Unmet needs associated with non-androgen injectable long-term prophylaxis therapies for HAE

Document Type

Conference Proceeding

Publication Date

10-25-2024

Publication Title

Allergy

Keywords

androgen, complement component C1s inhibitor, lanadelumab, adult, angioneurotic edema, child, conference abstract, controlled study, contusion, cross-sectional study, discomfort, drug dose regimen, drug therapy, female, gastrointestinal discomfort, human, infusion related reaction, injectable, major clinical study, male, pain, prophylaxis, side effect, subcutaneous tissue

Abstract

Background: The majority of non-androgen long-term prophylaxis therapies (LTPs) for hereditary angioedema (HAE) are injectable (lanadelumab, C1 esterase inhibitor [C1-INH]). We aimed to characterize the most common physician-reported unmet needs associated with injectable non-androgen LTPs. Method: Data were collected from the Adelphi HAE Disease Specific Programme™, a real-world cross-sectional survey of HAE patients and their treating physicians in the United-States (January-November 2023). Physicians utilized patients' medical charts and their clinical judgement to provide data on patient demographics, treatments, and unmet needs/issues of injectable LTPs. Patients included in this analysis were 12+ years of age with HAE type 1 or 2 and receiving injectable non-androgen LTP as their current treatment. Descriptive statistics were reported. Results: Overall, 76 physicians provided data for 288 patients treated with non-androgen LTPs, of which 133 were prescribed injectable LTPs. Mean (± standard deviation) age was 35.0±12.20 years and 50.0% were female. Most frequently, patients were prescribed lanadelumab (n = 76; 57%), followed by subcutaneous C1-INH (n = 33; 25%), and intravenous C1-INH (n = 24; 18%). The mean duration of non-androgen injectable LTP treatment was 2.5±2.0 years. The most frequently physician-selected unmet needs identified were different route of administration (n = 48; 36%), dosing required too frequently (n = 26; 20%), infusion site reactions (n = 24; 18%), discomfort upon administration (n = 21; 16%), gastrointestinal side-effects (n = 20; 15%), too expensive for patients (n = 17; 13%), frequent side effects (n = 13; 10%), high administrative burden (n = 13; 10%), pain/bruising upon administration (n = 12; 9%), and not effective at reducing attack frequency (n = 11; 8%). Physicians reported that there were no unmet needs with non-androgen injectable LTP for 34 (26%) patients. Conclusion: Despite the availability of non-androgen LTPs, their use is associated with a high treatment burden. This study provides real-world insights regarding physicians' perceptions of unmet needs associated with non-androgen injectable LTPs. Current route of administration, frequent dosing schedules, side effects, and cost were the most common unmet needs/issues reported.

Volume

79

First Page

561

Last Page

562

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