Safety and efficacy of sucroferric oxyhydroxide in pediatric patients with chronic kidney disease

• Authors: Larry Greenbaum, Emory University; Nikola Jeck, Philipps University; Günter Klaus, Philipps University; Marc Fila, CHU Hop Arnaud de Villeneuve; Cristina Stoica, Institutul Clinic Fundeni; Sahar Fathallah-Shaykh, University of Alabama Birmingham; Ana Paredes, Nicklaus Childrens Hospital; Larysa Wickman, CS Mott Childrens Hospital; Raoul Nelson, University of Utah; Rita D. Swinford, University of Texas Medical School at Houston; Carolyn Larkins Abitbol, University of Miami; Mihaela Balgradean, Spitalul Clin Urgenta Pentru Copii Maria Sklodows; Augustina Jankauskiene, Vilnius University; Amandine Perrin, Vifor Pharma Management Ltd; Milica Enoiu, Vifor Pharma Management Ltd; Sun-Young Ahn, George Washington University
• Journal Title: Pediatric Nephrology
• Volume: Volume 36, Number 5
• Publisher: Springer | 2020-10-27, Pages 1233-1244
• Type of Work: Article | Final Publisher PDF
• Publisher DOI: http://doi.org/10.1007/s00467-020-04805-y
• Subjects: Health Sciences, Human Development; Health Sciences, Health Care Management; Health Sciences, Public Health
• Keywords: Science & Technology; Life Sciences & Biomedicine; Pediatrics; Urology & Nephrology; Chronic kidney disease; Hyperphosphatemia; Children; Phosphate binder; Sucroferric oxyhydroxide; Safety profile; Oral calcium carbonate; Phosphate binder; Hemodialysis patients; Lanthanum deposition; Serum phosphorus; Mortality risk; Young adults; Children; Metabolism

Abstract

Background Pediatric patients with advanced chronic kidney disease (CKD) are often prescribed oral phosphate binders (PBs) for the management of hyperphosphatemia. However, available PBs have limitations, including unfavorable tolerability and safety. Methods This phase 3, multicenter, randomized, open-label study investigated safety and efficacy of sucroferric oxyhydroxide (SFOH) in pediatric and adolescent subjects with CKD and hyperphosphatemia. Subjects were randomized to SFOH or calcium acetate (CaAc) for a 10-week dose titration (stage 1), followed by a 24-week safety extension (stage 2). Primary efficacy endpoint was change in serum phosphorus from baseline to the end of stage 1 in the SFOH group. Safety endpoints included treatment-emergent adverse events (TEAEs). Results Eighty-five subjects (2–18 years) were randomized and treated (SFOH, n = 66; CaAc, n = 19). Serum phosphorus reduction from baseline to the end of stage 1 in the overall SFOH group (least squares [LS] mean ± standard error [SE]) was − 0.488 ± 0.186 mg/dL; p = 0.011 (post hoc analysis). Significant reductions in serum phosphorus were observed in subjects aged ≥ 12 to ≤ 18 years (LS mean ± SE − 0.460 ± 0.195 mg/dL; p = 0.024) and subjects with serum phosphorus above age-related normal ranges at baseline (LS mean ± SE − 0.942 ± 0.246 mg/dL; p = 0.005). Similar proportions of subjects reported ≥ 1 TEAE in the SFOH (75.8%) and CaAc (73.7%) groups. Withdrawal due to TEAEs was more common with CaAc (31.6%) than with SFOH (18.2%). Conclusions SFOH effectively managed serum phosphorus in pediatric patients with a low pill burden and a safety profile consistent with that reported in adult patients.