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Angioedema hereditário
ORPHA:91378CID-10 · D84.1CID-11 · 4A00.14PCDT · SUSDOENÇA RARA

Angioedema Hereditário (AEH) é uma doença genética caracterizada pelo aparecimento de inchaços (edemas) que são temporários e voltam a acontecer, atingindo a pele (por baixo dela) e/ou as mucosas (membranas que revestem partes do corpo, como o intestino ou a boca). Esses inchaços resultam em partes do corpo inchadas e/ou dor de barriga.

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Introdução

O que você precisa saber de cara

📋

Angioedema Hereditário (AEH) é uma doença genética caracterizada pelo aparecimento de inchaços (edemas) que são temporários e voltam a acontecer, atingindo a pele (por baixo dela) e/ou as mucosas (membranas que revestem partes do corpo, como o intestino ou a boca). Esses inchaços resultam em partes do corpo inchadas e/ou dor de barriga.

Pesquisas ativas
30 ensaios
173 total registrados no ClinicalTrials.gov
Publicações científicas
3.099 artigos
Último publicado: 2026 Apr 7
Medicamentos
8 registrados
ECALLANTIDE, ICATIBANT ACETATE, HUMAN C1-ESTERASE INHIBITOR

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8 medicamentos registrados
Ver detalhes, fases e interações →
ECALLANTIDEICATIBANT ACETATEHUMAN C1-ESTERASE INHIBITORCONESTAT ALFALANADELUMABAVORALSTATTRANEXAMIC ACIDICATIBANT

Escala de raridade

CLASSIFICAÇÃO ORPHANET · BRASIL 2024
1-9 / 100 000
Ultra-rara
<1/50k
Muito rara
1/20k
Rara
1/10k
Pouco freq.
1/5k
Incomum
1/2k
Prevalência
5.0
Europe
Início
All ages
🏥
SUS: Cobertura parcialScore: 50%
PCDT disponível3 medicamentos CEAFCID-10: D84.1
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Sinais e sintomas

O que aparece no corpo e com que frequência cada sintoma acontece

Partes do corpo afetadas

🫃
Digestivo
9 sintomas
🧬
Pele e cabelo
5 sintomas
🫁
Pulmão
5 sintomas
🦴
Ossos e articulações
3 sintomas
😀
Face
3 sintomas
💪
Músculos
2 sintomas

+ 29 sintomas em outras categorias

Características mais comuns

Morfologia anormal da epiglote
Eritema
Edema do dorso das mãos
Diarreia
Inchaço articular
Edema facial
59sintomas
Sem dados (59)

Os sintomas variam de pessoa para pessoa. Abaixo estão as 59 características clínicas mais associadas, ordenadas por frequência.

Morfologia anormal da epigloteAbnormal epiglottis morphology
EritemaErythema
Edema do dorso das mãosEdema of the dorsum of hands
DiarreiaDiarrhea
Inchaço articularJoint swelling

Linha do tempo da pesquisa

Publicações por ano — veja quando o interesse científico cresceu
Anos de pesquisa1desde 2026
Total histórico3.099PubMed
Últimos 10 anos200publicações
Pico2025110 papers
Linha do tempo
2026Hoje · 2026🧪 1994Primeiro ensaio clínico📈 2025Ano de pico
Publicações por ano (últimos 10 anos)

Encontrou um erro ou informação desatualizada? Sugira uma correção →

Genética e causas

O que está alterado no DNA e como passa nas famílias

Genes associados

7 genes identificados com associação a esta condição. Padrão de herança: Autosomal dominant.

F12Coagulation factor XIIDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Factor XII is a serum glycoprotein that participates in the initiation of blood coagulation, fibrinolysis, and the generation of bradykinin and angiotensin. Prekallikrein is cleaved by factor XII to form kallikrein, which then cleaves factor XII first to alpha-factor XIIa and then trypsin cleaves it to beta-factor XIIa. Alpha-factor XIIa activates factor XI to factor XIa (PubMed:2019570, PubMed:21304106, PubMed:8427954)

LOCALIZAÇÃO

Secreted

VIAS BIOLÓGICAS (1)
Defective factor XII causes hereditary angioedema
MECANISMO DE DOENÇA

Factor XII deficiency

An asymptomatic anomaly of in vitro blood coagulation. Its diagnosis is based on finding a low plasma activity of the factor in coagulating assays. It is usually only accidentally discovered through pre-operative blood tests. Factor XII deficiency is divided into two categories, a cross-reacting material (CRM)-negative group (negative F12 antigen detection) and a CRM-positive group (positive F12 antigen detection).

EXPRESSÃO TECIDUAL(Tecido-específico)
Fígado
376.1 TPM
Esôfago - Mucosa
10.0 TPM
Brain Nucleus accumbens basal ganglia
6.6 TPM
Skin Sun Exposed Lower leg
5.4 TPM
Vagina
5.3 TPM
OUTRAS DOENÇAS (3)
congenital factor XII deficiencyhereditary angioedema type 3F12-associated cold autoinflammatory syndrome
HGNC:3530UniProt:P00748
PLGPlasminogenDisease-causing germline mutation(s) inRestrito
FUNÇÃO

Plasmin dissolves the fibrin of blood clots and acts as a proteolytic factor in a variety of other processes including embryonic development, tissue remodeling, tumor invasion, and inflammation. In ovulation, weakens the walls of the Graafian follicle. It activates the urokinase-type plasminogen activator, collagenases and several complement zymogens, such as C1, C4 and C5 (PubMed:6447255). Cleavage of fibronectin and laminin leads to cell detachment and apoptosis. Also cleaves fibrin, thrombosp

LOCALIZAÇÃO

Secreted

VIAS BIOLÓGICAS (1)
Platelet degranulation
MECANISMO DE DOENÇA

Plasminogen deficiency

A disorder characterized by decreased serum plasminogen activity. Two forms of the disorder are distinguished: type 1 deficiency is additionally characterized by decreased plasminogen antigen levels and clinical symptoms, whereas type 2 deficiency, also known as dysplasminogenemia, is characterized by normal, or slightly reduced antigen levels, and absence of clinical manifestations. Plasminogen deficiency type 1 results in markedly impaired extracellular fibrinolysis and chronic mucosal pseudomembranous lesions due to subepithelial fibrin deposition and inflammation. The most common clinical manifestation of type 1 deficiency is ligneous conjunctivitis in which pseudomembranes formation on the palpebral surfaces of the eye progresses to white, yellow-white, or red thick masses with a wood-like consistency that replace the normal mucosa.

EXPRESSÃO TECIDUAL(Tecido-específico)
Fígado
323.0 TPM
Rim - Córtex
19.1 TPM
Rim - Medula
3.6 TPM
Testículo
0.2 TPM
Cervix Endocervix
0.1 TPM
OUTRAS DOENÇAS (3)
hypoplasminogenemiaangioedema, hereditary, 4PLG-related hereditary angioedema with normal C1inh
HGNC:9071UniProt:P00747
SERPING1Plasma protease C1 inhibitorDisease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Serine protease inhibitor, which acrs as a regulator of the classical complement pathway (PubMed:10946292, PubMed:11527969, PubMed:3458172, PubMed:6416294). Forms a proteolytically inactive stoichiometric complex with the C1r or C1s proteases (PubMed:10946292, PubMed:3458172, PubMed:6416294). May also regulate blood coagulation, fibrinolysis and the generation of kinins (PubMed:8495195). Very efficient inhibitor of FXIIa. Inhibits chymotrypsin and kallikrein (PubMed:8495195)

LOCALIZAÇÃO

Secreted

VIAS BIOLÓGICAS (3)
Platelet degranulation Intrinsic Pathway of Fibrin Clot FormationRegulation of Complement cascade
MECANISMO DE DOENÇA

Angioedema, hereditary, 1

An autosomal dominant disorder characterized by episodic local swelling involving subcutaneous or submucous tissue of the upper respiratory and gastrointestinal tracts, face, extremities, and genitalia. Hereditary angioedema due to C1 esterase inhibitor deficiency is comprised of two clinically indistinguishable forms. In hereditary angioedema type 1, serum levels of C1 esterase inhibitor are decreased, while in type 2, the levels are normal or elevated, but the protein is non-functional.

