Health-Related Quality of Life with Subcutaneous C1-Inhibitor for Prevention of Attacks of Hereditary Angioedema William R. Lumry, MD, Timothy Craig,

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The Journal of Allergy and Clinical Immunology: In Practice

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Presentation transcript:

Health-Related Quality of Life with Subcutaneous C1-Inhibitor for Prevention of Attacks of Hereditary Angioedema William R. Lumry, MD, Timothy Craig, DO, Bruce Zuraw, MD, Hilary Longhurst, MD, PhD, James Baker, MD, H. Henry Li, MD, PhD, Jonathan A. Bernstein, MD, John Anderson, MD, Marc A. Riedl, MD, Michael E. Manning, MD, Paul K. Keith, MD, MSc, Donald S. Levy, MD, Teresa Caballero, MD, PhD, Aleena Banerji, MD, Richard G. Gower, MD, Henriette Farkas, MD, PhD, DSc, John-Philip Lawo, MSc, Ingo Pragst, PhD, Thomas Machnig, MD, MBA, Douglas J. Watson, PhD The Journal of Allergy and Clinical Immunology: In Practice Volume 6, Issue 5, Pages 1733-1741.e3 (September 2018) DOI: 10.1016/j.jaip.2017.12.039 Copyright © 2018 The Authors Terms and Conditions

Figure 1 COMPACT study design and timing of health-related quality of life and productivity assessments. C1-INH(SC), Subcutaneous C1-inhibitor; COMPACT, Clinical Studies for Optimal Management of Preventing Angioedema with Low-Volume Subcutaneous C1-Inhibitor Replacement Therapy; EQ-5D, European Quality of Life-5 Dimensions; HADS, Hospital Anxiety and Depression Scale; HAE, hereditary angioedema; IGART/SGART, Investigator and Subject Global Assessments of Response to Therapy; TSQM, Treatment Satisfaction Questionnaire for Medication; WPAI, Work Productivity and Activity Impairment Questionnaire. The Journal of Allergy and Clinical Immunology: In Practice 2018 6, 1733-1741.e3DOI: (10.1016/j.jaip.2017.12.039) Copyright © 2018 The Authors Terms and Conditions

Figure 2 Mean difference in within-subject health-related quality of life scores between C1-INH(SC) (both doses combined) and placebo treatments and 95% CI at the last assessment visit. The gray shading indicates differences considered statistically significant (95% CI does not overlap with 0); aTSQM results for the side effects domain not shown; n = 1 in each sequence. Numerical data for this figure are provided in Table E2 (available in this article's Online Repository at www.jaci-inpractice.org). C1-INH(SC), Subcutaneous C1-inhibitor; CI, confidence interval; EQ-5D, European Quality of Life-5 Dimensions; HADS, Hospital Anxiety and Depression Scale; n, number of patients with data; TSQM, Treatment Satisfaction Questionnaire for Medication; WPAI, Work Productivity and Activity Impairment Questionnaire. The Journal of Allergy and Clinical Immunology: In Practice 2018 6, 1733-1741.e3DOI: (10.1016/j.jaip.2017.12.039) Copyright © 2018 The Authors Terms and Conditions

Figure 3 Percentage of patients demonstrating minimal clinically important difference (MCID) improvements in health-related quality of life scores—change from screening to the last assessment visit with C1-INH(SC) or placebo. C1-INH(SC), Subcutaneous C1-inhibitor; EQ-5D, European Quality of Life-5 Dimensions; HADS, Hospital Anxiety and Depression Scale; TSQM, Treatment Satisfaction Questionnaire for Medication; WPAI, Work Productivity and Activity Impairment Questionnaire. The Journal of Allergy and Clinical Immunology: In Practice 2018 6, 1733-1741.e3DOI: (10.1016/j.jaip.2017.12.039) Copyright © 2018 The Authors Terms and Conditions

Figure E1 Mean scores over time by treatment group (combined placebo vs combined C1-INH(SC) 40 IU/kg and 60 IU/kg) for (A) quality of life/health status (EQ-5D), (B) anxiety and depression (HADS), (C) absenteeism and presenteeism (WPAI), (D) work productivity loss and activity impairment over time (WPAI), and (E) effectiveness and overall satisfaction (TSQM). C1-INH(SC), Subcutaneous C1-inhibitor; EQ-5D, European Quality of Life-5 Dimensions; HADS, Hospital Anxiety and Depression Scale; SD, standard deviation; TSQM, Treatment Satisfaction Questionnaire for Medication; WPAI, Work Productivity and Activity Impairment Questionnaire. The Journal of Allergy and Clinical Immunology: In Practice 2018 6, 1733-1741.e3DOI: (10.1016/j.jaip.2017.12.039) Copyright © 2018 The Authors Terms and Conditions