PATIENT-REPORTED ANXIETY IMPACTS UTILIZATION OF INJECTABLE ON-DEMAND TREATMENT OF HEREDITARY ANGIOEDEMA ATTACKS
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Hereditary Angioedema
Hereditary Angioedema
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Hereditary angioedema is an autosomal dominant disease characterized by recurrent angioedema attacks with the involvement of multiple organs.The disease is unknown to many health professionals and is therefore underdiagnosed.Patients who are not adequately diagnosed and treated have an estimated mortality rate ranging from 25% to 40% due to asphyxiation by laryngeal angioedema.Intestinal angioedema is another important and incapacitating presentation that may be the main or only manifestation during an attack.In this article, a group of experts from the ''Associac¸a ˜o Brasileira de Alergia e Imunologia (ASBAI)'' and the ''Grupo de Estudos Brasileiro em Angioedema Heredita ´rio (GEBRAEH)'' has updated the Brazilian guidelines for the diagnosis and treatment of hereditary angioedema.
Hereditary Angioedema
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Hereditary Angioedema
Omalizumab
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Past medical history
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Hereditary Angioedema
Prophylactic treatment
C1-inhibitor
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Complement component analysis is valuable for differentiating the various types of angioedema. Patients with hereditary angioedema have decreased levels of C1 esterase inhibitor and C4 in the presence of normal amounts of C3 and C1q. Acquired C1 esterase inhibitor deficiency secondary to malignant disease is also manifested by depressed C1 esterase inhibitor and C4, but decreased C1q levels distinguish it from hereditary angioedema. Normal values for these complement components are found in persons with allergic angioedema.
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Complement component 5
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BACKGROUND: Hereditary angioedema with C1 inhibitor deficiency is a rare disease caused by a deficiency and/or a decrease in the functional activity of the C1 inhibitor. The primary symptom of this condition is recurrent angioedema of various localizations. According to the modern concept of treatment, the therapy aims to stop emerging angioedema and prevent death as well as achieve complete control of the disease and a high quality of life. Lanadelumab is a modern medicine developed and used to prevent attacks in patients with hereditary angioedema aged 12 years.
AIM: A retrospective study (IISR-2021-200085) was conducted to evaluate the efficiency and safety of lanadelumab in real-life practice in Russia.
MATERIALS AND METHODS: In all, 16 patients with hereditary angioedema and C1 inhibitor deficiency were enrolled at the initiation of lanadelumab treatment. The patients were predominantly female (81%; 13/16). The average age of patients was 29.9 years; 19% (3/16) of the patients were adolescents. The effectiveness was evaluated by comparing the patient-reported attack rates. The following PROs for the adults only were assessed initially and during the treatment: angioedema activity score, angioedema control test (AECT), angioedema quality of life questionnaire (AE-QoL), and hereditary angioedema activity score. The incidences of adverse events were evaluated.
RESULTS: Before lanadelumab, 69% (11/16) of the patients received alternative long-term prophylaxis, which was canceled after the start of lanadelumab treatment. The average number of attacks per month and treated attacks per month prelanadelumab were 10 and 4.7 per patient, respectively. After 6 months of treatment, these values were 0.26 and 0.09, respectively (10 patients were symptom free at 6 months after the initiation of the treatment). After 3 months of treatment, the mean AECT values improved from 5.6 to 14.2 (p 0.001), and all patients showed adequate disease control. After 6 months of treatment, AE-QoL decreased from 58 to 19 (p 0.001). No serious adverse events related to lanadelumab were observed.
CONCLUSION: Our study demonstrated that the composite effect of lanadelumab minimizes the attack rate and improves the quality of life in patients with hereditary angioedema. A good safety profile of lanadelumab is shown.
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BL Zuraw, PJ Busse, M White. N Engl J Med. 2010;363(6):513–522
To determine the efficacy of nanofiltered C1 inhibitor concentrate in the management of hereditary angioedema (HAE).
Subjects were from 2 studies; the lowest age was 6 years (median age: 36 years).
Both studies compared an intravenous dose of 1000 U of nanofiltered C1 inhibitor concentrate in the management of HAE. The first study end point was the time to unequivocal relief of symptoms during an acute attack of HAE. The second study was …
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Isolated angioedema, without urticaria or itching, occurs as a result of an inherited or acquired defect in C1 esterase inhibitor activity. Most cases of isolated angioedema are caused by one of two types of hereditary angioedema (HAE). We present a case of the much rarer type II HAE with abdominal pain as the sole presenting symptom. Hereditary angioedema should be suspected in young adults with episodic abdominal pain for which common causes have been excluded. A history of HAE or episodic abdominal pain in family members is not necessary for diagnosis.
