Materiały źródłowe

• #1 Prevention of Recurrent Attacks of Hereditary Angioedema (HAE): Berotralstat and Its Oral Bioavailability

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10069425/

  Hereditary angioedema (HAE) is a condition characterized by episodes of cutaneous and submucosal edema. […] To minimize the difficulties of this pathology and to improve patients quality of life, prevention of this condition is essential. Berotralstat is a unique option for oral administration for routine prophylaxis. This drug acts by binding to kallikrein and reducing its plasma activity, lowering bradykinin levels. […] Clinical guidelines recommend, as a first line therapy for long-term prophylactic treatment of HAE type 1 and 2, plasma-derived C1-INH, lanadelumab, and berotralstat. […] Long-term prophylactic treatment is one of the goals of treatment of HAE. Preventing attacks can decrease complications of an acute attack but also allow patients to have a normal lifestyle. […] Clinical guidelines for long-term prophylaxis in HAE recommend plasma-derived C1-INH, lanadelumab, and berotralstat as first line medications.

• #2 Angioedema treatment and prevention | Angioedema NewsEnvelope icon

  https://angioedemanews.com/angioedema-treatments/

  Angioedema treatment mainly aims to control and prevent swelling, or edema, that occurs in the deeper layers of the skin or in mucus membranes. […] While there are a number of different treatment options available, most of them medications, their use depends on many factors, such as the type of angioedema and whether the goal is prevention or on-demand treatment. […] In all forms of angioedema, a key aspect in disease management is taking steps to lower the risk of future swelling attacks. […] For some patients, preventing future angioedema attacks may include resorting to medications that are taken regularly to reduce the risk of swelling. […] The guidelines also suggest that patients should consider long-term prophylactic (preventive) angioedema treatment to reduce the risk of swelling attacks.

• #3 Angioedema treatment and prevention | Angioedema NewsEnvelope icon

  https://angioedemanews.com/angioedema-treatments/

  Angioedema treatment mainly aims to control and prevent swelling, or edema, that occurs in the deeper layers of the skin or in mucus membranes. […] While there are a number of different treatment options available, most of them medications, their use depends on many factors, such as the type of angioedema and whether the goal is prevention or on-demand treatment. […] In all forms of angioedema, a key aspect in disease management is taking steps to lower the risk of future swelling attacks. […] For some patients, preventing future angioedema attacks may include resorting to medications that are taken regularly to reduce the risk of swelling. […] The guidelines also suggest that patients should consider long-term prophylactic (preventive) angioedema treatment to reduce the risk of swelling attacks.

• #4

  https://link.springer.com/article/10.1007/s40629-022-00223-8

  Hereditary angioedema (HAE) is a chronic condition which severely impacts those afflicted and is characterized by edema in mucosal and submucosal tissue or in dermal and subcutaneous structures, caused by vasodilatation. […] Due to the severity of impact of symptoms and the risks of an undetected or untreated HAE attack, early detection, diagnosis, and effective prevention or treatment of all attacks are particularly important. […] We will also summarize the current knowledge on symptoms caused by HAE, offer specific tips for early detection in everyday practice, and highlight the key role of long-term prophylaxis and patient-reported outcome measures (PROMs) as part of optimum management of HAE in line with the updated WAO/EAACI guidelines. […] The most important innovation in the range of HAE treatments, by expert consensus and in accordance with the latest international guidelines on HAE management, is the emphasis on long-term prophylaxis.

• #5 Management and Prevention of Hereditary Angioedema Attacks

  https://www.ajmc.com/view/ace010_lumry2_s111to18

  Hereditary angioedema (HAE) is a rare genetic syndrome caused by a deficiency in functional C1 inhibitor that results in recurrent episodes of nonpruritic swelling of the hands, feet, arms, legs, trunk, face, genitalia, bowels, and larynx beginning in childhood or adolescence and continuing throughout the patients lifetime. […] Treatment for acute HAE attacks in the United States has been transformed by new therapies that inhibit the underlying mechanisms of angioedema notably ecallantide, a potent and specific inhibitor of plasma kallikrein, and icatibant, a selective bradykinin receptor antagonist. […] This article reviews the current therapeutic landscape for HAE, including treatment for acute angioedema attacks, short- and long-term HAE prophylaxis, and home-based therapy. […] Contemporary medical management of HAE is divided between treatment of swelling attacks, short-term prophylaxis to avoid attacks during times of increased risk (eg, after medical or dental procedures), and long-term prophylaxis to reduce both the frequency and severity of attacks.

• #6

  https://link.springer.com/article/10.1007/s12016-024-09006-1

  Long-term prophylaxis (LTP) has been shown to reduce the frequency of hereditary angioedema (HAE) attacks; however, attacks occurring in patients receiving LTP have not been well characterized. […] LTP agents have been demonstrated to reduce the frequency of HAE attacks. […] The overarching goal of HAE treatment is to normalize patients’ lives by achieving complete disease control. […] This review confirms that a substantial proportion of patients who receive LTP do not achieve the goal of an attack-free status and experience attacks which are unpredictable with regard to severity, anatomical location, and duration. […] Attack-free rates were generally low (45%) in patients who received LTP with IV or SC pdC1INH, berotralstat, danazol, and TA. […] Most attacks that occurred in patients who received LTP were treated with 1 dose of an on-demand therapy, and unrestricted access to effective on-demand therapy remains essential for all people with HAE-C1INH, including patients receiving LTP.

• #7 Angioedema treatment and prevention | Angioedema NewsEnvelope icon

  https://angioedemanews.com/angioedema-treatments/

  Angioedema treatment mainly aims to control and prevent swelling, or edema, that occurs in the deeper layers of the skin or in mucus membranes. […] While there are a number of different treatment options available, most of them medications, their use depends on many factors, such as the type of angioedema and whether the goal is prevention or on-demand treatment. […] In all forms of angioedema, a key aspect in disease management is taking steps to lower the risk of future swelling attacks. […] For some patients, preventing future angioedema attacks may include resorting to medications that are taken regularly to reduce the risk of swelling. […] The guidelines also suggest that patients should consider long-term prophylactic (preventive) angioedema treatment to reduce the risk of swelling attacks.

