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Safety Evaluation of Oral Sirolimus in the Treatment of Childhood Diseases: A Systematic Review

Background: Sirolimus, a mammalian target of rapamycin inhibitor, has been widely used in pediatric patients, but the safety of sirolimus in pediatric patients has not been well determined. Objective: The objective of this study was to systematically evaluate prospective studies reporting the safety...

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Autores principales: Zhang, Zixin, Li, Yanan, Zhang, Guangyue, Yang, Kaiying, Qiu, Tong, Zhou, Jiangyuan, Gong, Xue, Ji, Yi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9497617/
https://www.ncbi.nlm.nih.gov/pubmed/36138604
http://dx.doi.org/10.3390/children9091295
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author Zhang, Zixin
Li, Yanan
Zhang, Guangyue
Yang, Kaiying
Qiu, Tong
Zhou, Jiangyuan
Gong, Xue
Ji, Yi
author_facet Zhang, Zixin
Li, Yanan
Zhang, Guangyue
Yang, Kaiying
Qiu, Tong
Zhou, Jiangyuan
Gong, Xue
Ji, Yi
author_sort Zhang, Zixin
collection PubMed
description Background: Sirolimus, a mammalian target of rapamycin inhibitor, has been widely used in pediatric patients, but the safety of sirolimus in pediatric patients has not been well determined. Objective: The objective of this study was to systematically evaluate prospective studies reporting the safety of sirolimus in the treatment of childhood diseases. Methods: The following data were extracted in a standardized manner: study design, demographic characteristics, intervention, and safety outcomes. Results: In total, 9 studies were included, encompassing 575 patients who received oral sirolimus for at least 6 months. Various adverse events occurred. The most common adverse event was oral mucositis (8.2%, 95% CI: 0.054 to 0.110). Through comparative analysis of the subgroups based on the targeted concentration range, we discovered that many adverse events were significantly higher in the high concentration group (≥10 ng/mL) than in the low concentration group (<10 ng/mL) (p < 0.01). More interestingly, we found that oral mucositis was more frequently reported in children with vascular anomalies than tuberous sclerosis complex. Conclusions: This study shows that oral sirolimus in the treatment of childhood diseases is safe and reliable. However, sirolimus treatment in the pediatric population should be strictly monitored to reduce the occurrence of serious or fatal adverse events.
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spelling pubmed-94976172022-09-23 Safety Evaluation of Oral Sirolimus in the Treatment of Childhood Diseases: A Systematic Review Zhang, Zixin Li, Yanan Zhang, Guangyue Yang, Kaiying Qiu, Tong Zhou, Jiangyuan Gong, Xue Ji, Yi Children (Basel) Systematic Review Background: Sirolimus, a mammalian target of rapamycin inhibitor, has been widely used in pediatric patients, but the safety of sirolimus in pediatric patients has not been well determined. Objective: The objective of this study was to systematically evaluate prospective studies reporting the safety of sirolimus in the treatment of childhood diseases. Methods: The following data were extracted in a standardized manner: study design, demographic characteristics, intervention, and safety outcomes. Results: In total, 9 studies were included, encompassing 575 patients who received oral sirolimus for at least 6 months. Various adverse events occurred. The most common adverse event was oral mucositis (8.2%, 95% CI: 0.054 to 0.110). Through comparative analysis of the subgroups based on the targeted concentration range, we discovered that many adverse events were significantly higher in the high concentration group (≥10 ng/mL) than in the low concentration group (<10 ng/mL) (p < 0.01). More interestingly, we found that oral mucositis was more frequently reported in children with vascular anomalies than tuberous sclerosis complex. Conclusions: This study shows that oral sirolimus in the treatment of childhood diseases is safe and reliable. However, sirolimus treatment in the pediatric population should be strictly monitored to reduce the occurrence of serious or fatal adverse events. MDPI 2022-08-26 /pmc/articles/PMC9497617/ /pubmed/36138604 http://dx.doi.org/10.3390/children9091295 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Systematic Review
Zhang, Zixin
Li, Yanan
Zhang, Guangyue
Yang, Kaiying
Qiu, Tong
Zhou, Jiangyuan
Gong, Xue
Ji, Yi
Safety Evaluation of Oral Sirolimus in the Treatment of Childhood Diseases: A Systematic Review
title Safety Evaluation of Oral Sirolimus in the Treatment of Childhood Diseases: A Systematic Review
title_full Safety Evaluation of Oral Sirolimus in the Treatment of Childhood Diseases: A Systematic Review
title_fullStr Safety Evaluation of Oral Sirolimus in the Treatment of Childhood Diseases: A Systematic Review
title_full_unstemmed Safety Evaluation of Oral Sirolimus in the Treatment of Childhood Diseases: A Systematic Review
title_short Safety Evaluation of Oral Sirolimus in the Treatment of Childhood Diseases: A Systematic Review
title_sort safety evaluation of oral sirolimus in the treatment of childhood diseases: a systematic review
topic Systematic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9497617/
https://www.ncbi.nlm.nih.gov/pubmed/36138604
http://dx.doi.org/10.3390/children9091295
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