Patient satisfaction and experience with intravenously administered C1-inhibitor concentrates in the United States Marc A. Riedl, MD * ; Aleena Banerji, MD y ; Paula J. Busse, MD z ; Douglas T. Johnston, DO x ; Mark A. Davis-Lorton, MD k ; Shital Patel, BS { ; Howard Parr, BA(Hons) # ; Joseph Chiao, MD { ; Douglas J. Watson, PhD, MSPH { ; Earl Burrell, PhD, MPH { ; Thomas Machnig, MD { * University of CaliforniaeSan Diego, La Jolla, California y Massachusetts General Hospital, Boston, Massachusetts z Icahn School of Medicine at Mount Sinai, New York, New York x University of North CarolinaeCharlotte, Charlotte, North Carolina k Winthrop Rheumatology Allergy & Immunology, Mineola, New York { CSL Behring, King of Prussia, Pennsylvania # Phoenix Healthcare, Darlington, United Kingdom A R T IC L E IN F O Article history: Received for publication March 3, 2017. Received in revised form May 12, 2017. Accepted for publication May 18, 2017. A B ST R AC T 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 ¼ 47; 94%) were using C1-INH(IV) for HAE prophylaxis. Most patients reported administration of C1-INH(IV) through a peripheral vein (n ¼ 34) and 19 were currently (n ¼ 17) or previously (n ¼ 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 ther- apy, 47% of respondents with ports experienced problems such as occlusion, thrombosis, and infection. Re- spondents 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. Ó 2017 American College of Allergy, Asthma & Immunology. Published by Elsevier Inc. All rights reserved. Introduction Hereditary angioedema (HAE) is a rare autosomal dominant disorder that affects 1:10,000 to 1:50,000 people. 1,2 The condition is characterized by recurrent episodes of nonpruritic edema in various parts of the body, including the hands, feet, face, and airway. 1,3,4 In addition, abdominal attacks associated with swelling of the intestinal wall can cause severe abdominal pain and acute cramping that can be accompanied by nausea and vomiting. 4,5 Reprints: Marc A. Riedl, MD, Clinical Director, US Hereditary Angioedema Associ- ation Angioedema Center, Division of Rheumatology, Allergy and Immunology, University of CaliforniaeSan Diego, 8899 University Center Lane, Suite 230, San Diego, CA 92122; E-mail: marcriedl@yahoo.com. Disclosures: Dr Riedl has served as an investigator for and has received consultancy and speaker fees from CSL Behring, Pharming, and Shire and is a US Hereditary Angioedema Advisory Board member. Dr Banerji has served on an advisory board for Alnylam, CSL Behring, and Shire; she has received research grants from Shire. Dr Busse has received research support and grants from CSL Behring and Shire; she has received consultancy fees from Shire. Dr Johnston has served on advisory boards for CSL Behring, Shire, and Valeant; he served as a speaker for Shire; he has served as an investigator with Biocryst and Shire/Dyax; he serves as a chair for the American Academy of Allergy, Asthma & Immunology. Dr Davis-Lorton has served on an advisory board for CSL Behring and Shire and has served as an investigator for Shire. Dr Patel has no conflicts to disclose. Mr. Parr is an employee of Phoenix Healthcare, which received funding from CSL Behring to conduct this study. Dr Chiao and Dr Watson are employees of CSL Behring. Dr Burrell was an employee of CSL Behring during the time of study conduct. Dr Machnig is an employee and stock- holder at CSL Behring. Funding Sources: CSL Behring. Contents lists available at ScienceDirect http://dx.doi.org/10.1016/j.anai.2017.05.017 1081-1206/Ó 2017 American College of Allergy, Asthma & Immunology. Published by Elsevier Inc. All rights reserved. Ann Allergy Asthma Immunol 119 (2017) 59e64