Pediatric Reports · 2021 · 13 citations · 15 references
MedicinePediatricsPharmacotherapyNeonatologyPediatric CardiologyPediatric PharmacotherapyNeonatal Cardiac DisordersNeonatal CardiologyNeonatal PharmacologyNeonatal PharmacotherapyPediatric TumorsTuberous Sclerosis ComplexCardiac RhabdomyomaDosage StabilityDosing In Premature InfantsEverolimus PharmacokineticsEverolimus TreatmentIndividual Therapeutic RangeNeonate DosingTherapeutic Dose Range
Background: 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. Case presentation: 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. Conclusions: 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.
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