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Sirolimus for vascular anomalies in the first year of life: a systematic review
Rebeca Cavazos1, Monika S Patil2, Sharada H Gowda2
1Department of Pediatrics, Section of Neonatology, Baylor College of Medicine, Houston, TX, USA. rxcavazo@texaschildrens.org.
Insights
Sirolimus effectively treats congenital vascular anomalies (VA) in infants, with over half experiencing reduced lesion size and generally mild side effects. Further research in controlled studies is recommended for sirolimus in infant VA treatment.
Area of Science:
- Pediatric Medicine
- Vascular Surgery
- Pharmacology
Background:
- Congenital vascular anomalies (VA) in infants present a significant clinical challenge.
- Limited evidence exists regarding the efficacy and safety of sirolimus for treating infantile VA.
- Sirolimus, an mTOR inhibitor, shows potential in managing complex vascular malformations.
Conclusions:
- Current evidence suggests sirolimus is an effective and well-tolerated treatment for congenital vascular anomalies in infants.
- The safety profile appears favorable, with predominantly mild and temporary adverse events.
- Further evaluation of sirolimus effectiveness and safety is warranted through well-designed randomized controlled or observational studies.
Abstract:
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.
