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Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complex: A Spanish expanded access trial

Author
Robles, Nicolás Roberto; Peces, Ramón; Gómez-Ferrer, Álvaro; Villacampa, Felipe; Álvarez-Ossorio, Jose Luis; Pérez-Segura, Pedro; Morote, Juan; Herrera-Imbroda, Bernardo; Nieto, Javier; Carballido Rodríguez, Joaquínuntranslated; Anido, Urbano; Valero, Marian; Meseguer, Cristina; Torra, Roser
Entity
UAM. Departamento de Medicina; Instituto de Investigación Sanitaria Hospital Universitario de La Paz (IdiPAZ)
Publisher
BioMed Central
Date
2016-09-26
Citation
10.1186/s13023-016-0517-9
Orphanet Journal of Rare Diseases 11.1 (2016): 128
 
 
 
ISSN
1750-1172
DOI
10.1186/s13023-016-0517-9
Funded by
This work was funded by Novartis Farmacéutica S.A., which was involved in study design, data analysis and interpretation, and writing of the manuscript
Editor's Version
http://dx.doi.org/10.1186/s13023-016-0517-9
Subjects
Angiomyolipoma; Everolimus; Safety; Tuberous sclerosis complex; Medicina
URI
http://hdl.handle.net/10486/678072
Rights
© 2016 The Author(s)

Licencia Creative Commons
Esta obra está bajo una Licencia Creative Commons Atribución 4.0 Internacional.

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.
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Google™ Scholar:Robles, Nicolás Roberto - Peces, Ramón - Gómez-Ferrer, Álvaro - Villacampa, Felipe - Álvarez-Ossorio, Jose Luis - Pérez-Segura, Pedro - Morote, Juan - Herrera-Imbroda, Bernardo - Nieto, Javier - Carballido Rodríguez, Joaquín - Anido, Urbano - Valero, Marian - Meseguer, Cristina - Torra, Roser

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