Refractory Head and Neck Lymphatic Malformation in Infants Treated With Sirolimus: A Case Series

BackgroundExtensive and complex head and neck lymphatic malformations (LMs) are challenging to manage through traditional therapy. The purpose of this retrospective study was to assess the efficacy and safety of sirolimus in infants with refractory head and neck LMs.MethodsSirolimus was administered...

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Main Authors: Changhua Wu, Dan Song, Lei Guo, Liang Wang
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-07-01
Series:Frontiers in Oncology
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fonc.2021.616702/full
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author Changhua Wu
Dan Song
Lei Guo
Liang Wang
author_facet Changhua Wu
Dan Song
Lei Guo
Liang Wang
author_sort Changhua Wu
collection DOAJ
description BackgroundExtensive and complex head and neck lymphatic malformations (LMs) are challenging to manage through traditional therapy. The purpose of this retrospective study was to assess the efficacy and safety of sirolimus in infants with refractory head and neck LMs.MethodsSirolimus was administered orally on a continuous dosing schedule. Patients were seen every month for the first three months and then subsequently every three months. The primary endpoints were safety and efficacy based on clinical and radiological evaluations.ResultsEight patients, refractory to standard care, were enrolled and received sirolimus continuously. After 12 months of follow-up, the response and safety to medication was evaluated: all patients experienced reductions in LMs bulk, ranging from modest to significant. Some minor adverse effects were reported: mouth sores, eczema, gastrointestinal reaction, dyslipidemia, and neutropenia.ConclusionSirolimus was efficient in children with refractory head and neck LMs and was well tolerated.
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spelling doaj.art-7fa259ba00a3462582727c69a3ea74e62022-12-21T18:21:40ZengFrontiers Media S.A.Frontiers in Oncology2234-943X2021-07-011110.3389/fonc.2021.616702616702Refractory Head and Neck Lymphatic Malformation in Infants Treated With Sirolimus: A Case SeriesChanghua WuDan SongLei GuoLiang WangBackgroundExtensive and complex head and neck lymphatic malformations (LMs) are challenging to manage through traditional therapy. The purpose of this retrospective study was to assess the efficacy and safety of sirolimus in infants with refractory head and neck LMs.MethodsSirolimus was administered orally on a continuous dosing schedule. Patients were seen every month for the first three months and then subsequently every three months. The primary endpoints were safety and efficacy based on clinical and radiological evaluations.ResultsEight patients, refractory to standard care, were enrolled and received sirolimus continuously. After 12 months of follow-up, the response and safety to medication was evaluated: all patients experienced reductions in LMs bulk, ranging from modest to significant. Some minor adverse effects were reported: mouth sores, eczema, gastrointestinal reaction, dyslipidemia, and neutropenia.ConclusionSirolimus was efficient in children with refractory head and neck LMs and was well tolerated.https://www.frontiersin.org/articles/10.3389/fonc.2021.616702/fulllymphatic malformationsirolimushead and necksclerotherapyinfant
spellingShingle Changhua Wu
Dan Song
Lei Guo
Liang Wang
Refractory Head and Neck Lymphatic Malformation in Infants Treated With Sirolimus: A Case Series
Frontiers in Oncology
lymphatic malformation
sirolimus
head and neck
sclerotherapy
infant
title Refractory Head and Neck Lymphatic Malformation in Infants Treated With Sirolimus: A Case Series
title_full Refractory Head and Neck Lymphatic Malformation in Infants Treated With Sirolimus: A Case Series
title_fullStr Refractory Head and Neck Lymphatic Malformation in Infants Treated With Sirolimus: A Case Series
title_full_unstemmed Refractory Head and Neck Lymphatic Malformation in Infants Treated With Sirolimus: A Case Series
title_short Refractory Head and Neck Lymphatic Malformation in Infants Treated With Sirolimus: A Case Series
title_sort refractory head and neck lymphatic malformation in infants treated with sirolimus a case series
topic lymphatic malformation
sirolimus
head and neck
sclerotherapy
infant
url https://www.frontiersin.org/articles/10.3389/fonc.2021.616702/full
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