Sebetralstat for breakthrough attacks in patients with hereditary angioedema receiving long-term prophylaxis in KONFIDENT-S.
Although long-term prophylaxis (LTP) reduces attack frequency in hereditary angioedema, patients may experience breakthrough attacks. Oral sebetralstat demonstrated favorable safety and efficacy compared with placebo in the randomized phase 3 KONFIDENT trial (NCT05259917), including in patients rece…
Management of hereditary angioedema in the UK: a comparison with international guidelines.
Hereditary angioedema (HAE) is a rare genetic disorder characterised by recurrent episodes of swelling, which can potentially be life-threatening, resulting in substantial physical, psychological, educational and occupational burden. Treatment of HAE is divided into on-demand treatment for acute att…
Enacted and internalised stigma and perceived discrimination in hereditary angioedema due to C1-inhibitor deficiency: A pilot study.
Stigma and perceived discrimination are understudied psychosocial components in hereditary angioedema due to C1-inhibitor deficiency (HAE-C1INH). This cross-sectional pilot study assessed stigma and perceived discrimination in 20 HAE-C1INH patients, comparing those with (HAE A+) and without (HAE A-)…
Improving capability, opportunity and motivation to support hereditary angioedema patients experiencing life threatening attacks: Pilot evaluation of a video-based training tool for healthcare professionals.
Hereditary angioedema (HAE) is a rare inherited illness which causes swelling and can be life-threatening without urgent treatment. Patients who experience life-threatening throat swellings report poor emergency care experiences and long-term psychological distress from these encounters. A > 5-min v…
International Guideline on the Diagnosis and Management of Pediatric Patients With Hereditary Angioedema.
Hereditary angioedema (HAE) with C1 inhibitor deficiency is a rare disease characterized by unpredictable episodes of tissue swelling (angioedema), which, in most cases, occur first under the age of 18 years, and entail a significant burden of disease not only for the patients but also for th…
Multimodal imaging evaluation of pre-systemic sclerosis patients presenting with Raynaud's Phenomenon from a reference microcirculatory clinic versus healthy controls: a cross-sectional study.
To determine whether multimodal imaging tools can detect dermal and microvascular abnormalities in pre-systemic sclerosis (pre-SSc) patients compared with matched healthy controls (HC). Non-selected pre-SSc patients (n = 20, fulfilling LeRoy's criteria) from the Ghent University (hospital) Raynaud's…
Clinical expressions, disease course, quality of life, and resilience in subgroups of patients with angioedema.
Patients with angioedema caused by C1INH deficiency (HAE-C1INH) experience recurrent episodes of unpredictable swelling. Recently, new genetic variants have been identified, whereas patients with unknown causes and no identifiable genetic mutations constitute a distinct subgroup. As a lifelong condi…
Transient C1-Inhibitor Functional Abnormalities During Acute Idiopathic Pancreatitis: A Diagnostic Challenge in Hereditary Angioedema Evaluation.
Hereditary angioedema (HAE) is an important but uncommon differential diagnosis in patients presenting with unexplained recurrent abdominal attacks, including idiopathic acute pancreatitis (IAP). However, whether acute IAP itself transiently affects complement test results and complicates the evalua…