title: An institutional experience of pre-emptive liver transplantation for pediatric primary hyperoxaluria type 1.
abstract: Primary hyperoxaluria type 1 (PH1) is an inherited metabolic disease that culminates in ESRF. Pre-emptive liver transplantation (pLTx) treats the metabolic defect and avoids the need for kidney transplantation (KTx). An institutional experience of pediatric PH1 LTx is reported and compared to the literature. Between 2004 and 2015, eight children underwent pLTx for PH1. Three underwent pLTx with a median GFR of 40 (30-46) mL/min/1.73 m(2) and five underwent sequential combined liver-kidney transplantation (cLKTx); all were on RRT at the time of cLKTx. In one case of pLTx, KTx was required eight and a half yr later. pLTx was performed in older (median 8 vs. 2 yr) and larger children (median 27 vs. 7.75 kg) that had a milder PH1 phenotype. In pediatric PH1, pLTx, ideally, should be performed before renal and extrarenal systemic oxalosis complications have occurred, and pLTx can be used "early" or "late." Early is when renal function is preserved with the aim to avoid renal replacement. However, in late (GFR < 30 mL/min/1.73 m(2) ), the aim is to stabilize renal function and delay the need for KTx. Ultimately, transplant strategy depends on PH1 phenotype, disease stage, child size, and organ availability.
authors.affiliation: Institute of Liver Studies, King's College Hospital NHS Foundation Trust, London, UK.
authors.forename: Shirin Elizabeth
authors.identifier: 0000-0003-1624-4467
authors.initials: SE
authors.lastname: Khorsandi
authors.affiliation: Institute of Liver Studies, King's College Hospital NHS Foundation Trust, London, UK.
authors.forename: Marianne
authors.initials: M
authors.lastname: Samyn
authors.affiliation: Institute of Liver Studies, King's College Hospital NHS Foundation Trust, London, UK.
authors.forename: Akhila
authors.initials: A
authors.lastname: Hassan
authors.affiliation: Institute of Liver Studies, King's College Hospital NHS Foundation Trust, London, UK.
authors.forename: Hector
authors.initials: H
authors.lastname: Vilca-Melendez
authors.affiliation: Evelina London Children's Hospital, St Thomas's Hospital, London, UK.
authors.forename: Simon
authors.initials: S
authors.lastname: Waller
authors.affiliation: Great Ormond Street Hospital, London, UK.
authors.forename: Rukshana
authors.initials: R
authors.lastname: Shroff
authors.affiliation: Evelina London Children's Hospital, St Thomas's Hospital, London, UK.|Great Ormond Street Hospital, London, UK.
authors.forename: Geoff
authors.initials: G
authors.lastname: Koffman
authors.affiliation: Great Ormond Street Hospital, London, UK.
authors.forename: William
authors.initials: W
authors.lastname: Van't Hoff
authors.affiliation: Institute of Liver Studies, King's College Hospital NHS Foundation Trust, London, UK.
authors.forename: Alastair
authors.initials: A
authors.lastname: Baker
authors.affiliation: Institute of Liver Studies, King's College Hospital NHS Foundation Trust, London, UK.
authors.forename: Anil
authors.initials: A
authors.lastname: Dhawan
authors.affiliation: Institute of Liver Studies, King's College Hospital NHS Foundation Trust, London, UK.
authors.forename: Nigel
authors.initials: N
authors.lastname: Heaton
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D006960:Hyperoxaluria, Primary / Q000150:complications / Q000601:surgery*; D007223:Infant; D007676:Kidney Failure, Chronic / Q000209:etiology / Q000517:prevention & control*; D016030:Kidney Transplantation; D016031:Liver Transplantation; D008297:Male; D012189:Retrospective Studies; D013997:Time Factors; D016896:Treatment Outcome
keywords: liver transplantation; pediatric kidney transplant; pediatric liver transplantation; primary hyperoxaluria