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Hereditary angioedema (HAE) attacks are recurrent, unpredictable, debilitating, and potentially life-threatening. Current guidelines state HAE treatment goals are complete disease control and normalization of life. Garadacimab, a fully human, once-monthly, anti-activated factor XII monoclonal antibody, was evaluated as HAE long-term prophylaxis in the pivotal Phase 3 (VANGUARD) study, and 62% of garadacimab-treated patients were attack-free (all patients receiving placebo experienced attacks). This post hoc analysis evaluated potential association between baseline patient characteristics and attack-free status.

L’angioœdème héréditaire (AOH) est une maladie génétique rare, chronique et potentiellement mortelle qui se caractérise par des épisodes récurrents d’œdèmes affectant la peau, les intestins et les voies respiratoires supérieures. Ces symptômes imprévisibles ont un impact négatif sur la qualité de vie (QdV). Le facteur XII activé (FXIIa), un initiateur du système de contact, produit de la bradykinine, un médiateur clé de l’AOH. Le garadacimab, premier anticorps monoclonal de sa catégorie ciblant le FXIIa, a réduit de manière significative la fréquence mensuelle des crises d’AOH par rapport au placebo (PBO; p<0,001) dans le cadre d’un essai de phase 3 multicentrique randomisé, en double aveugle et contrôlé vs PBO (VANGUARD, NCT04656418). Nous présentons ici les données relatives à la QdV de l’étude VANGUARD.

Hereditary angioedema (HAE) causes recurrent, debilitating attacks. Prophylaxis with garadacimab (anti-activated factor XII monoclonal antibody) demonstrated efficacy with a favorable safety profile in double-blind Phase 2 and pivotal Phase 3 (VANGUARD) studies. We report results from an ongoing open-label extension (OLE) study (NCT04739059).

Replacement therapy with subcutaneous C1-esterase inhibitor (C1-INH[SC]) 60 IU/kg twice weekly is approved for routine prophylaxis to prevent hereditary angioedema (HAE) attacks. Coagulation and fibrinolytic parameters levels tend to be elevated in HAE patients. We evaluated the effects of C1-INH(SC) on these parameters.

Hereditary angioedema (HAE) due to C1-inhibitor (C1-INH) deficiency is characterized by symptom onset in childhood, with earlier onset associated with a more severe disease course. Subcutaneous (SC) C1-INH is indicated for routine prophylaxis to prevent HAE attacks in adolescents and adults. We analyzed the long-term efficacy of C1-INH (SC) in pediatric patients treated in an open-label extension (OLE) of the phase III COMPACT trial.

Low levels of C1-esterase inhibitor (C1-INH) and concomitant reduced complement 4 (C4) concentrations have been identified in patients with hereditary angioedema (HAE). C4 constitutes a promising candidate for a biomarker of disease activity. This investigation aims to determine the relationship between C1-INH functional activity levels (C1-INH[f]) and C4 antigen concentrations in patients with HAE treated with subcutaneous (SC) C1-INH, inferring significance for clinical practice.

Elderly patients ≥65 years old with hereditary angioedema (HAE) type I/II due to C1-inhibitor (C1-INH) deficiency may have an altered response to treatment and be at higher risk for treatment-related adverse events (AEs) due to comorbidities and polypharmacy. Subcutaneous (SC) C1-INH is indicated for routine prophylaxis to prevent HAE attacks in adolescents and adults. This post-hoc analysis examined the efficacy and safety of C1-INH (SC) in patients ≥65 years old treated in an open-label extension study.

In women with hereditary angioedema (HAE) due to C1-inhibitor (C1-INH) deficiency, pregnancy can have a variable effect on disease activity. C1-INH concentrate is recommended as first-line therapy for pregnant patients with HAE. Subcutaneous C1-INH (HAEGARDA®, CSL Behring) is indicated for routine prophylaxis to prevent HAE attacks in adolescents and adults. This analysis examines outcomes in patients who became pregnant during C1-INH (SC) treatment in an open-label safety study.