| Autor: |
Cristache, C., Andronesi, A., Sorohan, B., Obrisca, B., Baston, C., Sinescu, I., Ismail, G. |
| Předmět: |
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| Zdroj: |
Romanian Journal of Urology; 2018, Vol. 17 Issue 4, p27-32, 6p |
| Abstrakt: |
Introduction and Objectives. Tuberous sclerosis complex (TSC) is a rare autosomal dominant disease with multisystemic involvement. Renal angiomyolipomas (AMLs) affect up to 80% of TSC patients and AML hemorrhage risk increases with size. Everolimus (EVE) is a mTOR inhibitor and is the only approved drug for managing renal AMLs in the adult population. We studied a series of TSC patients who received EVE therapy to evaluate its renal efficiency and safety. Materials and Methods. 18 patients with TSC and renal AML were enrolled, and they received 10 mg Everolimus daily. AML measurements were performed yearly, by using either computed tomography (CT) or magnetic resonance imaging (MRI). The largest baseline diameter of the biggest renal tumor was compared with the subsequent measurements. Clinical examination was performed, and biological parameters were systematically evaluated. Results. Of the total of 17 patients who underwent efficiency assessment, 16 (94.1%) responded to EVE by renal tumor shrinkage. A significant AML reduction was observed after 12 months of treatment, from a mean of 74.5 mm to 57.3 mm (23% reduction). The primary endpoint (AML’s diameter shrinkage ≥ 25% from baseline) was attained by 8 (47%) patients. In the aspect of adverse events (AE), no treatment interruptions were necessary due to intolerance. The most frequent AE was dyslipidemia, affecting 14 patients (82.3%), followed by anemia in 10 patients (58.8%). Proteinuria was the most common renal AE, which was managed by temporarily lowering the EVE dose. There were no AML-related hemorrhage and no local interventions were necessary. Conclusions. In our experience, EVE is an efficient and well tolerated treatment for adult TSC patients and associated AML. The most important positive result observed was no new bleeding episodes, which improves both renal and general outcomes. [ABSTRACT FROM AUTHOR] |
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