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

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

Research output: Contribution to journalArticlepeer-review

34 Scopus citations


Background: Hereditary angioedema with C1-inhibitor deficiency (C1-INH-HAE) impairs health-related quality of life (HRQoL). Objective: The objective of this study was to assess HRQoL outcomes in patients self-administering subcutaneous C1-INH (C1-INH[SC]; HAEGARDA) for routine prevention of HAE attacks. Methods: Post hoc analysis of data from the placebo-controlled, crossover phase III COMPACT study (Clinical Studies for Optimal Management of Preventing Angioedema with Low-Volume Subcutaneous C1-Inhibitor Replacement Therapy). Ninety patients with C1-INH-HAE were randomized to 1 of 4 treatment sequences: C1-INH(SC) 40 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). Results: Compared 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. Conclusions: In 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).

Original languageEnglish (US)
Pages (from-to)1733-1741.e3
JournalJournal of Allergy and Clinical Immunology: In Practice
Issue number5
StatePublished - Sep 1 2018


  • C1-inhibitor protein
  • Hereditary angioedema
  • Patient-reported outcomes
  • Productivity
  • Quality of life
  • Satisfaction
  • Subcutaneous

ASJC Scopus subject areas

  • Immunology and Allergy


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