Table 1. Key clinical parameters at... | American Society of Hematology

Table 1.

Key clinical parameters at baseline, weeks 12 and 24

	Patients A			Patient B			Patient C			Patient D			Patient E			Patient F
	41 y/o, F			51 y/o, F			67 y/o, M			29 y/o, F			22 y/o, F			44 y/o, F
	BL	W12	W24	BL	W12	W24	BL	W12	W24	BL	W12	W24	BL	W12	W24	BL	W12	W24
Danicopan, mg oral TID	100	150	150	100	100	100	150	150	150	150	150	150	100	100	150	100	100	150
Eculizumab, mg IV q14d	900	900	900	1200	900	900	900	900	900	900	900	900	1200	1200	1200	900	900	900
Hgb, g/dL	5.00	7.70	8.50	9.80	11.8	13.3	7.60	9.00	9.70	10.4	11.7	11.5	8.60	7.60	9.40	7.20	9.30	10.6
LDH, ×ULN	1.7	1.2	1.1	1.0	0.8	0.9	0.8	0.9	0.9	1.1	1.2	0.9	1.2	1.3	1.2	0.9	0.9	0.9
Reticulocytes, 10³/μL	159	121	112	250	123	162	141	97.0	87.0	191	56	56	120	179	90	405	243	253
Total bilirubin, mg/dL	2.14	1.80	2.40	1.24	0.89	0.81	1.03	0.580	0.580	2.26	0.700	0.650	2.35	2.22	1.75	3.93	1.50	2.95
Direct bilirubin, mg/dL	0.44	0.33	0.48	0.29	0.18	0.20	0.24	0.16	0.16	0.43	0.15	0.17	0.72	0.40	0.60	0.82	0.49	0.78
GPI-deficient RBC population, %	57	83	—*	80	89	—*	22	59	51	52	99	100	21	37	53	95	99	98
GPI-deficient granulocyte cell population, %	84	87	—*	99	85	—*	81	89	98	99	97	99	82	88	82	98	99	99
C3d⁺ GPI-deficient RBCs, %	6.30	—†	2.20	8.20	—†	1.00	17.5	18.6	16.7	15.9	0.200	5.20	21.3	11.8	4.90	71.0	48.0	53.4
FACIT-Fatigue‡	33	48	52	45	49	48	17	36	26	18	41	41	42	25	38	9	48	52

	Patients A			Patient B			Patient C			Patient D			Patient E			Patient F
	41 y/o, F			51 y/o, F			67 y/o, M			29 y/o, F			22 y/o, F			44 y/o, F
	BL	W12	W24	BL	W12	W24	BL	W12	W24	BL	W12	W24	BL	W12	W24	BL	W12	W24
Danicopan, mg oral TID	100	150	150	100	100	100	150	150	150	150	150	150	100	100	150	100	100	150
Eculizumab, mg IV q14d	900	900	900	1200	900	900	900	900	900	900	900	900	1200	1200	1200	900	900	900
Hgb, g/dL	5.00	7.70	8.50	9.80	11.8	13.3	7.60	9.00	9.70	10.4	11.7	11.5	8.60	7.60	9.40	7.20	9.30	10.6
LDH, ×ULN	1.7	1.2	1.1	1.0	0.8	0.9	0.8	0.9	0.9	1.1	1.2	0.9	1.2	1.3	1.2	0.9	0.9	0.9
Reticulocytes, 10³/μL	159	121	112	250	123	162	141	97.0	87.0	191	56	56	120	179	90	405	243	253
Total bilirubin, mg/dL	2.14	1.80	2.40	1.24	0.89	0.81	1.03	0.580	0.580	2.26	0.700	0.650	2.35	2.22	1.75	3.93	1.50	2.95
Direct bilirubin, mg/dL	0.44	0.33	0.48	0.29	0.18	0.20	0.24	0.16	0.16	0.43	0.15	0.17	0.72	0.40	0.60	0.82	0.49	0.78
GPI-deficient RBC population, %	57	83	—*	80	89	—*	22	59	51	52	99	100	21	37	53	95	99	98
GPI-deficient granulocyte cell population, %	84	87	—*	99	85	—*	81	89	98	99	97	99	82	88	82	98	99	99
C3d⁺ GPI-deficient RBCs, %	6.30	—†	2.20	8.20	—†	1.00	17.5	18.6	16.7	15.9	0.200	5.20	21.3	11.8	4.90	71.0	48.0	53.4
FACIT-Fatigue‡	33	48	52	45	49	48	17	36	26	18	41	41	42	25	38	9	48	52

