Ferumoxytol for iron deficiency anemia in patients undergoing hemodialysis. The FACT randomized controlled trial

Abstract

Background: Patients with chronic kidney disease (CKD) undergoing dialysis often require intravenous iron for iron deficiency anemia (IDA).

Materials and methods: The Ferumoxytol for Anemia of CKD Trial (FACT), a randomized, multicenter, open-label, phase 4 study, compared the long-term safety and efficacy of ferumoxytol with iron sucrose for the treatment of IDA in patients with CKD undergoing hemodialysis. Patients with IDA and CKD undergoing hemodialysis were randomized 2:1 to ferumoxytol 1.02 g (2 × 510 mg) or iron sucrose 1.0 g (10 × 100 mg) for a 5-week treatment period (TP). Over 11 months, patients underwent additional 5-week TPs whenever IDA (hemoglobin < 11.5 g/dL and transferrin saturation < 30%) was detected. The primary efficacy endpoint was mean change in hemoglobin from baseline to week 5 for each TP. Adverse events were recorded during the study.

Results: Overall, 293 patients received ferumoxytol (n = 196) or iron sucrose (n = 97). Ferumoxytol was noninferior to iron sucrose regarding hemoglobin change from baseline to week 5. The mean change in hemoglobin in the ferumoxytol and iron sucrose groups was 0.5 and 0.4 g/dL, respectively, in TP 1 (least-squares mean difference, 0.13; 95% confidence interval, -0.11 to 0.36) and 0.6 and 0.3 g/dL, respectively, in TP 2 (0.30; 0.06 - 0.55). Treatment-related and serious adverse events were similar in both groups; no new safety signals emerged.

Conclusion: Long-term administration of ferumoxytol has noninferior efficacy and a similar safety profile to iron sucrose when used to treat IDA in patients with CKD undergoing hemodialysis. .

Figure 1.. Study design and treatment. D = day; Hb = hemoglobin; HD = hemodialysis; ICF = informed consent form; IV = intravenous; TP = treatment period; TSAT = transferrin saturation.

Figure 2.. a: Mean change in hemoglobin at week 5. b: Mean hemoglobin levels by month for each treatment group over the course of the study; c: Median monthly ESA dose by treatment group over the course of the study; d: Mean change in TSAT at week 5. For Panels a and b, TP includes only those periods for which n > 5 for both treatment groups. For each TP, the number of patients treated with ferumoxytol and iron sucrose, respectively, is shown in brackets. ESA = erythropoiesis-stimulating agent; TP = treatment period; TSAT = transferrin saturation. *p < 0.05 vs. comparator.