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Long-term efficacy and safety of sirolimus for retinal astrocytic hamartoma associated with tuberous sclerosis complex

Mammalian target of rapamycin (mTOR) inhibitors (sirolimus or everolimus) have been demonstrated effective in reducing the size of tuberous sclerosis complex (TSC)-associated retinal astrocytic hamartoma (RAH) in short term. To investigate the long-term efficacy and safety of sirolimus on TSC-associ...

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Autores principales: Zhang, Chen-Xi, Xu, Kai-Feng, Long, Qin, Zhang, Xiao, Yang, Zhi-Kun, Dai, Rong-Ping, Zhang, Zhi-Qiao
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9716018/
https://www.ncbi.nlm.nih.gov/pubmed/36467422
http://dx.doi.org/10.3389/fcell.2022.973845
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author Zhang, Chen-Xi
Xu, Kai-Feng
Long, Qin
Zhang, Xiao
Yang, Zhi-Kun
Dai, Rong-Ping
Zhang, Zhi-Qiao
author_facet Zhang, Chen-Xi
Xu, Kai-Feng
Long, Qin
Zhang, Xiao
Yang, Zhi-Kun
Dai, Rong-Ping
Zhang, Zhi-Qiao
author_sort Zhang, Chen-Xi
collection PubMed
description Mammalian target of rapamycin (mTOR) inhibitors (sirolimus or everolimus) have been demonstrated effective in reducing the size of tuberous sclerosis complex (TSC)-associated retinal astrocytic hamartoma (RAH) in short term. To investigate the long-term efficacy and safety of sirolimus on TSC-associated RAH, 13 TSC-associated RAH patients (59 RAH lesions) who received sirolimus therapy for at least 2 years were retrospectively enrolled in this study. Changes in the maximal thickness (MT) of RAH on optical coherence tomography and the longest base diameter (LBD) of RAH on color fundus photography were assessed. The results showed that for a mean follow-up of 39 months, sirolimus was associated with a mean reduction of 14.6% in MT and 6.8% in LBD of RAHs. The main impacts of sirolimus occurred within the first 6–12 months, with 14.8% reduction in MT and 4.7% reduction in LBD. Mouth ulceration (10 [76.9%]) and acne (9 [69.2%]) were the most common adverse events. These follow-up data support the long-term use of sirolimus in TSC-associated RAH patients, and persistent use of sirolimus possibly prevents tumor regrowth.
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spelling pubmed-97160182022-12-03 Long-term efficacy and safety of sirolimus for retinal astrocytic hamartoma associated with tuberous sclerosis complex Zhang, Chen-Xi Xu, Kai-Feng Long, Qin Zhang, Xiao Yang, Zhi-Kun Dai, Rong-Ping Zhang, Zhi-Qiao Front Cell Dev Biol Cell and Developmental Biology Mammalian target of rapamycin (mTOR) inhibitors (sirolimus or everolimus) have been demonstrated effective in reducing the size of tuberous sclerosis complex (TSC)-associated retinal astrocytic hamartoma (RAH) in short term. To investigate the long-term efficacy and safety of sirolimus on TSC-associated RAH, 13 TSC-associated RAH patients (59 RAH lesions) who received sirolimus therapy for at least 2 years were retrospectively enrolled in this study. Changes in the maximal thickness (MT) of RAH on optical coherence tomography and the longest base diameter (LBD) of RAH on color fundus photography were assessed. The results showed that for a mean follow-up of 39 months, sirolimus was associated with a mean reduction of 14.6% in MT and 6.8% in LBD of RAHs. The main impacts of sirolimus occurred within the first 6–12 months, with 14.8% reduction in MT and 4.7% reduction in LBD. Mouth ulceration (10 [76.9%]) and acne (9 [69.2%]) were the most common adverse events. These follow-up data support the long-term use of sirolimus in TSC-associated RAH patients, and persistent use of sirolimus possibly prevents tumor regrowth. Frontiers Media S.A. 2022-11-18 /pmc/articles/PMC9716018/ /pubmed/36467422 http://dx.doi.org/10.3389/fcell.2022.973845 Text en Copyright © 2022 Zhang, Xu, Long, Zhang, Yang, Dai and Zhang. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Cell and Developmental Biology
Zhang, Chen-Xi
Xu, Kai-Feng
Long, Qin
Zhang, Xiao
Yang, Zhi-Kun
Dai, Rong-Ping
Zhang, Zhi-Qiao
Long-term efficacy and safety of sirolimus for retinal astrocytic hamartoma associated with tuberous sclerosis complex
title Long-term efficacy and safety of sirolimus for retinal astrocytic hamartoma associated with tuberous sclerosis complex
title_full Long-term efficacy and safety of sirolimus for retinal astrocytic hamartoma associated with tuberous sclerosis complex
title_fullStr Long-term efficacy and safety of sirolimus for retinal astrocytic hamartoma associated with tuberous sclerosis complex
title_full_unstemmed Long-term efficacy and safety of sirolimus for retinal astrocytic hamartoma associated with tuberous sclerosis complex
title_short Long-term efficacy and safety of sirolimus for retinal astrocytic hamartoma associated with tuberous sclerosis complex
title_sort long-term efficacy and safety of sirolimus for retinal astrocytic hamartoma associated with tuberous sclerosis complex
topic Cell and Developmental Biology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9716018/
https://www.ncbi.nlm.nih.gov/pubmed/36467422
http://dx.doi.org/10.3389/fcell.2022.973845
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