Biliary atresia: intention-to-treat analysis in a pediatric liver transplant center
Joao Seda Neto1, Eduardo Fonseca A1, Adriana Porta1, Irene Miura K1, Paulo Chapchap1, Carolina Costa M1, Flavia Feier H1, Gilda Porta1.
1Liver Transplantation, Hospital Sirio-Libanes, São Paulo, Brazil
Background: In patients with Biliary Atresia (BA), the efforts at avoiding premature liver transplantation (LT) target early identification and diagnosis, timing of Kasai-portoenterostomy (KPE) before 45 days of age, and centralization of surgery at experienced centers. However, a large percentage of infants still require LT in the first 2 years of life, and many referals to liver transplant centers are still late.
Aim: The aim of this study was to analyze the outcomes of all patients with BA, with no previous treatment, referred to a single team, on an intent‐to‐treat basis.
Methods: Retrospective study in patients with no previous treatment refferred to our center from Jan/2001 to Jan/2021. All the patient’s care, which included diagnosis of BA, portoenterostomy (PE), clinical follow-up, transplantation, and post-transplant follow-up was provided by the same pediatric hepatology and transplant team throughout the years. All patients included in this study had BA. They were evaluated according to their age (days) at first assessment, clinical presentation (jaundice, coluria, acolic stools), physical examination (size of the liver and spleen, presence of ascites), laboratory values (liver function tests, infectious workup, serum protein electrophoresis), image studies (total abdominal ultrasound and hepatobiliary scintigraphy - DISIDA Scan), liver biopsy, and intraoperative cholangiography when necessary. The patients were divided in three groups according to the initial assessment: LT only, Kasai portoenterostomy (KPE) only, and KPE + LT group.
Results: Thirty-nine patients with biliary atresia were followed from an early age. Seven patients did not undergo Kasai-PE, and received liver grafts at various time points (LT-only Group). Thirty-two children underwent KPE. Nine of them composed the Kasai only Group: 8 patients survived with their native liver at a median time of 5 years (range 2y to 11y), but one patient in this group died during the follow-up. Twenty-three patients were transplanted after the PE (Kasai + LT Group). Median follow-up in this group was 11 years (range 2 to 20). The overall patient survival of the entire cohort was 94.8%, and the survival with native liver was 28.1%. Interstingly, 10 patients in this series (25.6%) were babies conceived through in vitro fertilization (IVF), distributed proportionaly in the observed groups.
Conclusion: This abstract shows that when all the treatment options are at hand and timely offered to patients with BA, the expected long-term results are excellent (95% patient survival). A interesting finding in this study, which has not been reported elsewhere, was the 25% association of BA and IVF.
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