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Table 2 Summary of pre-specified exploratory and post hoc endpoints

From: Subcutaneous C1 inhibitor for prevention of attacks of hereditary angioedema: additional outcomes and subgroup analysis of a placebo-controlled randomized study

 

40 IU/kg (N = 45)

60 IU/kg (N = 45)

C1-INH (SC)

Placebo

C1-INH (SC)

Placebo

Primary efficacy endpoint

 Number of time-normalized HAE attacks/montha—mean (95% CI)

1.19 (0.54, 1.85)

3.61 (2.96, 4.26)

0.52 (0.00, 1.04)

4.03 (3.51, 4.55)

Secondary efficacy endpoint

 Uses of rescue medication/montha—mean (95% CI)

1.13 (− 1.44, 3.69)

5.55 (3.10, 8.00)

0.32 (− 0.33, 0.97)

3.89 (3.23, 4.55)

Pre-specified exploratory endpoints

 Time-normalized sum of symptom severity scores

  Mean (SD)

0.10 (0.19)

0.44 (0.40)

0.07 (0.15)

0.45 (0.33)

  Median (range)

0.02 (0, 0.9)

0.36 (0, 2.0)

0.01 (0, 0.9)

0.38 (0, 1.5)

  N

43

44

43

42

 Time-normalized sum of symptom severity scores combined with rescue medication use

  Mean (SD)

0.17 (0.35)

0.73 (0.69)

0.09 (0.18)

0.73 (0.51)

  Median (range)

0.04 (0, 1.8)

0.59 (0, 3.5)

0.02 (0, 0.9)

0.65 (0.1, 2.4)

  N

43

44

43

42

 Duration of HAE attacks per patient, days

  Mean (SD)

1.80 (1.08)

2.08 (1.21)

1.58 (0.98)

1.64 (0.66)

  Median (range)

1.57 (1.0, 5.8)

1.71 (1.0, 7.0)

1.0 (1.0, 5.5)

1.45 (1.0, 4.0)

Post hoc analysis of treated versus untreated HAE attacks

 Number (%) of patients with HAE attacks

26 (57.8)

40 (88.9)

25 (55.6)

42 (93.3)

 Number (%) of patients without HAE attacks

17 (37.8)

4 (8.9)

18 (40.0)

0

 Number (%) of patients with missing data about occurrence of HAE attacks

2 (4.4)

1 (2.2)

2 (4.4)

3 (6.7)

 Number of all HAE attacks

145

503

71

472

 Number (%) of HAE attacks treated with rescue medication

99 (68.3)

421 (83.7)

35 (49.3)

358 (75.8)

  With 1 rescue medication

92 (92.9)

375 (89.1)

35 (100)

318 (88.8)

   C1-INH (IV) Berinert®

83 (90.2)

338 (90.1)

28 (80.0)

271 (85.2)

   C1-INH (not Berinert IV)

0

1 (0.3)

0

5 (1.6)

   Icatibant (Firazyr®)

8 (8.7)

58 (15.5)

7 (20.0)

63 (19.8)

   Other

1 (1.1)

11 (2.9)

0

1 (0.3)

  With 2 rescue medications

7 (7.1)

33 (7.8)

0

27 (7.5)

   C1-INH (IV) Berinert

7 (100)

14 (42.4)

0

6 (22.2)

   C1-INH (not Berinert IV)

0

0

0

0

   Icatibant (Firazyr)

0

10 (30.3)

0

17 (63.0)

   Other

0

1 (3.0)

0

0

  With ≥ 3 rescue medications

0

13 (3.1)

0

16 (3.6)

   C1-INH (IV) Berinert

0

5 (38.5)

0

2 (15.4)

   C1-INH (not Berinert IV)

0

0

0

1 (7.7)

   Icatibant (Firazyr)

0

4 (30.8)

0

7 (53.8)

   Other

0

0

0

0

  1. C1-INH: C1-esterase inhibitor; CI: confidence interval; HAE: hereditary angioedema; IV: intravenous; N: number of patients in the intention-to-treat population; n: number of patients with data; SC: subcutaneous; SD: standard deviation
  2. aLeast squares estimate from a mixed-model