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

Orphanet Journal of Rare Diseases

卷:16

on behalf of the COMPACT Investigators¹  William R. Lumry²  Teresa Caballero³  John-Philip Lawo⁴  Subhransu Prusty⁴  Thomas Machnig⁴  John Anderson⁵  Markus Magerl⁶  Bruce Zuraw⁷  Marc A. Riedl⁸  Henriette Farkas⁹  H. Henry Li¹⁰  Richard G. Gower¹¹  Aleena Banerji¹²  Paul K. Keith¹³  Michael Manning¹⁴  Timothy Craig¹⁵  Donald S. Levy¹⁶  Hilary Longhurst¹⁷  Jonathan A. Bernstein¹⁸  Marco Cicardi¹⁹ 

[1] ;

[2] AARA Research Center;

[3] Allergy Department, Hospital La Paz Institute for Health Research (IdiPaz), Biomedical Research Network On Rare Diseases (CIBERER U754);

[4] CSL Behring GmbH;

[5] Clinical Research Center of Alabama;

[6] Department of Dermatology and Allergy, Charité, Universitätsmedizin Berlin;

[7] Department of Medicine, UC San Diego;

[8] Division of Rheumatology, Allergy and Immunology, University of California, San Diego;

[9] Hungarian Angioedema Reference Center, 3Rd Department of Internal Medicine, Semmelweis University;

[10] Institute for Asthma and Allergy;

[11] Marycliff Clinical Research;

[12] Massachusetts General Hospital;

[13] McMaster University;

[14] Medical Research of Arizona;

[15] Penn State University College of Medicine;

[16] UC Irvine;

[17] University College Hospital;

[18] University of Cincinnati College of Medicine and Bernstein Clinical Research Center, LLC;

[19] University of Milan;

关键词: C1-inhibitor protein;    Hereditary angioedema;    Patient-reported outcomes;    Health-related quality of life;    Productivity;    Subcutaneous;   

DOI  :  10.1186/s13023-020-01658-4

来源: DOAJ

【 摘 要 】

Abstract 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 .

【 授权许可】

Unknown