• #8 The latest therapeutic reports on short-term and long-term prophylaxis in the treatment of hereditary angioedema

  https://www.termedia.pl/The-latest-therapeutic-reports-on-short-term-and-long-term-prophylaxis-in-the-treatment-of-hereditary-angioedema,123,52426,1,1.html

  In the prophylaxis of HAE, we can distinguish between drugs used for both short-term prophylaxis and long-term prevention. The former group includes plasma-derived C1-INH (pd-C1-INH) concentrate (Berinert) and the latter, the human monoclonal antibody, plasma kallikrein inhibitor lanadelumab (Takhzyro) and Ecallantide (Kalbitor). Plasma-derived C1-INH concentrate (Berinert) in Poland for long-term prophylaxis is still an off-label drug. We also have other off-label drugs: tranexamic acid (Exacyl) and a synthetic steroid, an ethinyl-testosterone derivative (Danazol the only androgen derivative preparation available in Poland) […] Short-term prophylaxis for HAE focuses on preventing and minimizing symptoms before specific situations that may trigger an attack, such as surgery, skin or mucosal disruption, or stress. First-line treatment includes the administration of pd-C1-INH at a dose of 15-30 U/kg body weight, up to 6 h before the scheduled surgery.

• #9 Management and Prevention of Hereditary Angioedema Attacks

  https://www.ajmc.com/view/ace010_lumry2_s111to18

  Short-term prophylactic therapy is recommended prior to exposure to a potential trigger for an HAE attack, particularly dental work or invasive medical or surgical procedures. […] As shown in Figure 2, plasma-derived C1INH, if readily available, is the preferred short-term preventive therapy for patients with HAE undergoing minor dental manipulation, but pretreatment may be reserved for those in whom dental procedures have previously precipitated an attack. […] If C1INH is not available, 17-a-alkylated anabolic androgen (eg, danazol, 200 mg 2-3 times per day) for 5 days before the procedure and 2 to 5 days after is recommended. […] Development of agents for the long-term prevention of HAE angioedema attacks has lagged behind that of acute therapy. […] Presently, only Cinryze and danazol are FDA approved for the routine prophylaxis of HAE.

• #10 Management and Prevention of Hereditary Angioedema Attacks

  https://www.ajmc.com/view/ace010_lumry2_s111to18

  Short-term prophylactic therapy is recommended prior to exposure to a potential trigger for an HAE attack, particularly dental work or invasive medical or surgical procedures. […] As shown in Figure 2, plasma-derived C1INH, if readily available, is the preferred short-term preventive therapy for patients with HAE undergoing minor dental manipulation, but pretreatment may be reserved for those in whom dental procedures have previously precipitated an attack. […] If C1INH is not available, 17-a-alkylated anabolic androgen (eg, danazol, 200 mg 2-3 times per day) for 5 days before the procedure and 2 to 5 days after is recommended. […] Development of agents for the long-term prevention of HAE angioedema attacks has lagged behind that of acute therapy. […] Presently, only Cinryze and danazol are FDA approved for the routine prophylaxis of HAE.

• #11 Hereditary angioedema: Short-term prophylaxis before procedures or stressful events to prevent angioedema episodes – UpToDate

  https://www.uptodate.com/contents/hereditary-angioedema-short-term-prophylaxis-before-procedures-or-stressful-events-to-prevent-angioedema-episodes

  Hereditary angioedema: Short-term prophylaxis before procedures or stressful events to prevent angioedema episodes […] The various prophylactic therapies that are used prior to prevent HAE attacks („preprocedural” prophylaxis) will be reviewed here. […] Short-term prophylaxis to prevent angioedema in hereditary angioedema (HAE)

• #12 The latest therapeutic reports on short-term and long-term prophylaxis in the treatment of hereditary angioedema

  https://www.termedia.pl/The-latest-therapeutic-reports-on-short-term-and-long-term-prophylaxis-in-the-treatment-of-hereditary-angioedema,123,52426,1,1.html

  In the prophylaxis of HAE, we can distinguish between drugs used for both short-term prophylaxis and long-term prevention. The former group includes plasma-derived C1-INH (pd-C1-INH) concentrate (Berinert) and the latter, the human monoclonal antibody, plasma kallikrein inhibitor lanadelumab (Takhzyro) and Ecallantide (Kalbitor). Plasma-derived C1-INH concentrate (Berinert) in Poland for long-term prophylaxis is still an off-label drug. We also have other off-label drugs: tranexamic acid (Exacyl) and a synthetic steroid, an ethinyl-testosterone derivative (Danazol the only androgen derivative preparation available in Poland) […] Short-term prophylaxis for HAE focuses on preventing and minimizing symptoms before specific situations that may trigger an attack, such as surgery, skin or mucosal disruption, or stress. First-line treatment includes the administration of pd-C1-INH at a dose of 15-30 U/kg body weight, up to 6 h before the scheduled surgery.

• #13 Management and Prevention of Hereditary Angioedema Attacks

  https://www.ajmc.com/view/ace010_lumry2_s111to18

  Short-term prophylactic therapy is recommended prior to exposure to a potential trigger for an HAE attack, particularly dental work or invasive medical or surgical procedures. […] As shown in Figure 2, plasma-derived C1INH, if readily available, is the preferred short-term preventive therapy for patients with HAE undergoing minor dental manipulation, but pretreatment may be reserved for those in whom dental procedures have previously precipitated an attack. […] If C1INH is not available, 17-a-alkylated anabolic androgen (eg, danazol, 200 mg 2-3 times per day) for 5 days before the procedure and 2 to 5 days after is recommended. […] Development of agents for the long-term prevention of HAE angioedema attacks has lagged behind that of acute therapy. […] Presently, only Cinryze and danazol are FDA approved for the routine prophylaxis of HAE.

• #14 Short-term prophylaxis in patients with angioedema due to C1-inhibitor deficiency undergoing dental procedures: An observational study | PLOS One

  https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0230128

  Most C1-INH-HAE patients reported hurdles in receiving dental care. STP protects against attacks after dental procedures. Treating oral diseases results in improvement in the frequency of attacks. […] STP should be considered in these patients before dental procedures. […] STP was effective in preventing post-procedural attacks. […] A considerable percentage of patients (21.05%) experienced a reduction in the frequency of angioedema attacks after receiving dental care. This highlights the importance of treating oral pathologies in patients affected by angioedema due to C1-INH deficiency. […] Therefore, STP is strongly recommended in these patients who are undergoing tooth extraction.

