ON-DEMAND TREATMENTS FOR HEREDITARY ANGIOEDEMA AND HEALTHCARE RESOURCE UTILIZATION IN PEDIATRIC (2-11 YEARS) PATIENTS: A US CLAIMS DATABASE ANALYSIS
Author(s)
Raffi Tachdjian, MD, MPH1, Adil Adatia, MD2, Emel Aygören-Pürsün, MD3, Mauro Cancian, MD4, Aharon Kessel, MD5, H Henry Li, MD6, Heloise Reumaux, MD7, H. James Wedner, MD8, Bob Geng, MD9, Aditya Sehgal, BSc10, Paul K. Audhya, MD, MBA11, Alice Wang, MA11;
1University of California, Los Angeles, School of Medicine, Los Angeles, CA, USA, 2University of Alberta, Edmonton, AB, Canada, 3University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt, Germany, 4Azienda Ospedale Università di Padova, Padova, Italy, 5Bnai Zion Medical Center, Haifa, Israel, 6Institute for Asthma and Allergy, Wheaton, MD, USA, 7Hôpital Jeanne de Flandre, Lille, France, 8Washington University School of Medicine, St. Louis, MO, USA, 9University of California, San Diego, La Jolla, CA, USA, 10Pharmsight, Haryana, India, 11KalVista Pharmaceuticals, Framingham, MA, USA
1University of California, Los Angeles, School of Medicine, Los Angeles, CA, USA, 2University of Alberta, Edmonton, AB, Canada, 3University Hospital Frankfurt, Goethe University Frankfurt, Frankfurt, Germany, 4Azienda Ospedale Università di Padova, Padova, Italy, 5Bnai Zion Medical Center, Haifa, Israel, 6Institute for Asthma and Allergy, Wheaton, MD, USA, 7Hôpital Jeanne de Flandre, Lille, France, 8Washington University School of Medicine, St. Louis, MO, USA, 9University of California, San Diego, La Jolla, CA, USA, 10Pharmsight, Haryana, India, 11KalVista Pharmaceuticals, Framingham, MA, USA
Presentation Documents
OBJECTIVES: This study aimed to characterize the real-world utilization of on-demand treatments and healthcare resource utilization (HRU) among pediatric (<12y) hereditary angioedema (HAE-C1INH) patients.
METHODS: Data were extracted from the IQVIA PharMetrics® Plus Closed Health Plan Claims Database (study period: January 1, 2019 - December 31, 2024), identifying patients aged 2-11y with ≥1 claims of HAE on-demand therapy. Index date was defined as the date of the first on-demand therapy claim during the study period; continuous enrollment for 12mo following index date was required. A standardized dose was defined as one icatibant syringe, one rhC1INH vial, three ecallantide vials, or two pdC1INH (Berinert) vials. On-demand utilization and HRU were evaluated during follow up.
RESULTS: Ninety pediatric patients (61% male; mean age 7.8y) met continuous enrollment criteria. Sixty-two percent had claims for pdC1INH, 24% icatibant, 9% rhC1INH, and 7% ecallantide. One patient had claims for pdC1INH and ecallantide while one had claims for pdC1INH and icatibant. There were a total of 469 on-demand doses: 40% icatibant, 40% pdC1INH, 16% rhC1INH, 4% ecallantide. Mean on-demand doses were 0.43 per patient per month (range: 0.25 ecallantide - 0.77 rhC1INH). HRU was substantial, with a large proportion of patients requiring outpatient (81%; mean 4.1 visits/year) or emergency room (47%; mean 3.2 visits/year) care; 17% (mean 2.3 visits/year) utilized home-health services, while 3% (mean 1.3 visits/year) required inpatient hospitalization.
CONCLUSIONS: On-demand treatment options are limited for the US pediatric HAE population with only IV pdC1INH being FDA-approved. This analysis found that most (62%) pediatric patients had claims of pdC1INH, which contributed 40% of total on-demand doses. This contrasts with icatibant where 24% of patients contributed to the same total on-demand dose share. Pediatric patients utilized substantial HRU including the emergency room at a high rate. These results suggest a need for less invasive dosing options for children with HAE.
METHODS: Data were extracted from the IQVIA PharMetrics® Plus Closed Health Plan Claims Database (study period: January 1, 2019 - December 31, 2024), identifying patients aged 2-11y with ≥1 claims of HAE on-demand therapy. Index date was defined as the date of the first on-demand therapy claim during the study period; continuous enrollment for 12mo following index date was required. A standardized dose was defined as one icatibant syringe, one rhC1INH vial, three ecallantide vials, or two pdC1INH (Berinert) vials. On-demand utilization and HRU were evaluated during follow up.
RESULTS: Ninety pediatric patients (61% male; mean age 7.8y) met continuous enrollment criteria. Sixty-two percent had claims for pdC1INH, 24% icatibant, 9% rhC1INH, and 7% ecallantide. One patient had claims for pdC1INH and ecallantide while one had claims for pdC1INH and icatibant. There were a total of 469 on-demand doses: 40% icatibant, 40% pdC1INH, 16% rhC1INH, 4% ecallantide. Mean on-demand doses were 0.43 per patient per month (range: 0.25 ecallantide - 0.77 rhC1INH). HRU was substantial, with a large proportion of patients requiring outpatient (81%; mean 4.1 visits/year) or emergency room (47%; mean 3.2 visits/year) care; 17% (mean 2.3 visits/year) utilized home-health services, while 3% (mean 1.3 visits/year) required inpatient hospitalization.
CONCLUSIONS: On-demand treatment options are limited for the US pediatric HAE population with only IV pdC1INH being FDA-approved. This analysis found that most (62%) pediatric patients had claims of pdC1INH, which contributed 40% of total on-demand doses. This contrasts with icatibant where 24% of patients contributed to the same total on-demand dose share. Pediatric patients utilized substantial HRU including the emergency room at a high rate. These results suggest a need for less invasive dosing options for children with HAE.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
SA17
Topic
Study Approaches
Disease
SDC: Pediatrics, SDC: Rare & Orphan Diseases