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. 2017 Oct;28(10):3055-3065.
doi: 10.1681/ASN.2016101121. Epub 2017 May 31.

Long-Term Outcome of Steroid-Resistant Nephrotic Syndrome in Children

Agnes Trautmann  1 , Sven Schnaidt  2 , Beata S Lipska-Ziętkiewicz  3 , Monica Bodria  4   5 , Fatih Ozaltin  6 , Francesco Emma  7 , Ali Anarat  8 , Anette Melk  9 , Marta Azocar  10 , Jun Oh  11 , Bassam Saeed  12 , Alaleh Gheisari  13 , Salim Caliskan  14 , Jutta Gellermann  15 , Lina Maria Serna Higuita  16 , Augustina Jankauskiene  17 , Dorota Drozdz  18 , Sevgi Mir  19 , Ayse Balat  20 , Maria Szczepanska  21 , Dusan Paripovic  22 , Alexandra Zurowska  23 , Radovan Bogdanovic  24 , Alev Yilmaz  25 , Bruno Ranchin  26 , Esra Baskin  27 , Ozlem Erdogan  28 , Giuseppe Remuzzi  29   30   31 , Agnieszka Firszt-Adamczyk  32 , Elzbieta Kuzma-Mroczkowska  33 , Mieczyslaw Litwin  34 , Luisa Murer  35 , Marcin Tkaczyk  36 , Helena Jardim  37 , Anna Wasilewska  38 , Nikoleta Printza  39 , Kibriya Fidan  40 , Eva Simkova  41 , Halina Borzecka  42 , Hagen Staude  43 , Katharina Hees  2 , Franz Schaefer  44 ; PodoNet Consortium
Affiliations

Long-Term Outcome of Steroid-Resistant Nephrotic Syndrome in Children

Agnes Trautmann et al. J Am Soc Nephrol. 2017 Oct.

Abstract

We investigated the value of genetic, histopathologic, and early treatment response information in prognosing long-term renal outcome in children with primary steroid-resistant nephrotic syndrome. From the PodoNet Registry, we obtained longitudinal clinical information for 1354 patients (disease onset at >3 months and <20 years of age): 612 had documented responsiveness to intensified immunosuppression (IIS), 1155 had kidney biopsy results, and 212 had an established genetic diagnosis. We assessed risk factors for ESRD using multivariate Cox regression models. Complete and partial remission of proteinuria within 12 months of disease onset occurred in 24.5% and 16.5% of children, respectively, with the highest remission rates achieved with calcineurin inhibitor-based protocols. Ten-year ESRD-free survival rates were 43%, 94%, and 72% in children with IIS resistance, complete remission, and partial remission, respectively; 27% in children with a genetic diagnosis; and 79% and 52% in children with histopathologic findings of minimal change glomerulopathy and FSGS, respectively. Five-year ESRD-free survival rate was 21% for diffuse mesangial sclerosis. IIS responsiveness, presence of a genetic diagnosis, and FSGS or diffuse mesangial sclerosis on initial biopsy as well as age, serum albumin concentration, and CKD stage at onset affected ESRD risk. Our findings suggest that responsiveness to initial IIS and detection of a hereditary podocytopathy are prognostic indicators of favorable and poor long-term outcome, respectively, in children with steroid-resistant nephrotic syndrome. Children with multidrug-resistant sporadic disease show better renal survival than those with genetic disease. Furthermore, histopathologic findings may retain prognostic relevance when a genetic diagnosis is established.

Keywords: children; immunosuppression; nephrotic syndrome; outcomes; podocytopathies; steroid resistance.

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Figures

Figure 1.
Figure 1.
Distribution of the selected PodoNet Registry cohort. Selection of patients for IIS response and renal survival analyses from the total PodoNet Registry cohort.
Figure 2.
Figure 2.
Renal survival, analysed by response to IIS, is excellent in children with SRNS achieving full remission following IIS compared to patients being resistant to IIS. Ten-year ESRD-free survival rates were 94% (95% CI, 87% to 97%) in patients achieving full remission, 72% (95% CI, 48% to 86%) in patients with partial remission, and 43% (95% CI, 35% to 51%) in patients resistant to IIS.
Figure 3.
Figure 3.
The analysis of renal survival by disease category showed an excellent long-term outcome in IIS sensitive SRNS patients with sporadic disease occurrence and poor long-term outcome in patients with genetic disease. IIS resistant patients with sporadic disease had a better renal survival compared to patients with genetic disease (patients with partial IIS responsiveness were classified IIS resistant for this analysis).
See this image and copyright information in PMC

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