Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complex: a Spanish expanded
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RESEARCH
Open Access
Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complex: a Spanish expanded access trial Nicolás Roberto Robles1, Ramón Peces2, Álvaro Gómez-Ferrer3, Felipe Villacampa4, Jose Luis Álvarez-Ossorio5, Pedro Pérez-Segura6, Juan Morote7, Bernardo Herrera-Imbroda8, Javier Nieto9, Joaquín Carballido10, Urbano Anido11, Marian Valero12, Cristina Meseguer12 and Roser Torra13*
Abstract Background: 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. Methods: 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. Results: 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. Conclusions: These findings support the benefit of everolimus for renal AML due to a manageable safety profile accompanied by reduced AML and kidney volumes. Trial registration: EudraCT number 2012-005397-63; date of registration 22 Nov 2012. Keywords: Angiomyolipoma, Everolimus, Safety, Tuberous sclerosis complex
* Correspondence: [email protected] 13 Inherited Renal Diseases, Department of Nephrology, Fundació Puigvert, REDINREN, IIB Sant Pau, Universitat Autónoma de Barcelona, Cartagena, 340-350, 08025 Barcelona, Spain Full list of author information is available at the end of the article © 2016 The Author(s). Open Access This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons l
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