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Advisor(s)
Abstract(s)
Introdução: A atresia biliar (AB) é uma doença do recém-nascido com elevada
mortalidade se nĂŁo tratada precocemente. Manifesta-se nos primeiros meses de vida como
uma obstrução completa do colédoco e evolui progressivamente para uma colangiopatia
intra-hepĂĄtica. O tratamento inicial consiste em uma portoenterostomia (PE) para
restaurar o fluxo biliar seguido de transplante hepĂĄtico (TxH) nos casos em que a PE falha
ou Ă© considerada inĂștil. AlĂ©m da PE, alguns tratamentos adjuvantes sĂŁo utilizados para
melhorar os resultados da AB e reduzir as complicaçÔes. Estes incluem corticosteroides,
ĂĄcido ursodesoxicĂłlico, antibiĂłticos, probiĂłticos, anti-fibrĂłtico e suporte nutricional. Novas
terapĂȘuticas encontram-se sob investigação, nomeadamente, inibidor do transportador de
ĂĄcido biliar ileal, agonista do recetor X farnesĂłide, N-acetilcisteĂna, antivĂrus,
imunoglobulina, fator estimulador de colónias de granulócitos e células-tronco
hematopoiĂ©ticas. Outras formas de melhorar os resultados na AB tĂȘm sido sugeridas. Estas
incluem a redução ou eliminação do impacto dos fatores que influenciam o sucesso da PE e
a adoção de TxH como tratamento cirĂșrgico inicial dado os excelentes resultados nos raros
casos em que este foi empregue.
Objetivo: Nesta dissertação, serĂŁo exploradas as abordagens terapĂȘuticas atuais na AB
com o objetivo de avaliar a sua eficĂĄcia. Ademais, serĂŁo enfatizados os fatores prognĂłsticos
no momento da PE que parecem estar envolvidos na sobrevida do fĂgado nativo.
Metodologia: Realizou-se uma recolha de artigos na base de dados online PubMed. A
pesquisa incluiu artigos de revisĂŁo, ensaios clĂnicos, meta-anĂĄlises, ensaios controlados
randomizados, revisĂŁo sistemĂĄtica, publicados nos Ășltimos 10 anos, redigidos na lĂngua
inglesa, portuguesa e espanhola. Foram excluĂdos estudos realizados em animais e relatos
de casos isolados.
Conclusão: A PE é a opção primåria de tratamento na AB, porém paliativa em
consequĂȘncia da colangiopatia intra-hepĂĄtica que persiste. E o TxH Ă© o tratamento
definitivo, porém se associa a riscos e estå dependente da disponibilidade de enxertos. A
taxa de sucesso da PE estĂĄ dependente de vĂĄrios fatores como a idade no momento da
cirurgia, a experiĂȘncia do centro hospitalar, o mĂ©todo cirĂșrgico, as complicaçÔes pĂłsoperatĂłrias e as caraterĂsticas da ĂĄrvore biliar do prĂłprio doente. Esforços devem ser feitos
para eliminar a influĂȘncia dos fatores que sĂŁo passĂveis de modificação. As terapĂȘuticas
adjuvantes utilizadas, na sua generalidade, carecem de evidĂȘncias cientĂficas suficientes
relativamente Ă sua eficĂĄcia, sendo necessĂĄrios mais estudos para estabelecer o seu valor terapĂȘutico. As novas propostas de tratamento deverĂŁo ser submetidas a uma avaliação
rigorosa da eficĂĄcia e dos eventos adversos. Estudos que visam o estabelecimento de
critérios de prognóstico que permitem prever a lesão hepåtica progressiva são precisos de
forma a melhorar o tratamento destas crianças enquanto a etiologia da AB permanece por
desvendar.