• #15 The latest therapeutic reports on short-term and long-term prophylaxis in the treatment of hereditary angioedema

  https://www.termedia.pl/The-latest-therapeutic-reports-on-short-term-and-long-term-prophylaxis-in-the-treatment-of-hereditary-angioedema,123,52426,1,1.html

  Long-term prophylaxis is aimed at preventing chronic and recurrent attacks of edema and is indicated in patients with attacks occurring 1-2 per week requiring medication. This strategy involves regular, ongoing therapy, which includes the administration of pd-C1-INH 1-2 times a week, registered in the European Union for intravenous infusion. In first-line treatment, there is also lanadelumab registered in the EU and used at a dose of 300 mg twice a month. […] The treatment plan should be tailored to the individual patient’s needs, with drug selection and dosage closely monitored by a specialist to minimize side effects and ensure optimal disease control. Long-term prophylaxis can significantly reduce the frequency and severity of attacks, allowing patients to lead more normal and active lives. Recent research and development of new therapies help to further expand treatment options, offering HAE patients increasingly better control over their condition.

• #16 Hereditary Angioedema: Novel Molecules for Treatment of Acute Attacks and Long-Term Prophylaxis

  https://www.mdpi.com/2673-9879/4/1/5

  According to the efficacy of available treatments that allow the efficient management of attacks or reduce the frequency of attacks in long-term prophylaxis, but cannot offer complete disease control or normalize patients’ lives, there are some unmet needs that can be met with new drugs. For this reason, several pharmaceutical companies are actively developing new therapeutic options for HAE patients. […] Long-term prophylaxis (LTP) consists of prophylactic treatment, and involves ongoing or maintenance treatment with the objective of reducing the occurrence, intensity, and duration of HAE attacks. […] The authors of the international WAO/EAACI guideline recommend that long-term prophylaxis (LTP) should be considered when patients experience significant life events that are linked to heightened disease activity. The 2021 revision and update highlight the importance of complete control of the disease, with the aim of normalizing patients’ lives.

• #17 Hereditary Angioedema Management | TAKHZYRO® (lanadelumab-flyo)

  https://www.takhzyro.com/hcp/why-takhzyro/

  Long-term HAE prevention should be individualized and considered in all patients with HAE. […] The 2020 US HAEA guidelines recommend: Reviewing management plans for patients with HAE on a regular basis, including the need for preventive treatment. […] The US HAEA guidelines recommend TAKHZYRO as one of the first-line preventive therapies for long-term prevention in adults and adolescents 12 years of age. […] Since most attacks are unpredictable and not prompted by triggers, the 2021 international WAO/EAACI guidelines suggest physicians should not support excessive avoidance of suspected triggers, which can limit a patients normal life. […] TAKHZYRO (lanadelumab-flyo) is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in patients 2 years of age.

• #18 Hereditary Angioedema

  https://www.clevelandclinicmeded.com/medicalpubs/diseasemanagement/allergy/hereditary-angioedema/

  Treatment of HAE relies on longterm prophylaxis, shortterm prophylaxis, and the treatment of acute attacks. The longterm goal is to minimize the frequency and severity of angioedema episodes. […] Long-term prophylaxis should be considered in patients with a history of lifethreatening attacks or serious attacks more than once every 3 months. […] Prior to U.S. Food and Drug Administration (FDA) approval of C1 inhibitor replacement therapy in 2008, prophylactic or longterm treatment of HAE relied on the use of synthetic 17-alkylated androgens: danazol and stanozolol (Winstrol). […] Antifibrinolytic agents, such as aminocaproic acid, are another option for longterm treatment. […] More recently, various C1 inhibitor concentrates have been studied and FDAapproved for treatment of HAE in the United States.

• #19 Prophylactic Therapy for HAE

  https://www.cinryze.com/hcp/understand-prophylactic-therapy-for-hae

  Routine prevention may help patients prevent and reduce the frequency and severity of hereditary angioedema (HAE) attacks. […] Guidelines support exploring preventive treatment as part of an HAE treatment plan. They recommend that patients be evaluated for long-term prevention at every visit, or at least once a year. […] It is recommended that all attacks are considered for on-demand treatment, especially for attacks affecting the upper airway. […] Collaborative treatment decisions between physicians and patients are critical. […] Potential risks and physical benefits of prevention.

• #20 Prevention of Recurrent Attacks of Hereditary Angioedema (HAE): Berotralstat and Its Oral Bioavailability

  https://pmc.ncbi.nlm.nih.gov/articles/PMC10069425/

  Hereditary angioedema (HAE) is a condition characterized by episodes of cutaneous and submucosal edema. […] To minimize the difficulties of this pathology and to improve patients quality of life, prevention of this condition is essential. Berotralstat is a unique option for oral administration for routine prophylaxis. This drug acts by binding to kallikrein and reducing its plasma activity, lowering bradykinin levels. […] Clinical guidelines recommend, as a first line therapy for long-term prophylactic treatment of HAE type 1 and 2, plasma-derived C1-INH, lanadelumab, and berotralstat. […] Long-term prophylactic treatment is one of the goals of treatment of HAE. Preventing attacks can decrease complications of an acute attack but also allow patients to have a normal lifestyle. […] Clinical guidelines for long-term prophylaxis in HAE recommend plasma-derived C1-INH, lanadelumab, and berotralstat as first line medications.

• #21 Establishing New Standards in Hereditary Angioedema: Improving Outcomes Through Routine Prophylaxis – European Medical Journal

  https://www.emjreviews.com/allergy-immunology/article/establishing-new-standards-in-hereditary-angioedema-improving-outcomes-through-routine-prophylaxis-j01121/

  Hereditary angioedema (HAE) is a rare but debilitating and potentially fatal disease that presents in various forms, and can be difficult to manage. […] Three prophylactic medications are now licenced for use and recommended in international treatment guidelines, which effectively prevent future HAE attacks. […] Kiani believes that more patients can benefit from access to prophylactic treatments to prevent HAE attacks. Now there are three first line prophylactic therapies licenced, we are moving towards being able to prevent attacks, and more patients can expect to live a more normal life. […] The 2021 International World Allergy Organization (WAO)/EAACI Guidelines recommend use of one of three long-term prophylactic treatments to prevent future HAE attacks, which can be interchanged if response to one is poor.

