ESPN 51th Annual Meeting

ESPN 2018

Is combined liver-kidney transplantation a good indication in autosomal recessive polycystic kidney disease ?
Guillaume Dorval 1 Florence Lacaille 2 Laurence Heidet 1 Dominique Debray 2 Pauline Krug 1 Muriel Girard 2 Marina Charbit 1 Saoussen Krid 1 Nathalie Biebuyck 1 Christophe Chardot 3 Remi Salomon 1 Olivia Boyer 1

1- Necker-Enfants Malades Hospital, Department of Pediatric Nephrology, MARHEA, Paris, France
2- Necker-Enfants Malades Hospital, Department of Gastroenterology, Hepatology and Nutrition, Paris, France
3- Necker-Enfants Malades Hospital, Department of Pediatric Surgery, Paris, France

Autosomal Recessive Polycystic Kidney  Disease (AR-PKD) is aciliopathy caused by mutations  in  the PKHD1 gene. It is characterized by congenital liver fibrosis and cystic kidney disease. The objectives of this study were to describe the evolutionof renal and hepatic function in children with AR-PKD and to discuss indications for renal or combined liver-kidney transplantation.

Material and methods:

We retrospectively analyzed clinico-biological data from 25 children with AR-PKD and followed at Necker Childrens Hospital since 1985.


In 24% of children, the diagnosis was suspected on prenatal ultrasonography. For the others, the median age at diagnosis was 1.8 ± 0.67 years. At diagnosis, 60% had isolated renal features, 8% liver features only, and 32% exhibited both liver and kidney features. Thirteen patients were < 1 year old, and presented the same clinico-biological characteristics as the others, apart from more frequent arterial hypertension (p = 0.01). During the follow-up, hepatic features were mainly portal hypertension (64%, n = 16) with oesophageal varices in 48% (n = 12). Four patients presented with acute cholangitis, and only one experienced more than 3 episodes / year. None presented a chronic elevation of transaminases or hepatocellular insufficiency. At the last follow-up (FU) (mean FU of 11.1 ± 1.5 years), 8 patients (32%) had stage 1-2 chronic kidney disease (CKD) (mean FU of 6.7 ± 2.4 years), 7 had stage 3, and 4 had stage 4-5. Six children (24%) received a kidney transplant with a mean age at first transplantation of 9.3 ± 2.6 years. No synchronous or asynchronous combined transplantation was reported during childhood. However one 20 years-old patient received a preemptive combined liver-kidney transplantation due to
repetitive cholangitis. Nineteen children (76%) were treated for high blood pressure at thelast follow-up, or at the last pre-transplant consultation.


Liver survival in patients with AR-PKD is good in our cohort at least until adolescence. Combined liver-kidney transplantation seems to be very rarely indicated during
childhood in this pathology. The evolution of children with early manifestations (< 1 year) is similar apart from a higher frequency of arterial hypertension.