Burgmaier K, Kilian S, Bammens B, Benzing T, Billing H, Buescher A, Galiano M, Grundmann F, Klaus G, Mekahli D, Michel-Calemard L, Milosevski-Lomic G, Ranchin B, Sauerstein K, Schaefer S, Shroff R, Sterenborg R, Verbeeck S, Weber LT, Wicher D, Wuehl E, Doetsch J, Schaefer F, Liebau MC (2019)
Publication Type: Journal article
Publication year: 2019
Book Volume: 9
Pages Range: 7919-
Journal Issue: 1
DOI: 10.1038/s41598-019-43488-w
Autosomal recessive polycystic kidney disease (ARPKD) is a severe pediatric hepatorenal disorder with pronounced phenotypic variability. A substantial number of patients with early diagnosis reaches adulthood and some patients are not diagnosed until adulthood. Yet, clinical knowledge about adult ARPKD patients is scarce. Here, we describe forty-nine patients with longitudinal follow-up into young adulthood that were identified in the international ARPKD cohort study ARegPKD. Forty-five patients were evaluated in a cross-sectional analysis at a mean age of 21.4 (±3.3) years describing hepatorenal findings. Renal function of native kidneys was within CKD stages 1 to 3 in more than 50% of the patients. Symptoms of hepatic involvement were frequently detected. Fourteen (31%) patients had undergone kidney transplantation and six patients (13%) had undergone liver transplantation or combined liver and kidney transplantation prior to the visit revealing a wide variability of clinical courses. Hepatorenal involvement and preceding complications in other organs were also evaluated in a time-to-event analysis. In summary, we characterize the broad clinical spectrum of young adult ARPKD patients. Importantly, many patients have a stable renal and hepatic situation in young adulthood. ARPKD should also be considered as a differential diagnosis in young adults with fibrocystic hepatorenal disease.
APA:
Burgmaier, K., Kilian, S., Bammens, B., Benzing, T., Billing, H., Buescher, A.,... Liebau, M.C. (2019). Clinical courses and complications of young adults with Autosomal Recessive Polycystic Kidney Disease (ARPKD). Scientific Reports, 9(1), 7919-. https://doi.org/10.1038/s41598-019-43488-w
MLA:
Burgmaier, Kathrin, et al. "Clinical courses and complications of young adults with Autosomal Recessive Polycystic Kidney Disease (ARPKD)." Scientific Reports 9.1 (2019): 7919-.
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