Oral Therapies Expand Options for Personalized Management of Hereditary Angioedema
Advances in nonbiologic therapies are reshaping the management of hereditary angioedema (HAE), with growing emphasis on personalization, patient preferences, and treatment burden, according to a state-of-the-art review of preventive and on-demand strategies.
Beyond physical risk, attack frequency and severity of HAE affect psychological well-being, productivity, and quality of life. The authors emphasized that treatment selection should account for these broader impacts, noting that “recurrence and severity of attacks may impact psychological life, expectations and productivity.”
Drawing on international guidelines and clinical trial data, the review underscores that all patients should have reliable access to on-demand therapy. “All HAE patients should have an effective on-demand treatment available in case of attacks,” the authors stated. This recommendation remains foundational regardless of whether patients are also receiving long-term prophylaxis.
Long-term prophylaxis, however, warrants ongoing reassessment. The authors advised that LTP “should be considered and individualized for all patients at every visit,” using a shared decision-making framework to balance disease control with safety and tolerability. This approach reflects variability in attack patterns, patient lifestyles, and tolerance for different treatment modalities.
A key focus of the review was the shift toward oral therapies. Parenteral prophylaxis, while effective, is associated with logistical challenges, injection-related adverse effects, and adherence barriers. The authors noted that “parenteral administration of LTP is associated with treatment complexities and barriers,” which may limit long-term use in some patients.
Oral agents, beginning with berotralstat, offer a different paradigm. According to the authors, oral treatment “could address practical needs for HAE patients both in preventive and on-demand setting, avoiding injection-related side effects, reducing treatment burden, and improving quality of life.” Several additional oral drugs are in development, signaling continued innovation in this space.
For immunologists, the key takeaway is that HAE management is entering a more flexible, patient-centered era. Regular reassessment of prophylaxis needs, universal access to on-demand therapy, and consideration of oral options may help optimize outcomes while aligning treatment with patient priorities.
Reference
Zanichelli A, De Angeli G, Baroni I, Mansi M, Caravella G, Caruso R. Hereditary angioedema treatment beyond biologics: current state of preventive and on-demand approaches and new perspectives. Expert Opin Pharmacother. 2025;26(10):1221-1228. doi:10.1080/14656566.2025.2509782
