Long-term outcomes after pre-emptive liver transplantation in primary hyperoxaluria type 1

Hadas Shasha-Lavsky, Aviv Avni, Ziv Paz, Limor Kalfon, Amiel A. Dror, Orly Yakir, Tzipora Falik Zaccai, Irith Weissman

Research output: Contribution to journalArticlepeer-review

Abstract

Background: Primary hyperoxaluria type 1 (PH1) is an autosomal recessive disease caused by the liver defect of oxalate metabolism, which leads to kidney failure and systemic manifestations. Until recently, liver transplantation was the only definitive treatment. The timing of liver transplantation can be early, while kidney function is still normal (pre-emptive liver transplantation—PLT), or when the patient reaches stage 5 chronic kidney disease (CKD) and needs combined liver-kidney transplantation. We aimed to determine the long-term kidney outcomes of PLT in PH1 patients. Methods: A retrospective single-center study of PH1 patients who were followed in our center between 1997 and 2017. We compared the kidney outcomes of patients who underwent PLT to those who presented with preserved kidney function and did not undergo PLT. Results: Out of 36 PH1 patients, 18 patients were eligible for PLT (eGFR > 40 mL/min/1.73 m2 at the time of diagnosis). Seven patients underwent PLT (PLT group), while 11 continued conservative treatments (PLTn group). In the PLT group, the median eGFR at the time of PLT and at the end of the follow-up period (14–20 years) was 72 (range 50–89) and 104 (range 86–108) mL/min/1.73 m2, respectively, and no patient died or reached stage 5 CKD. In the PLTn group, eight patients (72.7%) reached stage 5 CKD (median time to kidney replacement therapy was 11 years), and two patients died from disease complications (18.2%). Conclusions: Pre-emptive liver transplantation preserved kidney function in patients with PH1 in our cohort. Early intervention can prevent kidney failure and systemic oxalosis in PH1. Graphical abstract: [Figure not available: see fulltext.].

Original languageEnglish
Pages (from-to)1811-1820
Number of pages10
JournalPediatric Nephrology
Volume38
Issue number6
DOIs
StatePublished - Jun 2023

Bibliographical note

Publisher Copyright:
© 2022, The Author(s), under exclusive licence to International Pediatric Nephrology Association.

Keywords

  • Kidney transplantation
  • Oxalate
  • Pre-emptive liver transplantation (PLT)
  • Primary hyperoxaluria type 1 (PH1)

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