Secondary end points
| Secondary end points . | Pegcetacoplan (n = 35) . | Control, supportive care only∗ (n = 18) . | Difference (95% CI) . | P value . |
|---|---|---|---|---|
| Hemoglobin response, n (%)†,‡ | 25 (71.4) | 1 (5.6) | 54.1 (33.9-74.3) | < .0001 |
| CFB in ARC at wk 26, LS mean (SD), ×109 cells per L | −123.3 (9.2) | −19.4 (25.2) | −103.8 (–158.9 to −48.7) | .0002 |
| Hemoglobin CFB at wk 26, LS mean (SE), g/dL | 2.9 (0.4) | 0.3 (0.8) | 2.7 (1.0-4.4) | .0019 |
| Transfusion avoidance through wk 26,‡ n (%) | 32 (91.4) | 1 (5.6) | 72.4 (55.8-89.0) | < .0001 |
| FACIT-Fatigue score CFB at wk 26, LS mean (SE) | 7.8 (1.2) | 3.3 (2.1) | 4.5 (−0.2 to 9.2) | .0610 |
| Global health status/QoL score (EORTC QLQ-C30) CFB at wk 26, LS mean (SE) | 18.9 (2.9) | −2.9 (5.7) | 21.8 (9.4-34.2) | Nominal .0006 |
| ARC normalization, n (%)‡,§ | 21 (60.0) | 1 (5.6) | 46.4 (25.3-67.5) | Nominal .0002 |
| Patients with a clinically meaningful‖ improvement in FACIT-Fatigue score,‡ n (%) | 21 (60.0) | 2 (11.1) | 48.9 (27.1-70.7) | Nominal .0007 |
| Hemoglobin normalization at wk 26,‡,¶ n (%) | 16 (45.7) | 0 (0) | 36.5 (16.5-56.4) | Nominal .0010 |
| LDH normalization,‡,# n (%) | 23 (65.7) | 0 (0) | 55.9 (36.8-75.0) | Nominal < .0001 |
| Secondary end points . | Pegcetacoplan (n = 35) . | Control, supportive care only∗ (n = 18) . | Difference (95% CI) . | P value . |
|---|---|---|---|---|
| Hemoglobin response, n (%)†,‡ | 25 (71.4) | 1 (5.6) | 54.1 (33.9-74.3) | < .0001 |
| CFB in ARC at wk 26, LS mean (SD), ×109 cells per L | −123.3 (9.2) | −19.4 (25.2) | −103.8 (–158.9 to −48.7) | .0002 |
| Hemoglobin CFB at wk 26, LS mean (SE), g/dL | 2.9 (0.4) | 0.3 (0.8) | 2.7 (1.0-4.4) | .0019 |
| Transfusion avoidance through wk 26,‡ n (%) | 32 (91.4) | 1 (5.6) | 72.4 (55.8-89.0) | < .0001 |
| FACIT-Fatigue score CFB at wk 26, LS mean (SE) | 7.8 (1.2) | 3.3 (2.1) | 4.5 (−0.2 to 9.2) | .0610 |
| Global health status/QoL score (EORTC QLQ-C30) CFB at wk 26, LS mean (SE) | 18.9 (2.9) | −2.9 (5.7) | 21.8 (9.4-34.2) | Nominal .0006 |
| ARC normalization, n (%)‡,§ | 21 (60.0) | 1 (5.6) | 46.4 (25.3-67.5) | Nominal .0002 |
| Patients with a clinically meaningful‖ improvement in FACIT-Fatigue score,‡ n (%) | 21 (60.0) | 2 (11.1) | 48.9 (27.1-70.7) | Nominal .0007 |
| Hemoglobin normalization at wk 26,‡,¶ n (%) | 16 (45.7) | 0 (0) | 36.5 (16.5-56.4) | Nominal .0010 |
| LDH normalization,‡,# n (%) | 23 (65.7) | 0 (0) | 55.9 (36.8-75.0) | Nominal < .0001 |
LS, least squares; SE, standard error.
Control group patients received supportive care (eg, transfusions, corticosteroids, and supplements [iron, folate, and vitamin B12]).
Defined as ≥1-g/dL increase from baseline to week 26.
Patients who received a transfusion, escaped from the control group to pegcetacoplan treatment, withdrew from study before week 26, or were lost to follow-up were categorized as nonresponders.
ARC < sex-specific ULN (male, 10 × 109 to 140 × 109 cells per L; and female, 10 × 109 to 120 × 109 cells per L) at week 26.
Clinically meaningful improvement is defined as a ≥3-point FACIT-Fatigue score increase22 from baseline to week 26.
Hemoglobin levels ≥ sex-specific LLN: male, 13.6 g/dL; and female, 12.0 g/dL.
LDH levels ≤ ULN (226 U/L) at week 26.