Reasons for premature sirolimus discontinuation.
death
ADVERSE EVENTS
Globally, 45 patients stopped sirolimus before the planned duration of 2 years (including 25 before the 12 months time point). Nineteen patients (14%) stopped because of lack of efficacy, including 1 death due to progression of a severe generalized lymphatic anomaly (GLA). Another patient died in the context of pericardial drainage not thought to be related to sirolimus. Ten patients (8%) stopped due to adverse events (AEs) despite a benefit observed in 8 of them. Fifteen patients (11.5%) stopped for noncompliance, despite a benefit observed in 9 of them.