Optimizing outcomes for neonatal ARPKD

Abstract

A retrospective analysis was conducted on 10 consecutive cases of neonatal ARPKD, 9 of whom received kidney transplants (KT). All were diagnosed antenatally (n = 6) or at birth. In the first month of life 70% required ventilatory support. Pre-emptive bilateral nephrectomy and peritoneal dialysis (PD) catheter placement were performed in 9 at a mean age of 7.8 +/- 11.9 months. The indications for nephrectomy were massive kidneys, resulting in suboptimal nutrition and respiratory compromise. All patients received assisted enteral nutrition, with significant increase in mean tolerated feeds following nephrectomy (p < 0.05), with increase in mean normalized weight and height (0.92 and 1.2 delta SDS respectively), by one year post-transplantation. KT was performed at a mean age and weight of 2.5 +/- 1.4 years and 13.3 +/- 6.1 kg. The mean creatinine clearance at one year post-KT was 91.3 +/- 38.1 mls/min/1.73 m(2), with a projected graft life expectancy of 18.4 years. Patient survival was 89% and death censored graft survival was 100%, at a mean follow-up of 6.1 +/- 4.5 years post-transplant. Six patients demonstrated evidence of hepatic fibrosis, one of which required liver transplantation. In patients with massive kidneys from ARPKD, pre-emptive bilateral nephrectomy, supportive PD and early aggressive nutrition, can minimize early infant mortality, so that subsequent KT can be performed with excellent patient and graft survival.

Transplantation in autosomal recessive polycystic kidney disease: liver and/or kidney?

“…Fever, right upper quadrant pain, icterus, and abnormal transaminases could indicate cholangitis but often the presentation can be subtle. A significant number of children have succumbed to the complications of biliary disease after transplantation [18][19][20][21]. Extrahepatic biliary disease as the predominant manifestation has also been reported [9].…”

“…It is very difficult to predict the rate of progression of portal hypertension or intrahepatic ductal dilatation. In the case series described from Beunoyer et al, a significant proportion of patients had portal hypertension and cholangitis at baseline [18]. Their group opted to perform isolated kidney transplantation in all of them.…”

Combined paediatric liver-kidney transplantation: Analysis of our experience and literature review

“…Reported data on iKT show patient survival to be 89% and death censored graft survival at 100% at 5 years. [14] However, Khan et al [15] reported on 14 patients who had received iKT in the presence of hepatic disease; 4/5 deaths in this series were due to complications from hepatic disease (systemic sepsis secondary to cholangitis) and 56% of the remaining survivors had complications related to congenital hepatic fibrosis at a mean of 6.3 years post transplantation. Davis et al [13] published a retrospective study of 203 patients who received iKT for similar indications.…”

Other Cystic Kidney Diseases

“…Therefore, renal transplantation is the treatment of choice for ESRD in ARPKD patients, and at least one report advocates preemptive nephrectomy in neonates with markedly enlarged kidneys. 19 Because ARPKD is a recessive disorder, either parent may be a suitable kidney donor. Native nephrectomies may be warranted in patients with massively enlarged kidneys to allow allograft placement.…”