Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complex: a Spanish expanded access trial
Por:
Robles, NR, Peces, R, Gomez-Ferrer, A, Villacampa, F, Alvarez-Ossorio, JL, Perez-Segura, P, Morote, J, Herrera-Imbroda, B, Nieto, J, Carballido, J, Anido, U, Valero, M, Meseguer, C, Torra, R
Publicada:
26 sep 2016
Resumen:
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.
Filiaciones:
Robles, NR:
Hosp Univ Infanta Cristina, REDINREN, Dept Nephrol, Ave Elvas S-N, Badajoz 06006, Spain
Peces, R:
Hosp Univ La Paz, REDINREN, Dept Nephrol, Paseo Castellana 261, Madrid 28046, Spain
Gomez-Ferrer, A:
Inst Valenciano Oncol, Dept Urol, Calle Prof Beltran Baguena 8, Valencia 46009, Spain
Villacampa, F:
Hosp Univ 12 Octubre, Dept Urol, Ave Cordoba S-N, Madrid 28041, Spain
Alvarez-Ossorio, JL:
Hosp Univ Puerta del Mar, Dept Urol, Ave Ana Viya 21, Cadiz 11009, Spain
Perez-Segura, P:
Hosp Univ Clin San Carlos, Dept Oncol, Calle Prof Martin Lagos S-N, Madrid 28040, Spain
Morote, J:
Hosp Univ Vall dHebron, Dept Urol, Paseo Vall dHebron 119-129, Barcelona 08035, Spain
Herrera-Imbroda, B:
Hosp Univ Virgen de la Victoria, Dept Urol, Campus Teatinos S-N, Malaga 29010, Spain
Nieto, J:
Hosp Gen Univ Ciudad Real, Dept Nephrol, Calle Obispo Rafael Torija S-N, Ciudad Real 13005, Spain
Carballido, J:
Hosp Univ Puerta de Hierro Majadahonda, Dept Urol, Calle Manuel de Falla 1, Majadahonda 28222, Spain
Anido, U:
Univ Santiago de Compostela, Hosp Clin, Dept Oncol, Travesia Choupana S-N, Santiago De Compostela 15706, Spain
Valero, M:
Novartis Farmaceut SA, Dept Med Affairs, Gran Via Corts Catalanes 764, Barcelona 08013, Spain
Meseguer, C:
Novartis Farmaceut SA, Dept Med Affairs, Gran Via Corts Catalanes 764, Barcelona 08013, Spain
Torra, R:
Univ Autonoma Barcelona, IIB St Pau, Fundacio Puigvert, Inherited Renal Dis,Dept Nephrol,REDINREN, Cartagena 340-350, Barcelona 08025, Spain
Gold, Green Published
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