拉丁美洲ama阴性原发性胆道胆管炎：治疗反应较低的独特亚群

IF 4.4 3区医学 Q2 GASTROENTEROLOGY & HEPATOLOGY

Annals of hepatology Pub Date : 2025-09-01 DOI:10.1016/j.aohep.2025.102013

Claudia Alves Couto , Guilherme Grossi Lopes Cançado , María Lucía Ferraz Ferraz , Debora Raquel Terrabuio , Alejandra Villamil , Lorena Castro Solari , Graciela Elia Castro-Narro , Ezequiel Ridruejo , Cristiane A. Villela-Nogueira , Danay Guerrero , Daniela Chiodi , Carla Enrique , Paulo Lisboa Bittencourt , Gustavo Pereira , Fernando Bessone , Stefanny Cornejo Hernández , Rodrigo Zapata , Hugo Cheinquer , Nicolás Ortiz López , Manuel Mendizabal , Nélia Hernandez

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引用次数: 0

摘要

原发性胆道胆管炎（PBC）是一种自身免疫性胆汁淤积性疾病，通常通过抗线粒体抗体（AMA）诊断。ama阴性PBC是否代表一种独特的临床表型仍然存在争议。本研究旨在根据拉丁美洲AMA状况描述PBC的流行病学特征。材料和方法这项正在进行的、回顾性的、国际多中心队列研究，由ALEH赞助，包括来自多个拉丁美洲国家的PBC患者。根据AMA状态对患者进行分层；排除自身免疫性肝炎- pbc重叠者。结果分析1204例患者的数据：巴西（48.3%）、阿根廷（23.4%）、智利（10.8%）、墨西哥（7.4%）等。多数为女性（92.3%），平均诊断年龄53±13岁；22.2%基线时有肝硬化。排除重叠综合征。AMA阳性占76.8%。ama阳性和ama阴性患者的女性比例相似（各92.5%,p=0.963），基线肝硬化（22.4%对23.6%,p=0.706），症状表现（77.5%对79.4%,p=0.544）。MASLD在ama阴性患者中更为常见（7.5%比3.8%,p=0.024）， sp100阳性率（9.1%比2.5%,p= 0.001）和gp210阳性率（7.3比3.3%,p= 0.001）也更高。95.2%的患者接受了UDCA治疗，其中28.3%的患者由于UDCA不完全缓解而接受了二线治疗。ama阳性患者在12个月时对熊去氧胆酸（UDCA）的反应更高，包括ALP正常化（29.7% vs. 21.2%, p=0.035）和深度反应（17.5% vs. 8.6%, p=0.007）。贝特治疗12个月后观察到类似的结果（34.8%比9.4%,p=0.005）。无移植生存无差异（p=0.213）。结论拉丁美洲的sama阴性PBC患者具有相似的基线特征，但对UDCA和贝特类药物的反应率较低，支持了生物学上不同疾病亚群的假设。

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AMA-NEGATIVE PRIMARY BILIARY CHOLANGITIS IN LATIN AMERICA: A DISTINCT SUBSET WITH LOWER TREATMENT RESPONSE

Introduction and Objectives

Primary biliary cholangitis (PBC) is an autoimmune cholestatic disease, typically diagnosed by the presence of anti-mitochondrial antibodies (AMA). Whether AMA-negative PBC represents a distinct clinical phenotype remains controversial. This study aimed to characterize the epidemiological profile of PBC according to AMA status in Latin America.

Materials and Methods

This ongoing, retrospective, international multicenter cohort study, sponsored by ALEH, includes PBC patients from multiple Latin American countries. Patients were stratified by AMA status; those with autoimmune hepatitis-PBC overlap were excluded.

Results

Data from 1,204 patients were analyzed: Brazil (48.3%), Argentina (23.4%), Chile (10.8%), Mexico (7.4%), and others. Most were female (92.3%) with a mean age at diagnosis of 53±13 years; 22.2% had cirrhosis at baseline. Overlap syndrome was excluded. AMA were positive in 76.8%. AMA-positive and AMA-negative patients had similar rates of female sex (92.5% each, p=0.963), baseline cirrhosis (22.4% vs. 23.6%, p=0.706), and symptomatic presentation (77.5% vs. 79.4%, p=0.544). MASLD was more frequent among AMA-negative patients (7.5% vs. 3.8%, p=0.024), which also had higher rates of sp100 (9.1% vs 2.5%, p< 0.001) and gp210 (7.3 vs 3.3%, p< 0.001) positivity. Treatment with UDCA was performed in 95.2% of patients and, from those, 28.3% had second line treatment indicated due to incomplete response to UDCA. AMA-positive patients showed higher response to ursodeoxycholic acid (UDCA) at 12 months, including ALP normalization (29.7% vs. 21.2%, p=0.035) and deep response (17.5% vs. 8.6%, p=0.007). Similar findings were observed after 12 months of fibrate therapy (34.8% vs. 9.4%, p=0.005). No difference was found in transplant-free survival (p=0.213).

Conclusions

AMA-negative PBC patients in Latin America present similar baseline features but have lower response rates to UDCA and fibrates, supporting the hypothesis of a biologically distinct disease subset.

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来源期刊

Annals of hepatology 医学-胃肠肝病学

CiteScore

7.90

自引率

2.60%

发文量

183

审稿时长

4-8 weeks

期刊介绍： Annals of Hepatology publishes original research on the biology and diseases of the liver in both humans and experimental models. Contributions may be submitted as regular articles. The journal also publishes concise reviews of both basic and clinical topics.