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HAE Management and Treatment

CE / CME

Expert Guidance on HAE Management and the Evolving Landscape of Prophylaxis

Physician Assistants/Physician Associates: 0.50 AAPA Category 1 CME credit

Nurse Practitioners/Nurses: 0.50 Nursing contact hour

Physicians: maximum of 0.50 AMA PRA Category 1 Credit™

Released: March 25, 2025

Expiration: March 24, 2026

Marc Riedl
Marc Riedl, MD, MS
CME/CE Information

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General Treatment Strategies for HAE: C1INH

Shifting into treatment strategies, this slide outlines the international and US guidelines. There are 3 pillars (or strategies) that HCPs should employ when developing a management plan for patients with HAE. They are on-demand treatment, short-term prophylaxis, and long-term prophylaxis.

On-demand treatment is necessary for every patient with HAE. This is a therapy that is given at the time of an attack to stop progression and rapidly improve symptoms to prevent morbidity or mortality. Every patient should possess sufficient on-demand treatment to address at least 2 attacks. Further, they should always carry this treatment on their person.

Short-term prophylaxis is used selectively at times when patients have a known exposure to a trigger. The classic example here is a dental procedure, particularly tooth extractions or oral surgery, medical procedures that will lead to tissue trauma, or intubation for general anesthesia. HCPs should implement a short-term prophylaxis strategy in patients’ treatment plan to reduce or minimize the risk of an HAE attack in these medical settings. There also may be life events that warrant short-term prophylaxis. These can include a long international trip, an important business project, a final exam, or a wedding—times when patients cannot afford to have an HAE attack. Some call this situational prophylaxis. Here, patients can use a preventative medicine for a short duration to minimize the risk of an HAE attack.

The final pillar is long-term prophylaxis, which is increasingly used in HAE management. This is a regularly taken medication that reduces the frequency and severity of HAE attacks. Ideally, it would prevent attacks entirely from occurring. Because of these new agents, outcomes in HAE are focusing more on attack-free periods. Many patients are seeing reduced attack risk and control of their disease with long-term prophylaxis. Although there are several treatment options for the long-term prophylaxis of HAE, HCPs must discuss with patients, engage in shared decision-making, and evaluate the individual to best determine where, when, and which long-term prophylactic option makes sense for them. There is no magic formula or specific recipe for instituting long-term prophylaxis in HAE. This treatment strategy can be incredibly beneficial for patients, but it is not a guarantee for all.1,2

The take-home message here is that any and all of these treatment strategies must be individualized to each patient and should aim to normalize their quality of life.

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Considerations for Therapy Initiation

In determining the most appropriate HAE treatment, there are numerous factors and variables to consider and discuss with patients. The type of HAE a patient has mostly relates to whether they have C1-INH deficiency or not. There are no known significant differences between treatment efficacy or safety for HAE-1/2, however, there are nuances in how to approach HAE-nC1-INH.

There are currently no robust, randomized trials on HAE-nC1-INH; we are still learning about and trying to study the best way to manage this subset of patients. But HCPs can feel confident about the safety and efficacy of our treatment options for HAE-1/2.

Age plays a role in determining which agents are approved for use in each patient since pediatrics are a unique subset. Having a confirmed HAE diagnosis is, of course, critical for HAE management as well. HCPs should then look at patients’ attacks—their frequency, location, type, severity, and any related complications. It is vital to discern and discuss this with patients to determine which strategy and agents will best address the particular aspects of their condition.

Further, HAE directly impacts patients’ quality of life, so these measures also are being incorporated more often into clinical trials and practice outcomes, as there are some validated tools to measure disease activity and quality of life. Ultimately, quality of life is one of the most important things to consider in managing HAE.

Other factors to consider are resource availability at your clinic, coverage of medications, and patient preferences. These are incorporated into the evidence-based guidelines largely as expert opinions because it is important for patients to have a say in their care, as they are the ones who will need to adhere to the treatment plan. Patients also may have strong preferences regarding the type of strategy or agent prescribed, including the route of administration and safety profile. Therefore, patient preferences should always be considered as HCPs develop an HAE management plan.1,2

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On-Demand Therapy and Short-term Prophylaxis

There are 4 FDA-approved options for on-demand therapy, including icatibant, ecallantide, a plasma-derived C1-INH, and a recombinant C1-INH. These are the recommended first-line on-demand treatments based on current data. As previously mentioned, patients should always carry at least 1 of these agents, and the choice of treatment should consider its accessibility and the patient’s knowledge on how to use it.

There are some second- and third-line on-demand treatments that are recommended. These include solvent detergent-treated plasma or fresh frozen plasma. The evidence for these agents are not nearly as strong, but if first-line treatments are not available, either can be considered in an emergency setting.

Patients always should have access to enough of their on-demand therapy to treat at least 2 attacks, as these attacks are unpredictable. It takes time to refill prescriptions, especially since these treatments are made available through specialty pharmacies, and we do not want patients to be without any on-demand treatment for any amount of time.

Unlike on-demand treatment, short-term prophylaxis is used more selectively for medical or surgical procedures or for certain life events to prevent attacks in a shorter period. The strongest evidence here is for intravenous, plasma-derived C1-INH given before the suspected trigger. There are data that support recombinant C1-INH, fresh frozen plasma, or attenuated androgens if plasma-derived C1-INH is inaccessible or unavailable.1,2

HCPs should ensure patients understand that these agents continue to be the recommendation based on the available evidence. Patients should always inform their HCP about any upcoming procedures or life event so a short-term prophylactic plan can be established.

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