EXPRESSÃO TECIDUAL(Ubíquo)
Artéria tibial
1258.4 TPM
Aorta
1246.5 TPM
Ovário
1138.5 TPM
Fígado
1081.6 TPM
Artéria coronária
1061.1 TPM
OUTRAS DOENÇAS (4)
hereditary angioedema with C1Inh deficiencyC1 inhibitor deficiencyhereditary angioedema type 1hereditary angioedema type 2
HGNC:1228UniProt:P05155
KNG1Kininogen-1Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Kininogens are inhibitors of thiol proteases. HMW-kininogen plays an important role in blood coagulation by helping to position optimally prekallikrein and factor XI next to factor XII; HMW-kininogen inhibits the thrombin- and plasmin-induced aggregation of thrombocytes. LMW-kininogen inhibits the aggregation of thrombocytes. LMW-kininogen is in contrast to HMW-kininogen not involved in blood clotting The active peptide bradykinin is a potent vasodilatator that is released from HMW-kininogen sho

LOCALIZAÇÃO

Secreted, extracellular space

VIAS BIOLÓGICAS (2)
Post-translational protein phosphorylationRegulation of Insulin-like Growth Factor (IGF) transport and uptake by Insulin-like Growth Factor Binding Proteins (IGFBPs)
MECANISMO DE DOENÇA

High molecular weight kininogen deficiency

Autosomal recessive coagulation defect. Patients with HWMK deficiency do not have a hemorrhagic tendency, but they exhibit abnormal surface-mediated activation of fibrinolysis.

EXPRESSÃO TECIDUAL(Tecido-específico)
Fígado
414.4 TPM
Rim - Medula
180.7 TPM
Rim - Córtex
60.9 TPM
Brain Frontal Cortex BA9
0.3 TPM
Testículo
0.3 TPM
OUTRAS DOENÇAS (3)
congenital high-molecular-weight kininogen deficiencyangioedema, hereditary, 6hereditary angioedema with normal C1inh not related to F12 or PLG variant
HGNC:6383UniProt:P01042
MYOFMyoferlinDisease-causing germline mutation(s) inTolerante
FUNÇÃO

Calcium/phospholipid-binding protein that plays a role in the plasmalemma repair mechanism of endothelial cells that permits rapid resealing of membranes disrupted by mechanical stress. Involved in endocytic recycling. Implicated in VEGF signal transduction by regulating the levels of the receptor KDR (By similarity)

LOCALIZAÇÃO

Cell membraneNucleus membraneCytoplasmic vesicle membraneLate endosome membrane

MECANISMO DE DOENÇA

Angioedema, hereditary, 7

A form of angioedema, a disorder characterized by episodic local swelling involving subcutaneous or submucous tissue of the upper respiratory and gastrointestinal tracts, face, extremities, and genitalia. HAE7 is an autosomal dominant form characterized by onset of recurrent swelling of the face, lips, and oral mucosa in the second decade.

EXPRESSÃO TECIDUAL(Ubíquo)
Fibroblastos
232.1 TPM
Artéria tibial
85.2 TPM
Vagina
85.0 TPM
Bladder
82.8 TPM
Aorta
82.6 TPM
INTERAÇÕES PROTEICAS (4)
OUTRAS DOENÇAS (2)
angioedema, hereditary, 7hereditary angioedema with normal C1inh not related to F12 or PLG variant
HGNC:3656UniProt:Q9NZM1
ANGPT1Angiopoietin-1Disease-causing germline mutation(s) inAltamente restrito
FUNÇÃO

Binds and activates TEK/TIE2 receptor by inducing its dimerization and tyrosine phosphorylation. Plays an important role in the regulation of angiogenesis, endothelial cell survival, proliferation, migration, adhesion and cell spreading, reorganization of the actin cytoskeleton, but also maintenance of vascular quiescence. Required for normal angiogenesis and heart development during embryogenesis. After birth, activates or inhibits angiogenesis, depending on the context. Inhibits angiogenesis a

LOCALIZAÇÃO

Secreted

VIAS BIOLÓGICAS (2)
RAF/MAP kinase cascadeTie2 Signaling
MECANISMO DE DOENÇA

Angioedema, hereditary, 5

A form of angioedema, a disorder characterized by episodic local swelling involving subcutaneous or submucous tissue of the upper respiratory and gastrointestinal tracts, face, extremities, and genitalia. HAE5 is an autosomal dominant form characterized by onset of episodic swelling of the face, lips, hands, and abdomen in the second decade of life.

OUTRAS DOENÇAS (2)
angioedema, hereditary, 5hereditary angioedema with normal C1inh not related to F12 or PLG variant
HGNC:484UniProt:Q15389
HS3ST6Heparan sulfate glucosamine 3-O-sulfotransferase 6Disease-causing germline mutation(s) inTolerante
FUNÇÃO

Sulfotransferase that utilizes 3'-phospho-5'-adenylyl sulfate (PAPS) to catalyze the transfer of a sulfo group to heparan sulfate. The substrate-specific O-sulfation generates an enzyme-modified heparan sulfate which acts as a binding receptor to Herpes Simplex Virus-1 (HSV-1) and permits its entry. Unlike 3-OST-1, does not convert non-anticoagulant heparan sulfate to anticoagulant heparan sulfate

LOCALIZAÇÃO

Golgi apparatus membrane

VIAS BIOLÓGICAS (1)
HS-GAG biosynthesis
MECANISMO DE DOENÇA

Angioedema, hereditary, 8

A form of angioedema, a disorder characterized by episodic local swelling involving subcutaneous or submucous tissue of the upper respiratory and gastrointestinal tracts, face, extremities, and genitalia. HAE8 inheritance is autosomal dominant.

VIAS REACTOME (1)
EXPRESSÃO TECIDUAL(Tecido-específico)
Skin Sun Exposed Lower leg
117.9 TPM
Skin Not Sun Exposed Suprapubic
107.0 TPM
Vagina
14.8 TPM
Esôfago - Mucosa
13.7 TPM
Bladder
4.1 TPM
INTERAÇÕES PROTEICAS (1)
OUTRAS DOENÇAS (2)
angioedema, hereditary, 8hereditary angioedema with normal C1inh not related to F12 or PLG variant
HGNC:14178UniProt:Q96QI5

Medicamentos e terapias

ECALLANTIDEPhase 4

Mecanismo: Plasma kallikrein inhibitor

ICATIBANT ACETATEPhase 4

Mecanismo: Bradykinin B2 receptor antagonist

HUMAN C1-ESTERASE INHIBITORPhase 4

Mecanismo: Coagulation factor XI inhibitor

CONESTAT ALFAPhase 4

Mecanismo: Plasma protease C1 inhibitor exogenous protein

LANADELUMABPhase 4

Mecanismo: Plasma kallikrein inhibitor

AVORALSTATPhase 3

Mecanismo: Plasma kallikrein inhibitor

TRANEXAMIC ACIDPhase 3

Mecanismo: Plasminogen inhibitor

ICATIBANTPhase 3

Mecanismo: Bradykinin B2 receptor antagonist

Ver mais no OpenTargets

Variantes genéticas (ClinVar)

594 variantes patogênicas registradas no ClinVar.

