AB162. Impact of limited access to medications in hereditary angioedema on treatment timeliness, disease control, and health-related quality of life: findings from an international patient survey
Abstract

AB162. Impact of limited access to medications in hereditary angioedema on treatment timeliness, disease control, and health-related quality of life: findings from an international patient survey

Hugo W. F. Mak1, Cathryn McDougall2,3, Jane C. Y. Wong1, Dorothy L. Y. Lam1, Ricardo Zwiener4,5, Jonny Peter2,3, Philip H. Li1,6

1Division of Rheumatology and Clinical Immunology, Department of Medicine, Queen Mary Hospital, The University of Hong Kong, Hong Kong, China; 2Allergy and Immunology Unit, University of Cape Town Lung Institute, Cape Town, South Africa; 3Department of Allergology and Clinical Immunology, Department of Medicine, Groote Schuur Hospital, University of Cape Town, Cape Town, South Africa; 4Allergy and Immunology Department, Hospital Universitario Austral, Buenos Aires, Argentina; 5President Asociación Argentina de Angioedema Hereditario (AEH Argentina), Buenos Aires, Argentina; 6Division of Rheumatology & Clinical Immunology, Department of Medicine, University of Hong Kong-Shenzhen Hospital, Shenzhen, China

Background: Hereditary angioedema (HAE) is a rare genetic disorder characterised by recurrent episodes of swelling. In recent years, there are various emerging on-demand treatment (ODT) and prophylaxis options. However, access to these HAE-specific medications varies across regions, and its impact from a patient perspective remains elusive. The objective of this study is to investigate the access to treatment for HAE patients in Argentina, South Africa, and Hong Kong and how this affects patient outcomes.

Methods: We surveyed HAE patients from three localities, namely Argentina, South Africa, and Hong Kong in late 2024. Their demographic features, current treatment, factors related to treatment decisions, disease activity, control [by Angioedema Control Test (AECT)], and impact on health-related quality of life [HRQoL; by Angioedema Quality of Life Questionnaire (AE-QoL)] were collected and analyzed.

Results: A total of 98 HAE patients [32 (32.7%) from Argentina, 30 (30.6%) from Hong Kong, and 36 (36.7%) from South Africa] responded. In country without access to HAE-specific medications (South Africa), there were significantly more patients without ODT (22.2% vs. 0.0%, P<0.001) and required other secondary ODT (P<0.001), such as fresh frozen plasma (30.6% vs. 0.0%). There was also significantly more usage of second-line, non-HAE-specific prophylactic agents (P<0.001), including androgens/steroids (41.7% vs. 4.8%) and tranexamic acid (16.7% vs. 0.0%). The no access group was significantly less likely to always immediately treat their HAE attacks (0.0% vs. 20.4%, P=0.01), with access/cost-related reasons such as cost concerns (34.6% vs. 7.4%, P=0.004), and anxiety over inability to refill medications before next attack (65.4% vs. 25.9%, P<0.001). Regarding disease control, the no access group was significantly less likely to be attack-free in the past 3 months (16.7% vs. 46.8%, P<0.001). Their AECT and AE-QoL scores were also significantly worse (respectively 8.6±4.3 vs. 14.8±3.0 and 49.0±20.9 vs. 7.3±15.5, both P<0.001), with fewer patients having well-controlled HAE (AECT ≥10; 40.6% vs. 86.7%, P<0.001).

Conclusions: Limited access to HAE medications remains pervasive in certain regions, with substantial, negative impacts on treatment timeliness, disease control, and HRQoL.

Keywords: Hereditary angioedema; treatment access; disparities; quality of life

Footnote

Conflicts of Interest: P.H.L. was a speaker and/or advisor for, and/or has received research funding from CSL Behring, KalVista Pharmaceuticals, Pharvaris, and Takeda. R.Z. has been a speaker for Shire/Takeda, CSL Behring, Novartis, Sanofi, Panalab, Pint Pharma, and AstraZeneca; advisor for Shire/Takeda, CSL Behring, AbbVie, Bagó, KalVista, Pint Pharma, and Pharvaris; and has received financial support for research from Shire/Takeda, Sanofi, and Pharvaris. J.P. has received honoraria, speaker’s fees, advisory board membership and/or educational grants from the following pharmaceutical or biotech companies which manufacture medical products: AbbVie, AstraZeneca, Astria, BioCryst, Cipla, CSL Behring, Dr Reddy’s, Eye-Gene, Glenmark, Janssen, Johnson and Johnson, Novartis, Pharmadynamics, Pharvaris, Sanofi/Regeneron, and Takeda. The other authors have no conflicts of interest to declare.

doi: 10.21037/jphe-26-ab162
Cite this abstract as: Mak HWF, McDougall C, Wong JCY, Lam DLY, Zwiener R, Peter J, Li PH. AB162. Impact of limited access to medications in hereditary angioedema on treatment timeliness, disease control, and health-related quality of life: findings from an international patient survey. J Public Health Emerg 2026;10:AB162.

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