Demographic and disease characteristics of patients at baseline
| Characteristics . | APPLY-PNH∗ (N = 97) . | APPOINT-PNH (N = 40) . | |
|---|---|---|---|
| C5i (n = 35) . | Iptacopan (n = 62) . | Iptacopan . | |
| Sex, n (%) | |||
| Female | 24 (68.6) | 43 (69.4) | 17 (42.5) |
| Race, n (%) | |||
| White | 26 (74.3) | 48 (77) | 12 (30.0) |
| Asian | 7 (20.0) | 12 (19.4) | 27 (67.5) |
| Black or African American | 2 (5.7) | 2 (3.2) | 1 (2.5) |
| Age, mean (SD), y | 49.8 (16.7) | 51.7 (16.9) | 42.1 (15.9) |
| Time since diagnosis, mean (SD), y | 13.6 (10.9) | 11.9 (9.8) | 4.7 (5.5) |
| Duration of C5i therapy, mean (SD), y | 4.2 (3.9) | 3.8 (3.5) | — |
| C5i therapy in the 6 mo before randomization, n (%) | |||
| Eculizumab | 23 (65.7) | 40 (64.5) | — |
| Ravulizumab | 12 (34.3) | 22 (35.5) | — |
| RBC transfusions† | |||
| No. of patients (%) | 21 (60.0) | 35 (56.5) | 28 (70.0) |
| No. of transfusions, mean (SD) | 4.0 (4.4) | 3.1 (2.6) | 3.1 (2.1) |
| Hb,‡ mean (SD), g/dL | 8.9 (0.9) | 8.9 (0.7) | 8.2 (1.1) |
| FACIT-Fatigue score,‡ mean (SD) | 30.8 (11.5) | 34.7 (9.8) | 32.8 (10.2) |
| LDH,‡ mean (SD), U/L | 272.7 (84.8) | 269.1 (70.1) | 1698.8 (683.3) |
| LDH > 1.5 × ULN,‡ n (%) | 3 (8.6) | 4 (6.5) | 40 (100.0) |
| History of MAVEs,§ n (%) | 9 (25.7) | 12 (19.4) | 5 (12.5) |
| Characteristics . | APPLY-PNH∗ (N = 97) . | APPOINT-PNH (N = 40) . | |
|---|---|---|---|
| C5i (n = 35) . | Iptacopan (n = 62) . | Iptacopan . | |
| Sex, n (%) | |||
| Female | 24 (68.6) | 43 (69.4) | 17 (42.5) |
| Race, n (%) | |||
| White | 26 (74.3) | 48 (77) | 12 (30.0) |
| Asian | 7 (20.0) | 12 (19.4) | 27 (67.5) |
| Black or African American | 2 (5.7) | 2 (3.2) | 1 (2.5) |
| Age, mean (SD), y | 49.8 (16.7) | 51.7 (16.9) | 42.1 (15.9) |
| Time since diagnosis, mean (SD), y | 13.6 (10.9) | 11.9 (9.8) | 4.7 (5.5) |
| Duration of C5i therapy, mean (SD), y | 4.2 (3.9) | 3.8 (3.5) | — |
| C5i therapy in the 6 mo before randomization, n (%) | |||
| Eculizumab | 23 (65.7) | 40 (64.5) | — |
| Ravulizumab | 12 (34.3) | 22 (35.5) | — |
| RBC transfusions† | |||
| No. of patients (%) | 21 (60.0) | 35 (56.5) | 28 (70.0) |
| No. of transfusions, mean (SD) | 4.0 (4.4) | 3.1 (2.6) | 3.1 (2.1) |
| Hb,‡ mean (SD), g/dL | 8.9 (0.9) | 8.9 (0.7) | 8.2 (1.1) |
| FACIT-Fatigue score,‡ mean (SD) | 30.8 (11.5) | 34.7 (9.8) | 32.8 (10.2) |
| LDH,‡ mean (SD), U/L | 272.7 (84.8) | 269.1 (70.1) | 1698.8 (683.3) |
| LDH > 1.5 × ULN,‡ n (%) | 3 (8.6) | 4 (6.5) | 40 (100.0) |
| History of MAVEs,§ n (%) | 9 (25.7) | 12 (19.4) | 5 (12.5) |
MAVE, major adverse vascular event; RBC, red blood cell; SD, standard deviation.
The data here are from the final data sets at trial completion; the data previously reported in congress abstracts were from interim data sets generated while the trials were ongoing (at which point the databases were still live); therefore, minor numerical differences may exist between the final efficacy data reported here and the previously reported efficacy data.
In the 6 months before randomization in APPLY-PNH and in the 6 months before study treatment in APPOINT-PNH.
Baseline values were defined as the last result obtained at or before the start of the study treatment (day 1), except for baseline Hb, which was defined as the mean of 2 measurements taken during the screening period (for any patients who received an RBC transfusion during screening after the first confirmatory Hb measurement, the baseline is the first measurement).
History of ≥ 1 event.