Wang Xuguang, Wu Haijun, Lei Feng, Liu Zhigang, Shen Guanzhu, Hu Xuefeng, Ye Yijing, Zhu Manyi, Huang Huageng, Chen Boyu, Huang Runda, Zhao Chong, Miao Jingjing, Wang Lin
Sun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Guangdong Key Laboratory of Nasopharyngeal Carcinoma Diagnosis and Therapy, Guangzhou, People's Republic of China.
Department of Nasopharyngeal Carcinoma, Sun Yat-sen University Cancer Center, Guangzhou, People's Republic of China.
Nat Commun. 2025 Mar 23;16(1):2848. doi: 10.1038/s41467-025-57884-6.
Radiotherapy-induced oral mucositis (RTOM) is a common side effect of radiotherapy in locoregionally advanced nasopharyngeal carcinoma (LA-NPC) receiving concurrent chemoradiotherapy (CCRT). In this phase 3 trial, we aim to evaluate the efficacy and safety of Ulinastatin (UTI) for the prevention and treatment of RTOM in LA-NPC patients (NCT03387774). The primary endpoint is the incidence of grade ≥3 acute RTOM during radiotherapy. Secondary endpoints include cumulative incidence of RTOM, recovery rate, the onset time and duration of grade ≥3 RTOM, oral pain (severe), safety and survival outcomes. 179 eligible patients are randomly assigned to UTI Group (n = 89) or Control group (n = 90). All UTI group patients complete UTI treatment as planned, and both groups complete scheduled CCRT. The incidence of grade 3 RTOM is significantly lower in UTI group compared with control group (25.8% vs 41.1%, P = 0.030). The trial meet its prespecified primary endpoint. No Ulinastatin related adverse events are observed during treatment. The 3-year overall survival (OS), locoregional relapse-free survival (LRRFS), distant metastasis-free survival (DMFS) and progression-free survival (PFS) in UTI group and control group are similar between two groups. In this work, Ulinastatin can effectively reduce the severity of RTOM and oral pain without increasing toxicity and compromising survivals.
放射性口腔黏膜炎（RTOM）是局部晚期鼻咽癌（LA-NPC）同步放化疗（CCRT）过程中放疗常见的副作用。在这项3期试验中，我们旨在评估乌司他丁（UTI）预防和治疗LA-NPC患者RTOM的疗效和安全性（NCT03387774）。主要终点是放疗期间≥3级急性RTOM的发生率。次要终点包括RTOM的累积发生率、恢复率、≥3级RTOM的发病时间和持续时间、口腔疼痛（重度）、安全性和生存结局。179例符合条件的患者被随机分配到UTI组（n = 89）或对照组（n = 90）。所有UTI组患者均按计划完成UTI治疗，两组均完成预定的CCRT。UTI组3级RTOM的发生率显著低于对照组（25.8%对41.1%，P = 0.030）。该试验达到了预先设定的主要终点。治疗期间未观察到与乌司他丁相关的不良事件。UTI组和对照组的3年总生存（OS）、局部区域无复发生存（LRRFS）、远处转移无复发生存（DMFS）和无进展生存（PFS）在两组之间相似。在本研究中，乌司他丁可有效降低RTOM的严重程度和口腔疼痛，且不增加毒性和影响生存。
