Efficacy and safety of mufemilast in patients with Behçet's syndrome: a phase 2 randomised clinical trial.

Methods

Patients diagnosed with BS according to the International Diagnostic (Classification) Criteria for Behçet's Disease 2013 and with active oral ulcers were eligible.

根据2013年贝赫切特病国际诊断（分类）标准确诊的BS患者，且有活动性口腔溃疡者，符合入选条件。

Patients were randomly assigned to mufemilast (45 mg or 60 mg) or placebo, administered orally twice daily for 12 weeks.

患者被随机分配接受45毫克或60毫克的mufemilast或安慰剂，每天两次口服，持续12周。

The primary endpoint was the area under the curve (AUC) for the number of oral ulcers from baseline to week 12.

主要终点是基线至第12周口腔溃疡数量的曲线下面积（AUC）。

Safety was measured in all patients who received at least 1 dose of the study drug (ClinicalTrials: NCT04609397).

安全性评估包括所有至少接受一次研究药物的患者（ClinicalTrials: NCT04609397）。

Results

Ninety patients were randomly assigned to the mufemilast 45 mg, 60 mg, or placebo groups (29, 31, and 30, respectively).

90名患者被随机分配到mufemilast 45 mg、60 mg或安慰剂组（分别为29、31和30人）。

The least-squares mean difference in AUC0-12 for oral ulcers between the 45 mg and 60 mg groups and the placebo was -104.8 (95% CI, -156.3 to -53.2; P < .001) and -142.5 (95% CI, -192.4 to -92.7; P < .001), respectively.

45 mg和60 mg组与安慰剂组在口腔溃疡AUC0-12的最小二乘均值差异分别为-104.8（95%置信区间，-156.3至-53.2；P<.001）和-142.5（95%置信区间，-192.4至-92.7；P<.001）。

The visual analogue pain scores and time to ulcer-free remission were significantly improved with mufemilast (P < .001).

mufemilast在视觉模拟疼痛评分和溃疡愈合无复发生存时间上有显著改善（P<.001）。

PDE4-related side effects were transient and resolved spontaneously, and discontinuation rates were low (7% for 45 mg, 7% for 60 mg, and 3% for placebo).

PDE4相关的副作用是短暂的，并且能够自行缓解，停药率较低（45毫克组为7%，60毫克组为7%，安慰剂组为3%）。

No serious adverse events were reported related to the drug.

未报告与药物相关的严重不良事件。

Conclusions

Among patients with oral ulcers associated with BS, mufemilast significantly reduced the number of oral ulcers, improved pain scores, and induced significantly more ulcer-free remissions compared with placebo, with an acceptable safety profile.

在伴有贝赫切特综合征（BS）的口腔溃疡患者中，mufemilast显著减少了口腔溃疡的数量，改善了疼痛评分，并与安慰剂相比，诱导了显著更多的无溃疡缓解期，且安全性可接受。