The burden of illness of patients with paroxysmal nocturnal haemoglobinuria receiving C5 inhibitors: clinical outcomes and medical encounters from the patient perspective

Figures & data

Figure 1. Patient flow chart. ^aEthical considerations precluded capture of reasons for voluntary discontinuation and any further data on respondents who quit (see Methods, Ethics Statements and Data Confidentiality).

Table 1. Patient characteristics.

Characteristics: Treatment Subgroups	Total (n = 71)			Eculizumab (n = 49)			Ravulizumab (n = 22)
	Mean (SD)	Median	n (%)	Mean (SD)	Median	n (%)	Mean (SD)	Median	n (%)
Demographics
Age (years)	43.0 (13.1)	42.0	–	42.3 (12.6)	42.0	–	44.4 (14.3)	44.5	–
Sex (female)	–	–	47 (66.2)	–	–	32 (65.3)	–	–	15 (68.2)
Have caregiver (yes)	–	–	23 (32.4)	–	–	16 (32.7)	–	–	7 (31.8)
Live with caregiver (yes)	–	–	19 (82.6)	–	–	13 (81.3)	–	–	6 (85.7)
Clinical characteristics
Weight (kg)*	73.4 (14.9)	72.3	–	69.7^a (13.4)	68.9	–	78.2^a (15.6)	79.8	–
Age at diagnosis (years)	29.8 (11.6)	27.0	–	29.9 (11.5)	27.0	–	29.6 (12.3)	25.5	–
Time from diagnosis (years)	13.2 (8.8)	13.0	–	12.5 (8.5)	12.0	–	14.8 (9.5)	14.5	–
Serum hemoglobin (Hb) levels^b (N = 63; eculizumab = 41; ravulizumab = 22)
Total Hb (g/dL)	10.2 (2.0)	10.0	–	10.2 (2.1)	10.0	–	10.2 (1.8)	10.5	–
Among those treated with C5i for ≥12 months	10.2 (2.0)	10.1	–	10.2 (2.1)	10.0	–	10.3 (1.8)	10.6	–
Comorbidities
Aplastic anaemia /severe aplastic anaemia	–	–	26 (36.6)	–	–	17 (34.7)	–	–	9 (40.9)
Myelodysplastic syndrome	–	–	1 (1.4)	–	–	0 (0)	–	–	1 (4.5)
Other bone marrow disorders	–	–	4 (5.6)	–	–	4 (8.2)	–	–	0 (0)
Characteristics: Hb level subgroups	Total (n = 63)			<10.5 g/dL (n = 36)			≥10.5 g/dL (n = 27)
	Mean (SD)	Median	N (%)	Mean (SD)	Median	n (%)	Mean (SD)	Median	n (%)
Demographics
Age (years)	43.3 (12.5)	42.0	–	43.8 (12.7)	44.0	–	42.6 (12.6)	42.0	–
Sex (female)	–	–	45 (71.4)	–	–	30 (83.3)^c	–	–	15 (55.6)^c
Have caregiver (yes)	–	–	22 (34.9)	–	–	11 (30.6)	–	–	11 (40.7)
Live with caregiver (yes)	–	–	18 (81.8)	–	–	10 (90.9)	–	–	8 (72.7)
Clinical characteristics
Weight (kg)	73.4 (14.9)	72.3	–	74.7 (16.3)	73.9	–	72.1 (13.5)	70.8	–
Age at diagnosis (years)	29.7 (11.7)	27.0	–	30.1 (12.6)	26.0	–	29.2 (10.6)	27.0	–
Time from diagnosis (years)	13.5 (8.0)	13.0	–	13.6 (7.1)	14.0	–	13.4 (9.2)	12.0	–
Mean serum hemoglobin levels^b (among those treated for ≥12 months; n = 63) (g/dL)	10.2 (2.0)	10.0	–	8.9 (1.3)	9.1	–	12.0 (1.1)	11.9	–
Comorbidities
Aplastic anaemia (including severe)	–	–	26 (41.3)	–	–	15 (41.7)	–	–	11 (40.7)
Myelodysplastic syndrome	–	–	1 (1.6)	–	–	1 (2.8)	–	–	0 (0.0)
Other bone marrow disorder^d	–	–	4 (6.3)	–	–	0 (0.0)	–	–	4 (14.8)

Notes: C5i: C5 inhibitor; Hb: (serum) hemoglobin. *Weight was collected only from relevant respondents to assess dosing patterns per ravulizumab prescribing categories; answer choices included ‘prefer not to answer.’ ^aSignificant at P = 0.045. ^bMost recent, among patients with reported levels. ^cSignificant at P = 0.016. ^dChi-square tests may be invalid due to cell counts < 5.

Table 2. Dosage.

Last dose: treatment subgroups	Total		40 ≤ BW < 60		60 ≤ BW < 100		BW ≥ 100
	N	%	N	%	N	%	N	%
Ravulizumab	22		3		15		4
900 mg	1	4.5	0	0.0	1	6.7	0	0.0
2400 mg	1	4.5	0	0.0	1	6.7	0	0.0
2700 mg	1	4.5	0	0.0	1	6.7	0	0.0
3000 mg	4	18.2	2	66.7^a	2	13.3	0	0.0
3300 mg	10	45.5	0	0.0	7	46.7^a	3	75.0
3600 mg	2	9.1	0	0.0	1	6.7	1	25.0^a
Don’t know	3	13.6	1	33.3	2	13.3	0	0.0
Eculizumab	49
600 mg	2	4.1	–	–	–	–	–	–
900 mg	30	61.2^a	–	–	–	–	–	–
1200 mg	11	22.4	–	–	–	–	–	–
^bOther (specified)	3	6.1	–	–	–	–	–	–
Don’t know	3	6.1	–	–	–	–	–	–

Notes: BW: body weight (in kilograms). ^aLabel-recommended dosage. ^bAll patients specified 1500 mg.

Figure 2. Haematological outcomes. BTH, breakthrough haemolysis; Hb, (serum) hemoglobin.

Notes: Data reflect events in the past 12 months, among query respondents treated for ≥12 months. Breakthrough haemolysis was defined in the survey as: ‘ … the return of haemolytic disease activity e.g. return of (my) symptoms/ anaemia / major vascular event e.g. a blood clot.’

Figure 3. Medical encounters. Rx, treatment subgroups; Hb, haemoglobin subgroups; PNH, paroxysmal nocturnal haemoglubinuria. ^aEculizumab versus ravulizumab significant at p = 0.011.

Data availability

Anonymised patient source data are not available; all relevant analysed data are included in the manuscript and supplementary materials.

Data availability

Anonymised patient source data are not available; all relevant analysed data are included in the manuscript and supplementary materials.