Gene Therapy vs Cochlear Implantation in Restoring Hearing Function and Speech Perception for Individuals With Congenital Deafness

作者：Xiaoting Cheng, Jiake Zhong, Jiajia Zhang, Chong Cui, Luoying Jiang, Yang-Wenyi Liu, Yuxin Chen, Qi Cao, Daqi Wang, Guiqing Cheng, Yuxin Zong, Min Shen, Chunxin Xu, Jun Lv, Hui Wang, Longlong Zhang, Biyun Zhu, Honghai Tang, Jinghan Wang, Xintai Fan, Yanqing Fang, Luo Guo, Jiawei Guo, Liheng Chen, Yanbo Yin, Zijing Wang, Lei Han, Shaowei Hu, Shengyi Wang, Guoyou Qin, Xue-Zhong Liu, Jinqiu Sang, Fan‐Gang Zeng, Wuqing Wang, Bing Chen, Zheng‐Yi Chen, Huawei Li, Yilai Shu · 发表于：JAMA Neurology · 年份：2025 · DOI：10.1001/jamaneurol.2025.2053 · 被引用次数：17 · 研究领域：Hearing, Cochlea, Tinnitus, Genetics、Hearing Loss and Rehabilitation、Congenital Ear and Nasal Anomalies

Importance: OTOF gene therapy (GT) has been shown to improve hearing and speech. The efficacy of GT remains to be compared against cochlear implantation (CI), the current gold standard for congenital deafness. Objective: To evaluate treatment outcomes in auditory and speech perception between patients with congenital deafness treated with GT, CI, or both. Design, Setting, and Participants: This nonblind cohort study was conducted between December 2022 and November 2024. GT patients received follow-up at 3, 6, and 12 months; CI patients received 1-time evaluation at the corresponding time intervals or longer (3, 6, or 12 months). The study was conducted at a single class A tertiary hospital in China. Participants with congenital severe to complete hearing loss, aged 1 to 18 years, who received GT or CI were enrolled. They were matched on duration of deafness, hearing thresholds, and speech ability at the presurgical baseline. Of 1568 participants screened, 72 participants enrolled. Participants were excluded if they had inner ear malformations or vestibular-cochlear nerve abnormalities. Exposures: GT only vs CI; bimodal (unilateral GT plus contralateral CI) vs bilateral CI; GT (CI turned off [CI-off]) vs unilateral CI. Main Outcomes and Measures: The primary outcomes were auditory and speech perception evaluated by questionnaires, including the Infant-Toddler Meaningful Auditory Integration Scale/Meaningful Auditory Integration Scale (IT-MAIS/MAIS), and tests, including audiom...