• #22 Long term prophylaxis for HAE with C1 inhibitor | JAA

  https://www.dovepress.com/a-review-of-randomized-controlled-trials-of-hereditary-angioedema-long-peer-reviewed-fulltext-article-JAA

  Treatment of HAE should involve individualized management strategies addressing on-demand/acute therapy, short-term and long-term prophylaxis, to minimize disease burden and improve patient-related quality of life. […] Long-term prophylaxis is considered in patients with recurrent episodes of HAE to reduce the frequency, duration, and severity of attacks, as well as to minimize the impact of the disease on daily activities. […] First-line long-term prophylactic treatment for HAE includes intravenous and subcutaneous pdC1-INH; subcutaneous lanadelumab, a monoclonal antibody that targets plasma kallikrein; and oral berotralstat, a small-molecule inhibitor of plasma kallikrein. […] The RCT data support that C1-INH replacement therapy is an effective tool for long-term prophylaxis in the management of HAE. Reductions in HAE attack frequency of 90% are achievable and could represent freedom from disease activity in clinical practice. […] Data from C1-INH replacement RCTs suggest that with adequate dosing and frequency of administration, even for patients with highly symptomatic HAE activity, potentially minimizing the burden of this disease is achievable.

• #23 Hereditary Angioedema: Novel Molecules for Treatment of Acute Attacks and Long-Term Prophylaxis

  https://www.mdpi.com/2673-9879/4/1/5

  In the last 10–15 years there has been an increased use of long-term prophylaxis for HAE attacks, especially in patients that present with frequent attacks (>3–4/month) and severe attacks (laryngeal or abdominal). […] The available therapeutic options then include plasma-derived C1 inhibitor for IV infusion (Cinryze, Takeda) or for subcutaneous administration at a higher dosage (Haegarda or Berinert sc, CSL Behring); both types of preparations need a twice-weekly infusion of each drug and can obtain a reduction in the attack rate of between 50% to a maximum of 88% for the subcutaneous C1 inhibitor preparation. […] Another more recent option for long-term prophylaxis is represented by lanadelumab, a humanized monoclonal antibody against activated plasma kallikrein, which is administered subcutaneously once every two weeks in the induction and once every 28 days after stabilization of the therapeutic response or after 6 months if the patient is attack-free.

• #24 Angioedema treatment and prevention | Angioedema NewsEnvelope icon

  https://angioedemanews.com/angioedema-treatments/

  Both Cinryze, which is injected directly into the bloodstream, and Haegarda, which is given via a subcutaneous or under-the-skin injection, contain a functional version of the C1-INH protein. […] If these angioedema medications aren’t effective or well tolerated, steroid-based medicines that can boost C1-INH production in the body, such as the attenuated androgen danazol, may be used as a second-line routine preventive treatment. […] Takhzyro, an antibody-based treatment administered via a subcutaneous injection, and Orladeyo, an oral small molecule taken once daily, also are available as a prophylactic treatment for HAE. […] The association’s recommendations suggest that every person with HAE should have access to an on-demand treatment to manage swelling attacks if they occur.

• #25 Hereditary and Acquired C1 Inhibitor Deficiency – Immunology; Allergic Disorders – MSD Manual Professional Edition

  https://www.msdmanuals.com/professional/immunology-allergic-disorders/allergic-autoimmune-and-other-hypersensitivity-disorders/hereditary-and-acquired-c1-inhibitor-deficiency

  Medications used for long-term prophylaxis of hereditary C1 inhibitor deficiency episodes include […] Plasma-derived C1 inhibitor may be given in regular IV infusions or subcutaneous injections as long-term prophylaxis. Patients can be taught to self-administer. Plasma-derived C1 inhibitor is available for long-term prevention of hereditary angioedema in the United States, but recombinant C1 esterase inhibitor is not. […] Lanadelumab is a recombinant humanized monoclonal antibody that binds to plasma kallikrein and blocks its activity. […] Berotralstat is a synthetic small molecule developed to inhibit plasma kallikrein. […] Attenuated androgens (eg, stanozolol, danazol) are used to stimulate hepatic C1 inhibitor synthesis. This treatment may be less effective for the acquired form of angioedema.

• #26 Management and Prevention of Hereditary Angioedema Attacks

  https://www.ajmc.com/view/ace010_lumry2_s111to18

  The safety and efficacy of Cinryze for routine HAE prophylaxis was demonstrated in a randomized, double-blind, placebo-controlled crossover study in 24 patients with HAE who had a history of at least 2 angioedema attacks per month. […] Patients receiving androgens should be routinely monitored for hepatic abnormalities with liver function tests and abdominal ultrasound every 6 to 12 months. […] Antifibrinolytic agents, primarily tranexamic acid and aminocaproic acid, though generally considered less effective than androgens, can be used as an alternative for long-term HAE prophylaxis in certain patient groups for whom androgens are contraindicated (eg, pregnant or lactating women, children). […] Effective management of HAE requires a comprehensive approach to care and prevention. […] All patients with HAE should have an individualized treatment and action plan developed in close collaboration with their physician that address both acute (on demand) treatment needs and long-term prevention.

• #27 Garadacimab for the prevention of hereditary angioedema attacks – VJBM

  https://www.biomedicine.video/cardiopulmonary-vascular/can-new-treatment-garadacimab-prevent-attacks-in-patients-with-hereditary-angioedema

  This video article presents the results of the VANGUARD phase III study, a pivotal, multicentre, randomized, double-blind, placebo-controlled trial investigating the efficacy and safety of garadacimab (a novel, fully human monoclonal antibody that inhibits activated factor XII), for prevention of hereditary angioedema attacks. […] Despite advances in the management of hereditary angioedema, there is an unmet need for convenient prophylactic treatments that show sustained efficacy in preventing hereditary angioedema attacks with a favorable safety profile. […] The VANGUARD pivotal phase III study demonstrated that once-monthly subcutaneous administration of garadacimab significantly reduced the number of hereditary angioedema attacks in patients aged 12 years and older compared with placebo and had a favourable safety profile. […] These results support the use of garadacimab as a long-term prophylactic therapy for the treatment of hereditary angioedema in adolescents and adults.

• #28 Hereditary Angioedema: Novel Molecules for Treatment of Acute Attacks and Long-Term Prophylaxis

  https://www.mdpi.com/2673-9879/4/1/5

  The ultimate objective of any novel therapy for treating hereditary angioedema is curation of the disease. This can be obtained by manipulating the genome by inserting a new SERPING1 gene that will compensate for the mutated allele and, therefore, will restitute a condition similar to normality with two alleles producing a normal amount of the C1 inhibitor protein and, at the bottom, abrogating the predisposing condition for recurrence of angioedema attacks.

