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儿茶酚-O-甲基转移酶抑制剂在帕金森病治疗中的应用

COMT Inhibitors in the Management of Parkinson's Disease.

作者信息

Fabbri Margherita, Ferreira Joaquim J, Rascol Olivier

机构信息

Department of Neurosciences, Clinical Investigation Center CIC 1436, Parkinson Toulouse Expert Centre, NS-Park/FCRIN Network and NeuroToul COEN Center, Toulouse University Hospital, INSERM, University of Toulouse 3, Toulouse, France.

Laboratory of Clinical Pharmacology and Therapeutics, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal.

出版信息

CNS Drugs. 2022 Mar;36(3):261-282. doi: 10.1007/s40263-021-00888-9. Epub 2022 Feb 25.

Abstract

Levodopa treatment remains the gold standard for Parkinson's disease, but shortcomings related to the pharmacological profile, notably, oral administration and the consequent occurrence of motor complications, have led to the development of several add-on levodopa treatments or to research to improve the method of delivery. Motor fluctuations, and to a lesser extent non-motor fluctuations, concern half of the patients with Parkinson's disease after 5 years of disease and patients identified them as one of their most bothersome symptoms. Catechol-O-methyl transferase inhibitors (COMT-Is) are one of the recommended first-line levodopa add-on therapies for the amelioration of end-of dose motor fluctuations in patient with advanced Parkinson's disease. Currently, two peripheral COMT-Is are considered as first-line choices - entacapone (ENT), which was approved by the US Food and Drug Administration in 1999 and the European Committee in 1998; and opicapone (OPC), which was approved by the European Committee in 2016. A second-line COMT-I that requires regular hepatic monitoring, tolcapone (TOL), was approved by the Food and Drug Administration in 1998 and the European Committee in 1997. Of note, OPC also received Food and Drug Administration approval in 2021, but it is still only marketed in a few countries, including Germany, UK, Spain, Portugal, Italy, Japan, and USA, while ENT and TOL have a wider market. Our narrative review summarizes the pharmacokinetic/pharmacodynamic properties, clinical efficacy in terms of motor fluctuations, motor/non-motor symptoms, quality of life, and safety data of these three COMT-Is, as evidenced by randomized clinical trials, as well as by real-life observational studies. Overall, a phase III non-inferiority trial showed a similar effect between ENT and OPC on off-time (-60.8 min/day and -40.3 min/day, vs placebo, respectively), with a possible additional off-time reduction of 39 min/day, obtained when there is a switch from ENT to OPC. Concomitantly, TOL can reduce off-time by an average of 98 min/day. A significant though discrete concomitant reduction on the Unified Parkinson's Disease Rating Scale motor section (2-3 points) is obtained with all three drugs vs placebo. Data on quality of life are fewer and more heterogeneous, with positive results obtained especially in open-label studies. Effects on non-motor symptoms were investigated as secondary outcome only in a few studies, frequently by means of non-specific scales and a benefit was observed in open-label studies. Dopaminergic adverse effects were the most frequent, dyskinesia being the most common for the three drugs eventually requiring levodopa dose reductions. No urine discoloration and a very low incidence of diarrhea were found with OPC compared with ENT and TOL. Regular hepatic monitoring is needed only for TOL. A combination of COMT-Is with new formulations of levodopa, including the subcutaneous, intrajejunal, or new extended-release formulation, merits further exploration to improve the management of both mild and severe motor fluctuations.

摘要

左旋多巴治疗仍然是帕金森病的金标准,但与药理学特性相关的缺点,尤其是口服给药以及随之出现的运动并发症,促使人们研发了几种左旋多巴附加治疗方法,或开展研究以改进给药方式。运动波动,以及程度较轻的非运动波动,在病程5年的帕金森病患者中困扰着半数患者,患者将其视为最令人烦恼的症状之一。儿茶酚-O-甲基转移酶抑制剂(COMT-Is)是推荐用于改善晚期帕金森病患者剂末运动波动的一线左旋多巴附加治疗药物之一。目前,两种外周COMT-Is被视为一线选择——恩他卡朋(ENT),于1999年获美国食品药品监督管理局批准,1998年获欧洲委员会批准;奥匹卡朋(OPC),于2016年获欧洲委员会批准。一种需要定期进行肝脏监测的二线COMT-I托卡朋(TOL),于1998年获美国食品药品监督管理局批准,1997年获欧洲委员会批准。值得注意的是,OPC在2021年也获得了美国食品药品监督管理局的批准,但仍仅在包括德国、英国、西班牙、葡萄牙、意大利、日本和美国在内的少数国家上市,而ENT和TOL的市场更广泛。我们的叙述性综述总结了这三种COMT-Is的药代动力学/药效学特性、在运动波动、运动/非运动症状以及生活质量方面的临床疗效和安全性数据,这些数据来自随机临床试验以及真实世界观察性研究。总体而言,一项III期非劣效性试验表明,ENT和OPC在关期时间方面对安慰剂的效果相似(分别为-60.8分钟/天和-40.3分钟/天),从ENT转换为OPC时关期时间可能额外减少39分钟/天。同时,TOL可使关期时间平均减少98分钟/天。与安慰剂相比,所有三种药物在统一帕金森病评定量表运动部分均有显著但轻微的伴随降低(2 - 3分)。关于生活质量的数据较少且更具异质性,尤其是在开放标签研究中获得了积极结果。仅在少数研究中将对非运动症状的影响作为次要结局进行了调查,通常采用非特异性量表,在开放标签研究中观察到了益处。多巴胺能不良反应最为常见,异动症是这三种药物最终都需要减少左旋多巴剂量的最常见不良反应。与ENT和TOL相比,OPC未发现尿液变色且腹泻发生率极低。仅TOL需要定期进行肝脏监测。COMT-Is与左旋多巴的新剂型(包括皮下、空肠内或新的缓释剂型)联合使用,值得进一步探索以改善对轻、重度运动波动的管理。

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