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

• Authors: Larry Greenbaum, Emory University; N Jeck, Philipps Univ; G Klaus, Philipps Univ; M Fila, CHU Hop Arnaud de Villeneuve; C Stoica, Institutul Clinic Fundeni; S Fathallah-Shaykh, University of Alabama Birmingham; A Paredes, Nicklaus Childrens Hosp; L Wickman, CS Mott Childrens Hosp; R Nelson, University of Utah; RD Swinford, Univ Texas Med Sch Houston; CL Abitbol, University of Miami; M Balgradean, Spitalul Clin Urgenta Pentru Copii Maria Sklodows; A Jankauskiene, Vilnius University; A Perrin, Vifor Pharma Management Ltd; M Enoiu, Vifor Pharma Management Ltd; S-Y 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
• Publisher DOI: http://doi.org/10.1007/s00467-020-04805-y
• 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; ASSOCIATION; 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.