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Evolving Management of HAE
Expert Commentary on the Evolving Management of Hereditary Angioedema

Released: February 19, 2025

Expiration: February 18, 2026

Marc Riedl
Marc Riedl, MD, MS
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Key Takeaways
  • New and emerging therapies for hereditary angioedema (HAE) such as plasma kallikrein inhibitors, anti–factor XIIa antibodies, and emerging gene therapies have the potential to reduce treatment burdens felt by patients with HAE.
  • Transitions of care can be complex for patients with HAE; ensuring that programs are in place to support patients can improve treatment outcomes.

Introduction
This commentary addresses key points on the management of hereditary angioedema (HAE), a condition caused by C1 inhibitor deficiency. The mechanism of HAE type I (low levels of C1 inhibitor) and type II (adequate levels but improperly functioning C1 inhibitor) is well understood, but a small subset of the population has normal levels and functioning of the C1 inhibitor, making diagnosis and management much more challenging. Long-term prophylaxis can be an effective treatment option to reduce the frequency and severity of attacks. However, it is important to note that long-term prophylaxis must be individualized and incorporated into the shared decision-making process.

New HAE Therapies Reshape Management and Their Place in Therapy
New and emerging therapeutic options such as single-administration gene therapies, plasma kallikrein inhibitors, and humanized anti–factor XIIa monoclonal antibodies have the potential to reduce the treatment burden because they may be easier to administer and thus preferred by some patients. Patients may not have to take them as often, or the route of administration may be better tolerated. Patients want treatments that are, of course, effective, but that are also easy to take, convenient, and tolerable.

Treatment decisions depend in part on what patients find most tolerable or what they think they can best adhere to. The new and emerging agents generally work well for the majority of the population, but this does not mean they are effective for every patient. These agents have largely been found to be very safe, without serious adverse effects. Thus, treatment selection focuses on efficacy as well as the route and frequency of administration. Additional considerations should include less serious adverse effects that affect tolerability. These questions should be addressed during in-depth conversations with patients to create an individualized treatment plan.

Importance of Seamless Transitions of Care
The pediatric population with HAE has been relatively underserved in terms of medication availability. This is because approval for medications in pediatrics usually occurs after approval of medications for adults. This issue has improved somewhat following approvals of HAE prophylactic medicines in the past few years, but management can still be more challenging in children than in adults because there are fewer options. As children with HAE become teenagers and adults, the number of therapies that can be offered increases. Allergists and immunologists are usually cross-trained and able to treat both children and adults, which often makes transitions easier, because patients can continue to see the same specialists.

Something that can be challenging for younger patients as they move into adulthood is assuming responsibility and decision-making, especially as it pertains to treatment adherence. When patients go off to college or move out of their parents’ house and start a job, they become responsible for not only taking their medicine but also filling their prescriptions and completing relevant paperwork for insurance and patient support groups—all the complexities that go along with obtaining these medications. In short, this is a process. It is a process that starts early through discussions with not only the teenage patient but also the patient’s parents to ensure that the patient understands how important it is to continue with their treatment plan and to continue following up with their specialists. Sometimes patients stop visiting their healthcare provider or develop other non-HAE health issues that can affect their treatment plan. Therefore, it is important for healthcare professionals and patients to discuss the importance of regular follow-up to review and potentially adjust the HAE management plan for optimal results.

Your Thoughts?
What aspect of transitions of care do you find most challenging, and how do you address it? Join the discussion by posting a comment below.

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In your practice, how often are you facilitating transition-of-care discussions with patients with HAE?

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