Regression of Cardiac Rhabdomyomas in a Neonate after Everolimus Treatment

Abstract EN

Cardiac rhabdomyoma often shows spontaneous regression and usually requires only close follow-up.

However, patients with symptomatic inoperable rhabdomyomas may be candidates for everolimus treatment.

Our patient had multiple inoperable cardiac rhabdomyomas causing serious left ventricle outflow-tract obstruction that showed a dramatic reduction in the size after everolimus therapy, a mammalian target of rapamycin (mTOR) inhibitor.

After discontinuation of therapy, an increase in the diameter of masses occurred and everolimus was restarted.

After 6 months of treatment, rhabdomyomas decreased in size and therapy was stopped.

In conclusion, everolimus could be a possible novel therapy for neonates with clinically significant rhabdomyomas.