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Open Access Real-world cohort study of adult and pediatric patients treated for hereditary angioedema in the United States

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Background:

There is limited real-world evidence on hereditary angioedema (HAE) patient characteristics and health-care resource utilization (HCRU); in addition, pediatric patients have been described in small cohorts.

Objective:

To describe patient characteristics, treatment patterns, and HCRU among adult and pediatric patients treated for HAE in a large U.S. cohort.

Methods:

This retrospective cohort study used an administrative claims data base (January 2006 to September 2015). Eligible patients with either ≥1 pharmacy claim for HAE-indicated therapies (C1 inhibitors, ecallantide, icatibant) or ≥2 medical claims with codes associated with HAE (per medical billing codes), and ≥1 claim for androgens, fresh frozen plasma, tranexamic acid, or ε-aminocaproic acid formed a “treated cohort.” Three nonexclusive treated cohorts were assessed: overall, pediatric, and HCRU (≥2 years of continuous enrollment during 2010‐2015).

Results:

Overall, 1429 patients received treatment (mean ± standard deviation [SD] age, 38.8 ±15.7 years; 62.4% female patients; mean ± SD Charlson Comorbidity Index of 1.4 ± 2.4). Common comorbidities were allergy or anaphylaxis (51.4%) and anxiety or depression (35.8%). Diagnoses indicative of HAE attacks included swelling and/or angioedema (78.5%), abdominal pain (55.6%), and asphyxiation (27.2%). Use of HAE-indicated medication rose between 2006 and 2015 to 81.8%, whereas androgen use declined (from 91.5% to 24.9%). Similar trends were observed in the pediatric treated cohort (n = 143). In the HCRU treated cohort (n = 538), HAE-related claims for emergency department and inpatient admissions were observed for 36.6% and 22.3% of patients, respectively.

Conclusion:

In a large U.S. cohort of adult and pediatric patients who received treatments indicated or used for HAE, common comorbidities and trends in resource use denoted the substantial burden of attacks, which reflected a continued need that recently approved long-term prophylactic treatments may help to address.

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Keywords: C1 esterase inhibitor; comorbidities; disease burden; health care resource utilization; hereditary angioedema; hospitalization; pediatric; real-world data; retrospective cohort study; treatment patterns

Document Type: Research Article

Affiliations: 1: From the AIRE Medical of Los Angeles, and Division of Allergy & Clinical Immunology, David Geffen School of Medicine at University of California Los Angeles, Los Angeles, California 2: IQVIA, Epidemiology and Drug Safety Practice, Seattle, Washington 3: IQVIA, Epidemiology and Drug Safety Practice, New York, New York, and 4: Shire, a Takeda company, Division of Global Evidence and Outcomes, Lexington, Massachusetts

Publication date: May 1, 2020

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  • Allergy and Asthma Proceedings is a peer reviewed publication dedicated to distributing timely scientific research regarding advancements in the knowledge and practice of allergy, asthma and immunology. Its primary readership consists of allergists and pulmonologists.

    The goal of the Proceedings is to publish articles with a predominantly clinical focus which directly impact quality of care for patients with allergic disease and asthma and by having the potential to directly impact the quality of patient care. AAP welcomes the submission of original works including peer-reviewed original research and clinical trial results. Additionally, as the official journal of the Eastern Allergy Conference (EAC), AAP will publish content from EAC poster sessions as well as review articles derived from EAC lectures.

    Featured topics include asthma, rhinitis, sinusitis, food allergies, allergic skin diseases, diagnostic techniques, allergens, and treatment modalities. Published material includes peer-reviewed original research, clinical trials and review articles.

    Articles marked "F" offer free full text for personal noncommercial use only.

    The journal is indexed in Thomson Reuters Web of Science and Science Citation Index Expanded, plus the National Library of Medicine's PubMed service.
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