12145 Background: Radiation-induced oral mucositis (RIOM) is common during head and neck radiotherapy ( > 90% incidence), and 40–70% of patients develop severe oral mucositis (SOM; WHO grade 3–4), which markedly impairs quality of life and may lead to radiotherapy interruptions. Given the limited effective therapies and the lack of evidence-based standard of care, we assessed the efficacy and safety of MeiLianFuXin Liquid ( Periplaneta americana extract) for reducing SOM incidence. Methods: This multicenter, randomized, double-blind, placebo-controlled phase II trial was conducted across 7 hospitals in China from June 12, 2024, through October 31, 2025. Eligible patients (18–80 years) had non-metastatic head and neck cancer planned for radiotherapy (planned total dose ≥60 Gy), with cisplatin as the only allowed concurrent agent. Following investigator-confirmed WHO grade 1 oral mucositis, patients were randomized (1:1:1) via a centralized interactive web response system (IWRS) using a dynamic minimization algorithm to receive low-dose MeiLianFuXin Liquid, high-dose MeiLianFuXin Liquid, or a matched placebo. The study drug was administered three times daily until 14 days post-radiotherapy. The primary endpoint was SOM incidence. Adjusted proportion differences were estimated using logistic regression. Adverse events were graded according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0. Results: In the full analysis set (FAS), 215 patients received treatment (low-dose, n = 72; high-dose, n = 72; placebo, n = 71). The incidence of SOM was 22.2% (95% CI, 13.3 to 33.6) in the low-dose group, 29.2% (95% CI, 19.0 to 41.1) in the high-dose group, and 43.7% (95% CI, 31.9 to 56.0) in the placebo group. Compared with placebo, adjusted proportion differences were −22.5% (95% CI, −37.3 to −7.8; P = .0042) for the low-dose group and −16.4% (95% CI, −32.2 to −0.5; P = .0463) for the high-dose group. Adverse events and serious adverse events were generally similar across groups. Conclusions: To our knowledge, this is the first randomized, double-blind, placebo-controlled trial of a traditional Chinese medicine for RIOM showing that MeiLianFuXin Liquid reduces SOM incidence versus placebo with a favorable safety profile. These findings support MeiLianFuXin Liquid as a promising treatment strategy to mitigate RIOM. Clinical trial information: ChiCTR2400084288.
yang et al. (Wed,) studied this question.
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