Prenatal Sirolimus Treatment for Rhabdomyomas in Tuberous Sclerosis
Daniel Ebrahimi-Fakhari1, Gabrielle Stires2, Eunice Hahn3
1Division of Neurology, Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio; Department of General Pediatrics, University Children's Hospital Muenster, Muenster, Germany.
Pediatric Neurology
|October 8, 2021
Summary
Maternal sirolimus effectively reduced fetal cardiac rhabdomyomas in tuberous sclerosis complex cases. While generally safe, optimal dosing requires further study, with a recommended starting dose of 2-3.5 mg/m²/day for target trough levels.
Area of Science:
- Cardiology
- Genetics
- Pharmacology
Background:
- Tuberous sclerosis complex (TSC) can cause cardiac rhabdomyomas, which may require intervention.
- Mechanistic target of rapamycin (mTOR) inhibitors like sirolimus are effective for various conditions.
- Limited data exists on transplacental sirolimus for fetal rhabdomyomas in TSC.
