HEALTHCARE RESOURCE UTILIZATION (HCRU) IN PEDIATRIC PATIENTS WITH HEREDITARY ANGIOEDEMA (HAE) IN THE UNITED STATES: A MATCHED STUDY USING LARGE INSURANCE CLAIMS

Author(s)

Christina G. Kwong, MD1, Tracy Yee, PhD, MPH2, Jenny Zhou, PhD3, Aolin Wang, PhD3, Hannah H. Kim, PhD3, Ellen Sears, MPH3, Anyu Zhu, MSc3, Amy Storfer-Isser, PhD, MS, MA2, Sandra Nestler-Parr, PhD, MSc, MPhil2, Patrick Gillard, PharmD, MSc2, Kenneth Paris, MD4;
1Phoenix Children's Hospital, Phoenix, AZ, USA, 2Biocryst Pharmaceuticals, Inc., Durham, NC, USA, 3Analysis Group, Inc., Boston, MA, USA, 4Children's Hospital New Orleans, New Orleans, LA, USA
OBJECTIVES: This study characterized the demographic and clinical profile of young children with hereditary angioedema (HAE) and compared their healthcare resource utilization (HCRU) with that of children without HAE.
METHODS: This retrospective cohort study used Komodo claims data (2016-2024) to identify pediatric patients aged 2-<12 years with HAE based on use of exclusive HAE treatments or suspected/presumed HAE derived from diagnostic codes and use of non-exclusive HAE treatments. Non-HAE controls were matched 3:1 to HAE patients on key demographic characteristics. Baseline characteristics and comorbidities were summarized descriptively. Inpatient (IP), emergency room (ER), and outpatient (OP) utilization during follow-up were assessed per person-year. Comparative analyses used generalized estimating equations with two-part models for zero-inflated outcomes.
RESULTS: This study included 411 pediatric patients with HAE and 1,233 non-HAE matched controls. Baseline demographic characteristics were balanced. Children with HAE had a higher comorbidity burden (Pediatric Comorbidity Index 2.2 vs. 0.9). During follow-up, children with HAE had 3.4 times the rate of OP visits compared with controls (incidence rate ratio [IRR], 95% confidence interval [CI]: 2.7-4.4) and significantly higher odds of having any IP admission (odds ratio [OR], 95% CI: 10.9, 6.7-17.8) or ER visit (OR, 95% CI: 3.0, 2.4-3.7). Among patients with ≥1 event, children with HAE demonstrated significantly greater HCRU compared with controls, including more IP admissions (IRR, 95% CI: 3.1, 2.1-4.6), more ER visits (IRR, 95% CI: 2.0, 1.6-2.6), and longer IP stays (ratio of mean length of stay, 95% CI: 2.4, 1.5-4.0).
CONCLUSIONS: Young children with HAE experience greater HCRU than matched controls without HAE, including more frequent OP and ER visits, IP admissions, and longer hospital stays. These findings underscore a substantial disease burden and reinforce the importance of optimized disease management to reduce healthcare utilization in pediatric patients with HAE.

Conference/Value in Health Info

2026-05, ISPOR 2026, Philadelphia, PA, USA

Value in Health, Volume 29, Issue S6

Code

EE69

Topic

Economic Evaluation

Topic Subcategory

Cost/Cost of Illness/Resource Use Studies

Disease

No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Pediatrics, SDC: Rare & Orphan Diseases, SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)

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