Sirolimus can Increase the Disappearance Rate of Cardiac Rhabdomyomas Associated with Tuberous Sclerosis: A Prospective Cohort and Self-controlled Case Series Study.

Abstract Objectives To investigate the efficacy and safety of sirolimus in the treatment of cardiac rhabdomyomas ) associated with tuberous sclerosis complex (TSC) and the specific benefits in different subgroups. Study design The study was a prospective cohort and self-controlled case series (SCCS) study. Based on the prevalence of CR at different ages, we estimated the natural tumor disappearance rate (TDR). The subgroup analysis was done by Cox regression. SCCS method was used to assess the magnitude and duration of the drug effect. Adverse events were described. Results A total of 217 patients were included in the cohort study. TDR was higher in younger age groups (HD = 0.99, P = 0.027) and female patients (HD = 2.08, P = 0.015). The age-adjusted incidence ratio showed that the disappearance of rhabdomyomas between 3 and 6 months was more related to sirolimus. Adverse events were observed 60 times in 42 of 217 children, mainly stomatitis. Conclusion Sirolimus can increase the disappearance rate of CR in the TSC population. Efficacy varies by sex and age: female and younger patients have higher TDR. Sirolimus is well-tolerated.