🧬 F12: NM_000505.4(F12):c.1532-1G>A ()
🧬 F12: GRCh38/hg38 5q35.2-35.3(chr5:176144576-178014188)x1 ()
🧬 F12: GRCh37/hg19 5q35.2-35.3(chr5:176097556-180719789)x1 ()
🧬 F12: NM_000505.4(F12):c.849C>T (p.Asp283=) ()
🧬 F12: NM_000505.4(F12):c.312C>A (p.Cys104Ter) ()
Ver todas no ClinVar

Classificação de variantes (ClinVar)

Distribuição de 437 variantes classificadas pelo ClinVar.

350
87
Patogênica (80.1%)
VUS (19.9%)
VARIANTES MAIS SIGNIFICATIVAS
SERPING1: NM_000062.3(SERPING1):c.881dup (p.Tyr294Ter) [Pathogenic]
MAK: NC_000006.11:g.10791926_10791927insSVA [Pathogenic]
SERPING1: NM_000062.3(SERPING1):c.551-2A>G [Pathogenic]
SERPING1: NM_000062.3(SERPING1):c.964del (p.Val322fs) [Pathogenic]
SERPING1: NM_000062.3(SERPING1):c.329_341del (p.Pro110fs) [Pathogenic]

Diagnóstico

Os sinais que médicos procuram e os exames que confirmam

Carregando...

Tratamento e manejo

Remédios, cuidados de apoio e o que precisa acompanhar

Pipeline de tratamentos
Pipeline regulatório — de medicamentos já aprovados a drogas em pesquisa exploratória.
Aprovado6
3Fase 313
2Fase 23
·Pré-clínico6
Medicamentos catalogadosEnsaios clínicos· 8 medicamentos · 20 ensaios
✓ Aprovados — podem ser usados hoje
ECALLANTIDEICATIBANT ACETATEHUMAN C1-ESTERASE INHIBITORCONESTAT ALFALANADELUMAB
Carregando informações de tratamento...

Onde tratar no SUS

Hospitais de referência no Brasil e o protocolo oficial do SUS (PCDT)

🇧🇷 Atendimento SUS — Angioedema hereditário

🗺️

Selecione um estado ou use sua localização para ver resultados.

Dados de DATASUS/CNES, SBGM, ABNeuro e Ministério da Saúde. Sempre confirme a disponibilidade diretamente com o estabelecimento.

Pesquisa ativa

Ensaios clínicos abertos e novidades científicas recentes

🟢 Recrutando agora

13 pesquisas recrutando participantes. Converse com seu médico sobre a possibilidade de participar.

Outros ensaios clínicos

173 ensaios clínicos encontrados, 30 ativos.

Distribuição por fase
Ver todos no ClinicalTrials.gov
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Publicações mais relevantes

Timeline de publicações
1.805 papers (10 anos)

Mostrando amostra de 200 publicações de um total de 1.805

#1

Oral deucrictibant for on-demand treatment of hereditary angioedema attacks (RAPIDe-1): a randomised, double-blind, placebo-controlled, phase 2 trial.

The Lancet. Haematology2026 Mar 19

Hereditary angioedema is a bradykinin-mediated disorder characterised by recurrent painful swelling attacks. Treatment relies on medications to prevent attacks and on-demand therapies for attack manifestations. Parenteral administration associated with most available on-demand therapies often leads to treatment being delayed or forgone. Deucrictibant is an orally bioavailable bradykinin B2 receptor antagonist under development for prophylaxis and on-demand treatment of hereditary angioedema attacks. We aimed to investigate the efficacy and safety of deucrictibant for the on-demand treatment of hereditary angioedema attacks. RAPIDe-1 was a double-blind, randomised, placebo-controlled, crossover, dose-ranging, phase 2 trial that recruited adults aged 18-75 years with hereditary angioedema type 1 or 2 from 38 sites (eg, university hospitals and accredited angioedema centres) across North America, Europe, and Israel. Participants were required to have experienced two or more attacks within the past 2 months or three or more attacks within the past 4 months before screening. An interactive response technology system randomly assigned eligible patients (1:1:1) to receive a blinded dose of oral deucrictibant (immediate-release capsule) 10 mg, 20 mg, or 30 mg during an attack-free period to assess pharmacokinetics and safety in the part 1 of the study; and, subsequently, to receive a crossover treatment sequence of two administrations of the same deucrictibant dose (10 mg, 20 mg, or 30 mg) and one of placebo to treat three investigator-confirmed angioedema attacks in the part 2. Randomisation was stratified by whether the participant was willing to participate in full pharmacokinetic sampling. Participants, investigators, site personnel, and the sponsor were masked to treatment assignment and capsules of deucrictibant and placebo were identical. For each attack, patients self-administered the oral study drug within 3 h after at least one symptom (skin pain, skin swelling, or abdominal pain) reached a visual analog scale (VAS) individual score of 30 or more out of 100 and within 6 h from symptom onset. The primary endpoint was change in the patient-reported composite VAS-3 score measuring severity of attack manifestations from before treatment to 4 h post-treatment and assessed in the modified intention-to-treat and per-protocol populations. Safety was assessed in all patients who received any dose of study drug. RAPIDe-1 is registered with ClinicalTrials.gov (NCT04618211) and is completed. Between Feb 3, 2021, and June 23, 2022, 89 patients were screened, of whom 74 were randomly assigned between Feb 23, 2021, and July 26, 2022. For the primary analysis set, the median follow-up was 130·0 (IQR 92·5 to 212·5) days for the deucrictibant 10 mg group, 166·5 (80·0 to 275·0) days for the deucrictibant 20 mg group, and 172·0 (65·0 to 242·0) days for the deucrictibant 30 mg group. The primary efficacy analysis included 147 attacks in 62 patients, of whom 42 (68%) were female and 60 (97%) were White. Least squares mean differences of change in VAS-3 score between deucrictibant and placebo was -16·75 (95% CI -21·52 to -11·97) for 10 mg, -15·02 (-20·22 to -9·81; p<0·0001) for 20 mg, and -16·28 (-21·27 to -11·29; p<0·0001) for 30 mg. In part 1, all treatment-emergent adverse events were grades 1-2, with the most common in two or more patients being headache (two [8%] of 25) and nasopharyngitis (two [8%] of 25) in the deucrictibant 30 mg group. In part 2, no single treatment-emergent adverse event was reported by two or more patients in any treatment group. Most adverse events were considered unrelated to the study drug and there were no grade 3 or worse adverse events. Deucrictibant significantly reduced the severity of hereditary angioedema attacks compared with placebo; these results support continued investigation of antagonism of the bradykinin B2 receptor with an orally available agent as a potentially effective approach, with a safety profile similar to placebo, for on-demand treatment. Pharvaris.

#2

Oral deucrictibant for prophylaxis of hereditary angioedema attacks (CHAPTER-1): primary analysis of a randomised, double-blind, placebo-controlled, phase 2 trial.

