Long‐term Health‐related Quality of Life in Patients Treated With Subcutaneous C1‐Inhibitor Replacement Therapy for the Prevention of Hereditary Angioedema Attacks: 113

Abstract

William R. Lumry, MD, Timothy J. Craig, DO FAAAAI, Markus A. K. Magerl, MD, Henriette Farkas, John-Philip Lawo, Joseph Chiao, MD, and Thomas Machnig, MD, PhD; AARA Research Center, Dallas, TX, Penn State College of Medicine, Hershey, PA, Department of Dermatology and Allergy, Charit e – Universit€atsmedizin, Berlin, Germany, Hungarian Angioedema Reference Center, Third Department of Internal Medicine, Semmelweis University, Budapest, Hungary, CSL Behring GmbH, Marburg, Germany, CSL Behring, King of Prussia, PA. RATIONALE: Hereditary angioedema (HAE) due to C1-esterase inhibitor (C1-INH) deficiency is known to have a significant negative impact on patients’ health-related quality of life (HRQoL). We evaluated the impact of long-term prophylaxis with subcutaneous C1-INH ([C1-INH (SC)] Haegarda , CSL Behring) on HRQoL in HAE patients treated in an openlabel extension (OLE) of the pivotal phase III COMPACT trial. METHODS: In the OLE, patients with >_4 HAE attacks within a consecutive 2-month period (N5126) self-administered C1-INH (SC) 40 or 60 IU/kg twiceweekly for up to 52 or 140weeks (US only). HRQoLwas self-assessed by patients at various points during the OLE using several instruments, including the European Quality of Life-5 Dimensions (EQ5D) questionnaire, Hospital Anxiety and Depression Scale (HADS), and Work Productivity and Activity Impairment (WPAI) assessment. RESULTS: With C1-INH (SC) 60 IU/kg, significant improvements from baseline to the end-of-study visit were observed on the EQ-5D, HADS, and 3 of 4 WPAI domains. Clinically meaningful improvements versus baseline (mean [95% CI]) were observed on the Health State Value (0.07 [0.01, 0.12]) and Visual Analogue Scale (7.45 [3.29, 11.62]) of the EQ-5D. Improvements were also noted on the HADS Depression (-0.95 [-1.57, -0.34]) and Anxiety (-1.23 [-2.08, -0.38]) scales and WPAI domains of Presenteeism (-23.33 [-34.86, -11.81]), Work Productivity Loss (-26.68 [-39.92, -13.44]), and Activity Impairment (-16.14 [-26.36, -5.91]). CONCLUSIONS: Long-term prophylaxis with C1-INH (SC) leads to significant and clinically meaningful improvements in various HRQoL measures in patients with HAE.