• #29 Long-term Prophylaxis with Androgens in the management of Hereditary Angioedema (HAE) in emerging countries | Orphanet Journal of Rare Diseases | Full Text

  https://ojrd.biomedcentral.com/articles/10.1186/s13023-022-02536-x

  Hereditary angioedema (HAE) is a rare autosomal dominant genetic disease characterized by repetitive subcutaneous or submucosal angioedema, activation of the kinin system, and increased vascular permeability. Although androgens are not approved for HAE treatment in many countries, they are widely used in China and Brazil to reduce the frequency and severity of HAE attacks. The ultimate goals of HAE treatment are to achieve total control of the disease and improve patient quality of life (QoL). Androgens have been widely used as long-term prophylaxis treatment for HAE patients in many countries, such as Brazil and China. Indeed, androgens are the most widely used long-term prophylaxis treatment in Brazil, where major adverse effects associated with continued administration of high-dosage androgen treatment are hepatotoxicity and virilization. Previous studies demonstrated that danazol can reduce the frequency and severity of HAE attacks. The shortcomings of androgen use in HAE were also reported in several population-based studies. Considering these findings, it could be speculated that some adverse effects of androgens were related to variations in treatment methods and/or the severity of disease in HAE patients. Androgens and antifibrinolytic agents are the main preventive treatment for long-term prophylaxis in Brazil and China. However, in consideration of the adverse effects of androgen treatment and need to improve patient QoL, androgens are no longer the first treatment choice recommended by guidelines in the global consensus. Currently, the recommended treatment option for long-term prevention of attacks in children (12 years) is C1-INH concentrate and lanadelumab. Therefore, patients with HAE who are administered androgens long term should have regular physical examinations to adjust their medication regimen. Alternative options might be considered in certain cases, such as C1-INH concentrate for pregnant women, or C1-INH concentrate and lanadelumab for adolescents (12 years).

• #30 Hereditary angioedema: Types, treatment, and more

  https://www.medicalnewstoday.com/articles/preventative-treatment-hereditary-angioedema

  There is no cure for HAE. Treatments focus on both preventing attacks from occurring and treating symptoms. […] According to the US Hereditary Angioedema Association (HAEA), doctors currently use seven medications to treat HAE. […] Parents or caregivers should talk to their child’s doctor about the possibility of them having HAE, particularly if one or both biological parents are living with the condition. […] If the child does have HAE, parents or caregivers should talk to their doctor about available treatment options and work with them to create a treatment plan. […] People with HAE who are pregnant should also talk to their doctor about their treatment options.

• #31 BioCryst Reports First Quarter 2025 Financial Results and Provides Business Update | BioCryst Pharmaceuticals, Inc.

  https://ir.biocryst.com/news-releases/news-release-details/biocryst-reports-first-quarter-2025-financial-results-and

  ORLADEYO (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks […] The company has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) to expand the ORLADEYO label to children with HAE aged 2 to 11 using an oral granule formulation. ORLADEYO would be the first targeted oral prophylactic therapy for children with HAE. […] Today we are reporting significant milestones for each of the next three programs in our pipeline, demonstrating our focus on advancing medicines with the potential to provide differentiated outcomes for patients of all ages. As we move closer to delivering an urgently needed oral prophylactic therapy to children with HAE.

• #32 BioCryst Reports First Quarter 2025 Financial Results and Provides Business Update – BioSpace

  https://www.biospace.com/press-releases/biocryst-reports-first-quarter-2025-financial-results-and-provides-business-update

  ORLADEYO (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks […] The company has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) to expand the ORLADEYO label to children with HAE aged 2 to 11 using an oral granule formulation. ORLADEYO would be the first targeted oral prophylactic therapy for children with HAE. […] As we move closer to delivering an urgently needed oral prophylactic therapy to children with HAE, we are simultaneously progressing two clinical-stage programs in Netherton syndrome and DME towards the first patient data later this year.

• #33 Angioedema – StatPearls – NCBI Bookshelf

  https://www.ncbi.nlm.nih.gov/books/NBK538489/

  Treatment is with antihistamines, epinephrine, and glucocorticoids. […] Care should be taken to stop the offending ACE inhibitor, and the patient should be not rechallenged with any of the ACE inhibitors in the future. […] Majority cases are asymptomatic and respond to immunochemotherapy. […] Treatment of acute attacks with icatibant and plasma-derived C1 inhibitor concentrate, and prophylaxis is with rituximab with or without chemotherapy and splenectomy. […] In pregnancy, the recommended therapy is plasma-derived nano-filtered C1-inhibitor, however, in acute episodes, bradykinin receptor antagonist Icatibant can be used as it is safe with no maternal and fetal adverse effects. […] In the pediatric population, the doses include 500 units (10-25 kg weighed patients), 1000 units and 1500 Units in patients weighing more than 25kg.

• #34 Long-term Prophylaxis with Androgens in the management of Hereditary Angioedema (HAE) in emerging countries | Orphanet Journal of Rare Diseases | Full Text

  https://ojrd.biomedcentral.com/articles/10.1186/s13023-022-02536-x

  Hereditary angioedema (HAE) is a rare autosomal dominant genetic disease characterized by repetitive subcutaneous or submucosal angioedema, activation of the kinin system, and increased vascular permeability. Although androgens are not approved for HAE treatment in many countries, they are widely used in China and Brazil to reduce the frequency and severity of HAE attacks. The ultimate goals of HAE treatment are to achieve total control of the disease and improve patient quality of life (QoL). Androgens have been widely used as long-term prophylaxis treatment for HAE patients in many countries, such as Brazil and China. Indeed, androgens are the most widely used long-term prophylaxis treatment in Brazil, where major adverse effects associated with continued administration of high-dosage androgen treatment are hepatotoxicity and virilization. Previous studies demonstrated that danazol can reduce the frequency and severity of HAE attacks. The shortcomings of androgen use in HAE were also reported in several population-based studies. Considering these findings, it could be speculated that some adverse effects of androgens were related to variations in treatment methods and/or the severity of disease in HAE patients. Androgens and antifibrinolytic agents are the main preventive treatment for long-term prophylaxis in Brazil and China. However, in consideration of the adverse effects of androgen treatment and need to improve patient QoL, androgens are no longer the first treatment choice recommended by guidelines in the global consensus. Currently, the recommended treatment option for long-term prevention of attacks in children (12 years) is C1-INH concentrate and lanadelumab. Therefore, patients with HAE who are administered androgens long term should have regular physical examinations to adjust their medication regimen. Alternative options might be considered in certain cases, such as C1-INH concentrate for pregnant women, or C1-INH concentrate and lanadelumab for adolescents (12 years).

