Evaluation of a Single Dose of Ferric Carboxymaltose in Fatigued, Iron-Deficient Women – PREFER a Randomized, Placebo-Controlled Study

Summary & key facts

This was a single-blinded, randomized trial of 290 premenopausal women with unexplained fatigue and low iron who received one 1000 mg IV dose of ferric carboxymaltose (FCM) or saline placebo. By Day 56, more women given FCM had reduced fatigue and larger improvements in some quality-of-life and cognitive scores, and FCM raised markers of red blood cell production and iron measures; however, more side effects were reported with FCM. The authors conclude that treating iron deficiency (even when hemoglobin is near normal) may improve symptoms, but several study authors had financial ties to the drug maker and side effects were more frequent with FCM.

Key facts:

• 290 women were included in the analysis: 144 received a single 1000 mg IV infusion of ferric carboxymaltose (FCM) and 146 received placebo (saline).
• Participants had fatigue (Piper Fatigue Scale score ≥5) and iron deficiency defined as ferritin <50 µg/L with transferrin saturation <20%, or ferritin <15 µg/L; hemoglobin had to be ≥115 g/L at entry.
• The primary endpoint (≥1 point decrease in Piper Fatigue Scale by Day 56) occurred in 65.3% of FCM-treated women versus 52.7% with placebo (odds ratio 1.68, 95% CI 1.05–2.70; p = 0.03).
• A 50% drop in fatigue score was seen in 33.3% of FCM patients versus 16.4% of placebo patients (p < 0.001).
• At Day 56 all FCM-treated patients had hemoglobin ≥120 g/L (versus 87% at baseline); in the placebo group the proportion with Hb ≥120 g/L fell from 86% to 81%.
• Treatment-emergent adverse events were more frequent with FCM (209 events) than with placebo (114 events). The most common events reported were headache, nasopharyngitis (common cold), fever, and nausea, and most events were mild or moderat
• The trial was single-blinded, conducted at 21 sites in Austria, Germany, Sweden and Switzerland, and is registered as ClinicalTrials.gov NCT01110356.
• The authors reported improvements in mental quality-of-life and some cognitive measures with FCM, and noted a larger attention benefit in the subgroup with very low ferritin (<15 µg/L).
• Several authors disclosed financial relationships with Vifor Pharma, and medical writing support was funded by Vifor Pharma, which is relevant when interpreting the results.

Abstract

ClinicalTrials.gov NCT01110356.

Topics

Erythropoietin and Anemia Treatment Iron Metabolism and Disorders Pharmacological Effects and Toxicity Studies

Categories

Health Sciences Hematology Medicine

Tags

Adverse effect Alternative medicine Anemia Clinical endpoint Ferritin Gastroenterology Internal medicine Iron deficiency Medicine Nausea Pathology Placebo Randomization Randomized controlled trial Tolerability Transferrin saturation