Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2020 Sep 29;10(1):16025.
doi: 10.1038/s41598-020-71956-1.

Severe neurological outcomes after very early bilateral nephrectomies in patients with autosomal recessive polycystic kidney disease (ARPKD)

Kathrin Burgmaier  1 , Gema Ariceta  2 , Martin Bald  3 , Anja Katrin Buescher  4 , Mathias Burgmaier  5 , Florian Erger  6   7 , Michaela Gessner  8 , Ibrahim Gokce  9 , Jens König  10 , Claudia Kowalewska  11 , Laura Massella  12 , Antonio Mastrangelo  13 , Djalila Mekahli  14   15 , Lars Pape  4   16 , Ludwig Patzer  17 , Alexandra Potemkina  18 , Gesa Schalk  19 , Raphael Schild  20 , Rukshana Shroff  21 , Maria Szczepanska  22 , Katarzyna Taranta-Janusz  23 , Marcin Tkaczyk  24 , Lutz Thorsten Weber  1 , Elke Wühl  25 , Donald Wurm  26 , Simone Wygoda  27 , Ilona Zagozdzon  28 , Jörg Dötsch  1 , Jun Oh  20 , Franz Schaefer  25 , Max Christoph Liebau  29   30 ; ARegPKD consortium
Collaborators, Affiliations

Severe neurological outcomes after very early bilateral nephrectomies in patients with autosomal recessive polycystic kidney disease (ARPKD)

Kathrin Burgmaier et al. Sci Rep. .

Abstract

To test the association between bilateral nephrectomies in patients with autosomal recessive polycystic kidney disease (ARPKD) and long-term clinical outcome and to identify risk factors for severe outcomes, a dataset comprising 504 patients from the international registry study ARegPKD was analyzed for characteristics and complications of patients with very early (≤ 3 months; VEBNE) and early (4-15 months; EBNE) bilateral nephrectomies. Patients with very early dialysis (VED, onset ≤ 3 months) without bilateral nephrectomies and patients with total kidney volumes (TKV) comparable to VEBNE infants served as additional control groups. We identified 19 children with VEBNE, 9 with EBNE, 12 with VED and 11 in the TKV control group. VEBNE patients suffered more frequently from severe neurological complications in comparison to all control patients. Very early bilateral nephrectomies and documentation of severe hypotensive episodes were independent risk factors for severe neurological complications. Bilateral nephrectomies within the first 3 months of life are associated with a risk of severe neurological complications later in life. Our data support a very cautious indication of very early bilateral nephrectomies in ARPKD, especially in patients with residual kidney function, and emphasize the importance of avoiding severe hypotensive episodes in this at-risk cohort.

PubMed Disclaimer

Conflict of interest statement

ML has received honoraria for scientific lectures from Pfizer. Representing the University Hospital of Cologne ML has been counselling Otsuka in an advisory board. DM, represented by KU Leuven University, received an educational grant from Otsuka and participated in an advisory board. The other authors declare no potential conflict of interest.

Figures

Figure 1
Figure 1
Kaplan–Meier survival without severe neurological complications by patient group. Censored observations (last documented follow-up or death) are marked with a cross. P values were derived using log rank tests. EBNE early bilateral nephrectomies, TKV total kidney volume, VEBNE very early bilateral nephrectomies, VED very early dialysis.
See this image and copyright information in PMC

References

    1. Ward CJ, Hogan MC, Rossetti S, Walker D, Sneddon T, Wang X, et al. The gene mutated in autosomal recessive polycystic kidney disease encodes a large, receptor-like protein. Nat Genet. 2002;30:259–269. doi: 10.1038/ng833. - DOI - PubMed
    1. Onuchic LF, Furu L, Nagasawa Y, Hou X, Eggermann T, Ren Z, et al. PKHD1, the polycystic kidney and hepatic disease 1 gene, encodes a novel large protein containing multiple immunoglobulin-like plexin-transcription-factor domains and parallel beta-helix 1 repeats. Am J Hum Genet. 2002;70:1305–1317. doi: 10.1086/340448. - DOI - PMC - PubMed
    1. Bergmann C, Guay-Woodford LM, Harris PC, Horie S, Peters DJM, Torres VE. Polycystic kidney disease. Nat Rev Dis Primer. 2018;4:50. doi: 10.1038/s41572-018-0047-y. - DOI - PMC - PubMed
    1. Lu H, Galeano MCR, Ott E, Kaeslin G, Kausalya PJ, Kramer C, et al. Mutations in DZIP1L, which encodes a ciliary-transition-zone protein, cause autosomal recessive polycystic kidney disease. Nat Genet. 2017;49:1025–1034. doi: 10.1038/ng.3871. - DOI - PMC - PubMed
    1. Zurowska AM, Fischbach M, Watson AR, Edefonti A, Stefanidis CJ, European Paediatric Dialysis Working Group Clinical practice recommendations for the care of infants with stage 5 chronic kidney disease (CKD5) Pediatr. Nephrol. Berl. Ger. 2013;28:1739–1748. doi: 10.1007/s00467-012-2300-z. - DOI - PMC - PubMed

Publication types