Application of Everolimus in Preoperative Neoadjuvant Therapy of Tuberous Sclerosis Complex Associated With Renal Angiomyolipoma: A Single-Center Report of 5 Cases.

Abstract

The purpose of the study was to investigate the efficacy and safety of everolimus in preoperative neoadjuvant therapy of tuberous sclerosis complex (TSC) associated with renal angiomyolipoma (RAML). The clinical data of 5 patients with TSC associated with RAML who were admitted to our hospital from May 2014 to December 2017 were analyzed. All patients received once per day everolimus 10 mg oral treatment for 3 months, and the efficacy and safety were evaluated at 1 month and 3 months after drug treatment, respectively. All patients received partial nephrectomy (PN) 1 week after drug withdrawal. After 1 month of treatment with everolimus, the median greatest tumor diameter (GTD) decrease was 1.8 cm (range, 1.0-4.5 cm). After 3 months of treatment, the median GTD reduction was 2.5 cm (range, 1.0-5.0 cm). The main adverse reactions included oral mucositis in 4 cases, menstrual disorder in 2 cases, and urinary tract infection in 1 case. The median surgery time was 110 minutes (range, 80-130 minutes), the median intraoperative blood loss was 70 mL (range, 50-400 mL), and the median postoperative hospital stay was 6 days (range, 5-7 days). After a median follow-up of 24 months (range, 6-30 months), no tumor with a diameter of >4 cm was found except for 1 recurrence. The preservation of renal function is the most important thing for patients with TSC associated with bilateral RAML. For some patients who cannot receive PN, neoadjuvant therapy with everolimus might be considered before the surgery so as to give patients the chance of PN.