PP15.3 – 2545: Everolimus for patients with subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis complex (TSC): Results from an expanded access study

Abstract

Objective Everolimus For Fast Expanded aCcess in TSC SEGA (EFFECTS) study was designed to provide early access to everolimus for patients with SEGA associated with TSC and to further assess the safety and efficacy of everolimus in a real-world setting. Methods In this phase 3b, multicenter, expanded access study, patients ≥3 years of age with a definite diagnosis of TSC, with at least 1 SEGA lesion identified by MRI or CT scan received once daily everolimus, the dose of which was adjusted to attain a trough level of 5–15 ng/mL. Safety assessments included the collection of adverse events (AEs) and serious AEs (SAEs), with their severity and relationship to everolimus. Efficacy evaluation (secondary objective) was based on the best overall response at the end of the study. Results Overall, 100 of 120 (83.3%) patients completed the study. Median age of patients was 11 years (range 1–47); median daily dose of everolimus was 5.82 mg (range 2.0–11.8); median duration of exposure was 56.5 weeks (range 0.3–130). Overall, AEs were reported in 89 (74.2%) patients. The most common AEs were aphthous stomatitis (18, 15.0%), pyrexia (18, 15.0%), bronchitis (11, 9.2%), and stomatitis (10, 8.3%). Grade 3 and 4 AEs were reported in 25 (20.8%) and 3 (2.5%) patients, respectively. The most frequent grade 3 AE was stomatitis (4, 3.3%). A total of 62 (51.7%) patients had suspected drug-related AEs, of which 15 (12.5%) had grade 3 or 4 AEs. In 8 (6.7%) patients, AEs caused drug discontinuation. A total of 81 (67.5%) patients had a partial response, 35 (29.2%) had a stable disease, and 1 (0.8%) had progressive disease. Response was unknown in 3 (2.5%) patients. Conclusion Results confirm the acceptable safety profile and further support the efficacy of everolimus in patients with SEGA associated with TSC, in a real-world setting. Everolimus For Fast Expanded aCcess in TSC SEGA (EFFECTS) study was designed to provide early access to everolimus for patients with SEGA associated with TSC and to further assess the safety and efficacy of everolimus in a real-world setting. In this phase 3b, multicenter, expanded access study, patients ≥3 years of age with a definite diagnosis of TSC, with at least 1 SEGA lesion identified by MRI or CT scan received once daily everolimus, the dose of which was adjusted to attain a trough level of 5–15 ng/mL. Safety assessments included the collection of adverse events (AEs) and serious AEs (SAEs), with their severity and relationship to everolimus. Efficacy evaluation (secondary objective) was based on the best overall response at the end of the study. Overall, 100 of 120 (83.3%) patients completed the study. Median age of patients was 11 years (range 1–47); median daily dose of everolimus was 5.82 mg (range 2.0–11.8); median duration of exposure was 56.5 weeks (range 0.3–130). Overall, AEs were reported in 89 (74.2%) patients. The most common AEs were aphthous stomatitis (18, 15.0%), pyrexia (18, 15.0%), bronchitis (11, 9.2%), and stomatitis (10, 8.3%). Grade 3 and 4 AEs were reported in 25 (20.8%) and 3 (2.5%) patients, respectively. The most frequent grade 3 AE was stomatitis (4, 3.3%). A total of 62 (51.7%) patients had suspected drug-related AEs, of which 15 (12.5%) had grade 3 or 4 AEs. In 8 (6.7%) patients, AEs caused drug discontinuation. A total of 81 (67.5%) patients had a partial response, 35 (29.2%) had a stable disease, and 1 (0.8%) had progressive disease. Response was unknown in 3 (2.5%) patients. Results confirm the acceptable safety profile and further support the efficacy of everolimus in patients with SEGA associated with TSC, in a real-world setting.