Cançado Guilherme Grossi Lopes, Braga Michelle Harriz, Ferraz Maria Lúcia Gomes, Villela-Nogueira Cristiane Alves, Terrabuio Debora Raquel Benedita, Cançado Eduardo Luiz Rachid, Nardelli Mateus Jorge, Faria Luciana Costa, Gomes Nathalia Mota de Faria, de Oliveira Elze Maria Gomes, Rotman Vivian, de Oliveira Maria Beatriz, da Cunha Simone Muniz Carvalho Fernandes, Mazo Daniel Ferraz de Campos, Mendes Liliana Sampaio Costa, Ivantes Claudia Alexandra Pontes, Codes Liana, de Almeida E Borges Valéria Ferreira, Pace Fabio Heleno de Lima, Pessoa Mario Guimarães, Signorelli Izabelle Venturini, Coral Gabriela Perdomo, Bittencourt Paulo Lisboa, Levy Cynthia, Couto Cláudia Alves
Instituto Alfa de Gastroenterologia, Hospital das Clínicas da Universidade Federal de Minas Gerais, Av. Professor Alfredo Balena 110, Belo Horizonte 30130-100, Minas Gerais, Brazil; Hospital da Polícia Militar de Minas Gerais, Belo Horizonte, Minas Gerais, Brazil.
Departamento de Gastroenterologia, Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil.
Ann Hepatol. 2022 Jan-Feb;27(1):100546. doi: 10.1016/j.aohep.2021.100546. Epub 2021 Sep 30.
Little is known about primary biliary cholangitis (PBC) in non-whites. The purpose of this study was to evaluate clinical features and outcomes of PBC in a highly admixed population.
The Brazilian Cholestasis Study Group multicentre database was reviewed to assess demographics, clinical features and treatment outcomes of Brazilian patients with PBC.
562 patients (95% females, mean age 51 ± 11 years) with PBC were included. Concurrent autoimmune diseases and overlap with autoimmune hepatitis (AIH) occurred, respectively, in 18.9% and 14%. After a mean follow-up was 6.2 ± 5.3 years, 32% had cirrhosis, 7% underwent liver transplantation and 3% died of liver-related causes. 96% were treated with ursodeoxycholic acid (UDCA) and 12% required add-on therapy with fibrates, either bezafibrate, fenofibrate or ciprofibrate. Response to UDCA and to UDCA/fibrates therapy varied from 39%-67% and 42-61%, respectively, according to different validated criteria. Advanced histological stages and non-adherence to treatment were associated with primary non-response to UDCA, while lower baseline alkaline phosphatase (ALP) and aspartate aminotransferase (AST) levels correlated with better responses to both UDCA and UDCA/fibrates.
Clinical features of PBC in highly admixed Brazilians were similar to those reported in Caucasians and Asians, but with inferior rates of overlap syndrome with AIH. Response to UDCA was lower than expected and inversely associated with histological stage and baseline AST and ALP levels. Most of patients benefited from add-on fibrates, including ciprofibrate. A huge heterogeneity in response to UDCA therapy according to available international criteria was observed and reinforces the need of global standardization.
关于非白种人中原发性胆汁性胆管炎（PBC）的情况知之甚少。本研究旨在评估高度混合人群中PBC的临床特征和预后。
回顾巴西胆汁淤积研究组的多中心数据库，以评估巴西PBC患者的人口统计学、临床特征和治疗结果。
纳入562例PBC患者（95%为女性，平均年龄51±11岁）。并发自身免疫性疾病和与自身免疫性肝炎（AIH）重叠的发生率分别为18.9%和14%。平均随访6.2±5.3年后，32%发生肝硬化，7%接受肝移植，3%死于肝脏相关原因。96%接受熊去氧胆酸（UDCA）治疗，12%需要加用贝特类药物，包括苯扎贝特、非诺贝特或环丙贝特进行联合治疗。根据不同的有效标准，对UDCA治疗和UDCA/贝特类药物治疗的反应率分别为39%-67%和42%-61%。组织学晚期和不坚持治疗与对UDCA的原发性无反应相关，而较低的基线碱性磷酸酶（ALP）和天冬氨酸转氨酶（AST）水平与对UDCA和UDCA/贝特类药物的更好反应相关。
高度混合的巴西人中PBC的临床特征与白种人和亚洲人报道的相似，但与AIH重叠综合征的发生率较低。对UDCA的反应低于预期，且与组织学分期以及基线AST和ALP水平呈负相关。大多数患者从加用贝特类药物（包括环丙贝特）中获益。观察到根据现有国际标准，对UDCA治疗的反应存在巨大异质性，这强化了全球标准化的必要性。
