) patients experienced grade 3 adverse events, none was grade 4, and only one (5.3 %) was serious (pneumonia). AML volume was reduced ≥30 % in 11 (57.9 %) patients and ≥50 % in 9 (47.4 %); none progressed. Right and left kidney sizes decreased in 16 and 14 patients, respectively. These findings support the benefit of everolimus for renal AML due to a manageable safety profile accompanied by reduced AML and kidney volumes. EudraCT number ; date of registration 22 Nov 2012.
publishDate 2016
dc.date.none.fl_str_mv 2
2016-01-01
2016
2016-01-01
dc.type.none.fl_str_mv Article
http://purl.org/coar/resource_type/c_6501
VoR
http://purl.org/coar/version/c_970fb48d4fbd8a85
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv https://ddd.uab.cat/record/254237
https://dx.doi.org/urn:doi:10.1186/s13023-016-0517-9
url https://ddd.uab.cat/record/254237
https://dx.doi.org/urn:doi:10.1186/s13023-016-0517-9
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
https://creativecommons.org/licenses/by/4.0/
dc.rights.openaire.fl_str_mv info:eu-repo/semantics/openAccess
rights_invalid_str_mv open access
http://purl.org/coar/access_right/c_abf2
https://creativecommons.org/licenses/by/4.0/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.source.none.fl_str_mv reponame:Dipòsit Digital de Documents de la UAB
instname:Universitat Autònoma de Barcelona
instname_str Universitat Autònoma de Barcelona
reponame_str Dipòsit Digital de Documents de la UAB
collection Dipòsit Digital de Documents de la UAB
repository.name.fl_str_mv
repository.mail.fl_str_mv
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