The Lancet. Haematology2026 Mar 19

Hereditary angioedema is a bradykinin-mediated, rare condition characterised by recurrent and potentially life-threatening attacks of subcutaneous and submucosal swelling. Bradykinin B2 receptor antagonism is a proven mechanism for on-demand treatment of attacks, but no evidence exists on its effects when used prophylactically. Deucrictibant is an investigational orally bioavailable bradykinin B2 receptor antagonist. We aimed to evaluate the efficacy, safety, and tolerability of two dose regimens of oral deucrictibant administered as prophylaxis against hereditary angioedema attacks. CHAPTER-1 was a multicentre, double-blind, placebo-controlled, randomised, phase 2 trial conducted in two parts, a double-blind placebo controlled first part and an open-label second part, with only part 1 reported here. Part 1 recruited adults (aged 18-75 years) with hereditary angioedema type 1 or 2 from 37 sites (university hospitals and accredited angioedema centres) across North America, Europe, and Israel. Patients required a documented history of three or more attacks within the last 3 consecutive months before screening or two or more during the screening period (up to 8 weeks) to be eligible. An interactive response technology system randomised eligible patients 1:1:1 to receive oral deucrictibant 20 mg daily, 40 mg daily, or matching placebo for 12 weeks. Randomisation to treatment groups was stratified by the baseline attack rate. Patients, investigators, site personnel, and the sponsor were blinded to treatment assignment. Masking was achieved with identically appearing deucrictibant and placebo capsules. The primary endpoint was the time-normalised number of investigator-confirmed attacks per 4 weeks (monthly attack rate) from weeks 1 to 12 and was assessed using the intention-to-treat set. The endpoint was analysed by comparing each deucrictibant group with the placebo group using a Poisson generalised linear model with a log link function and Pearson's χ2 scaling of SEs to account for potential dispersion. The safety analysis set included all patients who were randomly assigned and who received one or more doses of study drug (deucrictibant or placebo). CHAPTER-1 is registered with ClinicalTrials.gov (NCT05047185) and is now complete. Between March 9, 2022, and June 19, 2023, 44 patients were screened. Of 34 patients who were randomly assigned, 11 patients received deucrictibant 20 mg, 12 patients received deucrictibant 40 mg, and 11 patients received the placebo, with a median follow-up of 85·0 days (IQR 84·0-86·0). 21 (62%) patients were female, 13 (38%) were male, and 34 (100%) patients were White. The least squares mean monthly attack rate (primary analysis) was 0·40 (95% CI 0·18-0·92) for deucrictibant 20 mg, 0·30 (0·11-0·81) for deucrictibant 40 mg, and 1·93 (1·30-2·88) for placebo; percent reduction in attack rate compared with placebo was 79·2% (95% CI 47·2-91·8) for deucrictibant 20 mg (p=0·0010) and 84·5% (95% CI 53·8-94·8) for deucrictibant 40 mg (p=0·0008). Treatment-related treatment-emergent adverse events were experienced by two (18%) patients receiving deucrictibant 20 mg, one (8%) patient receiving deucrictibant 40 mg, and one (9%) patient receiving the placebo; all were mild in severity (grade 1) and did not require dosing modification of the study drug. There were no serious adverse events or deaths in any treatment group. To the best of our knowledge, this trial provides the first clinical evidence and proof-of-concept for bradykinin B2 receptor antagonism as a therapeutic approach for the prevention of hereditary angioedema attacks and supports further investigation of oral deucrictibant for bradykinin-mediated angioedema. Pharvaris.

#3

Characterization of a unique pathogenic variant in the SERPING1 gene of a patient with hereditary angioedema type I.

Journal of dermatological science2026 Mar 09

Hereditary angioedema (HAE) is a rare genetic disease characterized by sudden onset of edema involving various organs. Among the three subtypes of the disease, HAE types I and II are caused by heterozygous variants in SERPING1 gene encoding C1 inhibitor (C1INH). The pathogenicity of each variant, however, has not fully been revealed. To assess the mechanism how a SERPING1 gene-variant identified in a patient with HAE type I caused the disease. Genetic analysis was conducted for a Japanese patient with HAE type I. The consequences resulting from the SERPING1 gene-variant were analyzed at mRNA levels. Overexpression studies in cultured cells were performed to analyze behavior of the mutant C1INH proteins. Effect of a small interfering RNA specific to the mutant SERPING1-mRNA on expression of wild-type C1INH was also tested. We identified a recurrent heterozygous variant c.820 A>G (p.Ile274Val) in the patient's SERPING1 gene. While we did not find any abnormalities in C1INH with the p.Ile274Val-variant, we found that the variant c.820 A>G caused an aberrant splicing event leading to a frameshift and a premature termination codon. This truncated protein clearly showed a dominant-negative effect against the wild-type C1INH. Finally, we showed that knock-down of the mutant SERPING1-mRNA recovered expression of the wild-type C1INH. A unique pathogenic mechanism for the SERPING1 gene-variant c.820 A>G has been disclosed. Furthermore, our findings have raised the possibility that RNA interference could be a new therapeutic tool for the disease.

#4

CRISPR in Medicine: A Systematic Review of Clinical Trials and Therapeutic Applications.

Human gene therapy2026 Mar

Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR associated protein 9 (CRISPR/Cas9) technology has become a revolutionary tool in medicine, offering substantial potential for treating a wide range of diseases, including hematological disorders, cancers, genetic conditions, and ophthalmological diseases. This systematic review evaluates the efficacy, safety, and applicability of CRISPR/Cas9 in clinical trials. A comprehensive search of the PubMed, Scopus, Web of Science, and Cochrane databases was conducted. All studies, up to November 2024, meeting the eligibility criteria assessing the application of CRISPR for the treatment of diseases were included. A quality assessment of the included studies was conducted using the Cochrane risk of bias tool. The Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 statement for systematic reviews and meta-analyses was followed, and a total of 17 studies were included. This systematic review of CRISPR/Cas9 technology focused on its effectiveness and safety across various diseases. In nonmalignant hematological disorders, CRISPR successfully treated β-thalassemia and sickle cell disease, resulting in high transfusion independence and the elimination of disease crises. In malignant hematological disorders, B-cell acute lymphoblastic leukemia, CRISPR-engineered chimeric antigen receptor T (CAR-T) cells achieved an 83.3% complete remission rate. Furthermore, CRISPR-based CAR-T cells showed promising results in B-cell non-Hodgkin's lymphoma. In oncology, lung cancer and other solid tumors are among the diseases that have been safely engineered using CRISPR gene editing technology. For genetic disorders, CRISPR improved vision in retinal degeneration and reduced symptoms in hereditary angioedema and transthyretin amyloidosis with mild side effects. The results demonstrated CRISPR's potential across a wide range of conditions. In conclusion, the findings underscore the potential role of CRISPR/Cas9 technology across a wide range of diseases. However, challenges remain, including optimizing delivery systems, minimizing off-target effects, addressing immunogenicity concerns, and ethical considerations.

#5

Elevated serum zonulin is associated with high attack frequency in hereditary angioedema: providing insight into the gut-angioedema axis.

