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...
Ausführliche Beschreibung

Gespeichert in:

Autor*in:	Luisa Federica Nespoli [verfasserIn] Elena Albani [verfasserIn] Carla Corti [verfasserIn] Luigina Spaccini [verfasserIn] Enrico Alfei [verfasserIn] Irene Daniele [verfasserIn] Gian Vincenzo Zuccotti [verfasserIn] Gianluca Lista [verfasserIn] Valeria Calcaterra [verfasserIn] Savina Mannarino [verfasserIn]

Format:	E-Artikel
Sprache:	Englisch

Erschienen:	2021

Schlagwörter:	cardiac rhabdomyoma tuberous sclerosis everolimus neonate low dose

Übergeordnetes Werk:	In: Pediatric Reports - MDPI AG, 2009, 13(2021), 1, Seite 104-112
Übergeordnetes Werk:	volume:13 ; year:2021 ; number:1 ; pages:104-112

Links:	Link aufrufen Link aufrufen Link aufrufen Journal toc

DOI / URN:	10.3390/pediatric13010015

Katalog-ID:	DOAJ007585160

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allfieldsSound	10.3390/pediatric13010015 doi (DE-627)DOAJ007585160 (DE-599)DOAJ91809e1898114c06b371c4b97e8b9bbf DE-627 ger DE-627 rakwb eng RJ1-570 Luisa Federica Nespoli verfasserin aut Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature Review 2021 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier <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. cardiac rhabdomyoma tuberous sclerosis everolimus neonate low dose Medicine R Pediatrics Elena Albani verfasserin aut Carla Corti verfasserin aut Luigina Spaccini verfasserin aut Enrico Alfei verfasserin aut Irene Daniele verfasserin aut Gian Vincenzo Zuccotti verfasserin aut Gianluca Lista verfasserin aut Valeria Calcaterra verfasserin aut Savina Mannarino verfasserin aut In Pediatric Reports MDPI AG, 2009 13(2021), 1, Seite 104-112 (DE-627)634760505 (DE-600)2572005-3 20367503 nnns volume:13 year:2021 number:1 pages:104-112 https://doi.org/10.3390/pediatric13010015 kostenfrei https://doaj.org/article/91809e1898114c06b371c4b97e8b9bbf kostenfrei https://www.mdpi.com/2036-7503/13/1/15 kostenfrei https://doaj.org/toc/2036-7503 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2005 GBV_ILN_2009 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2055 GBV_ILN_2111 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 13 2021 1 104-112
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callnumber-first	R - Medicine
author	Luisa Federica Nespoli
spellingShingle	Luisa Federica Nespoli misc RJ1-570 misc cardiac rhabdomyoma misc tuberous sclerosis misc everolimus misc neonate misc low dose misc Medicine misc R misc Pediatrics Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature Review
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topic_title	RJ1-570 Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature Review cardiac rhabdomyoma tuberous sclerosis everolimus neonate low dose
topic	misc RJ1-570 misc cardiac rhabdomyoma misc tuberous sclerosis misc everolimus misc neonate misc low dose misc Medicine misc R misc Pediatrics
topic_unstemmed	misc RJ1-570 misc cardiac rhabdomyoma misc tuberous sclerosis misc everolimus misc neonate misc low dose misc Medicine misc R misc Pediatrics
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title	Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature Review
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title_full	Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature Review
author_sort	Luisa Federica Nespoli
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author_browse	Luisa Federica Nespoli Elena Albani Carla Corti Luigina Spaccini Enrico Alfei Irene Daniele Gian Vincenzo Zuccotti Gianluca Lista Valeria Calcaterra Savina Mannarino
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title_sort	efficacy of everolimus low-dose treatment for cardiac rhabdomyomas in neonatal tuberous sclerosis: case report and literature review
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title_auth	Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature Review
abstract	<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.
abstractGer	<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.
abstract_unstemmed	<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.
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title_short	Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature Review
url	https://doi.org/10.3390/pediatric13010015 https://doaj.org/article/91809e1898114c06b371c4b97e8b9bbf https://www.mdpi.com/2036-7503/13/1/15 https://doaj.org/toc/2036-7503
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Efficacy of Everolimus Low-Dose Treatment for Cardiac Rhabdomyomas in Neonatal Tuberous Sclerosis: Case Report and Literature Review

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