Conclusions
autism disorders, intellectual disabilities, seizure, epilepsy
EVENTS, HYDROCEPHALUS, ADVERSE EFFECTS, EPILEPSY
The effectiveness of everolimus in epilepsy was acceptable and in line with EXIST-3. In this study, however, most patients also changed their concomitant ASM treatments. This may suggest that everolimus was not the only cause of the improvement in the seizure situation. Treatment effectiveness with everolimus was associated with a younger age. The results indicate that everolimus treatment reduces or stabilises rAML lesions, reduces risk of renal events, and reduces SEGA volume and risk of hydrocephalus.Most adverse effects were generally mild to moderate, but some tended to be more frequent than in EXIST and other clinical practice studies. Careful monitoring of adverse effects is needed, and benefits against adverse effects should be carefully considered and discussed with patients and parents before the start of treatment and during follow-up. It is our opinion that awareness in vulnerable patients, such as children and patients with intellectual disabilities and autism disorders, is of special importance.