Marc A Riedl1, Aleena Banerji2, Paula J Busse3, Douglas T Johnston4, Mark A Davis-Lorton5, Shital Patel6, Howard Parr7, Joseph Chiao6, Douglas J Watson6, Earl Burrell6, Thomas Machnig6. 1. University of California-San Diego, La Jolla, California. Electronic address: marcriedl@yahoo.com. 2. Massachusetts General Hospital, Boston, Massachusetts. 3. Icahn School of Medicine at Mount Sinai, New York, New York. 4. University of North Carolina-Charlotte, Charlotte, North Carolina. 5. Winthrop Rheumatology Allergy & Immunology, Mineola, New York. 6. CSL Behring, King of Prussia, Pennsylvania. 7. Phoenix Healthcare, Darlington, United Kingdom.
Abstract
BACKGROUND: Hereditary angioedema (HAE) is a rare genetic disorder with substantial morbidity and mortality. Despite expanded choices for effective acute treatment, prophylactic options are more limited. Intravenous C1 esterase inhibitor (C1-INH[IV]) is licensed and used to prevent HAE symptoms. OBJECTIVE: To better understand patient experiences with using C1-INH(IV), including level of satisfaction and types and frequency of complications. METHODS: Fifty adult members (≥18 years of age) of the US HAE Association who had HAE type I or II completed a self-administered internet survey. Eligible participants were experiencing at least 1 HAE attack per month and must have been receiving treatment with C1-INH(IV) as prophylaxis or acute therapy. RESULTS: Almost all respondents (n = 47; 94%) were using C1-INH(IV) for HAE prophylaxis. Most patients reported administration of C1-INH(IV) through a peripheral vein (n = 34) and 19 were currently (n = 17) or previously (n = 2) using a central venous port. Most respondents (62%) who used a peripheral vein to administer treatment reported having difficulty finding a usable vein or getting the infusion to work properly at least some of the time. Issues accessing veins, exhausted veins, and frequency of attacks were the main reasons physicians recommended ports to respondents. Although ports allow easier administration of therapy, 47% of respondents with ports experienced problems such as occlusion, thrombosis, and infection. Respondents using C1-INH prophylaxis reported a mean of 2.3 attacks per month during the previous 6 months. CONCLUSION: The survey results identified clinical challenges with IV HAE medication use, including venous access issues and ongoing monthly attack occurrence despite prophylactic C1-INH(IV) administration.
BACKGROUND: Hereditary angioedema (HAE) is a rare genetic disorder with substantial morbidity and mortality. Despite expanded choices for effective acute treatment, prophylactic options are more limited. Intravenous C1 esterase inhibitor (C1-INH[IV]) is licensed and used to prevent HAE symptoms. OBJECTIVE: To better understand patient experiences with using C1-INH(IV), including level of satisfaction and types and frequency of complications. METHODS: Fifty adult members (≥18 years of age) of the US HAE Association who had HAE type I or II completed a self-administered internet survey. Eligible participants were experiencing at least 1 HAE attack per month and must have been receiving treatment with C1-INH(IV) as prophylaxis or acute therapy. RESULTS: Almost all respondents (n = 47; 94%) were using C1-INH(IV) for HAE prophylaxis. Most patients reported administration of C1-INH(IV) through a peripheral vein (n = 34) and 19 were currently (n = 17) or previously (n = 2) using a central venous port. Most respondents (62%) who used a peripheral vein to administer treatment reported having difficulty finding a usable vein or getting the infusion to work properly at least some of the time. Issues accessing veins, exhausted veins, and frequency of attacks were the main reasons physicians recommended ports to respondents. Although ports allow easier administration of therapy, 47% of respondents with ports experienced problems such as occlusion, thrombosis, and infection. Respondents using C1-INH prophylaxis reported a mean of 2.3 attacks per month during the previous 6 months. CONCLUSION: The survey results identified clinical challenges with IV HAE medication use, including venous access issues and ongoing monthly attack occurrence despite prophylactic C1-INH(IV) administration.
Authors: Ting Qiu; Maria J Chiuchiolo; Adele S Whaley; Anthony R Russo; Dolan Sondhi; Stephen M Kaminsky; Ronald G Crystal; Odelya E Pagovich Journal: Allergy Date: 2019-03-19 Impact factor: 13.146
Authors: Konrad Bork; John T Anderson; Teresa Caballero; Timothy Craig; Douglas T Johnston; H Henry Li; Hilary J Longhurst; Cristine Radojicic; Marc A Riedl Journal: Allergy Asthma Clin Immunol Date: 2021-04-19 Impact factor: 3.406
Authors: Donald Levy; Teresa Caballero; Iftikhar Hussain; Avner Reshef; John Anderson; James Baker; Lawrence B Schwartz; Marco Cicardi; Subhransu Prusty; Henrike Feuersenger; Ingo Pragst; Michael E Manning Journal: Pediatr Allergy Immunol Pulmonol Date: 2020-09-16 Impact factor: 1.349