scholarly journals Long‐term safety and effectiveness of berotralstat for hereditary angioedema: The open‐label APeX‐S study

2021 ◽  
Vol 11 (4) ◽  
Author(s):  
Henriette Farkas ◽  
Marcin Stobiecki ◽  
Jonny Peter ◽  
Tamar Kinaciyan ◽  
Marcus Maurer ◽  
...  
Keyword(s):  
Hereditary Angioedema ◽  
Open Label

scholarly journals 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

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
William R. Lumry ◽  
◽  
Bruce Zuraw ◽  
Marco Cicardi ◽  
Timothy Craig ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Hereditary Angioedema ◽  
Depression Scale ◽  
Health State ◽  
Life Questionnaire ◽  
Open Label ◽  
Activity Impairment ◽  
Open Label Extension

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.

scholarly journals Efficacy of Lanadelumab in Hereditary Angioedema Patients With and Without Prior Long-Term Prophylaxis Use: Interim Results From the HELP Open-Label Extension Study

2020 ◽  
Vol 145 (2) ◽  
pp. AB102
Author(s):  
Kim Paes ◽  
Timothy Craig ◽  
Jonathan Bernstein ◽  
Hilary Longhurst ◽  
Emel Aygoren-Pursun ◽  
...  
Keyword(s):  
Hereditary Angioedema ◽  
Extension Study ◽  
Open Label ◽  
Open Label Extension

Open-Label Study (ARIDO) Evaluating Long-Term Safety of Topical Glycopyrronium Tosylate (GT) in Patients with Axillary Hyperhidrosis

2017 ◽  
Vol 1 ◽  
pp. s95 ◽  
Author(s):  
Dee Anna Glaser ◽  
Adelaide A Hebert ◽  
Alexander Nast ◽  
William P Werschler ◽  
Stephen Shideler ◽  
...  
Keyword(s):  
Axillary Hyperhidrosis ◽  
Open Label ◽  
Open Label Study ◽  
Label Study

Abstract Not AvailableDisclosure: Study supported by Dermira.

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