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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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Detalles Bibliográficos
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
Descripción
Sumario: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.