EFFECTS: an expanded access program of everolimus for patients with subependymal giant cell astrocytoma associated with tuberous sclerosis complex

Abstract

[...]recently, surgery was the only option available for the treatment of SEGAs. Abbreviations AEs, adverse events; CT, computed tomography; CTCAE, Common Terminology Criteria for Adverse Events; EFFECTS, Everolimus For Fast Expanded aCcess in TSC SEGA; EMA, European Medicines Agency; EXIST, EXamining everolimus In a Study of Tuberous sclerosis complex; FAS, full analysis set, MRI, magnetic resonance imaging; mTOR, mammalian target of rapamycin; SEGA, subependymal giant cell astrocytoma; TSC, tuberous sclerosis complex; USFDA, United States Food and Drug Administration Declarations Acknowledgments This study was funded by Novartis Pharma AG. We thank Kshama Chitnis (ex-employee) and Rama Mylapuram, Novartis Healthcare Pvt. Ltd. for providing medical editorial assistance with this manuscript.