The Brazilian Alagille syndrome study: New insights from a multicenter national cohort.
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История · было → стало
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Carvalho Elisa, Porta Gilda, Miura Irene Kazue, Ferreira Cristina Helena Targa, Silva Luciana Rodrigues, Sandy Natascha Silva, Fagundes Eleonora Druve Tavares, Oliveira Eliene, Freitas Aline Falleiros, de Tommaso Adriana Maria Alves, Sawamura Regina, Melere Melina Utz, Vieira Sandra Maria Goncalves, Seixas Renata Belém Pessoa de Melo, Trevizoli Isadora de Carvalho, Ferreira Alexandre Rodrigues, Hirschfeld Adriana Porta Miche, Pugliese Renata Pereira Sustovich, Marques Cibele Dantas Ferreira, Brandão Maria Angela Bellomo, Pinto Raquel Borges, Lopes Leticia Helena Caldas, Maia Jussara Melo de Cerqueira, de Melo Ana Cristina Vieira, Silva Carolina Sores, Nader Luiza Salgado, Guiotti Maria Tereza, Alcântara Roberta Vacari, Resende Leonardo, Rocha Bruno Pereira Ribeiro, Perin Nilza Maria Medeiros, Rodrigues Adriana Teixeira, Danesi Vera Lúcia Baggio, Hessel Gabriel, Silveira Themis Reverbel
Аннотация
METHODS: Multicenter retrospective cohort study of children with ALGS. Descriptive statistics summarized clinical data. Overall survival was estimated using Kaplan-Meier curves. NLS was analyzed with competing risk models. Associations between laboratory parameters and NLS were tested using Pearson's chi-square.
RESULTS: From 14 Brazilian reference centers, 120 children (52.5% male) were included, with mean age at diagnosis of 17.8 months and a median follow-up time of 88.5 months. Most presented characteristic facies (89.9%), jaundice (88.2%), pruritus (87.9%), and neonatal cholestasis (79.2%). Thirty percent underwent liver transplantation (LT) and 6.7% died. Jaundice, xanthomas, hepatomegaly, splenomegaly, and presence of excoriations were significantly associated with the need for LT or death. Direct bilirubin (DB) > 6 mg/dL, cholesterol >300 mg/dL, increased aspartate aminotransferase (AST) > 5 times the upper limit of normal (ULN), and AST-to-platelet ratio index (APRI) > 1 were associated with a significantly increased risk of LT or death, with hazard ratios of 18.2 (p < 0.001, 95% confidence interval [95% CI] = 5.1-64.5), 3.4 (p = 0.016, 95% CI = 1.3-9.2), 3.9 (p = 0.018, 95% CI = 1.2-12.4), and 10.2 (p = 0.013, 95% CI = 1.3-82.2), respectively.
CONCLUSIONS: The main clinical manifestations included characteristic facies, jaundice, and pruritus. Higher rates of LT or death were associated with jaundice, xanthomas, hepatomegaly, splenomegaly and presence of excoriations. Higher rates of NLS were associated with DB levels below 6 mg/dL, cholesterol levels below 300 mg/dL, AST levels <5x ULN, and an APRI < 1.