Introduction
Methods
Study Design
Patients
Study Assessments and Endpoints
Statistical Analysis
Results
Patient Disposition and Demographics
Demographic characteristic | Ferric carboxymaltose (n = 41) | Placebo (n = 40) |
---|---|---|
Mean age, years (SD) | 41.2 (11.1) | 43.9 (10.8) |
Sex, n (%) | ||
Female | 40 (97.6) | 40 (100.0) |
Male | 1 (2.4) | 0 |
Race, n (%) | ||
White | 33 (80.5) | 30 (75) |
Black | 7 (17.1) | 7 (17.5) |
Hispanic | 0 | 2 (5.0) |
Asian | 1 (2.4) | 0 |
Other | 0 | 1 (2.5) |
History of iron intolerance, n (%) | ||
No | 35 (85.4) | 37 (92.5) |
Yesa
| 6 (14.6) | 3 (7.5) |
History of drug allergy, n (%) | ||
No | 21 (51.2) | 17 (42.5) |
Yes | 20 (48.8) | 23 (57.5) |
Mean IRLS score, kg, mean (SD) | 13.4 (13.2) | 16.3 (14.9) |
Efficacy
Patient-Reported Outcomes
Outcomes | Ferric carboxymaltose |
n
| Placebo |
n
|
P valuea
|
---|---|---|---|---|---|
Patient-reported outcomes | |||||
FIQR | |||||
Baseline, mean (SD) | 82.8 (9.5) | 40 | 83.1 (9.4) | 40 | |
Day 42, mean change from baseline (SD) | − 45.0 (29.5) | 38 | − 29.7 (27.0) | 39 | 0.015 |
BPI, Pain Severity | |||||
Baseline, mean (SD) | 6.9 (1.2) | 40 | 7.0 (1.4) | 40 | |
Day 42, mean change from baseline (SD) | − 3.1 (2.7) | 38 | − 1.8 (2.1) | 39 | 0.022 |
BPI, Pain Interference | |||||
Baseline, mean (SD) | 8.1 (1.2) | 40 | 7.9 (1.3) | 40 | |
Day 42, mean change from baseline (SD) | − 4.1 (3.3) | 38 | − 2.3 (2.6) | 39 | 0.010 |
MOS Sleep scale | |||||
Baseline, mean (SD) | 72.7 (13.1) | 39 | 79.5 (12.4) | 40 | |
Day 42, mean change from baseline (SD) | − 13.6 (18.0) | 37 | − 9.5 (17.1) | 37 | 0.083 |
Fatigue Visual Numeric Scale | |||||
Baseline, mean (SD) | 9.2 (1.0) | 40 | 9.3 (0.9) | 40 | |
Day 42, mean change from baseline (SD) | − 3.7 (3.2) | 37 | − 1.7 (2.2) | 39 | < 0.001 |
Iron indices | |||||
Serum ferritin, µg/l | |||||
Screening, mean (SD) | 19.0 (12.4) | 40 | 18.1 (11.6) | 40 | |
Day 42, mean change from baseline (SD) | 292.5 (145.9) | 39 | 2.0 (13.8) | 38 | < 0.001 |
Transferrin saturation, % | |||||
Screening, mean (SD) | 14.6 (5.3) | 40 | 13.6 (5.5) | 40 | |
Day 42, mean change from baseline (SD) | 24.4 (11.4) | 39 | 5.8 (15.1) | 38 | < 0.001 |
Hematology parameters | |||||
Hemoglobin, g/dl | |||||
Screening, mean (SD) | 12.3 (1.2) | 41 | 12.3 (1.2) | 40 | |
