Table 2.
Primary end point and selected secondary end points (ITT population)a
| High-ferritin FCM (n = 153) | Low-ferritin FCM (n = 152) | Oral iron (n = 308) | |
|---|---|---|---|
| Primary end point | |||
| Time to initiation of other anaemia management or Hb trigger (central monitoring)b,e | |||
| n (%) | 36 (23.5) | 49 (32.2) | 98 (31.8) |
| HR (95% CI)c | Reference | 0.68 (0.45, 1.05) | 0.65 (0.44, 0.95) |
| P-value (log-rank) | Reference | 0.082 | 0.026 |
| Events contributing to primary end point | |||
| Hb triggerb | 10 (6.5) | 19 (12.5) | 36 (11.7) |
| ESA only | 16 (10.5) | 18 (11.8) | 24 (7.8) |
| Other iron therapy only | 0 | 5 (3.3) | 20 (6.5) |
| ESA and iron therapy | 0 | 2 (1.3) | 2 (0.6) |
| Transfusion | 9 (5.9) | 5 (3.3) | 14 (4.5) |
| ESA and transfusion | 1 (0.7) | 0 | 2 (0.6) |
| Primary end point | |||
| Time to initiation of other anaemia management or Hb trigger (local monitoring)b,d,e | |||
| n (%) | 40 (26.1) | 55 (36.2) | 115 (37.3) |
| HR (95% CI)c | Reference | 0.68 (0.45, 1.03) | 0.62 (0.43, 0.88) |
| P-value (log-rank) | Reference | 0.064 | 0.008 |
| Secondary end points | |||
| Blood transfusion | |||
| n (%) | 11 (7.2) | 11 (7.2) | 26 (8.4) |
| Odds ratio (95% CI) | 0.89 (0.42, 1.88) | 0.97 (0.46, 2.04) | Reference |
| P-value | 0.77 | 0.94 | Reference |
| Time to Hb increase ≥1 g/dLe,f,g | |||
| n (%) | 87 (56.9) | 52 (34.2) | 99 (32.1) |
| HR (95% CI) | Reference | 2.11 (1.49, 2.98) | 2.04 (1.52, 2.72) |
| P-value (log-rank) | Reference | <0.001 | <0.001 |
| Hb, LS mean (SE) (g/dL)f,h | |||
| Baseline | 10.1 (0.1) | 10.2 (0.1) | 10.2 (0.1) |
| Month 12 | 12.0 (0.1) | 11.5 (1.1) | 11.5 (0.1) |
| Change from baseline | 1.4 (0.1) | 0.9 (0.1) | 1.0 (0.1) |
| P-value (change from baseline to Month 12 versus oral ironi) | 0.014 | 0.26 | Reference |
| Ferritin, LS mean (SE) (µg/L)f,h | |||
| Baseline | 54 (9) | 48 (9) | 53 (6) |
| Month 12 | 503 (11) | 125 (11) | 184 (8) |
| Change from baseline | 451 (10) | 81 (11) | 137 (8) |
| P-value (change from baseline to Month 12 versus oral ironi) | <0.001 | <0.001 | Reference |
| TSAT, LS mean (SE) (%)f,h | |||
| Baseline | 16.2 (1.0) | 16.1 (1.0) | 15.5 (0.7) |
| Month 12 | 31.2 (1.3) | 24.2 (1.3) | 28.6 (1.0) |
| Change from baseline | 15.8 (1.3) | 8.5 (1.3) | 13.8 (1.0) |
| P-value (change from baseline to Month 12 versus oral ironi) | 0.20 | 0.001 | Reference |
| eGFR, LS mean (SE) (mL/min/1.73 m2)j | |||
| Baseline | 32.1 (1.1) | 31.8 (1.1) | 33.2 (0.8) |
| Month 12 | 35.3 (1.4) | 31.1 (1.4) | 33.7 (1.0) |
| Change from baseline | 0.4 (0.8) | −1.6 (0.8) | −1.1 (0.6) |
| P-value (change from baseline to Month 12 versus oral ironi) | 0.14 | 0.64 | Reference |
| Requirement for dialysis | |||
| n % | 5 (3.3) | 1 (0.7) | 10 (3.2) |
| Odds ratio (95% CI) | 1.01 (0.34, 3.00) | 0.20 (0.03, 1.56) | Reference |
| P-value | 0.99 | 0.12 | Reference |
eGFR, estimated glomerular filtration rate; FCM, ferric carboxymaltose; Hb, haemoglobin; LS, least squares; TSAT, transferrin saturation.
aContinuous variables are shown as mean (SD).
bTime to initiation of other anaemia management such as an ESA or transfusion, or an Hb trigger (two consecutive Hb values <10 g/dL on or after Week 8, without an increase of ≥0.5 g/dL between the two measurements).
cProportional hazards modelling.
dPre-specified sensitivity analysis.
eKaplan–Meier estimates.
fMeasured at local laboratory.
gPrior to first initiation of other anaemia management.
hMeasurements were included up to the point at which other anaemia therapy was initiated and/or the randomized study medication was discontinued.
iAnalysis of covariance analysis based on LS mean values.
jEstimated by MDRD-4 equation [22] at local laboratory.