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Management of children with congenital nephrotic syndrome: challenging treatment paradigms

S. Dufek, T. Holtta, A. Trautmann, E. Ylinen, H. Alpay, G. Ariceta, C. Aufricht, J. Bacchetta, SA. Bakkaloglu, A. Bayazit, RY. Cicek, I. Dursun, A. Duzova, M. Ekim, D. Iancu, A. Jankauskiene, G. Klaus, F. Paglialonga, A. Pasini, N. Printza, V....

. 2019 ; 34 (8) : 1369-1377. [pub] 20190801

Jazyk angličtina Země Velká Británie

Typ dokumentu časopisecké články, multicentrická studie

Perzistentní odkaz   https://www.medvik.cz/link/bmc20023796

Grantová podpora
CDF-2016-09-038 Department of Health - United Kingdom

BACKGROUND: Management of children with congenital nephrotic syndrome (CNS) is challenging. Bilateral nephrectomies followed by dialysis and transplantation are practiced in most centres, but conservative treatment may also be effective. METHODS: We conducted a 6-year review across members of the European Society for Paediatric Nephrology Dialysis Working Group to compare management strategies and their outcomes in children with CNS. RESULTS: Eighty children (50% male) across 17 tertiary nephrology units in Europe were included (mutations in NPHS1, n = 55; NPHS2, n = 1; WT1, n = 9; others, n = 15). Excluding patients with mutations in WT1, antiproteinuric treatment was given in 42 (59%) with an increase in S-albumin in 70% by median 6 (interquartile range: 3-8) g/L (P < 0.001). Following unilateral nephrectomy, S-albumin increased by 4 (1-8) g/L (P = 0.03) with a reduction in albumin infusion dose by 5 (2-9) g/kg/week (P = 0.02). Median age at bilateral nephrectomies (n = 29) was 9 (7-16) months. Outcomes were compared between two groups of NPHS1 patients: those who underwent bilateral nephrectomies (n = 25) versus those on conservative management (n = 17). The number of septic or thrombotic episodes and growth were comparable between the groups. The response to antiproteinuric treatment, as well as renal and patient survival, was independent of NPHS1 mutation type. At final follow-up (median age 34 months) 20 (80%) children in the nephrectomy group were transplanted and 1 died. In the conservative group, 9 (53%) remained without dialysis, 4 (24%; P < 0.001) were transplanted and 2 died. CONCLUSION: An individualized, stepwise approach with prolonged conservative management may be a reasonable alternative to early bilateral nephrectomies and dialysis in children with CNS and NPHS1 mutations. Further prospective studies are needed to define indications for unilateral nephrectomy.

A and P Kyriakou Children's Hospital Athens Greece

Ankara University Hospital Ankara Turkey

Azienda Ospedaliero Universitaria Sant'Orsola Malpighi Bologna Italy

Center for Nephrology University College London London UK

Center for Pediatric and Adolescent Medicine Heidelberg Germany

Center of Pediatrics Vilnius University Vilnius Lithuania

Centro Materno Infantil do Norte Porto Portugal

Department of Pediatric Nephrology Cerrahpasa Medical Faculty Istanbul Turkey

Department of Pediatric Nephrology Cukurova University Adana Turkey

Department of Pediatric Nephrology Erciyes University Kayseri Turkey

Department of Pediatric Nephrology Gazi University Hospital Ankara Turkey

Department of Pediatric Nephrology University of Helsinki and Helsinki University Hospital Helsinki Finland

Department of Pediatrics Mater Dei Hospital Malta Msida Malta

Department of Pediatrics University Hospital of Padova Padova Italy

Division of Pediatric Nephrology Hacettepe University Faculty of Medicine Sihhiye Ankara Turkey

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan Italy

Great Ormond Street Hospital for Children NHS Foundation Trust London UK

Hippokratio General Hospital Aristotle University Thessaloniki Greece

Hôpital Femme Mère Enfant Lyon France

Hospital MaternoInfantil de la Vall d'Hebron Barcelona Spain

IRCCS Giannina Gaslini Genova Italy

KfH Pediatric Kidney Center Marburg Germany

Medical University of Vienna Vienna Austria

Pediatric Nephrology University Hospital Motol Prague Czech Republic

School of Medicine Marmara University Istanbul Turkey

Citace poskytuje Crossref.org

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