Orphanet journal of rare diseases2026 Feb 25

Zonulin is a key regulator of epithelial barrier permeability and has been implicated in barrier dysfunction across various inflammatory conditions. To elucidate the potential role of intestinal permeability in HAE, this study investigated serum zonulin levels and evaluated their association with disease activity and psychiatric status. The study included 28 patients with HAE type 1 (HAE-C1INH) and 30 age- and sex-matched healthy controls. Serum zonulin levels were measured by ELISA. Patients were stratified according to attack frequency (high: ≥2 vs. low: <2 attacks/month). Psychiatric status was assessed using the Hospital Anxiety and Depression Scale (HADS). Multivariable linear regression analysis was performed to evaluate the association between zonulin levels and attack frequency after adjustment for body mass index (BMI). Overall serum zonulin levels did not differ significantly between patients with HAE and healthy controls (p = 0.06). However, stratified analyses revealed that patients with high attack frequency exhibited significantly higher zonulin levels compared with both low-attack-frequency patients and controls (p < 0.01), whereas zonulin levels in the low-attack-frequency group were comparable to controls. In multivariable analysis, attack frequency remained independently associated with serum zonulin levels after adjustment for BMI. Although zonulin levels were higher among patients with clinically significant anxiety and/or depressive symptoms based on HADS, this association was weaker than that observed for attack frequency. This pilot study provides preliminary evidence that elevated serum zonulin levels in HAE are associated with higher attack burden, rather than representing a uniform feature of the disease. These findings suggest that zonulin elevation reflects inflammatory activity independent of psychiatric symptom burden and position intestinal barrier integrity as a relevant mechanistic component within the proposed Gut-Angioedema Axis. Future prospective studies are warranted to validate these findings. Not applicable.

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Real-world quality of life in patients with hereditary angioedema receiving lanadelumab or other long-term prophylaxis.

Allergy and asthma proceedings
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Zhonghua nei ke za zhi
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International archives of allergy and immunology
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Human gene therapy
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Allergologie select
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An International Delphi Study on Barriers to On-Demand Treatment of Hereditary Angioedema Attacks.

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Clinical reviews in allergy &amp; immunology
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Donidalorsen for Long-Term Prophylaxis of Hereditary Angioedema Attacks: Results from the OASISplus Open-Label Extension Cohort at Year 1.

Journal of asthma and allergy
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Frontiers in immunology
2026

2025 FDA TIDES (Peptides and Oligonucleotides) Harvest.

Pharmaceuticals (Basel, Switzerland)
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Elevated serum zonulin is associated with high attack frequency in hereditary angioedema: providing insight into the gut-angioedema axis.

Orphanet journal of rare diseases
2026

Management of pregnancy, delivery and breast-feeding in hereditary angioedema: an analysis of 15 pregnancies with conventional treatment approaches and a case of Lanadelumab use.

Orphanet journal of rare diseases
2026

Burden of disease in adult patients with hereditary angioedema: results from a multinational survey.

Orphanet journal of rare diseases
2026

Rethinking the management of hereditary angioedema.

Allergy and asthma proceedings
2026

Updates in Hereditary Angioedema and Chronic Spontaneous Urticaria.

The Journal of allergy and clinical immunology
2026

Burden of hereditary angioedema: results from a multinational survey of caregivers for adult and pediatric patients.

Orphanet journal of rare diseases
2026

Is hereditary angioedema associated with deficits in emotion regulation? A quantitative study in adult patients.

Orphanet journal of rare diseases
2026

"Emerging role of plasma kallikrein inhibitors in preventing hereditary angioedema flares in pregnancy".

Annals of medicine and surgery (2012)
2026

The power of a pill: Ekterly (Sebetralstat): first oral on-demand therapy for hereditary angioedema-a paradigm shift in treatment.

Annals of medicine and surgery (2012)
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Clinical features of hereditary angioedema involving the gastrointestinal tract: A retrospective analysis.

The World Allergy Organization journal
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Novel SERPING1 Genetic Variant in Two Family Members with Hereditary Angioedema.

Acta medica portuguesa
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Developing a patient journey map to improve care and experience in Chinese patients with hereditary angioedema.

The World Allergy Organization journal
2026

Updates on the Current and Evolving Treatment for Hereditary Angioedema.

The journal of allergy and clinical immunology. In practice
2026

Sebetralstat (Ekterly) for treatment of hereditary angioedema attacks.

The Medical letter on drugs and therapeutics
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The patient experience of hereditary angioedema: findings from a racially diverse sample of adult patients.

Orphanet journal of rare diseases
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Recent advances in factor XII(a) inhibitors: an updated patent landscape (2020-present).

Expert opinion on therapeutic patents
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Algorithms in Allergy: Hereditary Angioedema.

Allergy
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Population Pharmacokinetic/Pharmacodynamic Modeling of Donidalorsen, an Antisense Oligonucleotide in Development for Prophylaxis of Hereditary Angioedema.

CPT: pharmacometrics &amp; systems pharmacology
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International Guideline on the Diagnosis and Management of Pediatric Patients With Hereditary Angioedema.

Allergy
2026

The International/Canadian hereditary angioedema guideline.

Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology
2025

Ecological momentary assessments for patients with hereditary angioedema: a feasibility and acceptability controlled study.

Frontiers in digital health
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Classification of angioedema types using decision tree modeling.

Frontiers in immunology
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Comparison of Clinical Characteristics Between Hereditary Angioedema Patients Aged 65 Years and Older and Those Under 65: A Perspective on Elderly Patients.

Life (Basel, Switzerland)
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Perioperative Hereditary Angioedema Triggered by Laryngeal Mask Airway: A Case Report and Anesthetic Implications.

Cureus
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Attack rate reductions following berotralstat initiation among US patients with hereditary angioedema in the real-world.

Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology
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Pain management in patients with hereditary angioedema: A case report.

Medicine
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Functional physiological, psychological, and biochemical reactivity to socially evaluated cold pressor test in hereditary angioedema patients (FRoSEn).

Frontiers in immunology
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Long-term effectiveness and safety of lanadelumab in Canadian patients with hereditary angioedema: a subanalysis of the EMPOWER study.

Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology
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Patient Preferences for Long-Term Prophylactic Treatment in Hereditary Angioedema: A Discrete-Choice Experiment.

The patient
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Type II hereditary angioedema with an apparently de novo SERPING1 mutation in China: A case report and family screening.

Medicine
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Human Plasma-Derived C1 Inhibitor for Short-Term Prophylaxis in Hereditary Angioedema With Normal C1 Inhibitor.

Clinical and experimental allergy : journal of the British Society for Allergy and Clinical Immunology
2026

Expanding the Diagnostic Lens: Mast Cell Activation Syndromes and the Hidden Spectrum of Angioedema Associated Mortality in COVID-19.

Academic forensic pathology
2026

Efficacy and acceptability of lanadelumab for long-term prophylaxis in hereditary angioedema: A Chinese multicenter real-world analysis.

The World Allergy Organization journal
2026

Non-peptide bradykinin B2 receptor ligands possessing the substituted quinolinyl moiety: Pharmacological properties and prospective clinical uses.

Peptides
2026

Patient-reported disease burden and health care utilization of HAE-nl-C1INH: insights from a real-world survey.

Clinical and experimental medicine
2026

Association Between the Angioedema Control Test and Attack Frequency in Hereditary Angioedema.

Clinical and translational allergy
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Bradykinin reduces wound healing in human umbilical vein endothelial cells via downregulation of vascular endothelial growth factor A.

Journal of inflammation (London, England)
2025

Lanadelumab in Hereditary Angioedema: Real-World Outcomes and Implications for Access Practices in Europe.

Journal of clinical medicine
2026

Donidalorsen (Dawnzera) for prevention of hereditary angioedema attacks.

The Medical letter on drugs and therapeutics
2026

A Comprehensive Assessment of Health-Related Quality of Life in Hereditary Angioedema as a Patient-Centered Approach.

