TY - JOUR
T1 - Management of children with congenital nephrotic syndrome
T2 - challenging treatment paradigms
AU - ESPN Dialysis Working Group
AU - Dufek, Stephanie
AU - Holtta, Tuula
AU - Trautmann, Agnes
AU - Ylinen, Elisa
AU - Alpay, Harika
AU - Ariceta, Gema
AU - Aufricht, Christoph
AU - Bacchetta, Justine
AU - Bakkaloglu, Sevcan A.
AU - Bayazit, Aysun
AU - Cicek, Rumeysa Yasemin
AU - Dursun, Ismail
AU - Duzova, Ali
AU - Ekim, Mesiha
AU - Iancu, Daniela
AU - Jankauskiene, Augustina
AU - Klaus, Günter
AU - Paglialonga, Fabio
AU - Pasini, Andrea
AU - Printza, Nikoleta
AU - Said Conti, Valerie
AU - do Sameiro Faria, Maria
AU - Schmitt, Claus Peter
AU - Stefanidis, Constantinos J.
AU - Verrina, Enrico
AU - Vidal, Enrico
AU - Vondrak, Karel
AU - Webb, Hazel
AU - Zampetoglou, Argyroula
AU - Bockenhauer, Detlef
AU - Edefonti, Alberto
AU - Shroff, Rukshana
PY - 2019/8/1
Y1 - 2019/8/1
N2 - 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.
AB - 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.
KW - bilateral nephrectomies
KW - congenital nephrotic syndrome
KW - genotype–phenotype correlation
KW - management approach, NPHS1
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U2 - 10.1093/ndt/gfy165
DO - 10.1093/ndt/gfy165
M3 - Article
C2 - 30215773
AN - SCOPUS:85078339378
VL - 34
SP - 1369
EP - 1377
JO - Nephrology Dialysis Transplantation
JF - Nephrology Dialysis Transplantation
SN - 0931-0509
IS - 8
ER -