Introduction: Biliary atresia (BA) is a newborn disease with high mortality if not treated early. It manifests itself in the first months of life as a complete obstruction of the common bile duct and progressively evolves into an intrahepatic cholangiopathy. Initial treatment consists of a portoenterostomy (PE) to restore bile flow followed by liver transplantation (LTx) in cases where PE fails or is deemed useless. In addition to PE, some adjuvant treatments are used to improve AB outcomes and reduce complications. These include corticosteroids, ursodeoxycholic acid, antibiotics, probiotics, anti-fibrotics and nutritional support. New therapies are under investigation, namely, ileal bile acid transporter inhibitor, farnesoid X receptor agonist, N-acetylcysteine, antivirus, immunoglobulin, granulocyte colony stimulating factor and hematopoietic stem cells. Other ways to improve results in AB have been suggested. These include the reduction or elimination of the impact of factors that influence the success of PE and the adoption of LTx as an initial surgical treatment given the excellent results in the rare cases in which it has been used. Objectives: In this dissertation, current therapeutic approaches in AB will be explored to assess their effectiveness. Furthermore, prognostic factors at the time of PE that seem to be involved in native liver survival will be emphasized. Methodology: Articles were collected from the PubMed online database. The research included review articles, clinical trials, meta-analyses, randomized controlled trials, systematic review, published in the last 10 years, written in English, Portuguese, and Spanish. Animal studies and reports of isolated cases were excluded. Conclusions: PE is the primary treatment option in BA, but palliative because of persistent intrahepatic cholangiopathy. And LTx is the definitive treatment, but it is associated with risks and is dependent on the availability of grafts. The success rate of PE is dependent on several factors such as age at the time of surgery, the experience of the hospital, the surgical method, postoperative complications, and the characteristics of the patient's own biliary tree. Efforts must be made to eliminate the influence of variables that are subject to modification. The adjuvant therapies used, in general, lack sufficient scientific evidence regarding their effectiveness, requiring further studies to establish their therapeutic value. New treatment proposals should be subject to a rigorous evaluation of efficacy and adverse events. Studies aimed at establishing prognostic criteria that allow predicting progressive liver damage are needed to improve the treatment of these children while the etiology of BA remains to be revealed.
Introduction: Biliary atresia (BA) is a newborn disease with high mortality if not treated early. It manifests itself in the first months of life as a complete obstruction of the common bile duct and progressively evolves into an intrahepatic cholangiopathy. Initial treatment consists of a portoenterostomy (PE) to restore bile flow followed by liver transplantation (LTx) in cases where PE fails or is deemed useless. In addition to PE, some adjuvant treatments are used to improve AB outcomes and reduce complications. These include corticosteroids, ursodeoxycholic acid, antibiotics, probiotics, anti-fibrotics and nutritional support. New therapies are under investigation, namely, ileal bile acid transporter inhibitor, farnesoid X receptor agonist, N-acetylcysteine, antivirus, immunoglobulin, granulocyte colony stimulating factor and hematopoietic stem cells. Other ways to improve results in AB have been suggested. These include the reduction or elimination of the impact of factors that influence the success of PE and the adoption of LTx as an initial surgical treatment given the excellent results in the rare cases in which it has been used. Objectives: In this dissertation, current therapeutic approaches in AB will be explored to assess their effectiveness. Furthermore, prognostic factors at the time of PE that seem to be involved in native liver survival will be emphasized. Methodology: Articles were collected from the PubMed online database. The research included review articles, clinical trials, meta-analyses, randomized controlled trials, systematic review, published in the last 10 years, written in English, Portuguese, and Spanish. Animal studies and reports of isolated cases were excluded. Conclusions: PE is the primary treatment option in BA, but palliative because of persistent intrahepatic cholangiopathy. And LTx is the definitive treatment, but it is associated with risks and is dependent on the availability of grafts. The success rate of PE is dependent on several factors such as age at the time of surgery, the experience of the hospital, the surgical method, postoperative complications, and the characteristics of the patient's own biliary tree. Efforts must be made to eliminate the influence of variables that are subject to modification. The adjuvant therapies used, in general, lack sufficient scientific evidence regarding their effectiveness, requiring further studies to establish their therapeutic value. New treatment proposals should be subject to a rigorous evaluation of efficacy and adverse events. Studies aimed at establishing prognostic criteria that allow predicting progressive liver damage are needed to improve the treatment of these children while the etiology of BA remains to be revealed.
Description
Keywords
Fatores PrognĂłsticos Atresia Biliar Sobrevida do FĂgado Nativo Tratamento FarmacolĂłgico Tratamento MĂ©dico