Table 1. Patient characteristics Patient... | American Society of Hematology

Table 1.

Patient characteristics

Patient	Parent study	Sex	Medical history of AA/MDS	Prior acute BTH episodes on PEG∗,†	Before entering parent study						At OLE baseline
Patient	Parent study	Sex	Medical history of AA/MDS	Prior acute BTH episodes on PEG∗,†	ECU dose	LDH (U/L)	Hb (g/dL)	CH50 (U/mL)	RBC transfusions within 12 mo	Medical history of thrombosis	LDH (U/L)	Hb (g/dL)	Reticulocytes (× 10⁹/L)	Bilirubin (μmol/L)	PEG dose frequency before intensive treatment
1	PEGASUS	Female	no/no	no	900 mg every 2 wk	265	8.6	0	2	yes	168	12.5	70	7.4	BIW
2	PEGASUS	Female	no/no	yes	900 mg every 2 wk	177	8.1	9.43	1	yes	609	11.7	120	1.9	Q3D‡
3	PEGASUS	Female	no/no	yes	900 mg every 2 wk	159	8.2	2.42	1	yes	132	11.5	40	6.7	BIW
4	PEGASUS	Female	yes/no	yes	900 mg every 2 wk	214	7.1	4.9	27	no	187	10.1	60	16.1	BIW
5	PEGASUS	Female	no/no	yes	1200 mg every 2 wk	243	10.9	2.61	9	no	162	11.9	70	11.1	Q3D‡
6	PEGASUS	Male	yes/no	no	900 mg every 2 wk	213	8.7	0.13	6	no	154	11.6	80	8.2	BIW
7	PEGASUS	Male	no/no	yes	1200 mg every 2 wk	216	9.3	6.4	0	yes	181	13.5	120	19.0	Q3D‡
8	PEGASUS	Male	yes/no	no	900 mg every 2 wk	188	8.5	0	8	no	262	11.8	80	10.4	BIW
9	PEGASUS	Male	no/no	no	900 mg every 2 wk	503	6.7	22.35	3	no	336	13.9	120	5.3	BIW
10	PEGASUS	Male	no/no	no	1200 mg every 2 wk	225	5.5	8.21	12	no	224	12.6	NR	31.0	BIW
11	PADDOCK	Male	yes/no	yes	Treatment naïve	1866	10.4	589	11	no	343	15.0	80	11.1	TIW‡,§
12	PRINCE	Male	no/no	no	Treatment naïve	3195	9.7	143	16	yes	193	11.8	92	31.5	BIW
13	PRINCE	Male	yes/no	no	Treatment naïve	1371	8.6	72.17	15	no	283	8.0	78	16.6	Q3D§

Patient	Parent study	Sex	Medical history of AA/MDS	Prior acute BTH episodes on PEG∗,†	Before entering parent study						At OLE baseline
Patient	Parent study	Sex	Medical history of AA/MDS	Prior acute BTH episodes on PEG∗,†	ECU dose	LDH (U/L)	Hb (g/dL)	CH50 (U/mL)	RBC transfusions within 12 mo	Medical history of thrombosis	LDH (U/L)	Hb (g/dL)	Reticulocytes (× 10⁹/L)	Bilirubin (μmol/L)	PEG dose frequency before intensive treatment
1	PEGASUS	Female	no/no	no	900 mg every 2 wk	265	8.6	0	2	yes	168	12.5	70	7.4	BIW
2	PEGASUS	Female	no/no	yes	900 mg every 2 wk	177	8.1	9.43	1	yes	609	11.7	120	1.9	Q3D‡
3	PEGASUS	Female	no/no	yes	900 mg every 2 wk	159	8.2	2.42	1	yes	132	11.5	40	6.7	BIW
4	PEGASUS	Female	yes/no	yes	900 mg every 2 wk	214	7.1	4.9	27	no	187	10.1	60	16.1	BIW
5	PEGASUS	Female	no/no	yes	1200 mg every 2 wk	243	10.9	2.61	9	no	162	11.9	70	11.1	Q3D‡
6	PEGASUS	Male	yes/no	no	900 mg every 2 wk	213	8.7	0.13	6	no	154	11.6	80	8.2	BIW
7	PEGASUS	Male	no/no	yes	1200 mg every 2 wk	216	9.3	6.4	0	yes	181	13.5	120	19.0	Q3D‡
8	PEGASUS	Male	yes/no	no	900 mg every 2 wk	188	8.5	0	8	no	262	11.8	80	10.4	BIW
9	PEGASUS	Male	no/no	no	900 mg every 2 wk	503	6.7	22.35	3	no	336	13.9	120	5.3	BIW
10	PEGASUS	Male	no/no	no	1200 mg every 2 wk	225	5.5	8.21	12	no	224	12.6	NR	31.0	BIW
11	PADDOCK	Male	yes/no	yes	Treatment naïve	1866	10.4	589	11	no	343	15.0	80	11.1	TIW‡,§
12	PRINCE	Male	no/no	no	Treatment naïve	3195	9.7	143	16	yes	193	11.8	92	31.5	BIW
13	PRINCE	Male	yes/no	no	Treatment naïve	1371	8.6	72.17	15	no	283	8.0	78	16.6	Q3D§

AA, aplastic anemia; BIW, twice weekly; BTH, breakthrough hemolysis; ECU, eculizumab; Hb, hemoglobin; LDH, lactate dehydrogenase; MDS, myelodysplastic syndrome; OLE, open-label extension; NR, not reported; PEG, pegcetacoplan; Q3D, every 3 days; RBC, red blood cell; TIW, 3 times weekly.

∗

AE terms reported for acute BTH (hemolysis, BTH, IVH, and acute hemolysis) were used to assess prior acute BTH episodes.

†

Entire duration of PEG treatment.

‡

Patient had LDH >2× ULN and experienced an AE of BTH that was deemed related to PNH by the investigators.

Patient had LDH >2× ULN and experienced an AE of anemia that was deemed related to PNH by the investigators.

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