Treatment of hereditary angioedema: When the goal is having a normal life

Arruda, L. Karla; Ferriani, Mariana Paes Leme

doi:10.1016/j.jaci.2021.05.023

Cited by 5 publications

(14 citation statements)

References 10 publications

(26 reference statements)

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“… 1 Treatments for acute attacks must be given intravenously or subcutaneously, and available options include ecallantide (kallikrein inhibitor), intravenous C1 inhibitor protein, and icatibant (bradykinin B2 receptor antagonist). 1 , 4 Icatibant and intravenous C1 inhibitor protein can be self-administered; only treatment with ecallantide requires administration by a health-care professional. Some of these have undesirable side effects, including hypersensitivity reactions, anaphylaxis, and/or a burning sensation at the injection site.…”

Section: Mainstays Of Treatmentsmentioning

confidence: 99%

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

Diaz-Menindez¹,

Morgenstern-Kaplan²,

Cuervo‐Pardo³

et al. 2023

TCRM

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Hereditary angioedema (HAE) is a condition characterized by episodes of cutaneous and submucosal edema. Angioedema of the extremities and abdominal attacks are the most common manifestations of the disease. It can also affect the upper airways with the potential of becoming life-threatening. The two most common causes of HAE are a deficiency of C1 inhibitor (classified as type 1 HAE) or a dysfunction of C1 inhibitor (type 2 HAE). A malfunction or deficiency of C1 inhibitor leads to an overactivated plasma kallikrein (an inflammatory vasoactive peptide), that increases bradykinin, mediating the angioedema episodes in patients with HAE. 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. Open-label studies have demonstrated the effectiveness of a single daily dose of berotralstat 150 mg in preventing HAE attacks. This review aims to examine studies performed to elucidate the efficacy, safety, and tolerability of berotralstat.

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Section: Mainstays Of Treatmentsmentioning

confidence: 99%

“…Some of these have undesirable side effects, including hypersensitivity reactions, anaphylaxis, and/or a burning sensation at the injection site. 2 , 4 …”

Section: Mainstays Of Treatmentsmentioning

confidence: 99%

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

Diaz-Menindez¹,

Morgenstern-Kaplan²,

Cuervo‐Pardo³

et al. 2023

TCRM

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“…Despite advances in treatment, the burden of HAE remains heavy for patients. Indeed, there is a significant humanistic burden associated with HAE, particularly due to disease heterogeneity and the unpredictability of disabling HAE attacks, making them difficult to characterize and effectively manage [ 55 , 56 ]. Attacks can occur in the absence of an identifiable event.…”

Section: Quality Of Life (Qol) In Patients With Haementioning

confidence: 99%

“…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 [ 59 ]. The availability and cost of new treatments for HAE are important issues in China and Brazil [ 9 , 55 ]. In the last decade, several new drugs and new indications for old drugs have played a role in the management of HAE.…”

Section: Safe and Effective Long-term Prophylactic Treatment Alternat...mentioning

confidence: 99%

Long-term Prophylaxis with Androgens in the management of Hereditary Angioedema (HAE) in emerging countries

Guo

Zhang

et al. 2022

Orphanet J Rare Dis

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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. C1-inhibitor (C1-INH) deficiency, the main mechanism of HAE pathogenesis, occurs when abnormal activation of plasma kallikrein, bradykinin, and factor XII, or mutation of genes such as SERPING1 cause quantitative or functional C1-INH defects. 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 long-term adverse effects of androgen treatment are concerning for both physicians and patients. Virilization, weight gain, acne, hirsutism, liver damage, headache, myalgia, hematuria, menstrual disorders, diminished libido, arterial hypertension, dyslipidemia, and anxiety/depression are commonly observed during long-term treatment with androgens. These adverse effects can affect the quality of life of HAE patients and often lead to treatment interruption, especially in women and children. In-depth studies of the pathogenesis of HAE have led to the approval of alternative treatment strategies, including plasma-derived C1 inhibitor, recombinant human C1 inhibitor, plasma Kallikrein inhibitor (ecallantide; lanadelumab), and bradykinin B2 receptor antagonist (icatibant), some of which have achieved satisfactory results with mostly non-serious side effects. Therefore, a new standard of medical care may expand possibilities for the management of HAE in emerging countries.

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“…O Angioedema Hereditário (AEH) é um edema não inflamatório, assimétrico e de origem autossômica dominante, atingindo um em cada 50.000 indivíduos. Caracterizada por ser autolimitada, esta doença restringe-se ao tecido subcutâneo ou submucoso e ocorre devido ao aumento da permeabilidade vascular e da vasodilatação, causando o inchaço da região atingida (Giavina-Bianchi et al, 2018;Serpa et al, 2021;Arruda & Leme Ferriani, 2021). Os episódios de AEH são recorrentes, devido a mutação no gene SERPING1 que decorre em função da deficiência do inibidor de C1 (C1-INH), induzindo a produção, em excesso, do mediador vasoativo bradicinina, de modo a afetar diversos órgãos, sendo que um terço das crises se localizam na região abdominal e outro terço na região dos membros (Busse & Christiansen, 2020;.…”

Section: Introductionunclassified

Perfil clínico de pacientes com Angioedema Hereditário atendidos em um hospital escola da Cidade de Maceió, Alagoas

Tenório

Silva

Távora

et al. 2021

RSD

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O Angioedema Hereditário (AEH) é uma doença genética rara caracterizada por ataques recorrentes de angioedema cutâneo, dor abdominal intensa e comprometimento das vias aéreas. Este estudo teve por objetivo descrever o perfil clínico de pacientes portadores de AEH assistidos no Ambulatório de Imunologia Clínica e Alergia de um hospital escola da cidade de Maceió, Alagoas, Brasil. Trata-se de um estudoretrospectivo, transversal e descritivo, onde foram coletados dados como sexo, idade de início do AEH, manifestações, fatores desencadeantes, terapia profilática e terapia em momento de crise.Treze participantes compuseram a amostra total, sendo oito do sexo masculino e idade variando entre 08 e 40 anos. O principal fator desencadeante foi o trauma, sendo o edema nas extremidades o sinal mais frequente. O ácido tranexâmico foi a droga referenciada como agente terapêutico em momentos de crise. O fator histórico familiar esteve presente em 69,2% dos participantes e, mesmo o ácido tranexâmico não estando recomendado para profilaxia a longo prazo para o AEH, ainda assume referência entre os pacientes deste estudo, revelando a necessidade da disseminação do conhecimento acerca dos processos fisiopatológicos da doença direcionada às equipes de saúde, no intuito de orientar uma terapêutica adequada e, assim, diminuir a morbimortalidade assegurando uma maior qualidade de vida para os pacientes, como também para os seus familiares.

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Treatment of hereditary angioedema: When the goal is having a normal life

Cited by 5 publications

References 10 publications

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

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

Long-term Prophylaxis with Androgens in the management of Hereditary Angioedema (HAE) in emerging countries

Perfil clínico de pacientes com Angioedema Hereditário atendidos em um hospital escola da Cidade de Maceió, Alagoas

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