International archives of allergy and immunology
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Garadacimab-gxii-A Novel Prophylactic Treatment for Hereditary Angioedema: A Drug Review.

The Annals of pharmacotherapy
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Novel drug approval by the FDA: Ekterly (Sebetralstat), oral plasma kallikrein inhibitor for treating acute attacks of hereditary angioedema.

Annals of medicine and surgery (2012)
2026

Physiological evaluation of the emotional regulation of patients with hereditary angioedema.

BioPsychoSocial medicine
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Non-injectable versus injectable epinephrine treatment thresholds for acute allergic reactions in the community.

The journal of allergy and clinical immunology. In practice
2025

Cardiac and vascular autonomic control in patients with hereditary angioedema.

Frontiers in physiology
2026

Corrigendum to 'Hereditary angioedema-induced laryngeal edema: A diagnostic and therapeutic race against time' [American Journal of Emergency Medicine 93 (2025) 238.e5-238.e8].

The American journal of emergency medicine
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Long-term prophylactic treatment preferences and willingness to switch therapy in individuals with hereditary angioedema.

Annals of allergy, asthma &amp; immunology : official publication of the American College of Allergy, Asthma, &amp; Immunology
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Therapeutic Potential of C1-Inhibitor in Vascular Diseases and Beyond.

Arteriosclerosis, thrombosis, and vascular biology
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Treatment of Hereditary Angioedema With Plasma-Derived C1 Inhibitor: A Review.

Clinical and translational allergy
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From guidelines to global impact: updates to address disparities in hereditary angioedema care.

Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology
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Epinephrine and emergency medical services activation recommendations during acute allergic reactions in community settings: International consensus report.

The Journal of allergy and clinical immunology
2025

SAFE-CGRP study: multicenter retrospective evaluation of the safety of CGRP pathway-targeting monoclonal antibodies in migraine with relevant comorbidities or conditions excluded from trials.

Frontiers in neurology
2025

Sebetralstat: a paradigm shift in hereditary angioedema management.

Annals of medicine and surgery (2012)
2025

CRISPR-Cas9 gene editing for hereditary angioedema: current treatments and emerging therapies.

Annals of medicine and surgery (2012)
2025

Health-related quality of life in hereditary angioedema patients treated with subcutaneous C1 inhibitor (C1-INH) therapy in Canada.

Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology
2025

Urgent diagnosis of hereditary angioedema in the ICU and ED: the critical role of rapid complement testing - a case report.

International journal of emergency medicine
2026

Long-term Prophylaxis With Lanadelumab in Patients With Angioedema Due to C1 Inhibitor Deficiency: A Real-life Study in Spain.

Journal of investigational allergology &amp; clinical immunology
2025

Management of hereditary angioedema with normal C1Inh: a series of 163 French patients.

Orphanet journal of rare diseases
2025

Hereditary angioedema: A national investigation of associated comorbidities and surgical procedures.

The World Allergy Organization journal
2025

A sensitive and specific assay to characterize plasma kallikrein activity in plasma from patients with hereditary angioedema.

The World Allergy Organization journal
2025

Impact of injectable HAE on-demand treatments on health-related quality of life: a patient and caregiver interview study.

Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology
2025

Implementation of genetic diagnosis and personalized management of hereditary angioedema in a Chinese regional center: a community case study of three families.

Frontiers in allergy
2025

Hereditary Angioedema Prophylaxis Therapy: Berotralstat and Lanadelumab Safety Profile.

Medicina (Kaunas, Lithuania)
2025

On-demand treatment of hereditary angioedema attacks: Patient-reported utilization, barriers, and outcome.

Annals of allergy, asthma &amp; immunology : official publication of the American College of Allergy, Asthma, &amp; Immunology
2025

Berotralstat, the first oral prophylaxis for hereditary angioedema in children aged 2 to 12 years: The kids are alright!

Annals of allergy, asthma &amp; immunology : official publication of the American College of Allergy, Asthma, &amp; Immunology
2025

Abdominal Pain Attacks and Angioedema in a Patient With a Homozygous MEFV Mutation: Concurrent FXII Hereditary Angioedema.

Journal of investigational allergology &amp; clinical immunology
2025

Sebetralstat for On-Demand Treatment of Mucosal Hereditary Angioedema Attacks in KONFIDENT-S.

Clinical and translational allergy
2025

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Cureus
2025

Hereditary angioedema as a cause of recurrent abdominal pain in a pediatric patient with Crohn's disease.

JPGN reports
2025

Long-Term Attenuation of Vascular Hyperpermeability in a Hereditary Angioedema Mouse Model by Adenine Base Editing.

Allergy
2025

Common features of rare disease patients in the emergency department: a systematised literature review.

Orphanet journal of rare diseases
2026

Clinical and genetic study in factor XII hereditary angioedema in a population from Southern Spain.

Annals of allergy, asthma &amp; immunology : official publication of the American College of Allergy, Asthma, &amp; Immunology
2026

Donidalorsen: First Approval.

Drugs
2025

A Multicentric Clinical Study to Evaluate Pharmacokinetics, Efficacy, and Safety of Immune Globulin Subcutaneous 20% Weekly/Biweekly Dosing in Treatment-Experienced Patients and Loading/Weekly Maintenance Dosing in Treatment-Naïve Patients with Primary Immunodeficiency.

Journal of clinical immunology
2025

Delphi Consensus on Attenuated Androgen Use for Long-Term Prophylaxis in Hereditary Angioedema: AURA Project.

Clinical and translational allergy
2025

Exposure-Response Analysis of Donidalorsen for the Treatment of Hereditary Angioedema.

Clinical and translational science
2025

Unexpected genomic architecture in a sporadic case of C1-INH Hereditary Angioedema: the hidden heritability.

Orphanet journal of rare diseases
2026

Sebetralstat: FDA approves first oral on-demand therapy for hereditary angioedema.

Irish journal of medical science
2025

Garadacimab (Andembry) for hereditary angioedema prophylaxis.

The Medical letter on drugs and therapeutics
2025

Spinal Anesthesia With Short-Term Prophylaxis in a Patient With Hereditary Angioedema: A Case Report.

Cureus
2025

C1 esterase inhibitor (C1-INH) response as a supportive diagnostic criterion for patients with suspected hereditary angioedema with normal C1-INH.

The World Allergy Organization journal
2025

Hereditary Angioedema With Normal C1 Esterase Inhibitor: A Case of a Late Diagnosis in a 48-Year-Old Male.

Cureus
2025

Self-administered oral sebetralstat: a game-changer in treatment of hereditary angioedema.

Annals of medicine and surgery (2012)
2026

Berotralstat effectiveness and safety in patients with hereditary angioedema with normal C1 inhibitor.

The journal of allergy and clinical immunology. Global
2025

Leveraging lanadelumab: A phase 4 evaluation of hereditary angioedema by attack rate subgroups.

Annals of allergy, asthma &amp; immunology : official publication of the American College of Allergy, Asthma, &amp; Immunology
2026

Transient hepatitis as a novel withdrawal phenomenon after danazol discontinuation in hereditary angioedema.

The journal of allergy and clinical immunology. In practice
2025

Unveiling the impact of hereditary angioedema: a study on quality of life in Brazilian adults.

Anais da Academia Brasileira de Ciencias
2025

French protocol for the diagnosis and management of hereditary angioedema.

La Revue de medecine interne
2025

An Unusual Gastrointestinal Presentation of Sjogren's Syndrome: A Case Report.

