Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complex : a Spanish expanded access trial
Robles, Nicolás Roberto (Complejo Hospitalario Infanta Cristina (Badajoz))
Peces, Ramón (Hospital Universitario La Paz (Madrid))
Gómez-Ferrer, Álvaro (Fundació Institut Valencià d'Oncologia)
Villacampa, Felipe (Hospital Universitario 12 de Octubre (Madrid))
Álvarez-Ossorio, Jose Luis (Hospital Universitario Puerta del Mar (Cadis, Andalusia))
Morote Robles, Juan (Hospital Universitari Vall d'Hebron. Institut de Recerca)
Herrera-Imbroda, Bernardo (Hospital Universitario Virgen de la Victoria (Màlaga, Andalusia))
Nieto, Javier (Hospital General Universitario de Ciudad Real)
Carballido, Joaquín (Hospital Universitario Puerta de Hierro Majadahonda (Madrid))
Anido, Urbano (Hospital Clínico Universitario (Santiago de Compostela, Galícia))
Valero, Marian (Novartis Farmacéutica (Barcelona, Catalunya))
Meseguer, Cristina (Novartis Farmacéutica (Barcelona, Catalunya))
Torra Balcells, Roser (Institut d'Investigació Biomèdica Sant Pau)
Universitat Autònoma de Barcelona

Date:	2016
Abstract:	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.
Rights:	Aquest 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.
Language:	Anglès
Document:	Article ; recerca ; Versió publicada
Subject:	Angiomyolipoma ; Everolimus ; Safety ; Tuberous sclerosis complex
Published in:	Orphanet Journal of Rare Diseases, Vol. 11 (september 2016) , ISSN 1750-1172

DOI: 10.1186/s13023-016-0517-9
PMID: 27669821

9 p, 499.7 KB

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Research literature > UAB research groups literature > Research Centres and Groups (research output) > Health sciences and biosciences > Institut de Recerca Sant Pau
Articles > Research articles
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