Although oral iron is the initial treatment approach for iron deficiency anemia (IDA), some
patients fail to respond to or cannot tolerate oral iron. This double-blind safety and efficacy
study of the intravenous (IV) iron, ferumoxytol, randomized patients with a history of
unsatisfactory oral iron therapy, or in whom oral iron could not be used, to ferumoxytol
(n = 609) or placebo (n = 203). The proportion of
patients achieving the primary endpoint (hemoglobin increase ≥2.0 g/dL at Week 5) was
81.1% with ferumoxytol versus 5.5% with placebo (P < 0.0001).
The mean increase in hemoglobin from Baseline to Week 5, a secondary endpoint (also the alternative
preplanned primary efficacy endpoint for other health authorities), was 2.7 versus 0.1 g/dL
(P < 0.0001). Achievement of a hemoglobin ≥12 g/dL, time to a
hemoglobin increase ≥2.0 g/dL, and improvement in the Functional Assessment of Chronic
Illness Therapy Fatigue score also significantly favored ferumoxytol over placebo at Week 5
(P < 0.0001). Ferumoxytol treatment-emergent adverse events were mainly mild
to moderate. Ferumoxytol was effective and well tolerated in patients with IDA of any underlying
cause in whom oral iron was ineffective or could not be used. This trial was registered at http://www.clinicaltrials.gov as #NCT01114139.
Am. J. Hematol. 89:7–12, 2014. © 2013 Wiley Periodicals, Inc.