Last Updated : November 20, 2017
Details
FilesProject Line:
Health Technology Review
Project Sub Line:
Summary with Critical Appraisal
Project Number:
RC0943-000
Question
- What is the clinical effectiveness of sirolimus for the treatment of tuberous sclerosis complex?
- What is the clinical effectiveness of everolimus for the treatment of tuberous sclerosis complex?
Key Message
Eight publications were identified including four systematic reviews, two RCTs, a subgroup analysis of an RCT, and one cohort study. The evidence suggests that treatment with everolimus or sirolimus demonstrated a better response rate when compared to placebo for symptoms of TSC including reduction in cardiac rhabdomyolipoma, seizure frequency, and renal angiomyolipoma. Three unique primary studies were identified for everolimus and one study was identified for sirolimus. However, the studies were conducted in small sample sizes and have a follow-up of up to one year, making it difficult to understand the long term effectiveness and safety profile of these agents. Common adverse events include stomatitis, mouth ulcerations, nasopharynitis and upper respiratory tract infections and generally occur in at least 15% of those who are treated with everolimus or sirolimus which may result in discontinuation or dose reduction of the drug, likely requiring frequent monitoring and assessment.
Files
Last Updated : November 20, 2017