| Popis: |
The optimum starting dose of intravenous continuous erythropoietin receptor activator (C.E.R.A.) has been previously determined; this study ascertains the optimum starting dose of subcutaneous (SC) C.E.R.A. administration in pediatric patients.Phase 2, open-label, single-arm, multicenter study.Patients aged 3 months-17 years with renal anemia and chronic kidney disease (CKD) who were on, or not on, dialysis and receiving maintenance treatment with erythropoiesis-stimulating agents (ESAs).C.E.R.A. SC every 4 weeks (starting dose was based on defined conversion factors).The primary outcome was the change in hemoglobin (Hb) concentration between the baseline and evaluation period for each patient. Secondary efficacy measures and safety were also evaluated.Forty patients aged 0.4-17.7 years enrolled. The study achieved its primary outcome: the mean change in Hb concentration was +0.48 g/dL; 95% confidence intervals (0.15-0.82) were within prespecified -1 to 1 g/dL boundaries; standard deviation (±1.03) was1.5 g/dL. Mean Hb concentrations were maintained within the target 10-12 g/dL range in 24/38 patients and within ±1 g/dL of the baseline in 19/38 patients and the median C.E.R.A. SC dose decreased over time. Efficacy in key subgroups (age group, dialysis type, prior ESA treatment) was consistent with the primary outcome. Thirty-eight patients completed the core period; 25 chose to enter the safety-extension period. Safety was consistent with prior studies, with no new signals.Single-arm and open-label study; small sample size.Pediatric patients with anemia secondary to CKD who were on, or not on, dialysis could be safely and effectively switched from maintenance ESAs to C.E.R.A. SC every 4 weeks, using defined dose-conversion factors to determine the optimum starting dose. |
