International expert consensus on gene therapy for hereditary hearing loss: Based on clinical trials

作者：Xintai Fan, Ziwen Gao, Jiake Zhong, Yuxin Chen, Xiaoyun Chen, Lukas D. Landegger, Tobias Moser, Fan-Gang Zeng, Yu Sun, Xin Jin, Robert Nash, Wade W. Chien, Dan Jiang, John H. Greinwald, Manohar Bance, Manuel Manrique Rodríguez, Sang‐Yeon Lee, Guodong Feng, Haidi Yang, Chen‐Chi Wu, Lei Xu, Wei Yuan, Yong Feng, Yu Zhao, Barbara Vona, Nicola Strenzke, Dirk Beutner, Nikul Amin, James Arwyn–Jones, Deepak Chandrasekeharan, Dazhi Shi, Di Zhang, Jian‐Ming Yang, Jieyu Qi, Qin Wang, Yanbo Yin, Yen‐Fu Cheng, Yong Tao, Yongfu Yu, Daqi Wang, Luoying Jiang, Luo Guo, Liheng Chen, Xiaoting Cheng, Chong Cui, Jun Lv, Shuang Han, Wuqing Wang, Yongxin Li, Xia Gao, Xue Zhong Liu, Dingjun Zha, Haibo Shi, Bing Chen, Qiuju Wang, Huijun Yuan, Shiming Yang, Shankai Yin, Hao Wu, Zhengmin Wang, Huawei Li, Jay T. Rubinstein, Lawrence R. Lustig, Renjie Chai, Zheng‐Yi Chen, Yilai Shu · 发表于：Med · 年份：2025 · DOI：10.1016/j.medj.2025.100886 · 被引用次数：4 · 研究领域：Hearing, Cochlea, Tinnitus, Genetics、Genomics and Rare Diseases、Hearing Loss and Rehabilitation

Background Hereditary hearing loss is one of the most common disabling disorders in children and lacks effective pharmacological treatments. Recent breakthroughs in OTOF gene therapy clinical trials necessitate standardized frameworks to guide emerging therapies. This study aims to establish the first international consensus on the clinical application of gene therapy for hereditary hearing loss. Methods A modified Delphi process was conducted from March 2024 to March 2025, involving 46 multidisciplinary experts from several countries across otology, genetics, audiology, gene therapy, and hearing rehabilitation. After a systematic literature review, as well as integration of research and clinical expertise and experience, three iterative voting rounds (two anonymous surveys and one online consensus meeting) were performed. Statements required ≥75% agreement for inclusion. Findings From 9,093 publications, 69 were used to draft and support the consensus statements. A total of 30 statements relevant to six domains achieved consensus on gene therapy for hereditary hearing loss, including ethical review (1 statement), patient selection criteria (12 statements), diagnosis and preoperative evaluation (9 statements), gene therapy drug delivery (4 statements), follow-up (3 statements), and post-treatment auditory and speech rehabilitation (1 statement). Conclusions This consensus provides the first globally endorsed framework for gene therapy in hereditary hearing loss. It standardiz...