Clinical case reports
2025

Icatibant Use During Pregnancy: A South African Case Series.

Journal of investigational allergology &amp; clinical immunology
2026

Remibrutinib in chronic spontaneous urticaria: 52-week results from two phase 3 studies.

The Journal of allergy and clinical immunology
2025

Unraveling angioedema: diagnostic challenges and emerging therapies.

Frontiers in immunology
2025

C1 Esterase Inhibitor Deficiency in an Indian Female: A Rare Case Report and a Review of Literature with Treatment Update.

Current drug targets
2026

National Audit of Long-Term Real-World Outcomes of Berotralstat Use in UK Patients With Hereditary Angioedema.

Allergy
2025

Misdiagnosed Hereditary Angioedema with Recurrent Abdominal Pain: A Novel SERPING1 Frameshift Variant.

Journal of asthma and allergy
2026

Real-World Evidence on the Management of Hereditary Angioedema With Normal C1 Inhibitor.

The journal of allergy and clinical immunology. In practice
2025

Therapeutic Advances in Hereditary Angioedema: A Focus on Present and Future Options.

Advances in therapy
2025

Absolute Configuration of Key Intermediates and the Gram Scale Synthesis of Berotralstat and ent-Berotralstat.

The Journal of organic chemistry
2025

Barriers and promoters to adapting research findings to clinical care in hereditary angioedema in the United States: A qualitative study.

PloS one
2025

Improving the standards of medical case reports; insights from a hereditary angioedema example.

Journal of family medicine and primary care
2025

Measurement of cleaved high-molecular-weight kininogen in patients with hereditary angioedema due to C1-inhibitor deficiency: preanalytical and analytical optimization.

Journal of immunological methods
2025

[Acquired angioedema associated with non-Hodgkin lymphoma: a clinical case report].

Revista alergia Mexico (Tecamachalco, Puebla, Mexico : 1993)
2025

[Economic Burden of Hereditary Angioedema from the Perspective of the Public Health System in Mexico].

Revista alergia Mexico (Tecamachalco, Puebla, Mexico : 1993)
2025

[Drug addiction and hereditary angioedema type 1, a dangerous combination].

Revista alergia Mexico (Tecamachalco, Puebla, Mexico : 1993)
2025

[Effectiveness and safety of Lanadelumab according to disease activity: pooled analysis of EMPOWER/ENABLE].

Revista alergia Mexico (Tecamachalco, Puebla, Mexico : 1993)
2025

Plasma Kallikrein Inhibitors for Multiple Disorders: Current Advances and Perspectives.

Journal of medicinal chemistry
2025

Observations on quality of life and disease control in patients with hereditary angioedema.

Skin health and disease
2025

First report from the Czech national registry of inborn errors of immunity (2012-2025).

Frontiers in immunology
2026

Population-scale analysis reveals inherited C1-inhibitor deficiency is a polyphenotypic thrombotic disorder.

Blood advances
2025

Perinatal Management and Clinical Outcomes of Hereditary Angioedema in Japan: A Case Series and Comprehensive Literature Review.

The Journal of dermatology
2025

Sebetralstat: First Approval.

Drugs
2025

Pediatric angioedema without urticaria: A rare presentation of autoimmune thyroid disease with complete remission on levothyroxine therapy.

Journal of postgraduate medicine
2025

Anaesthesia Management of a Case with Hereditary Angioedema for Whom Tracheal Dilatation was Planned.

Turkish journal of anaesthesiology and reanimation
2025

Quality of life and burden of disease in patients with hereditary angioedema and their caregivers.

Allergy and asthma proceedings
2025

Real-world treatment patterns and burden-of-disease of sub-optimally controlled hereditary angioedema.

The World Allergy Organization journal
2025

Evaluating functional C1INH with multiple laboratory methods across Hereditary Angioedema types.

Frontiers in immunology
2025

FDA approves anti-prekallikrein drug for hereditary angioedema.

Nature reviews. Drug discovery
2025

One-year real-life outcomes of lanadelumab therapy in Romanian patients with hereditary angioedema due to C1-inhibitor deficiency.

Frontiers in allergy
2026

A Phase 1 Randomized Study: Garadacimab Pharmacokinetics, Safety, and Tolerability After Administration via Autoinjector/Pre-Filled Pen Versus Pre-Filled Syringe in Healthy Participants.

Journal of clinical pharmacology
2025

Insights From the First 820 Patients From the Brazilian Multicenter Registry of Hereditary Angioedema: The Key Role of Genetic Testing and Targeted Therapies.

The journal of allergy and clinical immunology. In practice
2025

C1 inhibitor: from complement system to bradykinin angioedema.

Current opinion in immunology
2025

Status quo and future developments in the diagnosis and treatment of hereditary angioedema.

Journal der Deutschen Dermatologischen Gesellschaft = Journal of the German Society of Dermatology : JDDG
2025

Three-year real-world outcomes of lanadelumab prophylaxis in hereditary angioedema: Complete disease suppression and psychosocial benefits in two East Asian patients.

Intractable &amp; rare diseases research
2025

Revealing hidden patterns in hereditary angioedema: Insights beyond claims data.

Annals of allergy, asthma &amp; immunology : official publication of the American College of Allergy, Asthma, &amp; Immunology
2025

COVID-19-Induced Flare of Hereditary Angioedema in a Twelve-Year-Old Female Patient.

Cureus
2025

Long-Term Safety and Effectiveness of Sebetralstat: Interim Analysis of KONFIDENT-S Open-label Extension.

The journal of allergy and clinical immunology. In practice
2025

Long-term prophylactic treatment with deucrictibant for angioedema due to acquired C1-inhibitor deficiency.

The Journal of allergy and clinical immunology
2025

Bradykinin-Mediated Angioedema Induced by Drugs.

Journal of clinical medicine
2025

Garadacimab in hereditary angioedema due to normal C1INH with F12/PLG mutations.

The Journal of allergy and clinical immunology
2025

Expanding the Genetic and Clinical Spectrum of Hereditary Angioedema with Normal C1 Inhibitor: Novel Variants and Treatment Insights.

Journal of clinical immunology
2025

A single-centre retrospective study on the clinical characteristics of patients with hereditary angioedema and the therapeutic effect of lanadelumab.

Orphanet journal of rare diseases
2025

Impact of providing education on the recognition and differential diagnosis of angioedema among emergency department physicians.

The World Allergy Organization journal
2025

Family Testing Reveals Asymptomatic Carriers in a Case of Long-Undiagnosed Type 1 Hereditary Angioedema.

The Journal of dermatology
2025

Recurrent angioedema manifestation and treatment response in two patients from different families caring the myoferlin gene mutation: case series.

Orphanet journal of rare diseases
2025

A Rare Case of Idiopathic Angioedema Associated With Amlodipine Use.

Cureus
2025

Lanadelumab's impact on hereditary angioedema control and quality of life across disease activity subgroups: Real-world evidence.

Annals of allergy, asthma &amp; immunology : official publication of the American College of Allergy, Asthma, &amp; Immunology
2025

Disease control and disease activity in hereditary angioedema: two sides of the same coin?

Frontiers in immunology
2025

Impact of hereditary angioedema attacks on health-related quality of life and work productivity.

The World Allergy Organization journal
2025

Clinical presentation and management of 239 children and adolescents with hereditary angioedema.

The journal of allergy and clinical immunology. In practice
2025

Unmet needs in hereditary angioedema: an international survey of physicians.

Orphanet journal of rare diseases
2025

Proactive identification of rare diseases: lessons from hereditary angioedema diagnosis using electronic medical records.

