RT Journal Article
T1 Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complex: a Spanish expanded access trial.
A1 Robles, Nicolás Roberto
A1 Peces, Ramón
A1 Gómez-Ferrer, Álvaro
A1 Villacampa, Felipe
A1 Álvarez-Ossorio, Jose Luis
A1 Pérez-Segura, Pedro
A1 Morote, Juan
A1 Herrera-Imbroda, Bernardo
A1 Nieto, Javier
A1 Carballido, Joaquín
A1 Anido, Urbano
A1 Valero, Marian
A1 Meseguer, Cristina
A1 Torra, Roser
K1 Angiomyolipoma
K1 Everolimus
K1 Safety
K1 Tuberous sclerosis complex
AB 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 2012-005397-63 ; date of registration 22 Nov 2012.
YR 2016
FD 2016-09-26
LK http://hdl.handle.net/10668/10476
UL http://hdl.handle.net/10668/10476
LA en
DS RISalud
RD Apr 5, 2025