Abstract: TH-PO145

Management of Children With Congenital Nephrotic Syndrome: Challenging Treatment Paradigms

Session Information

Category: Glomerular

  • 1005 Clinical Glomerular Disorders

Authors

  • 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
Background

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.

Methods

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.

Results

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.

Conclusion

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.