关键词: Autoimmune bullous diseases Connective tissue diseases Tofacitinib Vasculitis

Mesh : Humans Janus Kinases STAT Transcription Factors Signal Transduction Autoimmune Diseases Skin Diseases / drug therapy Biological Products / therapeutic use Multicenter Studies as Topic Piperidines Pyrimidines

来  源:   DOI:10.1007/s00228-024-03621-9

Abstract:
OBJECTIVE: Autoimmune bullous diseases, connective tissue diseases, and vasculitis represent a group of severe rare skin diseases. While glucocorticoids and immunosuppressive agents serve as standard treatments for these diseases, their efficacy is limited due to adverse side effects, indicating the need for alternative approaches. Biologics have been used in the management of some rare skin diseases. However, the use of biologics is associated with concerns, such as infection risk and high costs, prompting the quest for efficacious and cost-effective alternatives. This study discusses the safety issues associated with tofacitinib and its potential in treating rare skin diseases.
METHODS: This narrative review focuses on the pharmacodynamic properties of tofacitinib and its impact on the JAK/STAT pathway. In addition, we present a comprehensive discussion of the effects and mechanism of action of tofacitinib for each severe rare skin disease.
RESULTS: This role of tofacitinib in treating severe rare skin diseases has been discussed, shedding light on its promising prospects as a treatment modality. Few reports of serious adverse events are available in patients treated with tofacitinib.
CONCLUSIONS: We explored the mechanism of action, efficacy, and safety considerations of tofacitinib and found that it can be used as a treatment option for rare skin diseases. However, multicenter clinical studies are needed to confirm the efficacy and safety of JAK inhibitors.
摘要:
目的:自身免疫性大疱性疾病,结缔组织疾病,血管炎代表一组严重的罕见皮肤病。虽然糖皮质激素和免疫抑制剂是这些疾病的标准治疗方法,由于不良副作用,它们的功效有限,表明需要替代方法。生物制剂已用于管理一些罕见的皮肤病。然而,生物制剂的使用与担忧有关,如感染风险和高成本,促使人们寻求有效和具有成本效益的替代品。这项研究讨论了与托法替尼相关的安全性问题及其在治疗罕见皮肤病中的潜力。
方法:这篇叙述性综述集中于托法替尼的药效学特性及其对JAK/STAT通路的影响。此外,我们全面讨论了托法替尼对每种严重罕见皮肤病的疗效和作用机制。
结果:已经讨论了托法替尼在治疗严重罕见皮肤病中的作用,揭示了其作为一种治疗方式的有希望的前景。在使用托法替尼治疗的患者中,很少有严重不良事件的报告。
结论:我们探索了作用机制,功效,和托法替尼的安全性考虑,发现它可以用作罕见皮肤病的治疗选择。然而,需要多中心临床研究来证实JAK抑制剂的有效性和安全性.
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