Long-Term Outcome of Steroid-Resistant Nephrotic Syndrome in Children
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Long-Term Outcome of Steroid-Resistant Nephrotic Syndrome in Children. / Trautmann, Agnes; Schnaidt, Sven; Lipska-Ziętkiewicz, Beata S; Bodria, Monica; Ozaltin, Fatih; Emma, Francesco; Anarat, Ali; Melk, Anette; Azocar, Marta; Oh, Jun; Saeed, Bassam; Gheisari, Alaleh; Caliskan, Salim; Gellermann, Jutta; Higuita, Lina Maria Serna; Jankauskiene, Augustina; Drozdz, Dorota; Mir, Sevgi; Balat, Ayse; Szczepanska, Maria; Paripovic, Dusan; Zurowska, Alexandra; Bogdanovic, Radovan; Yilmaz, Alev; Ranchin, Bruno; Baskin, Esra; Erdogan, Ozlem; Remuzzi, Giuseppe; Firszt-Adamczyk, Agnieszka; Kuzma-Mroczkowska, Elzbieta; Litwin, Mieczyslaw; Murer, Luisa; Tkaczyk, Marcin; Jardim, Helena; Wasilewska, Anna; Printza, Nikoleta; Fidan, Kibriya; Simkova, Eva; Borzecka, Halina; Staude, Hagen; Hees, Katharina; Schaefer, Franz; PodoNet Consortium.
In: J AM SOC NEPHROL, Vol. 28, No. 10, 10.2017, p. 3055-3065.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Long-Term Outcome of Steroid-Resistant Nephrotic Syndrome in Children
AU - Trautmann, Agnes
AU - Schnaidt, Sven
AU - Lipska-Ziętkiewicz, Beata S
AU - Bodria, Monica
AU - Ozaltin, Fatih
AU - Emma, Francesco
AU - Anarat, Ali
AU - Melk, Anette
AU - Azocar, Marta
AU - Oh, Jun
AU - Saeed, Bassam
AU - Gheisari, Alaleh
AU - Caliskan, Salim
AU - Gellermann, Jutta
AU - Higuita, Lina Maria Serna
AU - Jankauskiene, Augustina
AU - Drozdz, Dorota
AU - Mir, Sevgi
AU - Balat, Ayse
AU - Szczepanska, Maria
AU - Paripovic, Dusan
AU - Zurowska, Alexandra
AU - Bogdanovic, Radovan
AU - Yilmaz, Alev
AU - Ranchin, Bruno
AU - Baskin, Esra
AU - Erdogan, Ozlem
AU - Remuzzi, Giuseppe
AU - Firszt-Adamczyk, Agnieszka
AU - Kuzma-Mroczkowska, Elzbieta
AU - Litwin, Mieczyslaw
AU - Murer, Luisa
AU - Tkaczyk, Marcin
AU - Jardim, Helena
AU - Wasilewska, Anna
AU - Printza, Nikoleta
AU - Fidan, Kibriya
AU - Simkova, Eva
AU - Borzecka, Halina
AU - Staude, Hagen
AU - Hees, Katharina
AU - Schaefer, Franz
AU - PodoNet Consortium
N1 - Copyright © 2017 by the American Society of Nephrology.
PY - 2017/10
Y1 - 2017/10
N2 - 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.
AB - 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.
KW - Journal Article
U2 - 10.1681/ASN.2016101121
DO - 10.1681/ASN.2016101121
M3 - SCORING: Journal article
C2 - 28566477
VL - 28
SP - 3055
EP - 3065
JO - J AM SOC NEPHROL
JF - J AM SOC NEPHROL
SN - 1046-6673
IS - 10
ER -
