Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature Review

• Main Authors: Luisa Federica Nespoli (Author), Elena Albani (Author), Carla Corti (Author), Luigina Spaccini (Author), Enrico Alfei (Author), Irene Daniele (Author), Gian Vincenzo Zuccotti (Author), Gianluca Lista (Author), Valeria Calcaterra (Author), Savina Mannarino (Author)
• Format: Book
• Published: MDPI AG, 2021-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Summary

<i>Background</i>: Cardiac rhabdomyomas (CRs) are the most common cardiac tumors in newborns. Approximately 80-90% of cases are associated with tuberous sclerosis complex (TSC). In selective cases, Everolimus has resulted in a remarkable tumoral regression effect in children with TS. The optimal dosage for neonates is still unknown. <i>Case presentation</i>: We describe the use of Everolimus in a neonate with multiple biventricular CRs, causing subaortic obstruction, in which a low-dose treatment (0.1 mg/die), in an effort to maintain serum trough levels of 3-7 ng/mL, was successfully used off-label, without adverse effects. <i>Conclusions</i>: We showed that a low-dose Everolimus regimen may be an effective and safe treatment for CR regression in TS neonates, when the minimum therapeutic range was maintained.

Item Description

10.3390/pediatric13010015
2036-7503