Long-Term Efficacy and Safety of Mammalian Target of Rapamycin Inhibitor Treatment for Subependymal Giant Cell Astrocytoma in Tuberous Sclerosis.
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BACKGROUND: Subependymal giant cell astrocytoma (SEGA) is a low-grade astrocytoma occurring in approximately 5%-20% of persons affected with tuberous sclerosis complex (TSC). Therapy options include surgical resection or medical treatment with mammalian target of rapamycin (mTOR) inhibitors ([mTORis] everolimus or sirolimus) to prevent further growth and neurosurgical interventions due to life-threatening hydrocephalus. Short-term follow-up of mTORi treatment has been described in earlier studies; however, data on long-term efficacy and safety are limited. METHODS: Patients from SEGA studies at Cincinnati Children's Hospital Medical Center with ongoing care at our center and consented to ongoing retrospective records collection were included in the study. Data was compiled from medical records. RESULTS: Our cohort consisted of 25 patients with TSC. All but one were currently receiving mTORi treatment with either sirolimus (N = 4, 20%) or everolimus (N = 20, 80%) at the time of evaluation. Median mTORi treatment duration was 15.5 years (range: 9.8-16.8 years, interquartile range: 0.9 years). One patient underwent SEGA resection after starting mTORi. None required cerebrospinal fluid diversion. In 19 patients (76%), SEGA was stable or decreased over time. In six patients, it increased slightly from pretreatment baseline. Ongoing adverse drug reactions (ADR) at their last visit were reported in the medical record of eight patients (33%) with intermittent aphthous ulcers being the most frequent (N = 7). Long-term ADRs were observed in 17 patients (68%), including hypercholesterolemia (N = 10), diabetes mellitus type 2 (N = 6), prediabetes (N = 1), hypertension (N = 6), and osteoporosis (N = 3). CONCLUSIONS: Long-term TSC-associated SEGA treatment with mTORi is safe, effective and well tolerated. Knowledge of adverse reactions is important for successful long-term therapy.