Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature Review
<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...
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MDPI AG
2021-03-01
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| Series: | Pediatric Reports |
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| Online Access: | https://www.mdpi.com/2036-7503/13/1/15 |
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| author | Luisa Federica Nespoli Elena Albani Carla Corti Luigina Spaccini Enrico Alfei Irene Daniele Gian Vincenzo Zuccotti Gianluca Lista Valeria Calcaterra Savina Mannarino |
| author_facet | Luisa Federica Nespoli Elena Albani Carla Corti Luigina Spaccini Enrico Alfei Irene Daniele Gian Vincenzo Zuccotti Gianluca Lista Valeria Calcaterra Savina Mannarino |
| author_sort | Luisa Federica Nespoli |
| collection | DOAJ |
| description | <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. |
| first_indexed | 2024-03-09T06:08:30Z |
| format | Article |
| id | doaj.art-91809e1898114c06b371c4b97e8b9bbf |
| institution | Directory Open Access Journal |
| issn | 2036-7503 |
| language | English |
| last_indexed | 2024-03-09T06:08:30Z |
| publishDate | 2021-03-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Pediatric Reports |
| spelling | doaj.art-91809e1898114c06b371c4b97e8b9bbf2023-12-03T12:01:24ZengMDPI AGPediatric Reports2036-75032021-03-0113110411210.3390/pediatric13010015Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature ReviewLuisa Federica Nespoli0Elena Albani1Carla Corti2Luigina Spaccini3Enrico Alfei4Irene Daniele5Gian Vincenzo Zuccotti6Gianluca Lista7Valeria Calcaterra8Savina Mannarino9Pediatric Cardiology Unit, “Vittore Buzzi” Children’s Hospital, 20154 Milano, ItalyDepartment of Pediatrics, “Vittore Buzzi” Children’s Hospital, 20154 Milano, ItalyPediatric Cardiology Unit, “Vittore Buzzi” Children’s Hospital, 20154 Milano, ItalyClinical Genetics Unit, Department of Obstetrics and Gynecology, “V. Buzzi” Children’s Hospital, University of Milano, 20154 Milano, ItalyPediatric Neurology Unit, “V. Buzzi” Children’s Hospital, Milano, 20154 Milano, ItalyNeonatal Pathology and Neonatal Intensive Care Unit, “V. Buzzi” Children’s Hospital, University of Milano, 20154 Milano, ItalyDepartment of Pediatrics, “Vittore Buzzi” Children’s Hospital, 20154 Milano, ItalyNeonatal Pathology and Neonatal Intensive Care Unit, “V. Buzzi” Children’s Hospital, University of Milano, 20154 Milano, ItalyDepartment of Pediatrics, “Vittore Buzzi” Children’s Hospital, 20154 Milano, ItalyPediatric Cardiology Unit, “Vittore Buzzi” Children’s Hospital, 20154 Milano, Italy<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.https://www.mdpi.com/2036-7503/13/1/15cardiac rhabdomyomatuberous sclerosiseverolimusneonatelow dose |
| spellingShingle | Luisa Federica Nespoli Elena Albani Carla Corti Luigina Spaccini Enrico Alfei Irene Daniele Gian Vincenzo Zuccotti Gianluca Lista Valeria Calcaterra Savina Mannarino Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature Review Pediatric Reports cardiac rhabdomyoma tuberous sclerosis everolimus neonate low dose |
| title | Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature Review |
| title_full | Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature Review |
| title_fullStr | Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature Review |
| title_full_unstemmed | Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature Review |
| title_short | Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature Review |
| title_sort | efficacy of everolimus low dose treatment for cardiac rhabdomyomas in neonatal tuberous sclerosis case report and literature review |
| topic | cardiac rhabdomyoma tuberous sclerosis everolimus neonate low dose |
| url | https://www.mdpi.com/2036-7503/13/1/15 |
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