HAE expert panel calls for more convenient on-demand treatments
Easier ways to treat attacks could help patients follow critical medical guidelines
Written by |
Patients with hereditary angioedema (HAE) should ideally receive on-demand treatment within one hour of their first symptoms, according to an international panel of 19 specialists. However, the experts warned that several obstacles, ranging from the difficulty in recognizing early symptoms to the physical burden of using needles, often prevent patients from meeting this critical window.
The findings, based on a consensus study, highlight a significant gap between medical guidelines and the daily reality of living with HAE. To close this gap, the experts are calling for more convenient treatment options, better patient education, and improved access to medication.
Overall, the results “demonstrate the need for accessible and convenient on‐demand treatments for HAE attacks that will enable patients with HAE to improve adherence to guidelines,” researchers wrote.
By addressing these barriers, doctors hope to reduce the physical and emotional impact of the sudden, painful swelling episodes that define the condition.
The study, “An International Delphi Study on Barriers to On-Demand Treatment of Hereditary Angioedema Attacks,” was published in Clinical and Translational Allergy. It was funded by Kalvista Pharmaceuticals, the company behind the oral on-demand treatment Ekterly (sebetralstat).
Challenges in recognizing and treating attacks
HAE is a genetic disorder that causes sudden episodes of swelling in the face, tongue, airways, and digestive tract. While many patients take preventive medicine, they still need “on-demand” therapy to stop active attacks. Current guidelines say every attack should be treated as early as possible, but many patients wait too long because they aren’t sure if an attack has truly started or if the symptoms are severe enough to justify a needle-based treatment.
To understand why delays happen, researchers used a “Delphi process,” which involves multiple rounds of surveys to reach a formal agreement among experts. In healthcare settings, this method is commonly used to develop clinical recommendations or identify best practices when evidence is limited or uncertain.
The panel included specialists from North and South America, Europe, and the Asia-Pacific region. Panelists completed three rounds of surveys. In the first round, they answered open-ended questions about on-demand treatment. Their responses informed the statements for the second round, in which panelists rated their level of agreement. Statements that did not reach consensus were revised and reassessed in a third round.
Consensus was defined as at least 75% agreement among at least 75% of panelists. By the end of the process, consensus was reached on 24 statements covering five main areas:
- defining early treatment
- barriers to timely treatment
- the burden of on-demand therapies
- issues related to tolerability and convenience
- the role of patient-physician communication
One key finding was a clear definition of early treatment. Experts agreed that on-demand therapy should ideally be given within 60 minutes of symptom onset. They also agreed that treatment should still be given if symptoms are progressing, even if the opportunity for early treatment is missed.
The panel identified several major roadblocks. Many patients struggle to recognize the start of an attack, especially when symptoms are mild or involve abdominal pain. Others feel anxiety or embarrassment about using injectable therapies in public. Additionally, some patients lack the confidence to inject themselves, leading them to wait until they can find a caregiver or a healthcare professional to help.
Logistics also play a major role in treatment delays. Because most current on-demand treatments are injectable, they can be difficult to store, transport, and use discreetly. The pain associated with injections and the requirement for specific storage conditions often lead patients to “wait and see” if an attack will go away on its own rather than treating it immediately.
Experts agreed that treatments that are easier to carry and self-administer, such as oral options, could significantly improve patients’ adherence to medical advice. They also noted that reducing costs and increasing the availability of these medications are essential for better patient outcomes.
Improving patient and physician communication
The panel emphasized that better communication between doctors and patients is vital. They recommended shared decision-making that considers a patient’s lifestyle, preferences, and specific fears about treatment. Education is also key; patients who understand the science of HAE and have the tools to track their symptoms, such as digital diaries, are much more likely to treat attacks early.
While the study relied on expert opinions rather than new clinical trial data, the researchers believe these insights provide a practical roadmap for the future. They concluded that providing more convenient on-demand options is the best way to help HAE patients manage their condition effectively.
“The statements agreed on by the panel demonstrate a need in this population for accessible and convenient on-demand treatments that will allow patients to improve adherence to HAE guidelines,” the team concluded.