Day 42, mean change from baseline (SD) | 1.2 (0.9) | 40 | − 0.1 (0.9) | 39 | NA |
Hematocrit, % | |||||
Screening, mean (SD) | 38.4 (3.2) | 41 | 38.4 (3.3) | 40 | |
Day 42, mean change from baseline (SD) | 3.7 (3.0) | 40 | − 0.8 (5.3) | 39 | NA |
Outcomesa
| Ferric carboxymaltose | Placebo | |||
---|---|---|---|---|---|
Baseline, mean (SD) n = 40 | Day 42, mean change from baseline (SD) n = 38 | Baseline, mean (SD) n = 40 | Day 42, mean change from baseline (SD) n = 39 |
P valueb
| |
Patient-reported outcomes | |||||
Function score | 70.8 (11.5) | − 40.5 (29.4) | 73.3 (10.6) | − 26.2 (26.0) | 0.019 |
Overall score | 17.3 (2.7) | − 9.5 (6.7) | 17.0 (2.4) | − 6.7 (6.6) | 0.072 |
Symptoms score | 83.9 (10.0) | − 44.0 (29.8) | 83.5 (10.5) | − 28.6 (26.2) | 0.019 |
Brush score | 6.0 (2.8) | − 3.3 (3.7) | 5.7 (2.5) | − 1.9 (3.0) | 0.067 |
Walk score | 8.4 (1.6) | − 4.5 (3.5) | 8.8 (1.6) | − 2.8 (3.1) | 0.015 |
Prepare score | 7.4 (1.7) | − 4.5 (3.7) | 7.6 (2.0) | − 2.7 (3.3) | 0.009 |
Vacuum score | 8.6 (1.7) | − 4.9 (3.5) | 8.7 (2.0) | − 2.9 (3.2) | 0.009 |
Lift score | 8.1 (2.0) | − 4.7 (3.6) | 8.6 (1.4) | − 3.5 (3.1) | 0.074 |
Climb score | 8.3 (1.6) | − 4.7 (3.2) | 9.0 (1.3) | − 3.4 (3.6) | 0.068 |
Bed score | 7.9 (1.5) | − 4.7 (3.5) | 8.0 (2.1) | − 2.6 (3.5) | 0.008 |
Sit score | 7.7 (1.9) | − 4.1 (3.7) | 8.3 (1.7) | − 2.6 (3.1) | 0.014 |
Shop score | 8.5 (1.4) | − 5.1 (3.6) | 8.7 (1.2) | − 3.6 (3.0) | 0.028 |
Goals score | 8.7 (1.4) | − 4.7 (3.6) | 8.5 (1.2) | − 3.2 (3.2) | 0.071 |
Overwhelm score | 8.6 (1.5) | − 4.8 (3.4) | 8.5 (1.6) | − 3.5 (3.5) | 0.086 |
Pain score | 7.9 (1.2) | − 4.1 (2.9) | 8.0 (1.5) | − 2.7 (3.0) | 0.022 |
Energy score | 9.0 (1.6) | − 4.7 (3.4) | 8.6 (2.2) | − 1.9 (3.5) | 0.003 |
Stiff score | 8.8 (1.0) | − 4.2 (3.2) | 8.6 (1.3) | − 2.5 (2.8) | 0.018 |
Sleep score | 9.2 (1.2) | − 4.5 (3.4) | 9.3 (1.0) | − 2.6 (3.0) | 0.009 |
Level score | 7.7 (2.2) | − 4.2 (3.6) | 8.0 (1.6) | − 3.4 (3.4) | 0.171 |
Memory score | 8.4 (1.7) | − 4.5 (3.3) | 8.1 (1.5) | − 2.8 (3.3) | 0.031 |
Anxiety score | 8.3 (1.9) | − 3.9 (3.3) | 8.4 (1.3) | − 2.7 (3.1) | 0.103 |
Tender score | 8.4 (1.5) | − 4.7 (3.5) | 8.7 (1.6) | − 3.2 (3.1) | 0.030 |
Balance score | 7.9 (1.6) | − 4.3 (3.5) | 7.8 (1.8) | − 3.2 (3.2) | 0.159 |
Sense score | 8.4 (1.4) | − 4.8 (3.2) | 8.1 (2.2) | − 3.5 (3.4) | 0.109 |