The Treatment of Primary Biliary Cholangitis: Time for Personalized Medicine.

IF 8.4 2区 医学 Q1 ALLERGY
Xinyi Men, Yansheng Liu, Han Zhao, Bingrui Xie, Changcun Guo, Patrick S C Leung, Suraj Timilsina, M Eric Gershwin, Yulong Shang, Ying Han
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Abstract

Primary Biliary Cholangitis is an autoimmune liver disease distinguished by Anti-mitochondrial Antibodies and chronic non-suppurative lymphocytic microcholangitis. UDCA remains the exclusively recommended initial therapy for PBC. However, 40% of patients experience either incomplete biochemical response or intolerance to UDCA, which represents poorer outcomes and increased mortality. Therefore, early identification of high-risk patients and timely intensive treatments are necessary to delay the progression of PBC and better improve the prognosis. Intensive therapeutic strategies based on more stringent treatment goals and early efficacy assessment criteria are elaborated in this review. To exclude AIH-PBC overlap syndrome, liver biopsy is required for cholestasis patients with negative AMAs or PBC patients who respond inadequately to UDCA, especially those with elevated ALT and IgG. Combined immunosuppressants are considered for patients with moderate-to-severe hepatitis. ALP normalization is considered the improved therapeutic goal for high-risk patients, which has been verified achievable in multiple treatment attempts. The control of pruritus and fatigue constitutes the key therapeutic targets in the symptom management of PBC. Bezafibrate, Seladelpar, and IBAT inhibitors have demonstrated significant therapeutic potential in pruritus. Last but not least, Liver Stiffness Measurement is substantiated as efficient in the fibrotic monitoring of PBC patients. OCA and fibrates are respectively useful for compensated and decompensated fibrotic patients. Moreover, the conventional efficacy assessment procedure, which is the "wait-to-fail" strategy, exhibits suboptimal sensitivity in the timely detection of treatment-responsive patients. Therefore, early prediction and evaluation criteria at baseline and 1-month treatment will help in timely interventions for patients with insufficient efficacy. This improved identification strategy is expected to provide precise and personalized treatment for PBC patients.

原发性胆道胆管炎的治疗:个性化治疗的时机。
原发性胆道胆管炎是一种以抗线粒体抗体和慢性非化脓性淋巴细胞微胆管炎为特征的自身免疫性肝病。UDCA仍然是PBC唯一推荐的初始治疗方法。然而,40%的患者出现不完全生化反应或对UDCA不耐受,这代表较差的结果和死亡率增加。因此,早期发现高危患者,及时强化治疗,延缓PBC的进展,更好地改善预后是必要的。本文阐述了基于更严格的治疗目标和早期疗效评估标准的强化治疗策略。为了排除AIH-PBC重叠综合征,对于AMAs阴性的胆汁淤积患者或对UDCA反应不充分的PBC患者,特别是ALT和IgG升高的患者,需要进行肝活检。联合免疫抑制剂可用于中重度肝炎患者。ALP正常化被认为是高危患者的改进治疗目标,经多次治疗尝试证实是可以实现的。瘙痒和疲劳的控制是PBC症状管理的关键治疗目标。贝扎布雷特、西拉得帕和IBAT抑制剂已显示出治疗瘙痒的显著潜力。最后但并非最不重要的是,肝硬度测量在PBC患者的纤维化监测中是有效的。OCA和贝特类药物分别适用于代偿性和失代偿性纤维化患者。此外,传统的疗效评估程序,即“等待失败”策略,在及时发现治疗反应性患者方面表现出次优的敏感性。因此,在基线和治疗1个月的早期预测和评估标准有助于对疗效不足的患者及时干预。这种改进的识别策略有望为PBC患者提供精确和个性化的治疗。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
CiteScore
22.30
自引率
1.10%
发文量
58
审稿时长
6-12 weeks
期刊介绍: Clinical Reviews in Allergy & Immunology is a scholarly journal that focuses on the advancement of clinical management in allergic and immunologic diseases. The journal publishes both scholarly reviews and experimental papers that address the current state of managing these diseases, placing new data into perspective. Each issue of the journal is dedicated to a specific theme of critical importance to allergists and immunologists, aiming to provide a comprehensive understanding of the subject matter for a wide readership. The journal is particularly helpful in explaining how novel data impacts clinical management, along with advancements such as standardized protocols for allergy skin testing and challenge procedures, as well as improved understanding of cell biology. Ultimately, the journal aims to contribute to the improvement of care and management for patients with immune-mediated diseases.
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