• #35 Angioedema – StatPearls – NCBI Bookshelf

  https://www.ncbi.nlm.nih.gov/books/NBK538489/

  Antihistamine, corticosteroids, and epinephrine are treatments of histaminergic angioedema. […] Treatment of Bradykinin-mediated angioedema is often resistant to standard therapies such as epinephrine, glucocorticoids or antihistamines. […] Short-term prophylaxis before high-risk procedures in high-risk individuals. […] Anesthesiologists must be aware of guideline-based treatment for hereditary and acquired angioedema. […] C1 Inhibitor is the first-line long-term prophylaxis, while the androgens are used as second-line agents. […] Discontinuing trigger factors (estrogen-containing oral contraceptives, hormonal replacement therapy, angiotensin-converting enzyme inhibitors) […] Treating with plasma-derived C1 inhibitor for acute attacks. Preventing attacks with progestins, tranexamic acid, and danazol.

• #36 Angioedema: Causes, Symptoms, Types & Treatments

  https://my.clevelandclinic.org/health/diseases/22632-angioedema

  If you have allergy-related angioedema, you can prevent occurrences by avoiding the food, medication or other triggers that cause allergic reactions. […] If you have non-allergic angioedema as a drug reaction to taking ACEIs, youll need to work with your healthcare provider to find another medication.

• #37 Angioedema – Australasian Society of Clinical Immunology and Allergy (ASCIA)

  https://www.allergy.org.au/patients/skin-allergy/angioedema

  Angioedema is a condition where small blood vessels leak fluid into the tissues under the skin, causing swelling in different parts of the body. […] There is no known cure, but with the right diagnosis and management with appropriate medication, it can usually be prevented. […] Severe throat swelling requires the early use of medication (adrenaline for anaphylaxis or medication for HAE) followed by immediate transfer to hospital by ambulance. […] People with repeated angioedema should be referred by their doctor to a clinical immunology/allergy specialist for diagnosis and management. […] Avoid the triggers that make symptoms worse. It may be possible to manage angioedema by avoiding exposure to triggers that include: Excessive heat, eating spicy foods, and alcohol consumption. […] Antihistamines are commonly used to treat angioedema. […] The aim of treatment is to stop the episodes of angioedema from happening, make them less frequent or make the symptoms less severe. […] Other medications may be used if antihistamines are unsuccessful in managing angioedema.

• #38 Eating Safely: Avoiding Angioedema Triggers

  https://www.beckerentandallergy.com/blog/managing-foods-trigger-angioedema

  Angioedema is a condition characterized by the swelling of deep layers of the skin and mucous membranes. […] Managing angioedema requires caution when it comes to food choices, as certain foods can trigger episodes of swelling. […] To effectively manage risk for angioedema and minimize the risk of triggering episodes, it is important to be cautious about the foods you consume. Here are some guidelines to follow: […] Avoiding foods that may cause allergic reactions or intolerances. […] Be cautious of tree nuts such as almonds, walnuts, and cashews, as they can often trigger angioedema. […] Certain food additives like preservatives and artificial sweeteners may cause allergic reactions in some individuals. […] Some individuals with angioedema may experience swelling after consuming specific green salad ingredients, such as spinach or arugula.

• #39 Eating Safely: Avoiding Angioedema Triggers

  https://www.beckerentandallergy.com/blog/managing-foods-trigger-angioedema

  NSAIDs like aspirin or ibuprofen can cause severe symptoms in individuals prone to angioedema. […] Spices like chili peppers or hot sauces can trigger angioedema in certain individuals. […] Some individuals may experience angioedema episodes after consuming alcohol. […] Opt for fresh fruits and vegetables that are not known triggers for angioedema. […] Choose lean proteins like chicken, fish, and tofu as they are less likely to cause allergic reactions or trigger angioedema. […] Incorporate whole grains such as brown rice, quinoa, and oats into your diet for a nutritious and safe option. […] Ensure you stay hydrated by drinking an adequate amount of water throughout the day. […] A healthcare professional specializing in allergies and immune system disorders can provide personalized advice and guidance for managing angioedema.

• #40 Eating Safely: Avoiding Angioedema Triggers

  https://www.beckerentandallergy.com/blog/managing-foods-trigger-angioedema

  Maintain a record of your food intake and any symptoms experienced. This can help identify specific triggers and guide your conversations with healthcare professionals. […] By understanding the different types of angioedema, identifying trigger factors, and making informed food choices, individuals can minimize the risk of angioedema episodes.

• #41 Management and Prevention of Hereditary Angioedema Attacks

  https://www.ajmc.com/view/ace010_lumry2_s111to18

  The safety and efficacy of Cinryze for routine HAE prophylaxis was demonstrated in a randomized, double-blind, placebo-controlled crossover study in 24 patients with HAE who had a history of at least 2 angioedema attacks per month. […] Patients receiving androgens should be routinely monitored for hepatic abnormalities with liver function tests and abdominal ultrasound every 6 to 12 months. […] Antifibrinolytic agents, primarily tranexamic acid and aminocaproic acid, though generally considered less effective than androgens, can be used as an alternative for long-term HAE prophylaxis in certain patient groups for whom androgens are contraindicated (eg, pregnant or lactating women, children). […] Effective management of HAE requires a comprehensive approach to care and prevention. […] All patients with HAE should have an individualized treatment and action plan developed in close collaboration with their physician that address both acute (on demand) treatment needs and long-term prevention.

• #42 Management and Prevention of Hereditary Angioedema Attacks

  https://www.ajmc.com/view/ace010_lumry2_s111to18

  Home-based management of HAE prophylaxis and acute attacks is becoming an increasingly important component of the care of patients with HAE, and is now formally endorsed by international consensus documents. […] Access to self-administered or assisted infusion of C1INH reduces the frequency and severity of attacks, improves quality of life, reduces sick time, is well tolerated, and is popular with patients. […] Good health practices and preventive care should also be incorporated into every HAE management plan. […] Finally, family members of patients with HAE, including siblings, parents, grandparents, and grandchildren, should be screened for the condition.

