Corporal Michael J. Crescenz VA Medical Center, Philadelphia, PA, USA.
Department of Dermatology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Expert Opin Investig Drugs. 2023 May;32(5):345-353. doi: 10.1080/13543784.2023.2212154. Epub 2023 May 15.
There are no U.S. Food and Drug Administration (FDA) approved therapies for cutaneous lupus erythematosus (CLE). Litifilimab is a monoclonal antibody against BDCA2, a plasmacytoid dendritic cell-specific antigen, currently under investigation for systemic lupus erythematosus (SLE) and CLE. The LILAC study, published in the New England Journal of Medicine, is a phase II randomized controlled trial for CLE which demonstrated superiority of Litifilimab over placebo using a skin directed outcome measure.
This review identifies challenges that have hindered the development of any approved treatments for CLE, recent SLE trials that include skin disease data, and the pharmacological properties of litifilimab. We review the clinical efficacy and safety of litifilimab for both SLE and CLE in the phase I and II clinical trials. This review aims to highlight the need for more CLE-specific clinical trials and examine the potential of litifilimab as the first FDA approved therapy for CLE. (Clinical trial registration: www.clinicaltrials.gov identifier is NCT02847598.).
Litifilimab demonstrated efficacy in a randomized phase II clinical trial as a standalone CLE trial using validated skin-specific outcome measures, making it the first successful clinical trial for a CLE targeted therapy. If approved, litifilimab will be a pivotal change in the landscape of CLE management especially for severe and refractory disease.
目前尚无美国食品和药物管理局 (FDA) 批准的治疗皮肤红斑狼疮 (CLE) 的方法。Litifilimab 是一种针对 BDCA2 的单克隆抗体，BDCA2 是一种浆细胞样树突状细胞特异性抗原，目前正在研究用于系统性红斑狼疮 (SLE) 和 CLE。发表在《新英格兰医学杂志》上的 LILAC 研究是一项针对 CLE 的 II 期随机对照试验，该试验表明 Litifilimab 优于安慰剂，使用皮肤定向结局测量。
本综述确定了阻碍任何批准的 CLE 治疗方法发展的挑战、最近包括皮肤疾病数据的 SLE 试验，以及 litifilimab 的药理学特性。我们回顾了 litifilimab 在 I 期和 II 期临床试验中用于 SLE 和 CLE 的临床疗效和安全性。本综述旨在强调需要更多针对 CLE 的临床试验，并探讨 litifilimab 作为首个获得 FDA 批准的 CLE 治疗方法的潜力。（临床试验注册：www.clinicaltrials.gov 标识符为 NCT02847598。）
Litifilimab 在一项针对 CLE 的随机 II 期临床试验中表现出疗效，该试验使用经过验证的皮肤特异性结局测量作为单独的 CLE 试验，使其成为首个针对 CLE 靶向治疗的成功临床试验。如果获得批准，litifilimab 将是 CLE 管理领域的一个重要变化，特别是对于严重和难治性疾病。
