评估和管理原发性硬化性胆管炎患者的症状负担和生活质量

IF 0.8 4区 医学 Q4 PHARMACOLOGY & PHARMACY Expert Opinion on Orphan Drugs Pub Date : 2021-02-01 DOI:10.1080/21678707.2021.1898370
Josiah D. McCain, D. Chascsa, K. Lindor
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引用次数: 0

摘要

摘要简介:原发性硬化性胆管炎(PSC)是一种进展性胆管疾病,目前尚无治愈方法。由于没有任何治疗方法被证明可以延缓这种进展,目前对这些患者的重点是症状管理。同样,筛查和预防这些患者风险增加的疾病是高收益护理的有效组成部分。涵盖领域:使用美国国家医学图书馆(NLM)的国家生物技术信息中心(NCBI)数据库进行文献检索。本文综述了PSC患者的症状评估、药物治疗以及相关疾病和筛查策略方面的文献。此外,对以往的治疗方法和未来的治疗方向进行了探讨。专家意见:通过靶向炎症过程来改变这种疾病进展的努力尚未产生有意义的结果。抗菌药物的有限成功提出了生态失调在PSC中的作用的问题,并标志着一个新的潜在的治疗目标有待研究。然而,正如许多进行性肝脏疾病一样,器官纤维化仍然是不可逆转的步骤,直到有效的抗纤维化药物被开发出来,PSC的最终治疗方法可能仍然难以捉摸。
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Assessing and managing symptom burden and quality of life in primary sclerosing cholangitis patients
ABSTRACT Introduction: Primary sclerosing cholangitis (PSC) is a progressive disease of the bile ducts for which there is no cure. As no therapy has proven to delay this progression, the current focus for these patients is symptom management. In the same vein, the screening for and prevention of diseases for which these patients carry increased risk is an effective part of high-yield care. Areas Covered: A literature search was conducted using the National Center for Biotechnology Information (NCBI) database at the U.S. National Library of Medicine (NLM). Herein is reviewed that literature with special attention to symptom assessment and medical therapies as well as associated disease and screening strategies for patients with PSC. Additionally, some of the previous therapies future directions of treatment are explored. Expert Opinion: Efforts to alter the progression of this disease by targeting the inflammatory process have not produced meaningful results. The limited success of antimicrobial agents raises the question of the role of dysbiosis in PSC and marks a new potential target for therapies to be studied. However, as in many progressive diseases of the liver, fibrosis of the organ remains the irreversible step, and until effective anti-fibrotic agents are developed a definitive cure for PSC may remain elusive.
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来源期刊
Expert Opinion on Orphan Drugs
Expert Opinion on Orphan Drugs PHARMACOLOGY & PHARMACY-
CiteScore
2.30
自引率
0.00%
发文量
8
期刊介绍: Expert Opinion on Orphan Drugs is an international, peer-reviewed journal that covers all aspects of R&D on rare diseases and orphan drugs.
期刊最新文献
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