利特昔替尼：重度斑秃综述。
完整摘要 · 中文译文
Ritlecitinib（Litfulo®）是一种口服双重抑制剂，可抑制Janus激酶（JAK）3和在肝细胞癌中表达的酪氨酸激酶（TEC）激酶家族。它在全球多个国家获批用于治疗12岁及以上成人和青少年的重型斑秃。在一项针对头皮毛发脱落≥50%的成人和青少年斑秃患者的关键性、随机、双盲、安慰剂对照、2b/3期ALLEGRO试验中，与安慰剂相比，利特昔替尼50 mg每日一次显著增加了第24周时达到头皮毛发脱落≤20%的患者比例。利特昔替尼还改善了患者报告结局，包括患者总体变化印象（Patient's Global Impression of Change）应答。在关键性试验中，关键疗效结局的应答率在48周内持续增加，并在开放标签的3期ALLEGRO-LT试验中维持长达3年。长期治疗还与健康相关生活质量的改善相关。利特昔替尼总体耐受性良好，大多数不良事件为轻度或中度。虽然需要进一步的数据来充分描述长期安全性，但目前可获得的数据表明，利特昔替尼是治疗重型斑秃的一个有价值的新选择，包括对于获批治疗选择有限的青少年患者。 斑秃是一种慢性免疫介导性疾病，可导致头皮、面部（包括眉毛和睫毛）及身体其他部位出现非瘢痕性脱发。它可能严重影响心理健康，许多患者会经历焦虑和抑郁。尽管糖皮质激素通常作为一线治疗，但对于病情更严重或受累范围更广的患者，可用治疗选择一直有限。利特希替尼（Litfulo®）是一种每日一次口服的药物，通过阻断免疫系统中与该疾病相关的特定信号来发挥作用。它已在多个国家获批用于12岁及以上患有严重斑秃的成人和青少年。在一项关键性临床试验中，利特希替尼治疗实现了具有临床意义的头皮毛发再生，以及眉毛和睫毛再生的改善。获益随时间推移而增加，并与生活质量改善相关。利特希替尼在临床试验中总体耐受性良好。尽管仍需更多长期安全性数据，利特希替尼为严重斑秃患者（包括既往获批疗法很少的青少年）提供了一种有价值的新治疗选择。
完整摘要 · 英文原文
Ritlecitinib (Litfulo®) is an oral dual inhibitor of Janus kinase (JAK) 3 and the tyrosine kinase expressed in hepatocellular carcinoma (TEC) family of kinases. It is approved in several countries worldwide for the treatment of severe alopecia areata in adults and adolescents 12 years of age and older. In the pivotal, randomized, double-blind, placebo-controlled, phase 2b/3 ALLEGRO trial in adults and adolescents with alopecia areata with ≥ 50% scalp hair loss, ritlecitinib 50 mg once daily significantly increased the proportion of patients achieving ≤ 20% scalp hair loss at week 24 compared with placebo. Ritlecitinib also improved patient-reported outcomes, including Patient's Global Impression of Change responses. Response rates for key efficacy outcomes in the pivotal trial continued to increase through 48 weeks and were sustained for up to 3 years in the open-label phase 3 ALLEGRO-LT trial. Longer-term treatment was also associated with improvements in health-related quality of life. Ritlecitinib was generally well tolerated, with most adverse events being of mild or moderate severity. While further data are needed to fully characterize long-term safety, currently available data suggest that ritlecitinib is a valuable new option for the treatment of severe alopecia areata, including in adolescents for whom approved treatment options are limited. Alopecia areata is a chronic immune-mediated disorder that causes non-scarring hair loss on the scalp, face (including eyebrows and eyelashes), and other parts of the body. It can substantially affect psychological wellbeing, with many patients experiencing anxiety and depression. Although corticosteroids are commonly used as first-line treatment, options for patients with more severe or extensive disease have been limited. Ritlecitinib (Litfulo®) is an oral drug taken once daily that works by blocking specific signals in the immune system that are involved in this condition. It is approved in several countries for adults and adolescents aged 12 years and older with severe alopecia areata. In a pivotal clinical trial, treatment with ritlecitinib resulted in clinically meaningful hair regrowth on the scalp, as well as improvements in eyebrow and eyelash regrowth. Benefits increased over time and were associated with improved quality of life. Ritlecitinib was generally well tolerated in clinical trials. Although more long-term safety data are still needed, ritlecitinib is a valuable new treatment option for patients with severe alopecia areata, including adolescents who previously had few approved therapies.