Unit of Immunology, Rheumatology, Allergy and Rare Diseases, IRCCS San Raffaele Hospital, Milan, Italy.
Vita-Salute San Raffaele University, Milan, Italy.
Rheumatology (Oxford). 2020 Jan 1;59(1):171-175. doi: 10.1093/rheumatology/kez267.
To evaluate the efficacy and safety of apremilast in treating oral ulcers (OUs), the cardinal and high-disabling feature of Behçet's disease (BD).
Twelve consecutive patients affected by BD with recurrent/relapsing OUs resistant and/or intolerant to conventional therapy were enrolled and prospectively followed. The primary endpoint was the number of OUs at week 12. Secondary endpoints were modification from baseline to week 12 in Behçet's Syndrome Activity Score (BSAS), Behçet's Disease Current Activity Form (BDCAF) score, Behçet's Disease Quality of Life (BDQOL) scale and pain of OUs, as measured by a visual analogue scale (VAS). All adverse events (AEs) were recorded during follow-up. Non-parametric tests (Wilcoxon rank test) were used and a P-value <0.05 was considered statistically significant.
After 12 weeks of apremilast, there was a significant reduction in the number of OUs [0.58 (s.d. 0.67) vs 3.33 (s.d. 1.45) at baseline, P = 0.02] that was paralleled by improvement in disease activity: BSAS was 16.8 (s.d. 9.1) [from 45.9 (s.d. 19.6) at baseline] (P = 0.02), BDCAF score was 0.72 (s.d. 0.65) [vs 2.45 (s.d. 1.0) at baseline] (P = 0.04) and the VAS score for pain decreased to 23.3 (s.d. 13.7) [vs 67.9 (s.d. 17.2) at baseline] (P = 0.02). Consistently, an improvement of BDQOL was assessed (P = 0.02). Clinical improvement led to complete steroid discontinuation in six patients and a tapering of the prednisone dose in two patients (P = 0.016). Colchicine was discontinued in six of nine patients (P = 0.031). AEs related to apremilast occurred in four patients (mainly due to gastrointestinal AEs), leading to drug discontinuation in all of them.
Our preliminary real-world data support the use of apremilast as an effective therapeutic strategy against BD-related recurrent OUs resistant or intolerant to first-line therapy.
评估阿普斯特治疗复发性/难治性口腔溃疡（OU）的疗效和安全性，OU 是贝赫切特病（BD）的主要特征和高度致残特征。
连续纳入 12 例复发性/难治性 OU 且对常规治疗不耐受/抵抗的 BD 患者，并进行前瞻性随访。主要终点为第 12 周 OU 数。次要终点为 Behçet 综合征活动评分（BSAS）、Behçet 疾病活动量表（BDCAF）评分、BD 生活质量量表（BDQOL）和 OU 疼痛从基线到第 12 周的变化，OU 疼痛采用视觉模拟量表（VAS）评估。所有不良事件（AE）在随访期间均记录。采用非参数检验（Wilcoxon 秩检验），P 值<0.05 认为具有统计学意义。
阿普斯特治疗 12 周后，OU 数显著减少[0.58（s.d. 0.67）vs 3.33（s.d. 1.45），P=0.02]，疾病活动度改善：BSAS 为 16.8（s.d. 9.1）[从基线的 45.9（s.d. 19.6）]（P=0.02），BDCAF 评分 0.72（s.d. 0.65）[从基线的 2.45（s.d. 1.0）]（P=0.04），OU 疼痛 VAS 评分降至 23.3（s.d. 13.7）[从基线的 67.9（s.d. 17.2）]（P=0.02）。BDQOL 也得到了改善（P=0.02）。6 例患者完全停用激素，2 例患者逐渐减少泼尼松剂量（P=0.016），临床改善导致临床改善。9 例中的 6 例患者停用秋水仙碱（P=0.031）。阿普斯特相关 AE 发生在 4 例患者中（主要为胃肠道 AE），导致所有患者停药。
我们的初步真实世界数据支持阿普斯特作为一种有效的治疗策略，用于治疗对一线治疗不耐受/抵抗的 BD 相关复发性 OU。
