Department of Gastroenterology, Alfred Health, 55 Commercial Road, Melbourne, 3004, VIC, Australia.
Monash University, Melbourne, Australia.
Dig Dis Sci. 2021 Oct;66(10):3250-3262. doi: 10.1007/s10620-020-06662-z. Epub 2020 Oct 19.
In recent years, with the increasing availability of biologic therapies and due to safety concerns, the role of thiopurines in the management of inflammatory bowel disease has been questioned. While acknowledging that the benefit/risk ratio of biologic therapies is very high, they are expensive and are not required by a majority of patients. Therefore, thiopurines do retain an important role as steroid-sparing and maintenance agents when used as monotherapy, and in combination therapy with biologics due to their clinical and pharmacokinetic optimization of anti-tumor necrosis factor agents in particular. Safety concerns with thiopurines are real but also relatively rare, and with careful pre-treatment screening and ongoing monitoring thiopurine benefits outweigh risks in the majority of appropriately selected patients. Measurement of newer pharmacogenomic markers such as nudix hydrolase 15 (NUDT15), when combined with knowledge of existing known mutations (e.g., thiopurine S-methyltransferase-TPMT), will hopefully minimize the risk of potentially life-threatening leukopenia by allowing for pre-treatment dosing stratification. Further optimization of thiopurine dosing via measurement of thiopurine metabolites should be performed routinely and is superior to weight-based dosing. The association of thiopurines with malignancies including lymphoproliferative disorders needs to be recognized in all patients and individualized in each patient. The decrease in lymphoma risk after thiopurine cessation provides an incentive for thiopurine de-escalation in appropriate patients after a period of prolonged deep remission. This review will summarize the current role of thiopurines in inflammatory bowel disease management and provide recommendations for commencing and monitoring therapy, and when to consider de-escalation.
近年来,随着生物治疗药物的可获得性增加,以及出于安全性方面的考虑,硫嘌呤类药物在炎症性肠病治疗中的作用受到了质疑。虽然生物治疗药物的获益/风险比非常高,但它们价格昂贵,且并非大多数患者所需。因此,硫嘌呤类药物在作为单药治疗以及与生物制剂联合治疗时仍具有重要作用,尤其是在优化抗肿瘤坏死因子药物的临床和药代动力学方面。硫嘌呤类药物的安全性问题确实存在,但也相对较少见,通过仔细的治疗前筛查和持续监测,在大多数经适当选择的患者中,硫嘌呤类药物的获益大于风险。测量新的药物基因组标记物,如核苷酸二磷酸水解酶 15(NUDT15),结合现有的已知突变(如硫嘌呤 S-甲基转移酶-TPMT)的知识,有望通过治疗前剂量分层来最大限度地降低潜在威胁生命的白细胞减少症的风险。通过测量硫嘌呤代谢物来进一步优化硫嘌呤的剂量应常规进行,且优于基于体重的剂量方案。在所有患者中都需要认识到硫嘌呤类药物与恶性肿瘤(包括淋巴增生性疾病)之间的关联,并在每位患者中进行个体化评估。硫嘌呤类药物停药后淋巴瘤风险降低,为在适当患者中实现延长缓解期后的硫嘌呤类药物降级提供了动力。本文将总结硫嘌呤类药物在炎症性肠病治疗中的当前作用,并为起始和监测治疗以及何时考虑降级提供建议。