	Patient G			Patient H			Patient I			Patient J			Patient K
	35 y/o, F			53 y/o, F			51 y/o, F			19 y/o, M			57 y/o, F
	BL	W12	W24	BL	W12	W24	BL	W12	W24	BL	W12	W24	BL	W12	W24
Danicopan, mg oral TID	100	100	100	100	200	200	100	150	200	100	100	200	100	100	150
Eculizumab, mg IV q14d	1500	1500	1500	900	900	900	900	900	900	1200	1200	1200	900	900	900
Hgb, g/dL	7.10	9.40	9.10	7.70	7.30	7.50	7.70	10.8	11.5	7.80	10.2	11.0	14.7	9.40	11.5
LDH, ×ULN	0.6	0.5	0.9	1.5	1.8	1.4	0.9	0.9	0.9	1.3	—*	1.2	0.9	1.2	1.1
Reticulocytes, 10³/μL	262	200	239	238	206	169	200	110	96	258	292	152	188	88	69
Total bilirubin, mg/dL	1.20	1.90	1.25	2.18	2.04	1.57	0.56	0.34	0.32	3.24	—*	1.14	3.76	1.85	1.41
Direct bilirubin, mg/dL	0.39	0.50	0.39	0.78	0.53	0.52	0.23	0.18	0.18	0.68	—*	0.23	0.64	0.39	0.31
GPI-deficient RBC population, %	63	90	96	19	71	—‡	73	95	—*	60	99	99	55	84	91
GPI-deficient granulocyte cell population, %	100	100	100	94	95	—*	98	98	—*	99	100	100	75	82	88
C3d⁺ GPI-deficient RBCs, %	80.0	29.4	57.0	38.8	14.9	22.8	13.6	0.30	0.10	34.3	57.9	28.7	20.2	44.2	6.30
FACIT-Fatigue‡	28	44	38	47	49	49	48	50	49	49	51	51	33	46	50

	Patient G			Patient H			Patient I			Patient J			Patient K
	35 y/o, F			53 y/o, F			51 y/o, F			19 y/o, M			57 y/o, F
	BL	W12	W24	BL	W12	W24	BL	W12	W24	BL	W12	W24	BL	W12	W24
Danicopan, mg oral TID	100	100	100	100	200	200	100	150	200	100	100	200	100	100	150
Eculizumab, mg IV q14d	1500	1500	1500	900	900	900	900	900	900	1200	1200	1200	900	900	900
Hgb, g/dL	7.10	9.40	9.10	7.70	7.30	7.50	7.70	10.8	11.5	7.80	10.2	11.0	14.7	9.40	11.5
LDH, ×ULN	0.6	0.5	0.9	1.5	1.8	1.4	0.9	0.9	0.9	1.3	—*	1.2	0.9	1.2	1.1
Reticulocytes, 10³/μL	262	200	239	238	206	169	200	110	96	258	292	152	188	88	69
Total bilirubin, mg/dL	1.20	1.90	1.25	2.18	2.04	1.57	0.56	0.34	0.32	3.24	—*	1.14	3.76	1.85	1.41
Direct bilirubin, mg/dL	0.39	0.50	0.39	0.78	0.53	0.52	0.23	0.18	0.18	0.68	—*	0.23	0.64	0.39	0.31
GPI-deficient RBC population, %	63	90	96	19	71	—‡	73	95	—*	60	99	99	55	84	91
GPI-deficient granulocyte cell population, %	100	100	100	94	95	—*	98	98	—*	99	100	100	75	82	88
C3d⁺ GPI-deficient RBCs, %	80.0	29.4	57.0	38.8	14.9	22.8	13.6	0.30	0.10	34.3	57.9	28.7	20.2	44.2	6.30
FACIT-Fatigue‡	28	44	38	47	49	49	48	50	49	49	51	51	33	46	50

F, female; M, male; q14d, every 14 days; TID, thrice daily; ULN, upper limit of normal; y/o, years old.

Stability of the sample was interrupted.

C3 fragment deposition was not tested at week 12 for this subject. No data entry.

Scores based on the FACIT-Fatigue Scale V4. Score ranges from 0 to 52; a score < 30 indicates severe fatigue.