Geng Bob, Craig Timothy J
Division of Rheumatology, Allergy, and Immunology, Department of Medicine, University of California San Diego, La Jolla, California.
Department of Medicine, Pediatrics, and Biomedical Sciences, Penn State University, Hershey Medical Center, Hershey, Pennsylvania.
Ann Allergy Asthma Immunol. 2022 Mar;128(3):263-268. doi: 10.1016/j.anai.2021.10.015. Epub 2021 Oct 18.
To review recent trends in the development of targeted small molecule drugs (SMDs) for the treatment of immunologically driven disorders, including atopic dermatitis, rheumatoid arthritis, and hereditary angioedema.
Data sources included peer-reviewed published literature from the PubMed database, published abstracts from scientific and medical meetings, and medication information from the Drugs@FDA database.
Articles with primary or retrospective trial results, articles with patient or physician survey results, articles providing expert perspectives, and commentary on chronic immunologic disorders, Food and Drug Administration package inserts, and abstracts from scientific meetings were selected.
Targeted biological therapies have greatly improved response rates and symptom relief for patients with long-term immunologically driven disorders over the past 2 decades. However, recent advances in the understanding of molecular pathways involved in the pathogenesis of these disorders have led to the development of novel targeted SMDs, such as tofacitinib and berotralstat, that can be delivered orally or topically. Few head-to-head studies that compare the safety and efficacy of biologics to SMDs in immunologically driven disorders exist, although some studies suggest that oral and topical modes of administration are preferred by patients and may improve patient quality of life over time.
Scientific advances have led to an increase in the development of targeted SMDs for the treatment of chronic immunologic disorders, which may revolutionize the management of these diseases. Head-to-head studies and real-world evidence are needed to fully compare treatment attributes between biologics and SMDs, including safety, efficacy, adherence, impact on quality of life, and cost-effectiveness.
回顾用于治疗免疫介导性疾病（包括特应性皮炎、类风湿性关节炎和遗传性血管性水肿）的靶向小分子药物（SMD）的近期发展趋势。
数据来源包括来自PubMed数据库的同行评审发表文献、科学和医学会议的发表摘要以及Drugs@FDA数据库的药物信息。
选择具有主要或回顾性试验结果的文章、具有患者或医生调查结果的文章、提供专家观点的文章以及关于慢性免疫疾病的评论、美国食品药品监督管理局包装说明书和科学会议摘要。
在过去20年中，靶向生物疗法极大地提高了长期免疫介导性疾病患者的缓解率和症状缓解情况。然而，最近对这些疾病发病机制中涉及的分子途径的认识进展导致了新型靶向SMD的开发，如托法替布和贝罗司他，可以口服或局部给药。虽然一些研究表明口服和局部给药方式更受患者青睐，并且随着时间推移可能改善患者生活质量，但在免疫介导性疾病中比较生物制剂与SMD安全性和有效性的直接对比研究很少。
科学进展导致用于治疗慢性免疫疾病的靶向SMD开发增加，这可能彻底改变这些疾病的管理方式。需要直接对比研究和真实世界证据来全面比较生物制剂和SMD之间的治疗属性，包括安全性、有效性、依从性、对生活质量的影响和成本效益。
