Health-Related Quality-of-Life with Subcutaneous C1-inhibitor for Prevention of Attacks of Hereditary Angioedema

Publication date: Available online 31 January 2018
Source:The Journal of Allergy and Clinical Immunology: In Practice
Author(s): William R. Lumry, Timothy Craig, Bruce Zuraw, Hilary Longhurst, James Baker, H.Henry Li, Jonathan A. Bernstein, John Anderson, Marc A. Riedl, Michael E. Manning, Paul K. Keith, Donald S. Levy, Teresa Caballero, Aleena Banerji, Richard G. Gower, Henriette Farkas, John-Philip Lawo, Ingo Pragst, Thomas Machnig, Douglas J. Watson
BackgroundHereditary angioedema with C1-INH deficiency (C1-INH-HAE) impairs health-related quality of life (HRQoL).ObjectiveTo assess HRQoL outcomes in patients self-administering subcutaneous C1-INH (C1-INH[SC]; HAEGARDA^®) for routine prevention of HAE attacks.MethodsPost-hoc analysis of data from a placebo-controlled, crossover phase III study (COMPACT). Ninety patients with C1-INH-HAE were randomized to 1 of 4 treatment sequences: C1-INH(SC) 40 IU/kg or 60 IU/kg twice-weekly for 16 weeks, preceded or followed by 16 weeks of twice-weekly placebo injections. All HAE attacks were treated with open-label on-demand treatment as necessary. HRQoL assessments at week 14 (last visit) included the European Quality of Life-5 Dimensions Questionnaire (EQ-5D-3L), the Hospital Anxiety and Depression Scale (HADS), the Work Productivity and Activity Impairment Questionnaire (WPAI), and the Treatment Satisfaction Questionnaire for Medication (TSQM).ResultsCompared with placebo (on-demand treatment alone), treatment with twice-weekly C1-INH(SC) (both doses combined) was associated with better EQ-5D visual analog scale general health, less HADS anxiety, less WPAI presenteeism, work productivity loss, and activity impairment, and greater TSQM effectiveness and overall treatment satisfaction. More patients self-reported a "good/excellent" response during routine prevention with C1-INH(SC) compared with on-demand only (placebo prophylaxis) management. For each HRQoL measure, a greater proportion of patients had a clinically meaningful improvement during C1-INH(SC) treatment compared with placebo.ConclusionsIn patients with frequent HAE attacks, a treatment strategy of routine prevention with self-administered twice-weekly C1-INH(SC) had a greater impact on improving multiple HAE-related HRQoL impairments, most notably anxiety and work productivity, compared with on-demand treatment alone (placebo prophylaxis).