富马酸酯治疗皮肤型红斑狼疮（CLE）：一项前瞻性、开放标签的II期试点研究。
Fumaric acid ester treatment in cutaneous lupus erythematosus (CLE): a prospective, open-label, phase II pilot study.
作者信息
Kuhn A, Landmann A, Patsinakidis N, Ruland V, Nozinic S, Perusquía Ortiz A M, Sauerland C, Luger T, Tsianakas A, Bonsmann G
机构信息
Interdisciplinary Center for Clinical Trials (IZKS), University Medical Center Mainz, Mainz, Germany Division of Immunogenetics, Tumor Immunology Program, German Cancer Research Center, Heidelberg, Germany
Division of Immunogenetics, Tumor Immunology Program, German Cancer Research Center, Heidelberg, Germany.
出版信息
Lupus. 2016 Oct;25(12):1357-64. doi: 10.1177/0961203316644335. Epub 2016 May 3.
OBJECTIVE
The aim of the study was to assess the efficacy and safety of fumaric acid esters (FAEs) in patients with cutaneous lupus erythematosus (CLE).
METHODS
In this 24-week, prospective, open-label, phase II pilot study, 11 patients with CLE, refractory to topical corticosteroids, were included. The primary endpoint of the study was the evaluation of the efficacy of FAEs after 24 weeks of treatment as assessed by the Revised Cutaneous Lupus Disease Area and Severity Index (RCLASI).
RESULTS
Compared to baseline, significant improvement in the mean total RCLASI activity score and the mean RCLASI activity score for skin lesions was observed in week 12 (p = 0.002, p = 0.002, respectively) and in week 24 (p = 0.009, p = 0.009, respectively). Most common adverse events included abdominal cramps and headache.
CONCLUSIONS
FAEs could be an alternative and safe treatment in patients with therapy-refractory CLE; however, randomized controlled trials are warranted to evaluate the efficacy and safety of FAEs in this disease.
目的
本研究旨在评估富马酸酯（FAEs）治疗皮肤型红斑狼疮（CLE）患者的疗效和安全性。
方法
在这项为期24周的前瞻性、开放标签的II期试验研究中，纳入了11例对局部皮质类固醇治疗无效的CLE患者。研究的主要终点是通过修订的皮肤狼疮疾病面积和严重程度指数（RCLASI）评估治疗24周后FAEs的疗效。
结果
与基线相比，在第12周（分别为p = 0.002，p = 0.002）和第24周（分别为p = 0.009，p = 0.009）观察到平均总RCLASI活动评分和皮肤病变的平均RCLASI活动评分有显著改善。最常见的不良事件包括腹部绞痛和头痛。
结论
对于治疗难治性CLE患者，FAEs可能是一种替代且安全的治疗方法；然而，需要进行随机对照试验来评估FAEs在该疾病中的疗效和安全性。
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