Transplantation procedures in children with primary hyperoxaluria type 1: outcome and longitudinal growth.
BACKGROUND
Cure of the metabolic defect in primary hyperoxaluria type 1 (PH1) is possible with liver transplantation (LTx). Preemptive LTx (PLTx) was promoted to prevent chronic kidney disease due to nephrocalcinosis and urolithiasis. However, timing of this procedure is difficult in view of the heterogeneity of PH1 and effective conservative treatment. Combined liver-kidney transplantation (LKTx) is able to cure metabolic defect and replace renal function at the same time and is effective and indicated for patients with or approaching end-stage renal disease (ESRD). Sometimes a sequential approach for LKTx (first liver, then kidney) has been recommended.


METHODS
We report on 13 patients with PH1 since 1995 who underwent transplantation procedures in our center for PH1 at a median age of 4.7 (range 1.4-8.9) years.


RESULTS
The first two patients, planned for a sequential strategy, died early after LTx because of infectious complications. Four patients underwent PLTx at a median glomerular filtration rate of 65 (range 27-98) mL/min/1.73 m/day (Hoppe et al., Pediatr Nephrol 1996; 10: 488), and three patients still have sufficient residual renal function after a follow-up of median 11.6 years. Seven patients with ESRD received a combined LKTx, including four with infantile oxalosis, and three weighing less than 10 kg. There was no mortality and catch-up growth was observed in most patients.


CONCLUSIONS
In summary and conclusion, transplantation procedures are challenging in PH1, but our results including growth data are encouraging. PLTx remains an option despite the difficulties in timing the procedure. LKTx is indicated for patients with ESRD and is possible even in patients with infantile oxalosis and may improve longitudinal growth.
Department of Pediatrics, Pediatric Gastroenterology and Hepatology, University Medical Centre Hamburg-Eppendorf, Hamburg, Germany. f.brinkert@uke.de
Florian
F
Brinkert
Rainer
R
Ganschow
Knut
K
Helmke
Egmond
E
Harps
Lutz
L
Fischer
Björn
B
Nashan
Bernd
B
Hoppe
Stephanie
S
Kulke
Dirk E
DE
Müller-Wiefel
Markus J
MJ
Kemper
D002648:Child; D002675:Child, Preschool; D005500:Follow-Up Studies; D006128:Growth / Q000502:physiology*; D006801:Humans; D006960:Hyperoxaluria, Primary / Q000601:surgery*; D007223:Infant; D007676:Kidney Failure, Chronic / Q000175:diagnosis; D016031:Liver Transplantation / Q000401:mortality / Q000502:physiology* / Q000706:statistics & numerical data; D008137:Longitudinal Studies; D016019:Survival Analysis; D017741:Survivors; D016896:Treatment Outcome