Orphanet journal of rare diseases
2025

Oral berotralstat for hereditary angioedema prophylaxis in patients aged 2 to <12 years: APeX-P interim results.

Annals of allergy, asthma &amp; immunology : official publication of the American College of Allergy, Asthma, &amp; Immunology
2025

Exercise-Induced Angioedema, Urticaria, and Anaphylaxis-A Narrative Review.

Sports (Basel, Switzerland)
2025

Characteristics and Comorbidities Influencing Mortality Risk Among Hereditary Angioedema Patients.

Journal of health economics and outcomes research
2025

Activation of the Complement/Lectin Pathway, Angiopoietin/Tie-2/VEGF-System, Cytokines and Chemokines in Different Angioedema Subtypes.

European journal of immunology
2025

Evaluating large language models for WAO/EAACI guideline compliance in hereditary angioedema management.

Allergologia et immunopathologia
2025

Bradykinin measurement by liquid chromatography tandem mass spectrometry in subjects with hereditary angioedema enhanced by cold activation.

The journal of allergy and clinical immunology. Global
2026

Long-Term Real-World Outcomes in Patients with Hereditary Angioedema Receiving Lanadelumab for 3 or More Years.

International archives of allergy and immunology
2025

Donidalorsen Treatment of Hereditary Angioedema in Patients Previously on Long-Term Prophylaxis.

The journal of allergy and clinical immunology. In practice
2025

Case reports of subcutaneous pdC1INH in pregnancy and lactation: expanding treatment options for hereditary angioedema in Portugal.

Frontiers in allergy
2025

Delays and Barriers Related to the Treatment of Hereditary Angioedema Attacks in Italy.

Allergy
2025

Treatment Patterns and Characteristics of Patients with Hereditary Angioedema Treated with Lanadelumab: A US Retrospective Chart Review.

Drugs - real world outcomes
2025

Effect of Fresh Frozen Plasma Infusion on Hospital Length of Stay for Patients With Hereditary Angioedema.

Journal of health economics and outcomes research
2025

ALERT (ChAracterizing uncontroLled sevERe asThma in Canada): Oral corticosteroid and short-acting β2-agonist overuse.

Annals of allergy, asthma &amp; immunology : official publication of the American College of Allergy, Asthma, &amp; Immunology
2025

Matching-adjusted indirect comparison between garadacimab and lanadelumab for the long-term prophylactic treatment of patients with hereditary angioedema.

Journal of comparative effectiveness research
2025

A Rare Case of Valsartan-Induced Angioedema.

Cureus
2025

[Pharmacological characteristics and clinical trial results of garadacimab (anti-activated factor XII monoclonal antibody) for long-term prophylaxis of hereditary angioedema].

Nihon yakurigaku zasshi. Folia pharmacologica Japonica
2025

Hereditary Angioedema Characterized by Laryngeal Obstruction.

The journal of allergy and clinical immunology. In practice
2025

Hereditary angioedema in a paediatric patient presenting with abdominal pain and duodenal dilation.

BMJ case reports
2025

Consensus on diagnosis and management of hereditary angioedema in Greece.

European annals of allergy and clinical immunology
Ver todos os 2.405 no EuropePMC

Associações

Organizações que acompanham esta doença — pra ter apoio e orientação

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Comunidades

Grupos ativos de quem convive com esta doença aqui no Raras

Ainda não existe comunidade no Raras para Angioedema hereditário

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Doenças relacionadas

Doenças com sintomas parecidos — ajudam quem ainda está buscando diagnóstico

Referências e fontes

Bases de dados externas citadas neste artigo

Publicações científicas

Artigos indexados no PubMed ligados a esta doença no grafo RarasNet — título, periódico e PMID direto da fonte, sem intermediação de IA.

  1. Oral deucrictibant for on-demand treatment of hereditary angioedema attacks (RAPIDe-1): a randomised, double-blind, placebo-controlled, phase 2 trial.
    The Lancet. Haematology· 2026· PMID 41865747mais citado
  2. Oral deucrictibant for prophylaxis of hereditary angioedema attacks (CHAPTER-1): primary analysis of a randomised, double-blind, placebo-controlled, phase 2 trial.
    The Lancet. Haematology· 2026· PMID 41865746mais citado
  3. Characterization of a unique pathogenic variant in the SERPING1 gene of a patient with hereditary angioedema type I.
    Journal of dermatological science· 2026· PMID 41850960mais citado
  4. CRISPR in Medicine: A Systematic Review of Clinical Trials and Therapeutic Applications.
    Human gene therapy· 2026· PMID 41810550mais citado
  5. Elevated serum zonulin is associated with high attack frequency in hereditary angioedema: providing insight into the gut-angioedema axis.
    Orphanet journal of rare diseases· 2026· PMID 41742205mais citado
  6. Factor XII in Thrombosis and Thromboinflammation: From Molecular Biology to Clinical Translation.
    Int J Mol Sci· 2026· PMID 41977511recente
  7. Workplace and Healthcare Stigma in Hereditary Angioedema: Links to Anxiety and Functional Impairment.
    Healthcare (Basel)· 2026· PMID 41975952recente
  8. 'What' and 'How' to Measure in Allergy and Clinical Immunology: A Systematic Review of Core Outcome Sets and Outcome Harmonisation Processes.
    Clin Exp Allergy· 2026· PMID 41974646recente
  9. Skeletal and dentoalveolar effects on the midpalatal suture and maxillary arch assessed by occlusal radiographs and three-dimensional digital models in patients treated with invisalign palatal expander and rapid palatal expander: a pilot study.
    Front Dent Med· 2026· PMID 41930095recente
  10. If It Is Not Immunoglobulin E-Mediated Allergy, What Is It?
    Br J Hosp Med (Lond)· 2026· PMID 41913989recente

Bases de dados e fontes oficiais

Identificadores e referências canônicas usadas para montar este verbete.

  1. ORPHA:91378(Orphanet)
  2. MONDO:0019623(MONDO)
  3. Angioedema Hereditario(PCDT · Ministério da Saúde)
  4. GARD:5979(GARD (NIH))
  5. Variantes catalogadas(ClinVar)
  6. Busca completa no PubMed(PubMed)
  7. Artigo Wikipedia(Wikipedia)
  8. Q2096745(Wikidata)

Dados compilados pelo RarasNet a partir de fontes abertas (Orphanet, OMIM, MONDO, PubMed/EuropePMC, ClinicalTrials.gov, DATASUS, PCDT/MS). Este conteúdo é informativo e não substitui avaliação médica.

Conteúdo mantido por Agente Raras · Médicos e pesquisadores podem colaborar

Angioedema hereditário
Compêndio · Raras BR

Angioedema hereditário

ORPHA:91378 · MONDO:0019623
🇧🇷 Brasil SUS
CEAF
1AIcatibantoLanadelumabeInibidor de C1-esterase
Geral
Prevalência
1-9 / 100 000
Herança
Autosomal dominant
CID-10
D84.1 · Defeitos no sistema complemento
CID-11
Ensaios
30 ativos
Medicamentos
8 registrados
Início
All ages
Prevalência
5.0 (Europe)
MedGen
UMLS
C0019243
Repurposing
25 candidatos
azosemideelectrolyte reabsorption inhibitor
benzthiazidecarbonic anhydrase inhibitor
bumetanidesolute carrier family member inhibitor
+17 outros
EuropePMC
Wikidata
Wikipedia
Papers 10a
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