Nespoli Luisa Federica, Albani Elena, Corti Carla, Spaccini Luigina, Alfei Enrico, Daniele Irene, Zuccotti Gian Vincenzo, Lista Gianluca, Calcaterra Valeria, Mannarino Savina
Pediatric Cardiology Unit, "Vittore Buzzi" Children's Hospital, 20154 Milano, Italy.
Department of Pediatrics, "Vittore Buzzi" Children's Hospital, 20154 Milano, Italy.
Pediatr Rep. 2021 Mar 1;13(1):104-112. doi: 10.3390/pediatric13010015.
: 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. : 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. : 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.
心脏横纹肌瘤（CRs）是新生儿最常见的心脏肿瘤。约80-90%的病例与结节性硬化症（TSC）相关。在部分病例中，依维莫司对患有TS的儿童产生了显著的肿瘤消退效果。新生儿的最佳剂量仍未知。
我们描述了在一名患有多个双心室CRs并导致主动脉下梗阻的新生儿中使用依维莫司的情况，为维持血清谷浓度在3-7 ng/mL，采用了低剂量治疗（0.1 mg/日），该治疗在标签外成功使用，且无不良反应。
我们表明，当维持最低治疗范围时，低剂量依维莫司方案可能是治疗TS新生儿CR消退的一种有效且安全的方法。