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Angioedema without wheals (urticaria) represents a heterogeneous group of clinically indistinguishable diseases of hereditary or acquired etiology. Hereditary angioedema is a rare inherited condition leading to recurrent, sometimes life-threatening angioedema attacks in subcutaneous tissues and gastrointestinal and oropharyngeal mucosa dating back to childhood or adolescence. Most of these patients have mutations in the SERPING1 gene, causing either low C1 inhibitor production (hereditary angioedema with C1 inhibitor deficiency type I) or the production of dysfunctional C1 inhibitor (hereditary angioedema with C1 inhibitor deficiency type II). Hereditary angioedema with normal C1 inhibitor has been defined later. Although C1 inhibitor concentration and function are in the normal range, it leads to typical hereditary angioedema symptoms owing to mutations in FXII, PLG, ANGPT1, KNG1, and MYOF genes. Patients who exhibit none of these genetic mutations despite having a similar clinical presentation are classified as having unknown hereditary angioedema. Fewer than 1 in 10 patients with C1 inhibitor deficiency have acquired angioedema with C1 inhibitor deficiency. The clinical presentation is very similar to that of hereditary angioedema, making it difficult to distinguish these 2 conditions clinically. Unlike hereditary angioedema, there are no genetic mutations, and family history and symptoms tend to appear later in life. Acquired angioedema with C1 inhibitor deficiency is commonly associated with lymphoproliferative and autoimmune diseases. Angioedema attacks might start 1 year before the underlying disease in acquired angioedema with C1 inhibitor deficiency. Approximately half of the patients admitted to the hospital for acute angioedema are patients receiving angiotensin-converting enzyme (ACE) inhibitor therapy. Angioedema typically occurs on the lips, tongue, mouth, pharynx, and subglottic regions. Patients may require hospitalization and intensive care monitoring owing to airway involvement. Idiopathic histaminergic acquired angioedema may be diagnosed only when any possible causes of histaminergic angioedema are excluded (foods, drugs, animal dander, aeroallergens, insect stings, latex, and others), and the symptoms respond well to antihistamine treatment. Idiopathic nonhistaminergic acquired angioedema should be considered when all other types of recurrent angioedema have been ruled out and patients do not respond to high-dose antihistamines. The lack of a standard biochemical laboratory test for patients with idiopathic histaminergic acquired angioedema, idiopathic nonhistaminergic acquired angioedema, angiotensin-converting enzyme inhibitor-induced acquired angioedema, and hereditary angioedema with normal C1 inhibitor makes the diagnosis more challenging. Future efforts should focus on increasing awareness of all the rare types of angioedema among physicians and developing more straightforward and more accessible diagnostic methods.
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要旨 症例は67歳の女性。64歳の時に遺伝性血管性浮腫（hereditary angioedema：HAE）と診断され，数日で軽快する顔面の発作性浮腫を数か月に1回の頻度で繰り返していた。受診の9時間前より誘因なく顔面の浮腫を認め，呼吸困難，悪心，嘔吐を伴ったため救急搬送された。来院時，SpO 2 100%（酸素マスク8L/分）で頻呼吸であり顔面に著明な浮腫を認めた。気管支鏡にて強い喉頭浮腫を認め，気道確保のため気管挿管を行った。血液検査では血清C4値とC1–インヒビター（C1–INH）活性値は高度に低下しており，嘔吐の原因精査のため施行した腹部CTでは十二指腸の著明な浮腫性肥厚を認めた。HAEの発作と診断し，C1–INH製剤1,500単位を静脈内投与した。第2病日には悪心は消失して十二指腸の浮腫も軽減していたが喉頭浮腫は増悪し，第3病日にも残存したためC1–INH製剤1,500単位を追加投与した。第4病日には喉頭浮腫も軽快したため抜管した。喉頭と腸管に同時に浮腫を来したHAEの報告は過去になく稀な症例であったが，C1–INHの局所的枯渇がHAEにおける発作性浮腫の原因であり，複数箇所に同時に浮腫を来すこともある。発作性浮腫は気道緊急，急性腹症，循環虚脱などに至ることもあるため，発作時には他の部位に合併する浮腫の有無を検索する必要がある。
Hereditary Angioedema
Laryngeal Edema
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