Long-term outcomes after pre-emptive liver transplantation in primary hyperoxaluria type 1.
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 c...
| Publicado en: | Pediatric Nephrology Vol. 38; no. 6; pp. 1811 - 1821 |
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| Autores principales: | , , , , , , , |
| Formato: | research tables/charts Journal Article |
| Publicado: |
Springer Nature
Jun2023
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| Acceso en línea: | Ver este registro en EBSCOhost |
| fields | @attributes: recordID: 1 pdfLink: plink: https://search.ebscohost.com/login.aspx?direct=true&db=ccm&AN=163449769&site=ehost-live header: @attributes: shortDbName: ccm uiTerm: 163449769 longDbName: CINAHL Complete uiTag: AN controlInfo: bkinfo: dissinfo: jinfo: jid: 0931041X EF1 jtl: Pediatric Nephrology issn: 0931041X maglogo: N pubinfo: dt: Jun2023 vid: 38 iid: 6 pid: 237 pub: Springer Nature place: New York, New York artinfo: ui: 163449769 163449769 163449769 10.1007/s00467-022-05803-y 163449769 ppf: 1811 ppct: 10 formats: fmt: – @attributes: type: T – @attributes: type: P tig: atl: Long-term outcomes after pre-emptive liver transplantation in primary hyperoxaluria type 1. aug: au: Shasha-Lavsky, Hadas Avni, Aviv Paz, Ziv Kalfon, Limor Dror, Amiel A. Yakir, Orly Zaccai, Tzipora Falik Weissman, Irith affil: Pediatric Nephrology Unit, Galilee Medical Center, Nahariya, Israel sug: subj: Liver Transplantation Carbohydrate Metabolism, Inborn Errors Surgery Treatment Outcomes Renal Insufficiency, Chronic Prevention and Control Early Intervention Oxalic Acids Urine Human Retrospective Design Conservative Treatment Comparative Studies Descriptive Statistics Glomerular Filtration Rate Renal Replacement Therapy Renal Insufficiency, Chronic Therapy Renal Insufficiency, Chronic Mortality Child Adolescence Adult Child: 6-12 years Adolescent: 13-18 years Adult: 19-44 years ab: 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. pubtype: Academic Journal doctype: research tables/charts Journal Article ougenre: Article language: English refInfo: holdings: @attributes: islocal: N |
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