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Application of topical gentamicin ointment in the treatment of Nagashima‐type palmoplantar keratosis in children with a nonsense mutation

IMPORTANCE: Nagashima‐type palmoplantar keratosis (NPPK) is a hereditary dermatosis mostly caused by a nonsense mutation in SERPINB7. Despite the increasing interest in readthrough gentamicin treatment of NPPK, clinical evidence for this treatment is limited. OBJECTIVE: This study aimed to provide f...

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Autores principales: Wang, Shan, Yang, Zhou, Liu, Ying, Zhang, Huan, Liu, Zongyang, Wang, Xiaoling, Li, Ying, Liu, Haihong, Yang, Yonghong, Ma, Lin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10509405/
https://www.ncbi.nlm.nih.gov/pubmed/37736370
http://dx.doi.org/10.1002/ped4.12389
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author Wang, Shan
Yang, Zhou
Liu, Ying
Zhang, Huan
Liu, Zongyang
Wang, Xiaoling
Li, Ying
Liu, Haihong
Yang, Yonghong
Ma, Lin
author_facet Wang, Shan
Yang, Zhou
Liu, Ying
Zhang, Huan
Liu, Zongyang
Wang, Xiaoling
Li, Ying
Liu, Haihong
Yang, Yonghong
Ma, Lin
author_sort Wang, Shan
collection PubMed
description IMPORTANCE: Nagashima‐type palmoplantar keratosis (NPPK) is a hereditary dermatosis mostly caused by a nonsense mutation in SERPINB7. Despite the increasing interest in readthrough gentamicin treatment of NPPK, clinical evidence for this treatment is limited. OBJECTIVE: This study aimed to provide further evidence for the use of topical gentamicin in the treatment of NPPK in children with nonsense mutations. METHODS: We designed a bilaterally controlled study of topical gentamicin ointment. Children diagnosed with NPPK carrying nonsense mutations were enrolled in this study. A 0.1% gentamicin ointment was applied to one hand and an emollient to the other for 3 months. A bilateral comparison of the visual analog scale scores for clinical manifestations and safety was performed. RESULTS: Ten children with NPPK were included in this study. In comparison with the emollient side, the topical gentamicin side showed significant improvements in hyperkeratosis, erythema, maceration, and desquamation after 1 and 3 months of treatment (P < 0.05). However, hyperhidrosis and odor did not improve significantly. No adverse events were observed during the systemic safety monitoring examinations. INTERPRETATION: Topical gentamicin ointment showed good safety in the treatment of NPPK with nonsense mutations, indicating that it is a promising therapeutic choice in children with NPPK.
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spelling pubmed-105094052023-09-21 Application of topical gentamicin ointment in the treatment of Nagashima‐type palmoplantar keratosis in children with a nonsense mutation Wang, Shan Yang, Zhou Liu, Ying Zhang, Huan Liu, Zongyang Wang, Xiaoling Li, Ying Liu, Haihong Yang, Yonghong Ma, Lin Pediatr Investig Original Article IMPORTANCE: Nagashima‐type palmoplantar keratosis (NPPK) is a hereditary dermatosis mostly caused by a nonsense mutation in SERPINB7. Despite the increasing interest in readthrough gentamicin treatment of NPPK, clinical evidence for this treatment is limited. OBJECTIVE: This study aimed to provide further evidence for the use of topical gentamicin in the treatment of NPPK in children with nonsense mutations. METHODS: We designed a bilaterally controlled study of topical gentamicin ointment. Children diagnosed with NPPK carrying nonsense mutations were enrolled in this study. A 0.1% gentamicin ointment was applied to one hand and an emollient to the other for 3 months. A bilateral comparison of the visual analog scale scores for clinical manifestations and safety was performed. RESULTS: Ten children with NPPK were included in this study. In comparison with the emollient side, the topical gentamicin side showed significant improvements in hyperkeratosis, erythema, maceration, and desquamation after 1 and 3 months of treatment (P < 0.05). However, hyperhidrosis and odor did not improve significantly. No adverse events were observed during the systemic safety monitoring examinations. INTERPRETATION: Topical gentamicin ointment showed good safety in the treatment of NPPK with nonsense mutations, indicating that it is a promising therapeutic choice in children with NPPK. John Wiley and Sons Inc. 2023-06-28 /pmc/articles/PMC10509405/ /pubmed/37736370 http://dx.doi.org/10.1002/ped4.12389 Text en © 2023 Chinese Medical Association. Pediatric Investigation published by John Wiley & Sons Australia, Ltd on behalf of Futang Research Center of Pediatric Development. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ (https://creativecommons.org/licenses/by-nc-nd/4.0/) License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.
spellingShingle Original Article
Wang, Shan
Yang, Zhou
Liu, Ying
Zhang, Huan
Liu, Zongyang
Wang, Xiaoling
Li, Ying
Liu, Haihong
Yang, Yonghong
Ma, Lin
Application of topical gentamicin ointment in the treatment of Nagashima‐type palmoplantar keratosis in children with a nonsense mutation
title Application of topical gentamicin ointment in the treatment of Nagashima‐type palmoplantar keratosis in children with a nonsense mutation
title_full Application of topical gentamicin ointment in the treatment of Nagashima‐type palmoplantar keratosis in children with a nonsense mutation
title_fullStr Application of topical gentamicin ointment in the treatment of Nagashima‐type palmoplantar keratosis in children with a nonsense mutation
title_full_unstemmed Application of topical gentamicin ointment in the treatment of Nagashima‐type palmoplantar keratosis in children with a nonsense mutation
title_short Application of topical gentamicin ointment in the treatment of Nagashima‐type palmoplantar keratosis in children with a nonsense mutation
title_sort application of topical gentamicin ointment in the treatment of nagashima‐type palmoplantar keratosis in children with a nonsense mutation
topic Original Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10509405/
https://www.ncbi.nlm.nih.gov/pubmed/37736370
http://dx.doi.org/10.1002/ped4.12389
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