Sirolimus for vascular anomalies in the first year of life: a systematic review

Evidence on effectiveness and safety of sirolimus in congenital vascular anomalies in infancy is lacking. We aim to systematically review the efficacy and safety of sirolimus in treating congenital VA in infancy. We searched for and included all studies evaluating sirolimus for VA in the first year of life. The primary outcome was effectiveness. The secondary outcome was safety. We included 84 case series and reports (172 participants). Sirolimus decreased the size of the VA in >50% of participants, most of whom had minor transient side effects, and 27% had no adverse effects at all. When categorized by age (<1 month, 1–5 months and 6–12 months), the effectiveness was similar in all age groups. Available evidence suggests that sirolimus is effective and well tolerated. The effectiveness of sirolimus should be evaluated in a well-designed randomized controlled or observational studies.