• #43 US Hereditary Angioedema Association

  https://www.haea.org/pages/p/treatments

  With eight products currently approved by the FDA for preventing and treating Hereditary Angioedema (HAE) attacks, and several therapies approved for generic use, people with HAE and their physicians have options for developing an HAE treatment plan tailored to meet each person’s unique needs. […] Cinryze is indicated for routine prevention against HAE attacks in adults, adolescents and pediatric patients (6 years of age and older). […] Heagarda is delivered by subcutaneous injection and is approved for self-administration. […] Prophylactic treatment is an option for people who wish to try to reduce the frequency and/or severity of HAE attacks by taking medication on a regular schedule. This can be done on a long-term or short-term basis. Long-term prophylaxis is typically given regularly for weeks, months, or years in an effort to minimize the overall attack burden. Short-term prophylaxis is typically given just prior to a surgical or dental procedure to prevent an HAE attack during or following the procedure.

• #44 Current and Emerging Therapies to Prevent Hereditary Angioedema Attacks

  https://www.ajmc.com/view/current-and-emerging-therapies-to-prevent-hae-attacks

  Hereditary angioedema (HAE) is a rare genetic disease defined by recurrent attacks of edema, causing a substantial burden for patients, with morbidity, mortality, and reduced quality of life. Several novel therapeutics have recently been approved or are currently under evaluation for prevention of HAE attacks. Therapy for HAE can be generally divided into 2 approaches, acute (or on-demand) treatment of attacks and prevention of attacks, which is broken down into short-term prophylaxis (STP) and long-term prophylaxis (LTP). The 2017 revision and update to the international World Allergy Organization (WAO)/European Academy of Allergy and Clinical Immunology (EAACI) guideline for the management of HAE provides evidence-based recommendations for both of these approaches. C1-INH replacement can be used for either treatment or prevention of attacks. In 2017, the pdC1-INH agent (Haegarda) was approved by the FDA for prophylaxis of HAE in adolescent and adult patients. This is the second agent approved for HAE prophylaxis and the first C1-INH for SC administration. The WAO/EAACI guideline recommends that all patients undergoing any medical, surgical, or dental procedure involving any mechanical impact to the upper aerodigestive tract receive C1-INH as preprocedural prophylaxis as close as possible to the start of the procedure. LTP should be considered in patients when HAE is particularly burdensome. Successful treatment requires a high degree of compliance, so LTP therapy must be individualized based on disease activity, frequency of attacks, patients quality of life, availability of healthcare resources, failure to achieve adequate control through use of on-demand treatments, and patient preference. The WAO/EAACI guideline recommends C1-INH currently available in both IV and SC formulations as first-line therapy for LTP if available. A key factor that should be considered when prescribing therapy for HAE is route of administration. Whether acute or prophylactic treatment, route of administration can affect patient satisfaction with treatment, which ultimately impacts compliance. The clinical, economic, and quality-of-life burden to patients with HAE is substantial. Treatment plans must be individualized in partnership among the patient, family, and physician in order to achieve optimal outcomes. According to the Hereditary Angioedema Association Medical Advisory Board and WAO/EAACI guidelines, individualized treatment plans should address preventive measures, home care, and self-administration. An emergency plan with clear instructions and medications to use should be included for, if, and when an acute attack occurs.

• #45 Management and Prevention of Hereditary Angioedema Attacks

  https://www.ajmc.com/view/ace010_lumry2_s111to18

  Home-based management of HAE prophylaxis and acute attacks is becoming an increasingly important component of the care of patients with HAE, and is now formally endorsed by international consensus documents. […] Access to self-administered or assisted infusion of C1INH reduces the frequency and severity of attacks, improves quality of life, reduces sick time, is well tolerated, and is popular with patients. […] Good health practices and preventive care should also be incorporated into every HAE management plan. […] Finally, family members of patients with HAE, including siblings, parents, grandparents, and grandchildren, should be screened for the condition.

• #46 US Hereditary Angioedema Association

  https://www.haea.org/pages/p/treatments

  The availability of multiple on-demand and prophylaxis treatment options that can be administered at home has significantly increased the HAEA community’s quality of life while decreasing emergency room visits and hospitalizations. […] The US HAEA Medical Advisory Board recommends that all people with HAE due to C1-Inhibitor deficiency should have access to at least two standard doses of a medication proven effective in treating HAE attacks. […] Self and home administration can reduce the time to treatment and allows medication to be given earlier in an attack. Delays could allow an attack to become more severe and take longer to resolve once medication is finally received. Self and home treatment allows for greater independence and more reliable management of HAE symptoms. […] The US HAEA Medical Advisory Board suggests that everyone with HAE keep a record of their prophylaxis and/or on-demand treatments.

• #47 US Hereditary Angioedema Association

  https://www.haea.org/pages/p/treatments

  The availability of multiple on-demand and prophylaxis treatment options that can be administered at home has significantly increased the HAEA community’s quality of life while decreasing emergency room visits and hospitalizations. […] The US HAEA Medical Advisory Board recommends that all people with HAE due to C1-Inhibitor deficiency should have access to at least two standard doses of a medication proven effective in treating HAE attacks. […] Self and home administration can reduce the time to treatment and allows medication to be given earlier in an attack. Delays could allow an attack to become more severe and take longer to resolve once medication is finally received. Self and home treatment allows for greater independence and more reliable management of HAE symptoms. […] The US HAEA Medical Advisory Board suggests that everyone with HAE keep a record of their prophylaxis and/or on-demand treatments.

• #48 Management and Prevention of Hereditary Angioedema Attacks

  https://www.ajmc.com/view/ace010_lumry2_s111to18

  Home-based management of HAE prophylaxis and acute attacks is becoming an increasingly important component of the care of patients with HAE, and is now formally endorsed by international consensus documents. […] Access to self-administered or assisted infusion of C1INH reduces the frequency and severity of attacks, improves quality of life, reduces sick time, is well tolerated, and is popular with patients. […] Good health practices and preventive care should also be incorporated into every HAE management plan. […] Finally, family members of patients with HAE, including siblings, parents, grandparents, and grandchildren, should be screened for the condition.

