Marc Riedl1, Richard G Gower, Carole Alison Chrvala. 1. Section of Clinical Immunology and Allergy, Department of Medicine, University of California, Los Angeles, David Geffen School of Medicine, 90095, USA. mriedl@mednet.ucla.edu
Abstract
BACKGROUND: Hereditary angioedema (HAE) is a potentially life-threatening condition that affects approximately 1 in 50,000 persons. There are no known surveys of US physicians' knowledge and experience regarding the epidemiology, diagnosis, and management of HAE. OBJECTIVE: This survey of physicians treating patients with HAE assessed physician characteristics, diagnostic and treatment practices, factors that influence physician and patient treatment choices, and physician awareness of new therapies. METHODS: From October 2009 to February 2010, physicians (N = 172) voluntarily completed an online survey developed by physician-investigators. Data were analyzed with descriptive statistics. RESULTS: Most physicians (73.8%) managed 5 patients or fewer in diverse practice settings. Laboratory testing was considered most important in establishing a diagnosis. Fresh frozen plasma and C1 esterase inhibitors were the most frequently administered treatments for acute events; nearly 50% of respondents prescribed C1 esterase inhibitor for acute attacks. More than 80% of respondents prescribed androgens for long-term prophylaxis. Approximately half of respondents were aware of, and likely to use, new therapies for HAE. Other than efficacy, adverse effects were the most important factor that influenced physicians' treatment recommendations, whereas physicians perceived that patients were most influenced by adverse effects and cost. CONCLUSIONS: Wide variability exists in the treatment of patients with HAE. Many patients experience acute attacks that require emergency care or hospitalization. Androgens and fresh frozen plasma are frequently used despite recent availability of effective condition-specific agents, and many physicians are only somewhat aware of medications newly approved by the US Food and Drug Administration. Because the survey was completed shortly after approval of additional HAE therapies by the US Food and Drug Administration, these data will be useful for tracking changes in HAE treatment over time.
BACKGROUND:Hereditary angioedema (HAE) is a potentially life-threatening condition that affects approximately 1 in 50,000 persons. There are no known surveys of US physicians' knowledge and experience regarding the epidemiology, diagnosis, and management of HAE. OBJECTIVE: This survey of physicians treating patients with HAE assessed physician characteristics, diagnostic and treatment practices, factors that influence physician and patient treatment choices, and physician awareness of new therapies. METHODS: From October 2009 to February 2010, physicians (N = 172) voluntarily completed an online survey developed by physician-investigators. Data were analyzed with descriptive statistics. RESULTS: Most physicians (73.8%) managed 5 patients or fewer in diverse practice settings. Laboratory testing was considered most important in establishing a diagnosis. Fresh frozen plasma and C1 esterase inhibitors were the most frequently administered treatments for acute events; nearly 50% of respondents prescribed C1 esterase inhibitor for acute attacks. More than 80% of respondents prescribed androgens for long-term prophylaxis. Approximately half of respondents were aware of, and likely to use, new therapies for HAE. Other than efficacy, adverse effects were the most important factor that influenced physicians' treatment recommendations, whereas physicians perceived that patients were most influenced by adverse effects and cost. CONCLUSIONS: Wide variability exists in the treatment of patients with HAE. Many patients experience acute attacks that require emergency care or hospitalization. Androgens and fresh frozen plasma are frequently used despite recent availability of effective condition-specific agents, and many physicians are only somewhat aware of medications newly approved by the US Food and Drug Administration. Because the survey was completed shortly after approval of additional HAE therapies by the US Food and Drug Administration, these data will be useful for tracking changes in HAE treatment over time.
Authors: C Wintenberger; I Boccon-Gibod; D Launay; O Fain; G Kanny; P Y Jeandel; L Martin; A Gompel; L Bouillet Journal: Clin Exp Immunol Date: 2014-10 Impact factor: 4.330
Authors: Aleena Banerji; Murat Baş; Jonathan A Bernstein; Isabelle Boccon-Gibod; Maria Bova; John Dempster; Anete Sevciovic Grumach; Markus Magerl; Kimberly Poarch; Manuel Branco Ferreira Journal: Allergy Rhinol (Providence) Date: 2016-09-22