Yi Robin C, Moran Shannon K, Gantz Hannah Y, Strowd Lindsay C, Feldman Steven R
Center for Dermatology Research, Department of Dermatology, Wake Forest University School of Medicine, Winston-Salem, NC 27157, USA.
Department of Pathology, Wake Forest University School of Medicine, Winston-Salem, NC 27157, USA.
Children (Basel). 2024 Jul 25;11(8):892. doi: 10.3390/children11080892.
The management of pediatric dermatological conditions such as alopecia areata (AA), psoriasis, atopic dermatitis (AD), and hidradenitis suppurativa (HS) has significantly evolved with the introduction of biologics and small molecule targeted therapies. The advancement in understanding the immunopathogenesis of these chronic skin conditions has led to the development and approval of novel biologics and small molecule therapies. Initially approved by the United States Food and Drug Administration (FDA) for adults, most of these therapies are now being evaluated in clinical trials for safety and efficacy in adolescents and children, expanding new treatment options for pediatric patients. The role of the FDA in drug approval is multifaceted from drug inception, ensuring that research, data, and evidence show that the proposed drug is effective and safe for the intended use.
The goal of this review article is to provide an overview of the recently FDA-approved and potential biologic and oral small molecule therapies in clinical trials for AA, psoriasis, AD, and HS in pediatric patients.
The search for this review included keywords in ClinicalTrials.gov, PubMed, and Google Scholar for the latest research and clinical trials relevant to these conditions and treatments without the PRISMA methodology.
For pediatric AA, ritlecitinib is FDA-approved, while baricitinib and updacitinib are in phase 3 clinical trials for pediatric approval. The FDA-approved drugs for pediatric psoriasis include secukinumab, ustekinumab, ixekizumab, etanercept, and apremilast. Other phase 3 clinical trials for pediatric psoriasis include risankizumab, guselkumab, tildrakizumab, brodalumab, and deucravacitinib. For pediatric AD, the FDA-approved drugs are dupilumab, tralokinumab, abrocitinib, and upadacitinib, with many other drugs in phase 3 trials. Adalimumab is an FDA-approved biologic for pediatric HS, with various clinical trials ongoing for adults. The approved biologics and small molecule therapies had higher efficacy and improved safety profiles compared to traditional medications.
With numerous ongoing trials, the success of these clinical trials could lead to their inclusion in treatment guidelines for these chronic skin conditions. Biologics and small molecule therapies offer new avenues for effective disease management, enabling personalized therapeutic interventions and improving pediatric health outcomes.
随着生物制剂和小分子靶向疗法的引入，斑秃（AA）、银屑病、特应性皮炎（AD）和化脓性汗腺炎（HS）等儿童皮肤病的治疗有了显著进展。对这些慢性皮肤病免疫发病机制认识的进步推动了新型生物制剂和小分子疗法的研发与获批。这些疗法最初由美国食品药品监督管理局（FDA）批准用于成人，目前大多正在进行青少年和儿童安全性及有效性的临床试验评估，为儿科患者拓展了新的治疗选择。FDA在药物审批中的作用是多方面的，从药物研发开始，确保研究、数据和证据表明所提议的药物对于预期用途是有效且安全的。
这篇综述文章的目的是概述近期FDA批准的以及在儿科患者AA、银屑病、AD和HS临床试验中的潜在生物制剂和口服小分子疗法。
本综述的检索在ClinicalTrials.gov、PubMed和谷歌学术中使用关键词，以查找与这些病症和治疗相关的最新研究及临床试验，未采用PRISMA方法。
对于儿童AA，利特昔替尼已获FDA批准，而巴瑞替尼和乌帕替尼正处于儿科批准的3期临床试验阶段。FDA批准的儿童银屑病药物包括司库奇尤单抗、乌司奴单抗、依奇珠单抗、依那西普和阿普米司特。儿童银屑病的其他3期临床试验包括瑞莎珠单抗、古塞库单抗、替拉珠单抗、布罗达单抗和氘可来昔替尼。对于儿童AD，FDA批准的药物是度普利尤单抗、曲罗芦单抗、阿布昔替尼和乌帕替尼，还有许多其他药物处于3期试验阶段。阿达木单抗是FDA批准用于儿童HS的生物制剂，针对成人的各种临床试验正在进行中。与传统药物相比，获批的生物制剂和小分子疗法具有更高的疗效和更好的安全性。
随着众多临床试验的进行，这些临床试验的成功可能会使其纳入这些慢性皮肤病的治疗指南。生物制剂和小分子疗法为有效管理疾病提供了新途径，能够实现个性化治疗干预并改善儿科健康结局。