• #49 Hereditary Angioedema – ICER

  https://icer.org/assessment/hereditary-angioedema-2018/

  Treatment for HAE attacks is very expensive, as are the prophylactic therapies used to prevent such attacks, and so the overall cost of prophylaxis includes the savings incurred from less on-demand treatment. […] The economic models produced for this report were very sensitive to small changes in assumptions about the frequency of attacks, the amount of on-demand treatment required, and the exact dosing regimens of prophylactic therapy. […] Similarly, relatively small changes in the price of prophylactic therapy greatly improved its cost-effectiveness. […] Insurers and manufacturers should work together to achieve appropriate access to these important therapies at a price that patients and society can afford. […] Cost-effectiveness and coverage considerations hinge on the frequency and intensity of attacks.

• #50 Hereditary Angioedema – ICER

  https://icer.org/assessment/hereditary-angioedema-2018/

  Treatment for HAE attacks is very expensive, as are the prophylactic therapies used to prevent such attacks, and so the overall cost of prophylaxis includes the savings incurred from less on-demand treatment. […] The economic models produced for this report were very sensitive to small changes in assumptions about the frequency of attacks, the amount of on-demand treatment required, and the exact dosing regimens of prophylactic therapy. […] Similarly, relatively small changes in the price of prophylactic therapy greatly improved its cost-effectiveness. […] Insurers and manufacturers should work together to achieve appropriate access to these important therapies at a price that patients and society can afford. […] Cost-effectiveness and coverage considerations hinge on the frequency and intensity of attacks.

• #51 Hereditary Angioedema – ICER

  https://icer.org/assessment/hereditary-angioedema-2018/

  Treatment for HAE attacks is very expensive, as are the prophylactic therapies used to prevent such attacks, and so the overall cost of prophylaxis includes the savings incurred from less on-demand treatment. […] The economic models produced for this report were very sensitive to small changes in assumptions about the frequency of attacks, the amount of on-demand treatment required, and the exact dosing regimens of prophylactic therapy. […] Similarly, relatively small changes in the price of prophylactic therapy greatly improved its cost-effectiveness. […] Insurers and manufacturers should work together to achieve appropriate access to these important therapies at a price that patients and society can afford. […] Cost-effectiveness and coverage considerations hinge on the frequency and intensity of attacks.

• #52 US Hereditary Angioedema Association

  https://www.haea.org/pages/p/treatments

  The availability of multiple on-demand and prophylaxis treatment options that can be administered at home has significantly increased the HAEA community’s quality of life while decreasing emergency room visits and hospitalizations. […] The US HAEA Medical Advisory Board recommends that all people with HAE due to C1-Inhibitor deficiency should have access to at least two standard doses of a medication proven effective in treating HAE attacks. […] Self and home administration can reduce the time to treatment and allows medication to be given earlier in an attack. Delays could allow an attack to become more severe and take longer to resolve once medication is finally received. Self and home treatment allows for greater independence and more reliable management of HAE symptoms. […] The US HAEA Medical Advisory Board suggests that everyone with HAE keep a record of their prophylaxis and/or on-demand treatments.

• #53 Establishing New Standards in Hereditary Angioedema: Improving Outcomes Through Routine Prophylaxis – European Medical Journal

  https://www.emjreviews.com/allergy-immunology/article/establishing-new-standards-in-hereditary-angioedema-improving-outcomes-through-routine-prophylaxis-j01121/

  Two of the recommended prophylactic medications are injectable. […] Berotralstat is the first oral prophylactic for HAE to be licenced and is a selective inhibitor of plasma kallikrein. […] Our real-world data from UK centres confirms that prophylaxis improves quality of life for most patients by reducing the number of HAE attacks and their severity, confirms Kiani, who emphasises the importance of ensuring management plans are discussed and agreed with patients to support their needs, preferences, and lifestyle. […] Shared decision-making between the patient and physician on appropriate prophylaxis is vital to ensure the best outcomes for individual patients with different preferences and quality of life goals, Kiani asserts. […] The availability of licenced long-term prophylactic treatments to prevent HAE attacks is changing the way HAE is treated, and is giving more patients hope for a future free from debilitating HAE attacks.

• #54 Establishing New Standards in Hereditary Angioedema: Improving Outcomes Through Routine Prophylaxis – European Medical Journal

  https://www.emjreviews.com/allergy-immunology/article/establishing-new-standards-in-hereditary-angioedema-improving-outcomes-through-routine-prophylaxis-j01121/

  Two of the recommended prophylactic medications are injectable. […] Berotralstat is the first oral prophylactic for HAE to be licenced and is a selective inhibitor of plasma kallikrein. […] Our real-world data from UK centres confirms that prophylaxis improves quality of life for most patients by reducing the number of HAE attacks and their severity, confirms Kiani, who emphasises the importance of ensuring management plans are discussed and agreed with patients to support their needs, preferences, and lifestyle. […] Shared decision-making between the patient and physician on appropriate prophylaxis is vital to ensure the best outcomes for individual patients with different preferences and quality of life goals, Kiani asserts. […] The availability of licenced long-term prophylactic treatments to prevent HAE attacks is changing the way HAE is treated, and is giving more patients hope for a future free from debilitating HAE attacks.

• #55 Establishing New Standards in Hereditary Angioedema: Improving Outcomes Through Routine Prophylaxis – European Medical Journal

  https://www.emjreviews.com/allergy-immunology/article/establishing-new-standards-in-hereditary-angioedema-improving-outcomes-through-routine-prophylaxis-j01121/

  Two of the recommended prophylactic medications are injectable. […] Berotralstat is the first oral prophylactic for HAE to be licenced and is a selective inhibitor of plasma kallikrein. […] Our real-world data from UK centres confirms that prophylaxis improves quality of life for most patients by reducing the number of HAE attacks and their severity, confirms Kiani, who emphasises the importance of ensuring management plans are discussed and agreed with patients to support their needs, preferences, and lifestyle. […] Shared decision-making between the patient and physician on appropriate prophylaxis is vital to ensure the best outcomes for individual patients with different preferences and quality of life goals, Kiani asserts. […] The availability of licenced long-term prophylactic treatments to prevent HAE attacks is changing the way HAE is treated, and is giving more patients hope for a future free from debilitating HAE attacks.

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