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Abstract: TH-PO145

Management of Children With Congenital Nephrotic Syndrome: Challenging Treatment Paradigms

Session Information

Category: Glomerular

  • 1005 Clinical Glomerular Disorders


  • Dufek, Stephanie, Great Ormond Street Hospital for Children NHS Trust, London, United Kingdom
  • Ylinen, Elisa, University Hospital Helsinki, Helsinki, Finland
  • Trautmann, Agnes, Center for Pediatrics and Adolescents Medcine, University Hospital Heidelberg, Heidelberg, Germany
  • Vidal, Enrico, University-Hospital of Padova, Padova, Italy
  • Holtta, Tuula M., University Hospital Helsinki, Helsinki, Finland
  • Shroff, Rukshana, Great Ormond Street Hospital for Children NHS Trust, London, United Kingdom

Group or Team Name

  • On behalf of ESPN Dialysis Working Group

Management of children with congenital nephrotic syndrome (CNS) is challenging and ranges from antiproteinuric treatment to uni- or bilateral nephrectomies followed by dialysis and transplantation. Early bilateral nephrectomies followed by dialysis and transplantation is currently practised in most centres, but conservative treatment may also be effective.


We conducted a 6-year survey across members of the European Society for Paediatric Nephrology Dialysis Working Group to compare management strategies and their outcomes in children with CNS.


81 children (51% male) across 17 tertiary nephrology units in Europe were included (NPHS1 n=57; NPHS n=2, WT1 n=10, others n=12; details of mutations were not examined). Antiproteinuric treatment was given in 48 (59%) with an increase in S-albumin in 68% by median 6 (interquartile range 3-8) g/L (p<0.001) and decrease of weekly albumin infusion dose by median 1 (0-3) g/kg/week (p=0.021). Unilateral nephrectomy (or first kidney removal) was performed in 16 children. In those, S-albumin increased by median 3 (1–7) g/L (p=0.021) and weekly albumin infusion dose decreased by median 4 (0-7) g/kg/week (p=0.018). The median age at bilateral nephrectomy was 9 (7-15) months. Dialysis was initiated in 53 (65%) at a median age of 9 (5.5-15) months, with PD in 91% of children.
Children with NPHS1 mutations and >12 months follow-up were divided into two groups and their outcomes were compared: bilateral nephrectomy (n=26) versus conservative management (no nephrectomy; n = 17). Nephrectomised children presented earlier (3 vs 29 days; p=0.01), with comparable S-albumin (p=0.21) and S-creatinine (p=0.19). There was no difference in the number of septic or thrombotic episodes and growth was comparable. At final follow-up (median age 34 months) 9 (53%) children in the conservative group remained without renal replacement therapy, 4 (24%) received a renal transplant and 2 died. Amongst nephrectomised children 21 (81%; p<0.01) were transplanted and 1 died.


An individualised, stepwise approach, with prolonged conservative management, followed by unilateral nephrectomy may be a reasonable alternative to early bilateral nephrectomies in children with CNS and NPHS1 mutation.