View clinical trials related to Biliary Atresia.
Filter by:Biliary atresia (BA) is a neonatal liver disease characterized by impaired bile flow and is the most common indication for pediatric liver transplantation. BA can be treated with the Kasai portoenterostomy (KP), a procedure that attempts to restore bile flow and slow disease progression. However, success of the KP procedure is quite variable, and lack of adjuvant medical therapies following KP is a major gap in pediatric hepatology. This study begins to explore oral N-acetylcysteine (NAC) as a potential medical therapy in BA by determining whether an oral formulation can be given to infants. The primary objective is to determine tolerability of the oral NAC formulation. The primary outcome is tolerating at least 3 out of 4 total doses without emesis. The Bayesian Optimal Interval Design (BOIN) trial design will be used to determine the maximum tolerated dose of oral NAC. Our secondary objective is to assess palatability of the oral NAC formulation by comparing facial expressions when taking oral NAC versus other medications commonly given to cholestatic infants.
This is a multi-center retrospective chart review to compile a data repository of the management and outcomes of children with biliary atresia. Overall, investigators aim to evaluate which specific factors contribute to improved patient outcomes, to help guide potential improvements in patient care and resource utilization.
This study will evaluate the efficacy, safety and tolerability, as well as PK/PD of OCA in eligible pediatric participants with biliary atresia with successful hepatoportoenterostomy (HPE, also known as a Kasai portoenterostomy). The double-blind period comprises of 2 phases: dose titration phase and age expansion treatment phase.
Biliary atresia is a serious congenital anomaly characterized by persistent and progressive cholestatic jaundice. The incidence of biliary atresia is more common in East Asia, especially China, with an incidence of 2 per 10,000 live births. Liver transplantation is the only effective way to treat end-stage liver disease. However, distant organ damage, affecting the heart, brain, kidneys, lungs, and intestines, is still an important factor affecting the long-term survival of children after surgery. Desflurane is a volatile anesthetic commonly used in surgery. In order to observe the effect of desflurane on the incidence of early postoperative pediatric acute respiratory distress syndrome (PARDS) with biliary atresia who underwent living donor liver transplantation, and explore the related mechanism, a total of 165 infant patients underwent living liver transplantation due to biliary atresia from March 2023 to October 2023 are included in our single-center prospective study. They are randomly divided into propofol group (n=55), propofol and desflurane group (n=55) and desflurane group (n=55) according to the difference of intraoperative anesthesia maintenance. Gender, age, height, weight, PELD scores and other preoperative basic data are recorded. Operation time, anhepatic time and intraoperative blood loss volume are recorded. The basic information of liver donors are also recorded. Postoperative mechanical ventilation time, ICU stay time, tacrolimus concentration, total length of hospital stay and mortality during hospitalization are recorded. According to the the definition of PARDS recommended by the 2015 Pediatric Acute Lung Injury Consensus Conference is used as the diagnostic and grading criteria for postoperative PARDS, and the incidence and grading of PARDS within the first seven days after surgery are evaluated in the three groups. Peripheral blood is collected immediately after anesthesia induction, 30min after reperfusion and at the end of surgery to detect serum levels of HMGB1, IL-6 and TNF-α by enzyme linked immunosorbent assay (ELISA).
In most babies with biliary atresia (BA) bile ducts on the inside and outside of the liver are affected eventually leading to a complete blockage of bile flow from the liver. This in turn causes scarring (fibrosis) in the liver. The surgery called the Kasai procedure and eventually, a liver transplant could treat biliary atresia. However, children with BA have poor liver function, poor renal reserve, and poor ability to regulate water and electrolytes. Therefore, A reasonable and effective perioperative fluid management is more significant. Considering the high chlorine of saline, the liver burden of Lactated Ringer's solution and Acetated Ringer's solution, the Bicarbonate Ringer's solution which composition is similar to plasma, could be the best choice for perioperative fluid management of BA children. Therefore, we hypothesize that compared with other crystalloids, Bicarbonate Ringer's solution is a better choice to maintain water, electrolyte and acid-base balance in BA children.
Our study aims to develop a screening test for biliary atresia (BA) using dry blood spot to improve patient survival by early diagnosis. Newborn screening dry blood spot will be examined for the direct bilirubin (DB), γ-GT or matrix metalloproteinase-7 (MMP-7) levels. These findings will promote early diagnosis for BA and hence improve the survival.
A prospective observational study in infants with biliary atresia and controls to determine whether the composition of the intestinal microbiome is specific for biliary atresia will be conducted. The hypothesis of the study is "infants with biliary atresia have a unique microbiome signature at the time of diagnosis and changes in population dynamics occur during disease progression". The microbiome will be determined at diagnosis and at well-defined time points during the natural history of the disease.