Long-term kidney and liver outcome in 50 children with autosomal recessive polycystic kidney disease
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ORIGINAL ARTICLE
Long-term kidney and liver outcome in 50 children with autosomal recessive polycystic kidney disease Guillaume Dorval 1,2 & Olivia Boyer 1,2 & Anne Couderc 3 & Jean-Daniel Delbet 4 & Laurence Heidet 1,2 & Dominique Debray 5 & Pauline Krug 1,2 & Muriel Girard 5 & Brigitte Llanas 6 & Marina Charbit 1,2 & Saoussen Krid 1,2 & Nathalie Biebuyck 1,2 & Marc Fila 7 & Cécile Courivaud 8 & Frances Tilley 9 & Nicolas Garcelon 10 & Thomas Blanc 11 & Christophe Chardot 11 & Rémi Salomon 1,2 & Florence Lacaille 5 Received: 1 April 2020 / Revised: 10 September 2020 / Accepted: 1 October 2020 # IPNA 2020
Abstract Background Autosomal recessive polycystic kidney disease (ARPKD) is a rare ciliopathy characterized by congenital hepatic fibrosis and cystic kidney disease. Lack of data about long-term follow-up makes it difficult to discuss timing and type of organ transplantation. Our objectives were to evaluate long-term evolution and indications for transplantation, from birth to adulthood. Methods Neonatal survivors and patients diagnosed in postnatal period with ARPKD between 1985 January and 2017 December from 3 French pediatric centers were retrospectively enrolled in the study. Results Fifty patients with mean follow-up 12.5 ± 1 years were enrolled. ARPKD was diagnosed before birth in 24%, and at mean age 1.8 years in others. Thirty-three patients were < 1 year of age at first symptoms, which were mostly kidney-related. These most often presented high blood pressure during follow-up. Portal hypertension was diagnosed in 29 patients (58%), 4 of them with bleeding from esophageal varices. Eight patients presented cholangitis (> 3 episodes in three children). Liver function was normal in all patients. Nine children received a kidney transplant without liver complications. A 20-year-old patient received a combined liver-kidney transplant (CLKT) for recurrent cholangitis, and a 15-year-old boy an isolated liver transplant for uncontrollable variceal bleeding despite portosystemic shunt. Conclusions Long-term outcome in patients with ARPKD is heterogeneous, and in this cohort did not depend on age at diagnosis except for blood pressure. Few patients required liver transplantation. Indications for liver or combined liver-kidney transplantation were limited to recurrent cholangitis or uncontrollable portal hypertension. Liver complications after kidney transplantation were not significant. Keywords ARPKD . PKHD1 . Polycystic kidney disease . Portal hypertension . Transplantation . Long-term outcome . Children
Abbreviations ALT Alanine aminotransferase AP Alkaline phosphatase AST Aspartate aminotransferase CKD Chronic kidney disease CHF Congenital hepatic fibrosis CLKT Combined liver-kidney transplantation Supplementary Information The online version of this article (https:// doi.org/10.1007/s00467-020-04808-9) contains supplementary material, which is available to authorized users. * Guillaume Dorval [email protected] Extended author information available on the last page of the article
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