Real-world treatment patterns and burden-of-disease of sub-optimally controlled hereditary angioedema.

BACKGROUND

Hereditary angioedema (HAE) is a rare disease characterized by unpredictable, recurrent subcutaneous or submucosal swelling that negatively impacts patients' health-related quality of life (HRQoL). Despite treatment goals aimed at achieving complete control of the disease and normalizing patients' lives, the disease remains poorly controlled for some patients.

OBJECTIVES

To describe the demographic, clinical and treatment characteristics, as well as the HRQoL impairment of patients with sub-optimally controlled HAE type I/II per treating physician's judgment, focusing on understanding the factors influencing the burden of illness.

METHODS

A chart review was conducted at 32 HAE care centers across 18 European countries, Canada, and Israel between April 2022 and January 2023 in 214 patients aged ≥12 years with HAE type I/II sub-optimally controlled with on-demand treatment (ODT) and/or long-term prophylaxis (LTP). Patients receiving lanadelumab were excluded, as it was not yet widely available during the eligibility period. A cross-sectional survey at patient enrollment included the Angioedema Quality of Life (AE-QoL) and EQ-5D-5L questionnaires to assess the impact of HAE on HRQoL.

RESULTS

Patients with uncontrolled HAE had a mean (standard deviation [SD]) of 9.9 (13) attacks per year, with a mean (SD) duration of 1.9 (1) days per attack. During the one-year observation period, 50.5% of patients were on ODT only, 36.0% used LTP and ODT concurrently, 6.5% used LTP without ODT, and 7.0% were untreated. Attenuated androgens (AA; stanozolol and danazol) in LTP were used by 24.7% of patients, while tranexamic acid (TA) and C1-esterase inhibitor (C1-INH) replacement products were used for LTP by 9.8% and 6.5%, respectively. The mean (SD) AE-QoL total score was 44.4 (24.0), indicating a moderate level of impairment, with women experiencing worse HRQoL (total score of 50.9 [SD 24] vs 37.3 [SD 23] in men) [where the minimal clinically important difference is 6 points] [1]. HRQoL worsened with increasing attack rates, from 41.4 (SD 24.0) among patients with one to 5 attacks/year, still indicating moderate impairment in HRQoL, to 73.0 (SD 27.0) for patients with >40 attacks/year.

CONCLUSIONS

Suboptimal disease control in HAE was associated with the use of ODT only, as well as LTP mainly with AA/TA. It imposes a substantial burden on patients' HRQoL, more particularly, but not exclusively, for those with frequent attacks and for women. The results suggest a need for improved HAE management.ClinicalTrial.gov study identifier NCT04957641.