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Optimizing modern HAE management
Optimizing Modern HAE Management With Novel Therapies and Patient-Centered Care

Released: August 27, 2025

Marc Riedl
Marc Riedl, MD, MS
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Key Takeaways
  • The treatment landscape for patients with hereditary angioedema (HAE) continues to evolve as more long-term prophylaxis therapies with novel mechanisms of action emerge.
  • HAE management strategies should always be individualized to each patient, considering preferences regarding treatment dosing, scheduling, route of administration, and the patient’s experience with this genetic condition.
  • Investigational RNA-targeted agents, monoclonal antibodies, and gene therapies are addressing the treatment burden of HAE by offering less frequent dosing with promising HAE management.

We continue to see an evolution in treatment for patients with hereditary angioedema (HAE) as new therapies emerge. The mainstay, however, is for healthcare professionals (HCPs) to always provide individualized and patient-centered care with optimized treatment for each patient with HAE.

Although currently approved HAE treatments—on-demand and prophylaxis therapies—are effective across many populations and have good safety profiles, patients respond differently to medications. Responses may be based on reported adverse effects, the agent’s mechanism of action, or its efficacy in general. In addition, the development of new therapies with novel mechanisms of action will allow us to improve our care delivery and provide more options to patients who may have strong preferences. HCPs should strongly consider patients’ preferences because they know what works best for them and their lives. Maybe it is the dosing schedule, the route of administration, or the mechanism of action for certain agents.

I think that we will see changes in HAE management happen slowly and steadily. The more treatment options we have available for long-term prophylaxis, the more treatment plan refinement we will see for each individual patient. Preventing HAE attacks is appealing because it lends predictability to life and allows patients to carry out their daily activities without worrying about the painful disruption of dangerous HAE attacks.

Patient-Centered HAE Management Strategies
The first question HCPs need to ask themselves is, “Does a long-term prophylactic treatment approach make sense for the patient?” And the approach, according to the guidelines, should be to discuss long-term prophylaxis with every patient. Even if they do not choose to use prophylactic treatment currently, it may be an important option for them in the future. In my practice, I have found that most patients are interested in long-term prophylaxis because their HAE is quite disruptive, and preventing attacks is incredibly appealing to them.

Next, I walk through the different therapies with patients, with safety and efficacy always at the top of the list. I ensure that patients understand what the study data show in terms of the efficacy in preventing HAE attacks, the possible adverse effects to watch for, and the overall safety of the agent. There are other things that are important to patients that HCPs should consider, such as the route of administration, dosing schedule, possible dose adjustments that may be made after establishing a response, and portability, especially for patients who travel or are away from home often. You want to ensure that patients understand exactly how their treatment works and how they must administer it.

These are all important aspects of patient-centered care. But much of the decision-making comes down to 3 things. The optimal treatment choice has to: (1) work for them, (2) be tolerable, and (3) fit best into their particular lifestyle and/or experience with managing their HAE. This is all part of the shared decision-making process that is so critical in HAE management.

Furthermore, as HCPs who are experts in HAE, it is our job to know about this disease and the current data on the available treatments. But patients certainly are the experts in their experience with HAE and various medications. That partnership with patients—talking through the treatment options and seeing what fits best for each patient—is really what allows us to individualize each treatment plan. And at the end of the day, the more options we have, the better we can tailor care and treatment to meet each patient's specific needs.

Emerging HAE Therapies and Research
There are some exciting things happening in HAE research now. One is based on the mechanisms of action for certain agents in development, in particular, RNA-targeted medicines like donidalorsen and gene therapy. These have the potential to provide a longer duration of effect with a single treatment or dose. That is appealing because patients might receive effective prevention with less treatment. Of course, safety is always important, and RNA-targeted therapies have been used safely in other areas of medicine. There are a few more questions about the safety of gene therapy since it is considered a potentially permanent and irreversible treatment. HCPs must closely monitor patients for safety in the short and long term. Yet some of the study data that we are seeing for these agents look quite promising.

Another exciting development considers the burden of treatment, which relates to the need for less frequent or burdensome dosing. Monoclonal antibodies, such as navenibart, offer prolonged plasma half-life, providing patients better dosing administration options (ie, every 3 or 6 months) and potentially a long duration of effect, which is being investigated in current studies. Oral targeted small molecules, such as deucrictibant, a bradykinin B2 receptor antagonist, may offer an additional noninjected prophylactic option if proven safe and effective in ongoing clinical trials.

Additional treatment options with variable routes of administration, dosing schedules, and mechanisms of action for novel therapies will improve our management strategies for HAE in the long run. Therapies should be effective and safe and also reduce the burden of treatment. Remember that HAE is a genetic disease, making it a chronic and lifelong condition. Currently, we do not have a cure for HAE. Therefore, any treatment plan developed should ensure tolerability so that it is something patients can adhere to over long periods of time, maybe with some adjustments along the way.

The burden of treatment is a real issue here, and new treatment options should reduce this burden. I think that it is exciting now that we can effectively manage HAE and make it much easier for patients to live their lives without disruption from an attack and have minimal interference from their treatment.

Your Thoughts
How often do you discuss long-term prophylaxis strategies with your patients with HAE? You can get involved in the discussion by answering the poll question and posting a comment below.

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How often do you discuss long-term prophylaxis strategies with your patients with HAE?

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