Growth of Angiomyolipomas in Patients with Tuberous Sclerosis Complex Treated with mTOR Inhibitor Differs by Indication for Treatment (P8-13.003)

Abstract

<h3>Objective:</h3> To determine whether growth rate differs for patients treated with mTOR inhibitor for SEGA or AML or no treatment. <h3>Background:</h3> Tuberous Sclerosis Complex (TSC) is characterized by epilepsy, renal angiomyolipoma (AML), subependymal giant cell astrocytoma (SEGA), and other cognitive, dermatologic, pulmonary, and cardiac manifestations. mTOR inhibitors are prescribed to reduce the size of AMLs or SEGA and reduce seizure frequency. Thus, the indication for initiating mTOR therapy differs for each patient. <h3>Design/Methods:</h3> Patients seen in the Atrium Health Wake Forest Baptist Adult Tuberous Sclerosis clinic were retrospectively evaluated. Patients were eligible if they were adults (age &gt;18 years), had at least 1 renal AML, and had at least two instances of imaging (CT or MRI) to evaluate renal AMLs. Data was collected on patient demographics, current/prior mTOR use, renal involvement, and AML size. AML size was defined as the largest diameter of the dominant lesion. Change in median diameter of the dominant lesions of each treatment was calculated. <h3>Results:</h3> 44 patients were reviewed (mean age = 35.6±13.8, 61% female); 22 met eligibility. AML growth differed for patients prescribed mTOR inhibitor to treat SEGA (n=3, mean treatment 25.5 months, 0.5 mm/year decrease on the right, 1.2 mm/year decrease on the left) compared to AML (n=5, mean treatment = 16.25 months, 0.96 mm/year decrease on the right, 3.5 mm/year decrease on the left). Patients who had never used an mTOR inhibitor displayed a 1.7 mm/year increase in AML size in the right kidney and 1.9 mm/year increase in AML size in the left kidney. <h3>Conclusions:</h3> In this cohort of adult TSC patients with AMLs, patients who did not receive mTOR therapy experienced annual growth in AML size. Those having ever taken an mTOR inhibitor experienced annual decrease in AML size. The median decrease in size differed between patients who initiated treatment for SEGA vs. AMLs. <b>Disclosure:</b> Miss Neal has nothing to disclose. Dr. Thakker has nothing to disclose. Mr. Mallett has nothing to disclose. Dr. Silvia has nothing to disclose. The institution of Dr. Boggs has received research support from Liva Nova. Dr. Boggs has a non-compensated relationship as a Chairman PAB with Epilepsy Alliance North Carolina that is relevant to AAN interests or activities. Theodore B Stem has nothing to disclose. Dr. Pathak has nothing to disclose. Dr. Strowd has received personal compensation for serving as an employee of Kaplan. Dr. Strowd has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Monteris Medical, Inc. Dr. Strowd has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novocure. Dr. Strowd has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for American Academy of Neurology. The institution of Dr. Strowd has received research support from Southeastern Brain Tumor Foundation. The institution of Dr. Strowd has received research support from Jazz Pharmaceuticals. The institution of Dr. Strowd has received research support from National Institutes of Health. The institution of Dr. Strowd has received research support from Alpha Omega Alpha. The institution of Dr. Strowd has received research support from American Board of Psychiatry and Neurology. Dr. Strowd has received publishing royalties from a publication relating to health care. Dr. Strowd has received publishing royalties from a publication relating to health care.