58 - Treatment of Congenital and Infantile Nephrotic Syndrome: Report from the Pediatric Nephrology Research Consortium (PNRC)
Sunday, April 24, 2022
3:30 PM – 6:00 PM US MT
Poster Number: 58 Publication Number: 58.340
Alex R. Constantinescu, Charles E. Schmidt College of Medicine at Florida Atlantic University, Hollywood, FL, United States; Tej K. Mattoo, Wayne State University School of Medicine, Detroit, MI, United States; Larry A. Greenbaum, Emory University, ATLANTA, GA, United States; William E. Smoyer, Nationwide Children's Hospital, Delaware, OH, United States; Jianli Niu, Joe DiMaggio Children's Hospital at Memorial Regional Hospital, Hollywood, FL, United States; Scott E. Wenderfer, Baylor College of Medicine, Houston, TX, United States; Noel Howard, Texas Tech University Health Sciences Center School of Medicine, Amarillo, TX, United States; Melissa Muff-Luett, Children's Hospital & Medical Center, Omaha, NE, United States; Ali Annaim, Medical University of South Carolina, Charleston, SC, United States; Elizabeth B. Benoit, Boston Children's Hospital, Boston, MA, United States; Avram Traum, Boston Children's Hospital, Boston, MA, United States; Emilee Plautz, University of Minnesota Medical School, Minneapolis, MN, United States; Michelle Rheault, University of Minnesota Masonic Children's Hospital, Minneapolis, MN, United States; Robert L. Myette, Children's Hospital of Eastern Ontario & Ottawa Hospital Research Institute, Ottawa, ON, Canada; Katherine Twombley, Medical University of South Carolina College of Medicine, Charleston, SC, United States; Belkis Wandique-Rapalo, Joe DiMaggio Children's Hospital at Memorial Regional Hospital, Hollywood, FL, United States; Mahmoud Kallash, Nationwide Children's Hospital, Columbus, OH, United States; TETYANA L. VASYLYEVA, USA, AMARILLO, TX, United States
Vice President, Clinical and Translational Research Nationwide Children's Hospital Bexley, Ohio, United States
Background: Nephrotic syndrome (NS) presenting in the first year of life is defined as congenital (CNS) if it occurs in the first 3 months of life, or infantile (INS) if it occurs between 3 and 12 months of life. This age-based classification is necessary because of the differences in etiology, management and the clinical outcomes.
Objective: The aim of our study was to identify and compare the distinct management differences between children with CNS and INS in North America.
Design/Methods: Eleven PNRC sites participated in the study, using retrospective chart review of patients from 1998 to 2019. The data collected included demographic details, eGFR, type and timing of nephrectomy (Nx), requirements for albumin infusions before RRT, and approach to RRT, with dialysis or pre-emptive kidney transplantation (KT).
Results: The study included data from 49 patients with CNS (60% females) and 20 with INS (55% females). Median age at diagnosis was 1 month for CNS and 6 months for INS. eGFR, estimated by modified Schwartz’ formula, and serum albumin concentrations can be seen in Figure 1. IV albumin 6 months preceding RRT was recorded in 25 of 49 (51%) with CNS and in 4 of 20 (25%) with INS (χ2 =5.75; p=.016) (Figure 2). Nx was performed in 38 of 49 (78%) cases with CNS and in 10 of 20 (50%) with INS (χ2 =5.09; p=.024) (Figure 2). It was unilateral in 7 cases with CNS and in 2 with INS, and bilateral in 31 and 8 cases (p=.909), respectively. Nx was preceded by IV albumin in 22 of 27 (81%) with CNS and only 3 of 7 (43%) with INS (χ2=4.26; p=.039). Dialysis was initiated in 36 of 49 (73%) children with CNS, mostly associated with bilateral Nx followed by KT, and in 11 of 20 (55%) with INS (p=.135). Pre-emptive KT was recorded in 3 of 36 (8%) with CNS and 1 of 10 (10%) with INS, respectively. Notably, 8 of 49 (16%) children with CNS did not require nephrectomy or RRT, compared to 8 of 20 (40%) with INS (χ2=4.47; p=.035).Conclusion(s): Children with CNS needed more aggressive management with Nx and RRT compared to children with INS, as their requirement for IV albumin was significantly higher in the 6 months preceding RRT. Despite the variability in the care of children with CNS/INS across North America, an increasing percentage are now being managed without either nephrectomy or RRT. These findings challenge the historical assumption that Nx and RRT are needed for successful management of children with NS in the first year of life, particularly the CNS. Figure 1. eGFR and serum albumin concentration at Dx, 12 months post-Dx, and at initiation of RRT.Dx = diagnosis; RRT = renal replacement therapy Figure 2. The number of patients with Nx and albumin infusions before RRT.Nx = nephrectomy; RRT = renal replacement therapy