Pellegrino Raffaele, Imperio Giuseppe, De Costanzo Ilaria, Izzo Michele, Landa Fabio, Tambaro Assunta, Gravina Antonietta Gerarda, Federico Alessandro
Hepatogastroenterology Division, Department of Precision Medicine, University of Campania Luigi Vanvitelli, Via L. de Crecchio, 80138 Naples, Italy.
Pharmaceuticals (Basel). 2025 Feb 23;18(3):308. doi: 10.3390/ph18030308.
Ulcerative colitis (UC) is an inflammatory bowel disease in which one-quarter of patients are at risk of developing a severe form of the disease known as acute severe UC (ASUC). This condition exposes patients to serious complications, including toxic megacolon, surgical intervention, and even death. The current therapeutic strategy relies on time-dependent, multi-step algorithms that integrate systemic corticosteroids, calcineurin inhibitors, and biologic agents (specifically infliximab) as medical therapy aimed at avoiding colectomy. Despite this approach, a significant proportion of patients fail to respond to either corticosteroids or infliximab and may require alternative therapeutic options if there is no urgent surgical necessity. These alternatives include other biologics or emerging small molecules, although the evidence supporting these treatments remains extremely low, even considering their well-documented and promising efficacy and safety in moderate-to-severe UC. Conversely, it is necessary to investigate whether infliximab can be effectively replaced or surpassed by other approved biological agents and small molecules as first-line therapy after steroid resistance. This review aims to summarise the available evidence on small molecules, specifically Janus kinase inhibitors and sphingosine-1-phosphate receptor modulators.
溃疡性结肠炎(UC)是一种炎症性肠病,其中四分之一的患者有发展为一种严重形式疾病的风险,即急性重症溃疡性结肠炎(ASUC)。这种情况会使患者面临严重并发症,包括中毒性巨结肠、手术干预,甚至死亡。当前的治疗策略依赖于基于时间的多步骤算法,该算法将全身性皮质类固醇、钙调神经磷酸酶抑制剂和生物制剂(特别是英夫利昔单抗)整合作为旨在避免结肠切除术的药物治疗。尽管采用了这种方法,但相当一部分患者对皮质类固醇或英夫利昔单抗均无反应,并且如果没有紧急手术必要性,可能需要其他治疗选择。这些替代方案包括其他生物制剂或新兴小分子,尽管支持这些治疗的证据仍然极低,即使考虑到它们在中度至重度UC中已充分记录且前景良好的疗效和安全性。相反,有必要研究在类固醇抵抗后,英夫利昔单抗是否可以被其他已批准的生物制剂和小分子有效地替代或超越作为一线治疗。本综述旨在总结关于小分子,特别是 Janus 激酶抑制剂和鞘氨醇-1-磷酸受体调节剂的现有证据。