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Abstract: TH-PO233

Real-World Experience with C.E.R.A. in Pediatric Dialysis Patients Fits Modeling-Based Predictions: Results from the IPDN Registries

Session Information

Category: Anemia and Iron Metabolism

  • 202 Anemia and Iron Metabolism: Clinical

Authors

  • Schaefer, Franz S., University of Heidelberg, Heidelberg, Germany
  • Chanu, Pascal, Roche/Genentech, Lyon, France
  • Benner, Laura, Institute of Medical Biometry and Informatics, University of Heidelberg, Heidelberg, Germany
  • Frey, Nicolas, F. Hoffmann-La Roche Ltd, Basel, Switzerland
  • Yap, Hui Kim, National University Hospital, Singapore, Singapore
  • Vondrak, Karel, University Hospital Prague-Motol, Prague 5, Czechia
  • Coccia, Paula A., Hospital Italiano de Buenos Aires, Buenos Aires, Argentina
  • Ha, IL-Soo, Seoul National University Children's Hospital, Seoul, Korea (the Republic of)
  • Büscher, Rainer, University Children’s Hospital, Essen, Germany
  • Billing, Heiko, University Tuebingen, Tuebingen, Germany
  • Schmitt, Claus Peter, Center for Pediatric and Adolescent Medicine, Heidelberg, Germany
  • Samaille, Charlotte, CHRU Lille, Lille, France
  • Warady, Bradley A., Children's Mercy Kansas City , Kansas City, Missouri, United States
Background

The International Pediatric Dialysis Network (IPDN, www.pedpd.org) maintains two global registries with children on chronic peritoneal dialysis (IPPN) and hemodialysis (IPHN). We compared registry data to PK/PD modeling and simulation results to validate the modeling approach for Continuous Erythropoiesis Receptor Activator (C.E.R.A.) in children and to examine the use of C.E.R.A. in pediatric patients (pts).

Methods

Among a total of 3225 (IPPN) and 673 (IPHN) pts registered by April 2018 with at least one update, 126 (IPPN) and 32 (IPHN) pts received C.E.R.A. sc (IPPN) or iv (IPHN) and were included in a retrospective assessment of pts characteristics, efficacy (Hb, ferritin) and safety (hospitalization in past 6 months). A model-based evaluation of clinical trial data was conducted in parallel. Subsequent prospective simulations in pediatric pts were performed, i.e. without adjusting for baseline characteristics of IPDN pts.

Results

The mean age (SD) of pts at last visit was 12.0 (6.4; IPPN) yrs and 14.2 (5.3; IPHN) yrs. With median [Q1;Q3] monthly doses (μg/kg body weight) of 3.0 [2.2;4.9] for IPPN and 1.7 [1.1;3.0] for IPHN, mean (SD) Hb levels (g/dL) were 10.8 (1.7; IPPN) and 10.5 (1.8; IPHN). Mean (SD) ferritin levels (μg/L) were 264 (289; IPPN) and 499 (3339; IPHN). In pts with longitudinal follow-up, Hb and ferritin levels were stable during C.E.R.A. treatment. The most common causes of 132 (IPPN) and 41 (IPHN) reported hospitalizations were hypertension and peritonitis (n=19, both) in IPPN and hypertension (n=7) and pyelonephritis (n=5) in IPHN. Results of prospective simulations were in excellent accordance with observed IPDN data: simulated mean [95% CI] Hb levels (g/dL) were 10.9 [10.5;11.3] for sc and 11.0 [10.6;11.3] for iv. Simulated median [95% CI] doses (μg/kg every 4 weeks) after Hb stabilization were 2.7 [2.0;3.9] for sc and 2.3 [1.6;3.2] for iv.

Conclusion

The real-world outcomes regarding steady state C.E.R.A. doses and Hb responses confirm simulations based on models developed with adult and pediatric clinical trial data. With C.E.R.A. the Hb and ferritin levels were within clinical target ranges in peritoneal and hemodialysis settings. No new safety signals were observed.

Funding

  • Commercial Support