Abstract
BackgroundPhysician surveys on hereditary angioedema (HAE) management in 2010 and 2013 revealed important trends in HAE care. ObjectiveTo evaluate current HAE management and the impact of new treatment options on physician practice patterns over time. MethodsDuring June and July 2019, 5382 physicians were contacted by means of postal mail to complete a 47-question survey; 177 responded (3%). ResultsAcross the 3 surveys, the home replaced the emergency department as the most typically reported setting for HAE attack treatment (54.3% vs 11.6% in 2010 and 32.5% in 2013; P < .001). Physicians reported C1 esterase inhibitor (C1-INH) as the most typically prescribed long-term prophylactic treatment (LTP) (60.0% vs 20.4% in 2010 and 56.7% in 2013; P < .001). Subcutaneous LTP medications were most typically prescribed over intravenous (C1-INH, 41.4%; subcutaneous lanadelumab, 21%; intravenous C1-INH, 18.6%). Danazol, the most frequently prescribed LTP treatment, dropped to 6.4% (55.8% in 2010 and 23.4% in 2013; P < .001). The strongest nonefficacy factor influencing clinician treatment choice changed over time, with cost and (or) insurance coverage increasing to 43.7% (from 24.4% in 2010 and 40.5% in 2013; P = .001), whereas the concern over adverse effects dropped to 16.2% (from 55.8% in 2010 and 29.5% in 2013; P < .001). Physician-reported patient satisfaction remains high, with only 1.5% of physicians indicating patients are not satisfied with treatment. ConclusionThe US physician survey data reflect improvements in the HAE management in recent years. Therapeutic advances in HAE have led to reported higher rates of home treatment of HAE attacks, reduced concern for adverse treatment effects, and high levels of patient satisfaction.
Highlights
Hereditary angioedema (HAE) is an autosomal dominant disorder caused by C1 esterase inhibitor (C1-INH) deficiency that affects an estimated 1 out of 10,000 to 50,000 people worldwide.[1,2]
Angioedema symptoms involving the face and airway remain the most common symptoms triggering referral; physicians reported that abdominal pain is a prominent symptom most troubling to affected individuals
The laboratory tests used to confirm the hereditary angioedema (HAE) diagnosis remain consistent, with C4 levels identified as the most important screening test, genetic testing for factor XII mutations was used by 16.9% of respondents in the diagnostic evaluation, reflecting advanced knowledge in the underlying pathophysiology for a subset of patients with HAE having normal C1-INH
Summary
Hereditary angioedema (HAE) is an autosomal dominant disorder caused by C1 esterase inhibitor (C1-INH) deficiency that affects an estimated 1 out of 10,000 to 50,000 people worldwide.[1,2] Type I HAE accounts for approximately 85% of patients and is characterized by low levels of endogenous C1-INH, whereas type II HAE (15%) is associated with normal C1-INH levels but decreased functional activity.[2,3,4] These 2 variants of HAE caused by C1-INH deficiency result in increased production of bradykinin, which increases vascular permeability, leading to angioedema.[1,4] A third variant of HAE (HAE with normal C1-INH) has more recently been defined in patients with normal, functional C1-INH levels.[5]. Physician surveys on hereditary angioedema (HAE) management in 2010 and 2013 revealed important trends in HAE care. Results: Across the 3 surveys, the home replaced the emergency department as the most typically reported setting for HAE attack treatment (54.3% vs 11.6% in 2010 and 32.5% in 2013; P < .001). Physicians reported C1 esterase inhibitor (C1-INH) as the most typically prescribed long-term prophylactic treatment (LTP) (60.0% vs 20.4% in 2010 and 56.7% in 2013; P < .001). Conclusion: The US physician survey data reflect improvements in the HAE management in recent years. Therapeutic advances in HAE have led to reported higher rates of home treatment of HAE attacks, reduced concern for adverse treatment effects, and high levels of patient satisfaction
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