Patient outcomes associated with subcutaneous C1INH prophylaxis for hereditary angioedema: a retrospective analysis

Autor: William Lumry, Timothy Craig, John Anderson, Marc Riedl, Henry Li, Raffi Tachdjian, Michael Manning, Paolo Bajcic, Frank Rodino, Sam Wang, Thomas R. Sexton, Jonathan A. Bernstein
Jazyk: angličtina
Rok vydání: 2023
Předmět:
Zdroj: Allergy, Asthma & Clinical Immunology, Vol 19, Iss 1, Pp 1-9 (2023)
Druh dokumentu: article
ISSN: 1710-1492
DOI: 10.1186/s13223-023-00861-9
Popis: Abstract Background Real-world data on subcutaneous C1INH (C1INH[SC]) usage and patient-level impacts on hereditary angioedema (HAE)-related outcomes and quality of life (QoL) are both lacking and challenging to generate using conventional study methodologies. Using a hybrid study design involving patient interviews supplemented by retrospective medical chart data review, we conducted a real-world assessment of the impact of C1INH(SC) prophylaxis on HAE attack patterns, QoL, and on-demand medication use. Methods The study was conducted at seven US sites and included 36 adults with HAE who had been treated with C1INH(SC) long-term prophylaxis following ≥ 12 months of on-demand management only. Patients underwent 30-min interviews, facilitated and analyzed by a trained qualitative research specialist. Medical records were reviewed for 12 months before (pre-index) and after (post-index) initiation of C1INH(SC). Using interview data with descriptive terms converted to numerical values, we compared pre- versus post-index attack frequency, severity, and rescue medication usage. Results Mean (SD) annualized attack frequency per patient decreased 82.0%, from 38.8 (38.8) attacks/year pre-index to 7.0 (15.3) attacks/year (P
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