Management of HAE

What are the ultimate goals of treatment?

The ultimate aim of HAE treatment is to allow you to live your life without any attacks. Recently, a panel of medical experts in the management of HAE and HAE patient representatives defined the ultimate goals for HAE treatment as:¹

If complete control of the disease cannot be achieved, the goal of treatment should be to reduce the number of attacks as much as possible and improve the patient’s quality of life.¹

Aiming to achieve complete control of the disease with your doctor will help you to be able to live your life as normally as possible.

When treating HAE, a distinction is made between immediate treatment of acute swelling attacks (on-demand treatment) and preventative (prophylactic) therapy. You should discuss which of these treatment options is most suitable for you with your HAE specialist.¹

On-demand treatment

HAE attacks can be unpredictable, painful, debilitating and may be life-threatening in some cases; for example, if they occur in your throat. Therefore, effective on-demand therapy can save lives.

Experts recommend that attacks are treated as early as possible, as this can shorten the duration of attacks.¹ As such, it is also recommended that you always carry on-demand medication, even if you also receive prophylactic treatment, and you should have enough on-demand treatment for at least two attacks.¹

If possible, it is also recommended that you know how to self-administer your own on-demand treatment, to be able to treat the attack as soon as possible. However, if there is swelling in the airway or upper respiratory tract, you should always also consult an emergency doctor.¹

Treatment options for on-demand therapy

HAE attacks are, in general, caused by low levels of a protein called C1 esterase inhibitor (C1-INH), or non-functional C1-INH, resulting in overproduction of bradykinin that causes the swelling in HAE attacks. More information on the cascade leading to HAE attacks can be found here.¹

There are several HAE-specific treatment options available that effectively target the HAE cascade to manage acute swelling attacks¹
These treatments are guideline recommended and are administered either intravenously (i.e., into the vein) or subcutaneously (i.e., below the skin) to resolve attacks¹

Consult your doctor to determine the most appropriate treatment option for you

Prophylactic therapy

Long-term prophylaxis (LTP)

LTP is treatment that is taken regularly to prevent or reduce the number of HAE attacks, and is currently the only treatment type that has the potential to completely control the disease and to normalise lives of patients with HAE.¹

Your doctor should discuss LTP with you and you should work together to tailor the choice of treatment for your individual needs and preferences – taking into account your attack frequency and severity, your quality of life and your response to on-demand treatment. The choice of which treatment option you should use, if any, should be made by shared decision-making.¹ Studies show that LTP can result in less frequent and less severe HAE attacks, with improved quality of life.^2-4

Nevertheless, on-demand treatment is irreplaceable. Effective on-demand treatment is key even if you use LTP as you can still experience ‘breakthrough’ attacks. It is important that you always carry on-demand treatment with you and that you have enough on-demand medication to treat at least two attacks.¹

Treatment options for long-term prophylaxis

In recent years, several new LTP treatments have become available and are now the first-line treatment recommendations of experts in the management of HAE.⁵

Similar to on-demand therapy, there are several guideline-recommended LTP treatments for HAE that target the HAE cascade to prevent attacks
These treatments offer various routes of administration to suit your needs, including intravenous (in the vein), subcutaneous (below the skin), or oral medications, which you or your caregiver can learn to administer

Consult your doctor to determine which options are available and appropriate for you

The availability of modern prophylactic treatments and personalised disease management means that complete control of HAE is now a realistic possibility for you and other patients with HAE.¹

Short-term prophylaxis (STP)

STP is treatment that is given in the lead up to an event that may trigger an attack. Certain medical procedures may cause attacks in patients with HAE:

Procedures in the mouth and throat (e.g., dental treatment)
Surgical operations
Intubation (artificial ventilation)
Minimal invasive procedures (e.g., microscopic imaging of the throat, stomach or colon)

Attacks do not always occur after a medical procedure; however, you should always inform your treating doctors about your HAE. The doctor can then decide whether to provide STP. If in doubt, the doctor can discuss and coordinate with your treating HAE specialist. You may also use STP prior to specific triggers such as stressful life events.¹

Creating your individual treatment plan with shared decision-making

When considering your individualised treatment and action plan, your physician should take into account your disease activity such as the frequency and severity of attacks you experience as well as the effects attacks have on your quality of life.

Your treatment plan should consider your on-demand therapy utilisation, as this will inform on how well your disease is controlled. To shape your individualised treatment plan, your physician should assess how your LTP is affecting your disease activity, disease burden, and disease control at least once a year so that the treatments can be adapted as needed. Your treatment plan should consider your preference for certain treatments and it is important that you are involved in this shared decision-making process.¹

Your individualised treatment and action plan should take into account preventative measures as well as home care and self-administration. Along with your LTP treatment, you should have an emergency on-demand treatment plan.¹

As you know, HAE attacks should be treated as soon as possible. Talk to your doctor if you don't know how to self-administer your treatment and always be sure to carry your treatment!

What are the benefits of shared decision-making?

Shared decision-making allows a collaborative approach to disease management involving both you and your doctor.⁶You are the expert on how HAE impacts your life and therefore should always be involved in disease management decisions. Your doctor can provide you with accurate information to help inform your choices.⁶

References

1. Maurer M, Magerl M, Betschel S, Aberer W, Ansotegui IJ, Aygören-Pürsün E, et al. The international WAO/EAACI guideline for the management of hereditary angioedema-The 2021 revision and update. Allergy. 2022;77(7):1961–90.

2. Zuraw B, Lumry WR, Johnston DT, Aygören-Pürsün E, Banerji A, Bernstein JA, et al. Oral once-daily berotralstat for the prevention of hereditary angioedema attacks: A randomized, double-blind, placebo-controlled phase 3 trial. J Allergy Clin Immunol. 2021;148(1):164-72.e9.

3. Banerji A, Riedl MA, Bernstein JA, Cicardi M, Longhurst HJ, Zuraw BL, et al. Effect of lanadelumab compared with placebo on prevention of hereditary angioedema attacks: A randomized clinical trial. JAMA. 2018;320(20):2108–21.

4. Longhurst H, Cicardi M, Craig T, Bork K, Grattan C, Baker J, et al. Prevention of hereditary angioedema attacks with a subcutaneous C1 inhibitor. N Engl J Med. 2017;376(12):1131–40.

5. Maurer M, Aygören-Pürsün E, Banerji A, Bernstein JA, Balle Boysen H, Busse PJ, et al. Consensus on treatment goals in hereditary angioedema: A global Delphi initiative. J Allergy Clin Immunol. 2021;148(6):1526–32.

6. Elwyn G, Frosch D, Thomson R, Joseph-Williams N, Lloyd A, Kinnersley P, et al. Shared decision making: A model for clinical practice. J Gen Intern Med. 2012;27(10):1361–7.