Efficacy and safety of IV ferumoxytol for iron deficiency anemia in patients with cancer

Figures & data

Figure 1 Study design of Study 1 and Study 2.

Abbreviations: EP, endpoint; ICF, informed consent; IV, intravenous; D-1, one day prior to randomization; D-14, 14 days prior to randomization.

Table 1 Baseline patient demographics by treatment group

Baseline characteristics	Treatment group
	Ferumoxytol (n=75)	Iron sucrose (n=13)	Placebo (n=10)
Age, y, mean (SD)	58.5 (14.1)	66.7 (11.3)	53.4 (14.0)
Female, n (%)	56 (75)	6 (46)	8 (80)
Height, cm, mean (SD)	161.5 (8.2)	165.0 (10.3)	152.3 (5.7)
Weight, kg, mean (SD)	68.4 (21.3)	73.8 (18.8)	55.5 (20.4)
History of IV iron exposure, n (%)	6 (8)	2 (15)	1 (10)
Hgb level, g/dL, mean (SD)	9.0 (0.8)	9.0 (0.9)	8.8 (1.1)
TSAT, %, mean (SD)	9.4 (10.7)	7.7 (3.4)	9.6 (7.3)
Serum ferritin, ng/mL, mean (SD)	120.8 (195.9)	151.3 (189.6)	80.8 (93.9)
Baseline Hgb level, g/dL, n (%)
>7.0 to ≤8.5	22 (29)	4 (31)	3 (30)
>8.5 to <10.0	53 (71)	9 (69)	7 (70)
Race, n (%)
White	47 (63)	13 (100)	4 (40)
Black/African American	6 (8)	0 (0)	0 (0)
Asian	22 (29)	0 (0)	5 (50)
Other/multiracial	0 (0)	0 (0)	1 (10)
Type of cancer, n (%)
Gastrointestinal	31 (41)	8 (62)	3 (30)
Breast	12 (16)	1 (8)	1 (10)
Other	9 (12)	2 (15)	0 (0)
Cervix	7 (9)	0 (0)	3 (30)
Lung	7 (9)	0 (0)	2 (20)
Bladder	3 (4)	1 (8)	0 (0)
Ovarian	3 (4)	0 (0)	1 (10)
Pancreas	2 (3)	0 (0)	0 (0)
Prostate	1 (1)	1 (8)	0 (0)

Notes: No significant difference between groups in sex, race, type of cancer, history of IV iron use, Hgb category (Fisher exact test), baseline TSAT, or ferritin (two sample t-test).

Abbreviations: Hgb, hemoglobin; IV, intravenous; TSAT, transferrin saturation.

Figure 2 Mean change from baseline hemoglobin (A) and FACIT-F (B).

Notes: Bars represent 95% CIs, demonstrating within-group changes from baseline.
Abbreviations: Hgb, hemoglobin; FACIT-F, Functional Assessment of Chronic Illness Therapy-Fatigue.

Figure 3 Mean TSAT (A) and median ferritin (B) levels by treatment group.

Abbreviation: TSAT, transferrin saturation.

Table 2 Adverse event summary: cancer subgroup and overall study population

AE category, n (%)	Cancer subgroup			Pooled overall study population
	Ferumoxytol (n=75)	Iron sucrose (n=13)	Placebo (n=10)	Ferumoxytol (n=1,014)	Iron sucrose (n=199)	Placebo (n=200)
All AEs	48 (64.0)	6 (46.2)	8 (80.0)	467 (46.1)	88 (44.2)	86 (43.0)
Related AEsa	6 (8.0)	0 (0.0)	1 (10.0)	147 (14.5)	32 (16.1)	15 (7.5)
Serious AEs	7 (9.3)	1 (7.7)	1 (10.0)	33 (3.3)	5 (2.5)	6 (3.0)
Related serious AEs	2 (2.7)	0 (0.0)	0 (0.0)	6 (0.6)	0 (0.0)	0 (0.0)
AEs of special interestb	4 (5.3)	1 (7.7)	0 (0.0)	33 (3.3)	10 (5.0)	2 (1.0)
AEs resulting in study discontinuation	0 (0.0)	0 (0.0)	0 (0.0)	6 (0.6)	2 (1.0)	2 (1.0)
Deathc	1 (1.3)	0 (0.0)	1 (10.0)	3 (0.3)	0 (0.0)	1 (0.5)

Notes:

a Classified by the investigator as related to study drug.

b Protocol defined – including hypotension and hypersensitivity.

c Assessed as unrelated by the investigator.

Abbreviation: AEs, adverse events.

Table 3 Treatment-emergent adverse events

AE preferred term, n (%)	Treatment groups
TEAEs occurring in >1 ferumoxytol-treated patient in the cancer subgroup
	Ferumoxytol (n=75)	Iron sucrose (n=13)	Placebo (n=10)
Abdominal pain	4 (5.3)	0 (0.0)	0 (0.0)
Diarrhea	4 (5.3)	0 (0.0)	1 (10.0)
Nausea	4 (5.3)	1 (7.7)	0 (0.0)
Cough	3 (4.0)	0 (0.0)	0 (0.0)
Dizziness	3 (4.0)	0 (0.0)	0 (0.0)
Hypokalemia	3 (4.0)	0 (0.0)	0 (0.0)
Leukopenia	3 (4.0)	0 (0.0)	0 (0.0)
Neutropenia	3 (4.0)	0 (0.0)	2 (20.0)
Vomiting	3 (4.0)	0 (0.0)	0 (0.0)
Back pain	2 (2.7)	0 (0.0)	0 (0.0)
Constipation	2 (2.7)	1 (7.7)	0 (0.0)
Dry mouth	2 (2.7)	0 (0.0)	0 (0.0)
Dysgeusia	2 (2.7)	0 (0.0)	0 (0.0)
Iron deficiency anemia	2 (2.7)	0 (0.0)	0 (0.0)
Malignant neoplasm progression	2 (2.7)	0 (0.0)	0 (0.0)
Pain in extremity	2 (2.7)	0 (0.0)	0 (0.0)
Pyrexia	2 (2.7)	0 (0.0)	1 (10.0)
Urinary tract infection	2 (2.7)	0 (0.0)	0 (0.0)
TEAEs occurring in ≥1.5% of ferumoxytol-treated patients in the pooled overall study population
	Ferumoxytol (n=1,014)	Iron sucrose (n=199)	Placebo (n=200)
Headache	54 (5.3)	11 (5.5)	12 (6.0)
Nausea	39 (3.8)	7 (3.5)	5 (2.5)
Dizziness	33 (3.3)	3 (1.5)	7 (3.5)
Diarrhea	21 (2.1)	0 (0.0)	6 (3.0)
Urinary tract infection	19 (1.9)	3 (1.5)	6 (3.0)
Nasopharyngitis	18 (1.8)	2 (1.0)	4 (2.0)
Dysgeusia	18 (1.8)	13 (6.5)	1 (0.5)
Back pain	17 (1.7)	1 (0.5)	2 (1.0)
Vomiting	16 (1.6)	4 (2.0)	2 (1.0)
Abdominal pain	15 (1.5)	3 (1.5)	5 (2.5)
Fatigue	15 (1.5)	2 (1.0)	3 (1.5)

Abbreviations: AEs, adverse events; TEAEs, treatment-emergent adverse events.