肥酸作为低脂相关胆固醇症候群的二线治疗
Pierre-Antoine Soret1,2,3,4, Sara Lemoinne1,2,3,4, Maxime Mallet5
1Reference Center for Inflammatory Biliary Diseases and Autoimmune Hepatitis, European Reference Network on Hepatological Diseases (ERN Rare-Liver), Saint-Antoine Hospital, Assistance Publique - Hôpitaux de Paris, Paris, France.
Alimentary pharmacology & therapeutics
|October 11, 2023
概括
在ursodeoxycholic acid (UDCA) 无效时,obeticholic acid (OCA) 在治疗低脂相关胆固醇病症 (LPAC) 综合征方面表现有前途. 在这项初步研究中,五分之四的患者经历了症状改善,没有报告任何不良反应.
科学领域:
- 肝病学 肝病学是一种肝病学.
- 遗传学 是一个遗传学.
- 药理学 药理学是指药理学的学科.
背景情况:
- 低脂相关胆病综合征 (LPAC) 是一种罕见的遗传疾病,导致肝病,通常与ABCB4基因变异有关.
- Ursodeoxycholic 酸 (UDCA) 是标准的治疗方法,但有些患者经历治疗失败或不耐受.
- 法尔内索伊德-X受体 (FXR) 激动剂,如肥酸 (OCA),正在研究它们对调节ABCB4并使LPAC患者受益的潜力.
研究的目的:
- 为了评估在被诊断患有LPAC综合征的患者中OCA的疗效.
- 评估OCA在那些对UDCA治疗表现出不充分反应或不耐受的人群中的有效性.
主要方法:
- 对五名被诊断患有LPAC综合征的患者进行了回顾性分析.
- 患者接受了OCA治疗,OCA是一种选择性FXR激动剂,每日剂量从5毫克到10毫克不等,通常与UDCA结合,平均为36个月.
主要成果:
- 五分之四的患者 (80%) 报告说症状有所改善,其中三例完全消失,1例部分改善.
- 在治疗期间没有报告任何不良反应.
- 虽然大多数患者的症状有所改善,但在三个响应者中发现了肝病的持续性放射性迹象,而肝功能异常在一名患者中持续存在.
结论:
- 对UDCA.不反应或不耐受患者的LPAC综合征进行有效治疗的潜力被证明是OCA.
- 这些初步发现需要在更大规模的前性研究中进一步调查,以确认OCA在LPAC综合征中的治疗作用.
相关概念视频
01:20Lipid-Lowering Drugs: Statins and Miscellaneous Agents
678
Hyperlipidemia, a medical condition often referred to as high cholesterol, is characterized by abnormally elevated levels of lipids in the bloodstream. When present in excess, these lipids, specifically cholesterol and triglycerides, can lead to serious health complications, often involving cardiovascular diseases. Illnesses like atherosclerosis, heart attacks, and pancreatitis have all been linked to untreated hyperlipidemia. This means controlling and regulating cholesterol and triglyceride...
678
01:21Drugs for Treatment of Constipation-Predominant IBS
183
Pharmacological therapies for IBS-C are designed to alleviate abdominal discomfort and enhance bowel function. In patients with IBS-C, fiber supplements may help soften stools and decrease straining, but may also lead to increased gas production and bloating. Osmotic laxatives like milk of magnesia are frequently used to soften stools and increase stool frequency in IBS-C patients. In addition, two drugs approved for use in severe IBS-C adult cases are linaclotide (Linzess) and lubiprostone...
183
01:26Peptic Ulcer Disease IV: Management
108
Medical treatment strategies for peptic ulcers encompass various methods. The primary goal of treatment is to diminish gastric acidity and strengthen mucosal defense mechanisms.
The therapeutic approach involves ensuring adequate rest, implementing drug therapy, promoting smoking cessation, making dietary modifications, and emphasizing long-term follow-up care.
Pharmacological management
The prevailing therapy for peptic ulcers involves a combination of managing the patient's current...
The therapeutic approach involves ensuring adequate rest, implementing drug therapy, promoting smoking cessation, making dietary modifications, and emphasizing long-term follow-up care.
Pharmacological management
The prevailing therapy for peptic ulcers involves a combination of managing the patient's current...
108
01:30Acute Pancreatitis II: Clinical Manifestations and Management
132
Acute pancreatitis presents a complex medical emergency characterized by rapid onset inflammation of the pancreas, demanding timely diagnosis and management to prevent complications. The condition primarily manifests through severe upper abdominal pain that often radiates to the back. This pain intensifies following the consumption of fatty foods. Accompanying symptoms such as nausea, vomiting, abdominal distention, fever, dyspnea, cyanosis, and jaundice can vary in intensity but significantly...
132
01:17Drugs for Treatment of Diarrhea-Predominant IBS
188
Diarrhea-predominant irritable bowel syndrome (IBS-D) is a subtype of IBS characterized primarily by frequent, loose, or watery stools, abdominal pain, and abdominal discomfort. Therapeutic approaches to managing IBS-D include dietary changes, stress management techniques, and pharmaceutical interventions.
Two specific drugs used in the treatment are alosetron (Lotronex) and eluxadoline (Viberzi). Alosetron, a 5-HT3 antagonist, works by slowing the movement of stools in the gut, reducing bowel...
Two specific drugs used in the treatment are alosetron (Lotronex) and eluxadoline (Viberzi). Alosetron, a 5-HT3 antagonist, works by slowing the movement of stools in the gut, reducing bowel...
188
01:29Chronic Pancreatitis II: Collaborative Care
97
The management of chronic pancreatitis is multifaceted, involving a comprehensive approach that includes thorough assessment, diagnostic testing, and a variety of management strategies.
Assessment:
Assessment:
97