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Background: Despite the use of long-term prophylaxis (LTP) for hereditary angioedema (HAE), the risk of having an attack remains and patients with HAE and on LTP may still experience attacks that can be life threatening. However, the behavioralpatterns and perspectives surrounding HAE attack management by patients on LTP are not fully understood., Objective: This survey aimed to better understand and compare the behavioral patterns and perspectives, including attitudesand perceptions associated with on-demand treatment among patients on LTP versus those using on-demand therapy only., Methods: People living with HAE were recruited by the US Hereditary Angioedema Association to complete a 20-minute online survey between September 6 and October 19, 2022. Participants were stratified by treatment (50% using LTP [+ondemand therapy], 50% on-demand therapy only)., Results: Respondents included 107 patients with HAE (mean age, 41 years [range, 16-83 years]). Patients using LTP reported treating a mean +/- standard deviation 84.8% +/- 23.8% of their HAE attacks compared with a mean +/- standard deviation75.6% +/- 27.5% for patients with on-demand only treatment. Similar percentages of patients on LTP versus patients on-demand only reported always carrying on-demand treatment when away from home (35% versus 38%) and modifying their daily lives to minimize the occurrence of HAE attacks, which included avoiding potential triggers (42.9% versus 45.5%)., Conclusion: Although patients on LTP treat a higher percentage of their attacks compared with patients with on-demand only treatment, both groups reported similar behaviors in terms of carrying on-demand treatment when away from home and modifying their daily lives to minimize the occurrence of HAE attacks. These findings highlight the importance of understanding patient perspectives and behaviors in the management of HAE.

Background: Hereditary angioedema (HAE) is a rare condition marked by swelling episodes in various body parts, including the extremities, upper airway, face, intestinal tract, and genitals. Long-term prophylaxis (LTP), prescribed to control recurring HAE attacks, is integral to its management. Previously, attenuated androgens (AAs) were the only oral LTP options. However, in 2020, berotralstat, an oral plasma kallikrein inhibitor, was approved in the United States. A 2018 survey of adults with HAE type I or type II showed that almost all the patients who used prophylactic HAE medication preferred oral treatment (98%) and felt that it fit their lifestyle better than injectable treatment (96%). Still, guidelines lack consensus on transitioning patients from AAs to alternative oral prophylactic therapy. Objective: This paper aims to share expert insights and patient feedback on transitioning from AAs to berotralstat, an alternative oral prophylactic therapy, from the perspective of clinicians with extensive experience in treating patients with HAE. Methods: A panel of five HAE specialists convened for a virtual half-day roundtable discussion in April 2023. Results: Discussions about transitioning from AAs to berotralstat were prompted by routine consultations, patient inquiries based on independent research, ineffective current treatment, or worsening AA-related adverse effects. For patients who switched from AAs, the physicians reported that the decision was influenced by the alternative therapy's ability to prevent HAE attacks, its safety, and the once-daily administration schedule. All expert panel members identified fewer AA-related adverse effects; better quality of life; and less severe, shorter, and less frequent HAE attacks as clinical or patient goals they hoped to achieve through the treatment switch. Conclusion: The emergence of new, highly specific LTP drugs for HAE calls for the development of comprehensive recommendations and guidelines for transitioning from AAs to alternative oral prophylactic therapy. The expert panel highlighted key factors to consider during the development of such guidelines.

Background: Intranasal corticosteroids (INCS) are the cornerstone of treatment for chronic rhinosinusitis. Although INCS are generally considered safe and effective, there is a concern that chronic use may lead to ocular adverse effects. Objective: To assess ocular safety of the exhalation delivery system with fluticasone propionate (EDS-FLU) in patients with chronic rhinosinusitis with nasal polyps. Methods: Ocular safety data were collected during two randomized, double-blind, placebo controlled studies with open-label extensions. Ophthalmologists performed tonometry, slit-lamp, and visual acuity examinations to assess intraocular pressure (IOP) and the presence of cataracts. Ocular examinations were conducted before double-blind treatment, at the end of the 16-week double-blind phase, and at the end of the 8-week open-label phase. The results of pooled data from patients who received EDS-FLU 186 µg (n = 160), EDS-FLU 372 µg (n = 161), and EDS-placebo (n = 161) twice daily are reported here. Results: At the end of the double-blind phase, six patients developed elevated average IOP > 21 mm Hg: two patients (1.2%) in the EDS-placebo group, three patients (1.9%) in the EDS-FLU 186 µg group, and one patient (0.6%) in the EDS-FLU 372 µg group. In addition, 6 of 482 patients developed cataracts: 3 patients in the EDS-placebo group, 2 patients in the EDS-FLU 186 µg group, and 1 patient in the EDS-FLU 372 µg group. At the end of the open-label phase, two additional patients showed IOP > 21 mm Hg and two additional patients developed cataracts. Conclusion: No increased risk of elevated IOP was detected with EDS-FLU; the rate of cataract development was similar to EDS-placebo and to that reported with other INCS.Clinical trials NCT01622569 and NCT01624662, www.clinicaltrials.gov.