Surgical Management of Pediatric Primary Hyperoxaluria Type 1: An Eight-Patient Case Series in the Pre-siRNA Era.

ABSTRACT:

Background

Primary hyperoxaluria type 1 (PH1) is a rare autosomal recessive disorder that leads to systemic oxalosis and end-stage renal disease (ESRD). Before the advent of siRNA therapy, liver transplantation, often combined with kidney transplantation, was the only definitive treatment.

Methods

We conducted a retrospective review of eight pediatric patients with PH1 who underwent liver transplantation at a single tertiary care institution between 1998 and 2018. Clinical records were reviewed to extract data on transplant strategy, timing and modality of dialysis, surgical technique, and long-term outcomes. Histopathological and imaging data were used to assess systemic oxalate deposition.

Results

Five patients underwent sequential liver-kidney transplantation (seqL

SUBMITTER: Finer G 

PROVIDER: S-EPMC12618270 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature