Table 1. Summary of end points of key... | American Society of Hematology

Table 1.

Summary of end points of key trials of novel anticomplement-directed therapies in patients with PNH

Anticomplement-directed therapy	Mean increase in Hb (g/dL)	Percent of participants achieving Hb stabilization, %	Decrease in LDH (U/L)	Transfusion avoidance, %	BTH, %	Mean change in FACIT-Fatigue scores
Iptacopan^17-20	2.0-3.59	—	≤1.5 × ULN	95.2	—	8.6-10.8
Pegcetacoplan⁷^,⁸^,^21-24	2.4-3.84	85	≤1.5 × ULN	85-91	10-19	11-32
Danicopan²⁵^,²⁶	2.4-2.94	—	1.8 × ULN	83-91	—	8.0-11
Crovalimab^27-29	1.0-1.2	56-63	≤1.5 × ULN	65-82	4.0-10.4	7.8
Eculizumab⁷^,¹¹^,¹⁵	0.8	49-60	1.25 × ULN	68-82	5.1-23	5.2-12
Ravulizumab⁴^,³⁰	—	68-78	≤1.5 × ULN	73-86	0-4	6-10

Anticomplement-directed therapy	Mean increase in Hb (g/dL)	Percent of participants achieving Hb stabilization, %	Decrease in LDH (U/L)	Transfusion avoidance, %	BTH, %	Mean change in FACIT-Fatigue scores
Iptacopan^17-20	2.0-3.59	—	≤1.5 × ULN	95.2	—	8.6-10.8
Pegcetacoplan⁷^,⁸^,^21-24	2.4-3.84	85	≤1.5 × ULN	85-91	10-19	11-32
Danicopan²⁵^,²⁶	2.4-2.94	—	1.8 × ULN	83-91	—	8.0-11
Crovalimab^27-29	1.0-1.2	56-63	≤1.5 × ULN	65-82	4.0-10.4	7.8
Eculizumab⁷^,¹¹^,¹⁵	0.8	49-60	1.25 × ULN	68-82	5.1-23	5.2-12
Ravulizumab⁴^,³⁰	—	68-78	≤1.5 × ULN	73-86	0-4	6-10

Hb, hemoglobin.