Renal angiomyolipoma and tuberous sclerosis complex: long-term safety and efficacy outcomes of Everolimus therapy.

Autor: Ruiz Guerrero E; Unidad de Uro-Oncología, Servicio Urología, Hospital Universitario Puerta del Mar, INIBICA (Instituto de Investigación e Innovación Biomédica), Cádiz, España. Electronic address: estefirg_8@msn.com., Ledo Cepero MJ; Unidad de Uro-Oncología, Servicio Urología, Hospital Universitario Puerta del Mar, INIBICA (Instituto de Investigación e Innovación Biomédica), Cádiz, España., Ojeda Claro AV; Unidad de Uro-Oncología, Servicio Urología, Hospital Universitario Puerta del Mar, INIBICA (Instituto de Investigación e Innovación Biomédica), Cádiz, España., Soto Delgado M; Unidad de Uro-Oncología, Servicio Urología, Hospital Universitario Puerta del Mar, INIBICA (Instituto de Investigación e Innovación Biomédica), Cádiz, España., Álvarez-Ossorio Fernández JL; Unidad de Uro-Oncología, Servicio Urología, Hospital Universitario Puerta del Mar, INIBICA (Instituto de Investigación e Innovación Biomédica), Cádiz, España.
Jazyk: English; Spanish; Castilian
Zdroj: Actas urologicas espanolas [Actas Urol Esp (Engl Ed)] 2021 May; Vol. 45 (4), pp. 264-272. Date of Electronic Publication: 2021 Feb 23.
DOI: 10.1016/j.acuro.2020.11.004
Abstrakt: Introduction: Renal angiomyolipoma is a frequent manifestation of Tuberous Sclerosis Complex (TSC), for which everolimus therapy has been recently established as a novel non-invasive therapeutic option. As there are limited real life and long-term data, the analysis of our experience provides added value in terms of safety and efficacy.
Material and Methods: Descriptive analysis of our experience in patients with giant bilateral renal angiomyolipomas, in the context of TSC, treated with 10 mg oral everolimus daily, during a median of 71.5 months. We evaluated the following parameters: response rate and duration, reduction of kidney size and lesions, prevention of complications and presentation of toxicity and its cause.
Results: We confirm the effectiveness of treatment in 4 young patients, with multiple, bilateral angiomyolipomas of a median of 12 (5-19) cm maximum diameter, from June 2013 to date, after continuous reduction in lesion size, a decrease of 30% of the volume in 75% at six months and 50% in half of the subjects at two years, still showing drug response. Absence of complications such as bleeding or glomerular filtration rate decline in the long term, with a favorable safety profile, without interruptions and with mild-moderate, non-cumulative adverse effects, mostly within the first year of treatment.
Conclusion: Everolimus is a safe and effective therapeutic option for renal angiomyolipoma and various manifestations of TSC, which has been reproduced in real life with six years of follow-up.
(Copyright © 2020 AEU. Publicado por Elsevier España, S.L.U. All rights reserved.)
Databáze: MEDLINE
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