Combined liver-kidney transplantation for primary hyperoxaluria type 1 in young children

Sally R. Ellis, Sally Anne Hulton, Patrick J. McKiernan, Jean Ville De De Goyet, Deirdre A. Kelly

Research output: Contribution to journalArticle

71 Citations (Scopus)

Abstract

Background. Primary hyperoxaluria type 1 (PH1) is a rare condition in which deficiency of the liver enzyme alanine: glyoxylate aminotransferase leads to renal failure and systemic oxalosis. Combined liver-kidney transplantation (LKT) is recommended for end-stage renal failure (ESRF) in adults, but management of infants and young children is controversial. We retrospectively reviewed six children who underwent LKT for PH1. Methods. The median age at diagnosis was 1.8 years (range 3 weeks to 7 years). Two children presented with severe infantile oxalosis at 3 and 9 weeks, five patients had ESRF with nephrocalcinosis and systemic oxalosis, (median duration of dialysis 1.3 years), and one had progressive chronic renal failure. Four children underwent combined LKT, one child staged liver then kidney, and one infant had an isolated liver transplant. The median age at transplantation was 8.9 years (range 1.7-15 years). Results. Overall patient survival was four out of six. The two infants with PH1 and severe systemic oxalosis died (2 and 3 weeks post-transplant) due to cardiovascular oxalosis and sepsis. The other four children are well at median follow-up of 10 months (range 6 months to 7.4 years). No child developed hepatic rejection and all have normal liver function. Renal rejection occurred in three patients. Despite maximal medical management, oxalate deposits recurred in all renal grafts, contributing to graft loss in one (one of the infants who died), and significant dysfunction requiring haemodialysis post-transplant for 6 months. Conclusions. LKT is effective therapy for primary oxalosis with ESRF but has a high morbidity and mortality rate in children who present in infancy with nephrocalcinosis and systemic oxalosis. We feel that earlier LKT, or pre-emptive liver transplantation, may be a better therapeutic strategy to improve the outlook for these patients.

Original languageEnglish
Pages (from-to)348-354
Number of pages7
JournalNephrology Dialysis Transplantation
Volume16
Issue number2
Publication statusPublished - 2001

Fingerprint

Liver Transplantation
Kidney Transplantation
Hyperoxaluria
Chronic Kidney Failure
Transplants
Liver
Nephrocalcinosis
Kidney
Primary Hyperoxaluria
Oxalates
Primary hyperoxaluria type 1
Renal Insufficiency
Renal Dialysis
Dialysis
Sepsis
Transplantation
Morbidity
Survival
Mortality
Enzymes

Keywords

  • Kidney
  • Liver
  • Nephrocalcinosis
  • Primary hyperoxaluria
  • Systemic oxalosis
  • Transplantation

ASJC Scopus subject areas

  • Nephrology
  • Transplantation

Cite this

Ellis, S. R., Hulton, S. A., McKiernan, P. J., De Goyet, J. V. D., & Kelly, D. A. (2001). Combined liver-kidney transplantation for primary hyperoxaluria type 1 in young children. Nephrology Dialysis Transplantation, 16(2), 348-354.

Combined liver-kidney transplantation for primary hyperoxaluria type 1 in young children. / Ellis, Sally R.; Hulton, Sally Anne; McKiernan, Patrick J.; De Goyet, Jean Ville De; Kelly, Deirdre A.

In: Nephrology Dialysis Transplantation, Vol. 16, No. 2, 2001, p. 348-354.

Research output: Contribution to journalArticle

Ellis, SR, Hulton, SA, McKiernan, PJ, De Goyet, JVD & Kelly, DA 2001, 'Combined liver-kidney transplantation for primary hyperoxaluria type 1 in young children', Nephrology Dialysis Transplantation, vol. 16, no. 2, pp. 348-354.
Ellis SR, Hulton SA, McKiernan PJ, De Goyet JVD, Kelly DA. Combined liver-kidney transplantation for primary hyperoxaluria type 1 in young children. Nephrology Dialysis Transplantation. 2001;16(2):348-354.
Ellis, Sally R. ; Hulton, Sally Anne ; McKiernan, Patrick J. ; De Goyet, Jean Ville De ; Kelly, Deirdre A. / Combined liver-kidney transplantation for primary hyperoxaluria type 1 in young children. In: Nephrology Dialysis Transplantation. 2001 ; Vol. 16, No. 2. pp. 348-354.
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abstract = "Background. Primary hyperoxaluria type 1 (PH1) is a rare condition in which deficiency of the liver enzyme alanine: glyoxylate aminotransferase leads to renal failure and systemic oxalosis. Combined liver-kidney transplantation (LKT) is recommended for end-stage renal failure (ESRF) in adults, but management of infants and young children is controversial. We retrospectively reviewed six children who underwent LKT for PH1. Methods. The median age at diagnosis was 1.8 years (range 3 weeks to 7 years). Two children presented with severe infantile oxalosis at 3 and 9 weeks, five patients had ESRF with nephrocalcinosis and systemic oxalosis, (median duration of dialysis 1.3 years), and one had progressive chronic renal failure. Four children underwent combined LKT, one child staged liver then kidney, and one infant had an isolated liver transplant. The median age at transplantation was 8.9 years (range 1.7-15 years). Results. Overall patient survival was four out of six. The two infants with PH1 and severe systemic oxalosis died (2 and 3 weeks post-transplant) due to cardiovascular oxalosis and sepsis. The other four children are well at median follow-up of 10 months (range 6 months to 7.4 years). No child developed hepatic rejection and all have normal liver function. Renal rejection occurred in three patients. Despite maximal medical management, oxalate deposits recurred in all renal grafts, contributing to graft loss in one (one of the infants who died), and significant dysfunction requiring haemodialysis post-transplant for 6 months. Conclusions. LKT is effective therapy for primary oxalosis with ESRF but has a high morbidity and mortality rate in children who present in infancy with nephrocalcinosis and systemic oxalosis. We feel that earlier LKT, or pre-emptive liver transplantation, may be a better therapeutic strategy to improve the outlook for these patients.",
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