Real-world evidence on the dosing and safety of C.E.R.A. in pediatric dialysis patients: findings from the International Pediatric Dialysis Network registries

Pediatr Nephrol. 2024 Mar;39(3):807-818. doi: 10.1007/s00467-023-05977-z. Epub 2023 Aug 11.

Abstract

Background: This retrospective real-world study used data from two registries, International Pediatric Peritoneal Dialysis Network (IPPN) and International Pediatric Hemodialysis Network (IPHN), to characterize the efficacy and safety of continuous erythropoietin receptor activator (C.E.R.A.) in pediatric patients with chronic kidney disease (CKD) on peritoneal dialysis (PD) or hemodialysis (HD).

Methods: IPPN and IPHN collect prospective data (baseline and every 6 months) from pediatric PD and HD centers worldwide. Demographics, clinical characteristics, dialysis information, treatment, laboratory parameters, number and causes of hospitalization events, and deaths were extracted for patients on C.E.R.A. treatment (IPPN: 2007-2021; IPHN: 2013-2021).

Results: We analyzed 177 patients on PD (median age 10.6 years) and 52 patients on HD (median age 14.1 years) who had ≥ 1 observation while being treated with C.E.R.A. The median (interquartile range [IQR]) observation time under C.E.R.A. exposure was 6 (0-12.5) and 12 (0-18) months, respectively. Hemoglobin concentrations were stable over time; respective means (standard deviation) at last observation were 10.9 (1.7) g/dL and 10.4 (1.7) g/dL. Respective median (IQR) monthly C.E.R.A. doses at last observation were 3.5 (2.3-5.1) µg/kg, or 95 (62-145) µg/m2 and 2.1 (1.2-3.4) µg/kg, or 63 (40-98) µg/m2. Non-elective hospitalizations occurred in 102 (58%) PD and 32 (62%) HD patients. Seven deaths occurred (19.8 deaths per 1000 observation years).

Conclusions: C.E.R.A. was associated with efficient maintenance of hemoglobin concentrations in pediatric patients with CKD on dialysis, and appeared to have a favorable safety profile. The current analysis revealed no safety signals.

Keywords: Chronic kidney disease; Continuous erythropoietin receptor activator; Hemodialysis; Peritoneal dialysis; Real-world study.

MeSH terms

  • Adolescent
  • Child
  • Erythropoietin*
  • Hemoglobins / analysis
  • Humans
  • Kidney Failure, Chronic* / therapy
  • Prospective Studies
  • Registries
  • Renal Dialysis / adverse effects
  • Renal Insufficiency, Chronic* / drug therapy
  • Renal Insufficiency, Chronic* / therapy
  • Retrospective Studies
  • Treatment Outcome

Substances

  • Hemoglobins
  • Erythropoietin