. 2023 Mar 9;16:269–277. doi: 10.2147/JAA.S396338

Table 3.

Additional Efficacy Endpoints in Randomized Controlled Trials of Long-Term HAE Prophylaxis with C1-INH Replacement Therapy

Parameter	Plasma-Derived C1-INH IV²²	Plasma-Derived C1-INH SC¹⁸	Recombinant Human C1-INH IV²¹
≥50% Reduction in attacks versus placebo (95% CI)	pdC1-INH 1000 U (n = 11): 50%	40 IU/kg (n = 43): 76% (62–87) 60 IU/kg (n = 43): 90% (77–96)	ITT (n = 31) rhC1-INH twice weekly: 74% (57–86) rhC1-INH once weekly: 42% (26–59) PP (n = 23) rhC1-INH twice weekly: 96% (79–99) rhC1-INH once weekly: 57% (37–74)
≥70% or ≥75% Reduction in attacks versus placebo (95% CI)	≥75% reduction pdC1-INH 1000 U (n = 11): 45%	≥70% reduction 40 IU/kg (n = 43): 67% (52–79) 60 IU/kg (n = 43): 83% (68–91)	≥75% reduction^a PP (n = 23) rhC1-INH twice weekly: 52% (33–71) rhC1-INH once weekly: 26% (12–46)
≥90% Reduction in attacks versus placebo (95% CI)	pdC1-INH 1000 U (n = 11): 23%	40 IU/kg (n = 43): 43% (29–58) 60 IU/kg (n = 43): 58% (42–72)	PP (n = 23)^a rhC1-INH twice weekly: 26% rhC1-INH once weekly: 9%

Notes: ^aData on file. Pharming Healthcare, Inc.

Abbreviations: C1-INH, C1 esterase inhibitor; HAE, hereditary angioedema; ITT, intention-to-treat; IV, intravenous; pdC1-INH, plasma-derived C1-INH; PP, per protocol; rhC1-INH, recombinant human C1-INH; SC, subcutaneous.