Cortés-Hernández Josefina, Ávila Gabriela, Vilardell-Tarrés Miquel, Ordi-Ros Josep
Arthritis Res Ther. 2012 Dec 7;14(6):R265. doi: 10.1186/ar4111.
Cutaneous lupus erythematosus (CLE) is a chronic disease characterized by disfigurement and a relapsing course. Thalidomide has proven its efficacy in refractory cutaneous lupus disease, although it is not exempt from significant side effects and frequent relapses after withdrawal. New thalidomide analogues have been developed but lack clinical experience. The aim of this preliminary phase II study was to evaluate the efficacy and safety of lenalidomide in patients with refractory CLE.
Fifteen patients with refractory cutaneous lupus disease were enrolled in this single-center, open-label, non-comparative pilot trial between January 2009 and December 2010. Oral lenalidomide (5 to 10 mg/day) was administered and tapered according to clinical response. Patients were followed up for a mean of 15 months (range: 7 to 30). Primary efficacy endpoint was the proportion of patients achieving complete response, defined by a Cutaneous Lupus Erythematosus Disease Area and Severity index (CLASI) activity score of 0. Other secondary endpoints included development of side effects, evaluation of cutaneous and systemic flares, and impact on the immunological parameters.
One patient discontinued treatment due to side effects. All remaining patients saw clinical improvement and this was already noticeable after 2 weeks of treatment. Twelve of those patients (86%) achieved complete response but clinical relapse was frequent (75%), usually occurring 2 to 8 weeks after lenalidomide's withdrawal. No influence on systemic disease, immunological parameters or CLASI damage score was observed. Side effects including insomnia, grade 2 neutropenia and gastrointestinal symptoms, were minor (13%). These resolved after withdrawing medication. Neither polyneuropathy nor thrombosis was observed.
Lenalidomide appears to be efficacious and safe in patients with refractory CLE, but clinical relapse is frequent after its withdrawal.
ClinicalTrials.gov: NCT01408199.
皮肤型红斑狼疮（CLE）是一种慢性疾病，其特征为毁容和病情复发。沙利度胺已在难治性皮肤狼疮疾病中证明了其疗效，尽管它并非没有显著副作用，且停药后频繁复发。新型沙利度胺类似物已研发出来，但缺乏临床经验。这项II期初步研究的目的是评估来那度胺对难治性CLE患者的疗效和安全性。
2009年1月至2010年12月期间，15例难治性皮肤狼疮疾病患者参加了这项单中心、开放标签、非对照试验。口服来那度胺（5至10毫克/天），并根据临床反应逐渐减量。患者平均随访15个月（范围：7至30个月）。主要疗效终点是达到完全缓解的患者比例，完全缓解定义为皮肤红斑狼疮疾病面积和严重程度指数（CLASI）活动评分为0。其他次要终点包括副作用的发生、皮肤和全身病情复发的评估以及对免疫参数的影响。
1例患者因副作用停药。其余所有患者均有临床改善，治疗2周后即可明显看出。其中12例患者（86%）达到完全缓解，但临床复发频繁（75%），通常在来那度胺停药后2至8周发生。未观察到对全身疾病、免疫参数或CLASI损伤评分的影响。副作用包括失眠、2级中性粒细胞减少和胃肠道症状，程度较轻（13%）。停药后这些副作用消失。未观察到多发性神经病或血栓形成。
来那度胺对难治性CLE患者似乎有效且安全，但停药后临床复发频繁。
ClinicalTrials.gov：NCT01408199。
