Phase III, Randomized, Placebo-controlled Evaluation of Ferumoxytol Treatment for Iron Deficiency Anemia in Patients Who Have a History of Unsatisfactory Oral Iron Therapy: Fatigue and Health-related Quality of Life in Patients with Gastrointestinal Disorders: Presidential Poster

Abstract

Purpose: Iron is essential for the function of key proteins including hemoglobin (Hgb) and myoglobin, cytochromes, various enzymes, and for immune function. Iron deficiency can, therefore, negatively impact patients' health-related quality of life (HRQL). Iron deficiency anemia (IDA) is common in patients with gastrointestinal (GI) disease due to multiple factors, such as blood loss, inflammation and malabsorption. Although oral iron is the preferred first-line treatment, many patients cannot take it, do not tolerate it, or do not adequately respond; many of these patients, therefore, live with chronic anemia and related negative effects on their HRQL. Ferumoxytol (FER) is an IV iron indicated for the treatment of IDA in adults with chronic kidney disease. To explore the impact of FER treatment on patient reported outcomes in IDA patients who have a history of unsatisfactory oral iron therapy or in whom oral iron cannot be used, this randomized, placebo-controlled, double-blinded, Phase 3 study included: Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue), SF-36, and LASA. Methods: Patients with Hgb <10 and >7 g/dL, and TSAT <20% were randomized 3:1 to either 2 injections of 510 mg of FER 5±3 days apart or normal saline (placebo). FACIT-Fatigue, SF-36 and LASA domains were assessed at baseline and weekly up to Week 5. This is a post-hoc analysis of the pre-specified subgroup of 231 patients whose underlying cause of IDA was GI disorders. Results: Baseline FACIT-Fatigue scores (22.4 ±11.7) were lower than general US population norms (40.1), and comparable to anemic cancer patients receiving chemotherapy (23.9). Baseline SF-36 Domain and Summary scores were also below general population norms (50). By Week 5, in parallel with a robust increase in Hgb, FER-treated patients demonstrated significantly greater improvements in FACIT-Fatigue scores than placebo, approaching general population norms, and significantly greater improvements than placebo in 9 of the 10 SF-36 Domain and Summary scores (p< 0.05). Improvements in LASA domains were similar to the overall IDA population. Conclusion: This analysis found that patients with IDA due to GI disorders, who had been unsuccessfully treated with oral iron, had very poor baseline HRQOL scores. In this study, treatment with FER resulted not only in increased Hgb, but also in significant improvements in measures of health-related quality of life. Disclosure - Naomi Dahl, William Strauss, Gloria Lau, Kristine Bernard - Employees: AMAG Pharmaceuticals; Kendra DeBusk -Employee: Oxford Outcomes Charles Barish - None David Ford - Speaker's Bureau: Takeda. This research was supported by an industry grant from AMAG Pharmaceuticals.Table