Long-term health-related quality of life in patients treated with subcutaneous C1-inhibitor replacement therapy for the prevention of hereditary angioedema attacks: findings from the COMPACT open-label extension study

Lumry, William R.; Zuraw, Bruce; Cicardi, Marco; Craig, Timothy; Anderson, John; Banerji, Aleena; Bernstein, Jonathan A.; Caballero, Teresa; Farkas, Henriette; Gower, Richard G.; Keith, Paul K.; Levy, Donald S.; Li, H. Henry; Magerl, Markus; Manning, Michael; Riedl, Marc A.; Lawo, John-Philip; Prusty, Subhransu; Machnig, Thomas; Longhurst, Hilary

doi:10.1186/s13023-020-01658-4

Orphanet Journal of Rare Diseases

Table 2 HRQoL assessments (EQ-5D, HADS, WPAI, TSQM) at baseline and end-of-study, both C1-INH(SC) doses combined (N = 126)

From: Long-term health-related quality of life in patients treated with subcutaneous C1-inhibitor replacement therapy for the prevention of hereditary angioedema attacks: findings from the COMPACT open-label extension study

HRQoL assessment	Scoring and interpretation	Baseline		End of study (week 53 or extension week 88)		Intra-subject change from baseline^a
HRQoL assessment	Scoring and interpretation	n	Mean (SD)	n	Mean (SD)	n	Mean (SD)
EQ-5D
Health state value	Health state value: scored from 0 (dead) to 1 (full health); population norm in the US = 0.825 [34] VAS: scored from 0 (worst) to 100 (best imaginable health state); population norm in the US = 80.0 [34]	114	0.90 (0.168)	100	0.95 (0.098)	92	0.05 (0.153)
VAS		114	81.32 (17.600)	100	87.72 (13.036)	92	5.83 (14.601)
HADS
Depression	Both domains: 0–7 (normal) 8–10 (suggestive of the mood disorder) > 11 (probable presence of the mood disorder) Maximum (worst) score possible per domain = 21	114	2.88 (2.930)	100	1.91 (2.629)	92	− 0.80 (2.641)
Anxiety		114	5.48 (3.863)	100	4.11 (3.533)	92	− 1.23 (3.089)
WPAI
Absenteeism (% work time missed)^b	All domains Higher % = greater impairment (maximum score = 100%)	69	6.15 (17.056)	68	2.67 (11.670)	52	− 4.89 (23.296)
Presenteeism (% impairment while working)^b		68	19.71 (26.653)	68	8.24 (18.604)	51	− 14.31 (31.765)
Work productivity loss (% overall work impairment)^b		68	22.11 (29.018)	68	9.45 (20.766)	51	− 15.97 (34.946)
Activity impairment (% activity (non-work) impairment		114	27.54 (29.436)	100	14.40 (24.917)	92	− 14.35 (32.013)
TSQM
Effectiveness	Each domain Scoring range is 0–100; higher scores indicate better outcomes	104	73.88 (25.000)	100	83.67 (27.237)	82	15.04 (29.854)
Convenience		104	70.94 (16.965)	100	80.56 (17.470)	82	8.81 (17.632)
Overall satisfaction		104	78.79 (22.414)	100	92.42 (13.253)	82	14.57 (23.016)

C1-INH(SC) subcutaneous C1-inhibitor, EQ-5D European Quality of Life-5 Dimensions Questionnaire, HADS Hospital Anxiety and Depression Scale, HRQoL health-related quality of life, TSQM Treatment Satisfaction Questionnaire for Medication, WPAI Work Productivity and Activity Impairment Questionnaire, SD standard deviation, VAS visual analog scale
^aThe Change from Baseline analysis included only patients with both Baseline and End of Study scores, thus the number of patients in this column may be smaller than that shown in the End of Study column as a consequence of missing baseline data
^bAssessment completed only by employed patients

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ISSN: 1750-1172

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