Introduction: Alagille syndrome (ALGS) is an autosomal dominant genetic cholestasis associated with variable extra hepatic involvement. Given the large research in this topic during recent years, we aim to add experience from a high-volume center, regarding indications and outcomes of liver transplantation (LT) in children with ALGS.
Methods: we retrospectively analyzed digital clinical charts from patients with ALGS who received LT from 2001 to 2024, regarding associated cardiac involvement, LT indications, early and long-term complications, and graft and patient’s survival rates.
Results: from 2001 to 2024, 11 out of 287 pediatric LT were performed in patients with ALGS (3.8%). Nine ALGS patients had associated cardiac involvement (90%), 8 out of them presented mild pulmonary branch stenosis that did not required treatment. Eight patients (80%) required LT due to untreatable itching. Median age at LT was 22 mo. Median weight at LT was 8 kg (only one of them weighed more than 10 kg). Two LT were from deceased donors; all the others were performed with related living donors (81.8%). In the post LT period, 4 patients had biliary complications that were solved by interventional radiology, 1 patient had hepatic artery thrombosis that led to re LT, 1 patient had gastrointestinal PTLD solved by diminishing immunosuppression. One patient achieved operational tolerance 1 year after LT. One patient died due to sepsis 7 months after LT. Median of ICU stay was 6 days, median of total hospitalization was 16 days.
Conclusions: our series shows that pruritus is a common cause of LT in patients with ALGS, outcomes are comparable to other etiologies and LT is a therapeutic alternative in this group of cholestatic liver diseases.