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1.
J Pediatr ; 209: 92-96.e1, 2019 06.
Artigo em Inglês | MEDLINE | ID: mdl-30878206

RESUMO

OBJECTIVE: To investigate patient factors predictive of gamma glutamyltransferase (GGT) normalization following ursodeoxycholic acid (UDCA) therapy in children with primary sclerosing cholangitis. STUDY DESIGN: We retrospectively reviewed patient records at 46 centers. We included patients with a baseline serum GGT level ≥50 IU/L at diagnosis of primary sclerosing cholangitis who initiated UDCA therapy within 1 month and continued therapy for at least 1 year. We defined "normalization" as a GGT level <50 IU/L without experiencing portal hypertensive or dominant stricture events, liver transplantation, or death during the first year. RESULTS: We identified 263 patients, median age 12.1 years at diagnosis, treated with UDCA at a median dose of 15 mg/kg/d. Normalization occurred in 46%. Patients with normalization had a lower prevalence of Crohn's disease, lower total bilirubin level, lower aspartate aminotransferase to platelet ratio index, greater platelet count, and greater serum albumin level at diagnosis. The 5-year survival with native liver was 99% in those patients who achieved normalization vs 77% in those who did not. CONCLUSIONS: Less than one-half of the patients treated with UDCA have a complete GGT normalization in the first year after diagnosis, but this subset of patients has a favorable 5-year outcome. Normalization is less likely in patients with a Crohn's disease phenotype or a laboratory profile suggestive of more advanced hepatobiliary fibrosis. Patients who do not achieve normalization could reasonably stop UDCA, as they are likely not receiving clinical benefit. Alternative treatments with improved efficacy are needed, particularly for patients with already-advanced disease.


Assuntos
Colangite Esclerosante/sangue , Colangite Esclerosante/tratamento farmacológico , Ácido Ursodesoxicólico/uso terapêutico , gama-Glutamiltransferase/sangue , Adolescente , Análise de Variância , Biomarcadores/sangue , Criança , Estudos de Coortes , Feminino , Seguimentos , Humanos , Testes de Função Hepática , Masculino , Valor Preditivo dos Testes , Estudos Retrospectivos , Índice de Gravidade de Doença , Fatores de Tempo , Falha de Tratamento , Resultado do Tratamento
2.
J Pediatr Gastroenterol Nutr ; 59(3): 417-22, 2014 Sep.
Artigo em Inglês | MEDLINE | ID: mdl-24979319

RESUMO

The requirements for and conditions of subspecialty training in paediatric gastroenterology, hepatology, and nutrition (PGHN) are rather variable across European countries. The European Society for Pediatric Gastroenterology, Hepatology, and Nutrition (ESPGHAN) agreed on a training syllabus aimed to foster a harmonised European PGHN curriculum and to support national PGHN societies and governmental bodies to promote and establish high-quality training programmes and levels of certification in the field. The document provides PGHN training prerequisites and objectives and the basic knowledge elements to acquire the clinical, technical, and management skills needed. Guidelines and instruments for self-monitoring and appraisal are proposed, and a logbook is available online. These training standards are a first step towards a European certification and recognition as a specialist in PGHN.


Assuntos
Currículo , Gastroenterologia/educação , Ciências da Nutrição/educação , Pediatria/educação , Sociedades Médicas , Especialização/normas , Certificação/normas , Competência Clínica , Europa (Continente) , Gastroenterologia/normas , Humanos , Pediatria/normas
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