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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
BACKGROUND: Long-term prophylaxis with subcutaneous C1-inhibitor (C1-INH[SC]; HAEGARDA, CSL Behring) in patients with hereditary angioedema (HAE) due to C1-INH deficiency (C1-INH-HAE) was evaluated in an open-label extension follow-up study to the international, double-blind, placebo-controlled COMP...
Autores principales: | , , , , , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7885603/ https://www.ncbi.nlm.nih.gov/pubmed/33588897 http://dx.doi.org/10.1186/s13023-020-01658-4 |
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author | 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 |
author_facet | 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 |
author_sort | Lumry, William R. |
collection | PubMed |
description | BACKGROUND: Long-term prophylaxis with subcutaneous C1-inhibitor (C1-INH[SC]; HAEGARDA, CSL Behring) in patients with hereditary angioedema (HAE) due to C1-INH deficiency (C1-INH-HAE) was evaluated in an open-label extension follow-up study to the international, double-blind, placebo-controlled COMPACT study. The current analysis evaluated patient-reported health-related quality of life (HRQoL) data from 126 patients in the open-label extension study randomized to treatment with C1-INH(SC) 40 IU/kg (n = 63) or 60 IU/kg (n = 63) twice weekly for 52 weeks. HRQoL was evaluated at the beginning of the open-label study and at various time points using the European Quality of Life-5 Dimensions Questionnaire (EQ-5D), the Hospital Anxiety and Depression Scale (HADS), the Work Productivity and Activity Impairment Questionnaire (WPAI), and the Treatment Satisfaction Questionnaire for Medication. The disease-specific Angioedema Quality of Life Questionnaire (AE-QoL) and HAE quality of life questionnaire (HAE-QoL) instruments were administered in a subset of patients. Statistical significance was determined by change-from-baseline 95% confidence intervals (CIs) excluding zero. No adjustment for multiplicity was done. RESULTS: Mean baseline EQ-5D scores (Health State Value, 0.90; Visual Analog Scale, 81.32) were slightly higher (better) than United States population norms (0.825, 80.0, respectively) and mean HADS anxiety (5.48) and depression (2.88) scores were within “normal” range (0–7). Yet, patients using C1-INH(SC) 60 IU/kg demonstrated significant improvement from baseline to end-of-study on the EQ-5D Health State Value (mean change [95% CI], 0.07 [0.01, 0.12] and Visual Analog Scale (7.45 [3.29, 11.62]). In the C1-INH(SC) 60 IU/kg group, there were significant improvements in the HADS anxiety scale (mean change [95% CI], − 1.23 [− 2.08, − 0.38]), HADS depression scale (− 0.95 [− 1.57, − 0.34]), and WPAI-assessed presenteeism (mean change [95% CI], − 23.33% [− 34.86, − 11.81]), work productivity loss (− 26.68% [− 39.92, − 13.44]), and activity impairment (− 16.14% [− 26.36, − 5.91]). Clinically important improvements were achieved in ≥ 25% of patients for all domains except WPAI-assessed absenteeism (which was very low at baseline). Mean AE-QoL total score by visit ranged from 13.39 to 17.89 (scale 0–100; lower scores = less impairment). Mean HAE-QoL global scores at each visit (115.7–122.3) were close to the maximum (best) possible score of 135. CONCLUSIONS: Long-term C1-INH(SC) replacement therapy in patients with C1-INH-HAE leads to significant and sustained improvements in multiple measures of HRQoL. Trial registration A Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema, NCT02316353. Registered December 12, 2014, https://clinicaltrials.gov/ct2/show/NCT02316353. |
format | Online Article Text |
id | pubmed-7885603 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | BioMed Central |
record_format | MEDLINE/PubMed |
spelling | pubmed-78856032021-02-22 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 Orphanet J Rare Dis Research BACKGROUND: Long-term prophylaxis with subcutaneous C1-inhibitor (C1-INH[SC]; HAEGARDA, CSL Behring) in patients with hereditary angioedema (HAE) due to C1-INH deficiency (C1-INH-HAE) was evaluated in an open-label extension follow-up study to the international, double-blind, placebo-controlled COMPACT study. The current analysis evaluated patient-reported health-related quality of life (HRQoL) data from 126 patients in the open-label extension study randomized to treatment with C1-INH(SC) 40 IU/kg (n = 63) or 60 IU/kg (n = 63) twice weekly for 52 weeks. HRQoL was evaluated at the beginning of the open-label study and at various time points using the European Quality of Life-5 Dimensions Questionnaire (EQ-5D), the Hospital Anxiety and Depression Scale (HADS), the Work Productivity and Activity Impairment Questionnaire (WPAI), and the Treatment Satisfaction Questionnaire for Medication. The disease-specific Angioedema Quality of Life Questionnaire (AE-QoL) and HAE quality of life questionnaire (HAE-QoL) instruments were administered in a subset of patients. Statistical significance was determined by change-from-baseline 95% confidence intervals (CIs) excluding zero. No adjustment for multiplicity was done. RESULTS: Mean baseline EQ-5D scores (Health State Value, 0.90; Visual Analog Scale, 81.32) were slightly higher (better) than United States population norms (0.825, 80.0, respectively) and mean HADS anxiety (5.48) and depression (2.88) scores were within “normal” range (0–7). Yet, patients using C1-INH(SC) 60 IU/kg demonstrated significant improvement from baseline to end-of-study on the EQ-5D Health State Value (mean change [95% CI], 0.07 [0.01, 0.12] and Visual Analog Scale (7.45 [3.29, 11.62]). In the C1-INH(SC) 60 IU/kg group, there were significant improvements in the HADS anxiety scale (mean change [95% CI], − 1.23 [− 2.08, − 0.38]), HADS depression scale (− 0.95 [− 1.57, − 0.34]), and WPAI-assessed presenteeism (mean change [95% CI], − 23.33% [− 34.86, − 11.81]), work productivity loss (− 26.68% [− 39.92, − 13.44]), and activity impairment (− 16.14% [− 26.36, − 5.91]). Clinically important improvements were achieved in ≥ 25% of patients for all domains except WPAI-assessed absenteeism (which was very low at baseline). Mean AE-QoL total score by visit ranged from 13.39 to 17.89 (scale 0–100; lower scores = less impairment). Mean HAE-QoL global scores at each visit (115.7–122.3) were close to the maximum (best) possible score of 135. CONCLUSIONS: Long-term C1-INH(SC) replacement therapy in patients with C1-INH-HAE leads to significant and sustained improvements in multiple measures of HRQoL. Trial registration A Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema, NCT02316353. Registered December 12, 2014, https://clinicaltrials.gov/ct2/show/NCT02316353. BioMed Central 2021-02-15 /pmc/articles/PMC7885603/ /pubmed/33588897 http://dx.doi.org/10.1186/s13023-020-01658-4 Text en © The Author(s) 2021 Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data. |
spellingShingle | Research 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 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 |
title | 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 |
title_full | 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 |
title_fullStr | 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 |
title_full_unstemmed | 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 |
title_short | 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 |
title_sort | 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 |
topic | Research |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7885603/ https://www.ncbi.nlm.nih.gov/pubmed/33588897 http://dx.doi.org/10.1186/s13023-020-01658-4 |
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