Ferumoxytol versus placebo in iron deficiency anemia: efficacy, safety, and quality of life in patients with gastrointestinal disorders

Abstract

Introduction: Iron deficiency anemia (IDA) is common in patients with gastrointestinal (GI) disorders and can adversely affect quality of life. Oral iron is poorly tolerated in many patients with GI disorders. Ferumoxytol is approved for the intravenous treatment of IDA in patients with chronic kidney disease. This study aimed to evaluate the efficacy and safety of ferumoxytol in patients with IDA and concomitant GI disorders.

Patients and methods: This analysis included 231 patients with IDA and GI disorders from a Phase III, randomized, double-blind, placebo-controlled trial evaluating ferumoxytol (510 mg ×2) versus placebo in patients who had failed or were intolerant of oral iron therapy. The primary study end point was the proportion of patients achieving a ≥20 g/L increase in hemoglobin (Hgb) from baseline to Week 5. Other end points included mean change in Hgb, proportion of patients achieving Hgb ≥120 g/L, mean change in transferrin saturation, and patient-reported outcomes (PROs).

Results: Significantly more patients with IDA receiving ferumoxytol achieved a ≥20 g/L increase in Hgb versus placebo (82.1% vs 1.7%, respectively; P<0.001). Mean increase in Hgb (28.0 g/L vs -1.0 g/L, respectively; P<0.001) significantly favored ferumoxytol treatment. Ferumoxytol-treated patients demonstrated significantly greater improvements than placebo-treated patients relative to their very poor baseline PRO scores posttreatment, including improvements in the Functional Assessment of Chronic Illness Therapy-Fatigue questionnaire and various domains of the 36-Item Short-Form Health Survey. Ferumoxytol-treated patients had a low rate of adverse events.

Conclusion: In this study, ferumoxytol was shown to be an efficacious and generally well-tolerated treatment option for patients with IDA and underlying GI disorders who were unable to use or had a history of unsatisfactory oral iron therapy.

Keywords: efficacy; hemoglobin; inflammatory bowel disease; intravenous iron; patient-reported outcomes; quality of life.

Figure 1

Patient disposition for the subgroup of patients with IDA and GI disorders. Abbreviations: IDA, iron deficiency anemia; GI, gastrointestinal; AE, adverse event.

Figure 2

Mean Hgb values (g/l) from baseline to Week 5 in patients with IDA and GI disorders (intent-to-treat population). Notes: ^#The first ferumoxytol dose was administered at baseline. *P<0.0001 for mean change from baseline. Abbreviations: Hgb, hemoglobin; IDA, iron deficiency anemia; GI, gastrointestinal.

Figure 3

Mean TSAT values from baseline to Week 5 in patients with IDA and GI disorders (intent-to-treat population). Notes: ^#The first ferumoxytol dose was administered at baseline. *P<0.0001 for mean change from baseline. Abbreviations: IDA, iron deficiency anemia; GI, gastrointestinal; TSAT, transferrin saturation.

Figure 4

SF-36 domain scores at baseline in patients with IDA and GI disorders (intent-to-treat population). Abbreviations: IDA, iron deficiency anemia; GI, gastrointestinal; SF-36, 36-item Short-Form Health Survey; SD, standard deviation.

Figure 5

Patient-reported outcomes at baseline and Week 5 in patients with IDA and GI disorders (intent-to-treat population). Notes: The figure shows mean change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)–Fatigue scores. *P<0.05 vs placebo. The first ferumoxytol dose was administered at baseline. FACIT-fatigue score is 0-52, based on 13 questions scored 0–4. Abbreviations: IDA, iron deficiency anemia; GI, gastrointestinal.

Figure 6

Patient-reported outcomes at baseline and Week 5 in patients with IDA and GI disorders (intent-to-treat population). Notes: The figure shows mean change from baseline in the 36-Item Short-Form Health Survey (SF-36) domain scores. *P<0.05 vs placebo. The first ferumoxytol dose was administered at baseline. Error bars indicate 95% confidence intervals. Abbreviations: IDA, iron deficiency anemia; GI, gastrointestinal.

Figure 7

Patient-reported outcomes at baseline, Week 3, and Week 5 in patients with IDA and GI disorders (intent-to-treat population). Notes: Mean change from baseline in linear Analog Scale Assessment (lASA) domain scores is shown. The first ferumoxytol dose was administered at baseline. Error bars indicate 95% confidence intervals. Abbreviations: IDA, iron deficiency anemia; GI, gastrointestinal.