Long-term outcome of steroid-resistant nephrotic syndrome in children
PubMedID: 28566477 ; Weinvestigated the value of genetic, histopathologic, and early treatment response information inprognosing longterm 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>3months and<20 years of age): 612 haddocumentedresponsiveness tointensifiedimmunosuppression (IIS),1155hadkidneybiopsy results, and212had 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 achievedwith 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. Childrenwithmultidrug-resistant sporadic disease showbetter renal survival than thosewith geneticdisease. Furthermore, histopathologic findings may retain prognostic relevance when a genetic diagnosis is established. © 2017 by the American Society of Nephrology. ; Seventh Framework Programme: 2012-305608 European Geosciences Union Deutsche Forschungsgemeinschaft: Scha 477/11-1 Ministry of Education and Science: N402631840 Türkiye Bilimsel ve Teknolojik Araştirma Kurumu: 108S417 Università degli Studi di Parma Celal Bayar Üniversitesi Uniwersytet Medyczny im. Karola Marcinkowskiego w Poznaniu M.S.I. Foundation: Serbia Isfahan University of Medical Sciences Akdeniz Üniversitesi United Arab Emirates University Semmelweis Egyetem: Hungary Ondokuz Mayis Üniversitesi Western University: Ukraine ; The PodoNet project has been made possible by support received from E-Rare (German Ministry of Education and Research), European Union (EU) Seventh Framework Programme (EURenOmics) grant 2012-305608, Polish Ministry of Science and Education grant N402631840, German Research Foundation grant Scha 477/11-1, and Scientific and Technological Research Council of Turkey grant 108S417. ; The PodoNet collaborators were M.A., Lily Quiroz (Roberto del Rio Children's Hospital, Santiago de Chile, Chile), L.M.S.H., Ji?rí Dušek (University Hospital Motol, Prague, Czech Republic), B.R., Michel Fischbach (University Children's Hospital, Strasbourg), Tinatin Davitaia (M.Iashvili Children Central Hospital, Tbilisi, Georgia), J.G., J.O., A.M., F.S., Marianne Wigger (University Children's Hospital, Rostock, Germany), N.P., Peter Sallay (Semmelweis University, Budapest, Hungary), Alaleh Gheissari (Isfahan University of Medical Science, St. Al Zahra Hospital, Isfahan, Iran), Marina Noris (IRCCS - Istituto di Richerche Farmacologiche "Mario Negri" and Azienda Ospedaliera Papa Giovanni XXXI, Bergamo, Italy), Andrea Pasini (S.Orsola-Malpighi Hospital, Bologna, Italy), Gian Marco Ghiggeri (Istituto Giannina Gaslini, Genoa, and University of Parma, Italy), Gianluigi Ardissino (IRCCS Ca'Granda, Osepdale Maggiore Policlinico, Milano, Italy), Elisa Benetti (Hospital of Padua, Italy), F.E., Bilal Aoun (Rafic Hariri University Hospital, Beirut, Lebanon), Pauline Abou-Jaoudé (Notre Dame de Secours University Hospital, Byblos, Lebanon), A.J., A.W., Ewa Gacka (Pediatrics and Oncology Center, Chorzow, Poland), A.Z., D.D., M.T., Malgorzata Stanczyk (Polish Mothers Memorial Hospital Research Institute, Lodz, Poland), H.B., Magdalena Silska (Poznan University of Medical Sciences, Poznan, Poland), Tomasz Urasinski (Pomeranian Academy of Medicine, Szczecin, Poland), A.F.-A., Joanna Ksiazek (Centrum Zdrowia Dziecka, Warsaw, Poland), E.K.-M., Anna Medynska (Medical University, Wroclaw, Poland), M.S., Alberto Caldas Afonso (Hospital S. Joao Pediatrics, Porto, Portugal), H.J., Amira Peco-Antic (University Children's Hospital, Belgrade, Serbia), R.B., Rafael T. Krmar (Karolinska University Hospital, Stockholm, Sweden), Giacomo D. Simonetti (University Children's Hospital, Bern, Switzerland), B.S., A.A., A.B., E.B., Nilgun Cakar (Diskapi Children's Hospital, Ankara, Turkey), O.E., Birsin Özcakar (Ankara University Medical School, Ankara, Turkey), F.O., Onur Sakallioglu (Gulhane Military Academy of Medicine, Ankara, Turkey), Oguz Soylemezoglu (Gazi University Hospital, Ankara, Turkey), Sema Akman (Akdeniz University, Antalya, Turkey), Faysal Gok (Gulhane Military Academy of Medicine, Gulhane, Turkey), S.C., Cengiz Candan (Gaztepe Hospital, Istanbul, Turkey), A.Y., S.M., Ipek Akil (Celal Bayar University, Manisa, Turkey), Pelin Ertan (Celal Bayar University, Manisa, Turkey), Ozan Özkaya (Ondokuz Mayis University, Samsun, Turkey), Mukaddes Kalyoncu (Karadeniz University, Trabzon, Turkey), E.S., Entesar Alhammadi (Al Qassimi Hospital, Sharjah, United Arab Emirates), and Roman Sobko (Western Ukrainian Specialized Medical Center, Lviv, Ukraine).