. 2022 Oct 22;40(1):211–232. doi: 10.1007/s12325-022-02339-3

Table 2.

Summary of efficacy endpoints through the randomized treatment period (full analysis set) and SUBQ treatment period (SUBQ treated full analysis set)

	Randomized treatment period (through day 71)	SUBQ treatment period (through SUBQ day 351)^a
Percentage change in LDH from baseline, mean (SD) [95% CI]	(n = 82) 2.6 (33.9) [− 4.9, 10.0]	(n = 43) 5.7 (29.7) [− 3.4, 14.9]	(n = 73) 1.7 (21.9) [− 3.4, 6.9]	(n = 34) − 0.8 (17.2) [− 6.8, 5.2]	(N = 107) 0.9 (20.5) [− 3.0, 4.9]
BTH, n (%) [95% CI]	1 (1.2) [0.0, 6.5]	1 (2.2) [0.1, 11.8]	3 (3.6) [0.7, 10.1]	2 (4.5)^c [0.6, 15.5]	5 (3.9) [1.3, 8.9]
Transfusion avoidance, n (%) [95% CI]	79 (94.0) [86.7, 98.0]	39 (86.7) [73.2, 95.0]	72 (85.7) [76.4, 92.4]	35 (79.5) [64.7, 90.2]	107 (83.6) [76.0, 89.6]
Hemoglobin stabilization, n (%) [95% CI]	(n = 78) 73 (93.6) [85.7, 97.9]	(n = 44) 36 (81.8) [67.3, 91.8]	(n = 79) 66 (83.5) [73.5, 90.9]	(n = 44) 32 (72.7) [57.2, 85.0]	(N = 123) 98 (79.7) [71.5, 86.4]
Clinical manifestations of PNH, n (%) [95% CI]^d	40 (47.6) [36.6, 58.8]	21 (46.7) [31.7, 62.1]	53 (63.1) [51.9, 73.4]	22 (50.0) [34.6, 65.4]	75 (58.6) [49.6, 67.2]
Percentage change reticulocyte count from baseline, mean (SD) [95% CI]	(n = 64) 5.2 (22.7) [− 0.4, 10.9]	(n = 39) 3.1 (18.8) [− 3.0, 9.2]	(n = 55) 6.1 (26.3) [− 1.0, 13.2]	(n = 29) 0.4 (19.5) [− 7.0, 7.8]	(N = 84) 4.1 (24.2) [− 1.1, 9.4]
Percentage change eGFR from baseline, mean (SD) [95% CI]	(n = 82) 3.7 (16.2) [0.1, 7.2]	(n = 43) 9.2 (20.0) [3.1, 15.4]	(n = 73) 8.0 (21.0) [3.1, 12.9]	(n = 34) − 2.1 (12.6) [− 6.6, 2.3]	(N = 107) 4.8 (19.3) [1.1, 8.5]
Total PNH RBC clone size, mean (SD) [95% CI]	(n = 59) 43.4 (34.5) [0.7, 100.0]	(n = 34) 55.9 (32.3) [4.2, 98.0]	NA	NA	NA

Randomized treatment period (through day 71)

SUBQ treatment period (through SUBQ day 351)^a

SUBQ ravulizumab (n = 84)

IV ravulizumab (n = 45)

SUBQ/SUBQ ravulizumab^b (n = 84)

IV/SUBQ ravulizumab^b (n = 44)

Total (N = 128)

Percentage change in LDH from baseline, mean (SD) [95% CI]

(n = 82)

2.6 (33.9)

[− 4.9, 10.0]

(n = 43)

5.7 (29.7)

[− 3.4, 14.9]

(n = 73)

1.7 (21.9)

[− 3.4, 6.9]

(n = 34)

− 0.8 (17.2)

[− 6.8, 5.2]

(N = 107)

0.9 (20.5)

[− 3.0, 4.9]

BTH, n (%) [95% CI]

1 (1.2)

[0.0, 6.5]

1 (2.2)

[0.1, 11.8]

3 (3.6)

[0.7, 10.1]

2 (4.5)^c

[0.6, 15.5]

5 (3.9)

[1.3, 8.9]

Transfusion avoidance, n (%) [95% CI]

79 (94.0)

[86.7, 98.0]

39 (86.7)

[73.2, 95.0]

72 (85.7)

[76.4, 92.4]

35 (79.5)

[64.7, 90.2]

107 (83.6)

[76.0, 89.6]

Hemoglobin stabilization, n (%) [95% CI]

(n = 78)

73 (93.6)

[85.7, 97.9]

(n = 44)

36 (81.8)

[67.3, 91.8]

(n = 79)

66 (83.5)

[73.5, 90.9]

(n = 44)

32 (72.7)

[57.2, 85.0]

(N = 123)

98 (79.7)

[71.5, 86.4]

Clinical manifestations of PNH, n (%) [95% CI]^d

40 (47.6)

[36.6, 58.8]

21 (46.7)

[31.7, 62.1]

53 (63.1)

[51.9, 73.4]

22 (50.0)

[34.6, 65.4]

75 (58.6)

[49.6, 67.2]

Percentage change reticulocyte count from baseline, mean (SD) [95% CI]

(n = 64)

5.2 (22.7)

[− 0.4, 10.9]

(n = 39)

3.1 (18.8)

[− 3.0, 9.2]

(n = 55)

6.1 (26.3)

[− 1.0, 13.2]

(n = 29)

0.4 (19.5)

[− 7.0, 7.8]

(N = 84)

4.1 (24.2)

[− 1.1, 9.4]

Percentage change eGFR from baseline, mean (SD) [95% CI]

(n = 82)

3.7 (16.2)

[0.1, 7.2]

(n = 43)

9.2 (20.0)

[3.1, 15.4]

(n = 73)

8.0 (21.0)

[3.1, 12.9]

(n = 34)

− 2.1 (12.6)

[− 6.6, 2.3]

(N = 107)

4.8 (19.3)

[1.1, 8.5]

Total PNH RBC clone size, mean (SD) [95% CI]

(n = 59)

43.4 (34.5)

[0.7, 100.0]

(n = 34)

55.9 (32.3)

[4.2, 98.0]

BTH breakthrough hemolysis, CI confidence interval, eGFR estimated glomerular filtration rate, IV intravenous, LDH lactate dehydrogenase, NA not applicable, PNH paroxysmal nocturnal hemoglobinuria, SD standard deviation, SUBQ subcutaneous

^aEndpoints for the SUBQ treatment period analyses were based on the start of SUBQ treatment for each patient

^bAll patients in both treatment groups received SUBQ ravulizumab during the SUBQ treatment period. Patients randomized to the SUBQ group during the randomized treatment period received their first dose of SUBQ ravulizumab on day 15. Patients randomized to receive IV ravulizumab during the randomized treatment period had their first dose of SUBQ ravulizumab at the start of the extension period (day 71)

^cIncludes one patient for whom response to “Breakthrough hemolysis experienced?” was entered as “Yes” using only a local laboratory LDH value

^dInvestigator or designee assessed each patient for signs and symptoms of PNH, which may include the following: fatigue, chest pain, abdominal pain, dyspnea, dysphagia, erectile dysfunction, and red/dark urine or hemoglobinuria