Management of hereditary angioedema attacks by patients on long-term prophylaxis versus on-demand therapy only.

Despite the use of long-term prophylaxis (LTP) for hereditary angioedema (HAE), the risk of having an attack remains and patients with HAE and on LTP may still experience attacks that can be life threatening. However, the behavioral patterns and perspectives surrounding HAE attack management by patients on LTP are not fully understood. This survey aimed to better understand and compare the behavioral patterns and perspectives, including attitudes and perceptions associated with on-demand treatment among patients on LTP versus those using on-demand therapy only.

Challenging dogmas: Intravenous versus oral beta-lactam antibiotic provocation tests.

Background: Drug provocation tests (DPT) are considered the gold standard procedure to ascertain the diagnosis of beta-lactam (BL) allergy. Regarding route of administration, current recommendations prioritize oral challenges, considering them safer, and reserving the intravenous route for drugs for which this is the only formulation.

Objective: To compare in terms of tolerance and safety two protocols of BL DPT, using an oral protocol (OR-DPT) and an intravenous protocol (IV-DPT).

Hereditary angioedema in Spain: medical care and patient journey.

Background: Hereditary angioedema due to C1 inhibitor deficiency (HAE-C1INH) is a genetic rare disease characterized by recurrent, transient and unpredictable episodes of cold, non-pruriginous oedema without associated urticaria. The characteristics of the disease have a considerable impact on the quality of life of patients. The aim of this study was to increase understanding of the patient journey of HAE in Spain.

A Core Outcome Set for Efficacy of Acute Treatment of Hereditary Angioedema.

Background: Clinical trials investigating drugs for the acute treatment of hereditary angioedema attacks have assessed many different outcomes. This heterogeneity limits the comparability of trial results and may lead to selective outcome reporting bias and a high burden on trial participants.

Objective: To achieve consensus on a core outcome set composed of key outcomes that ideally should be used in all clinical efficacy trials involving the acute treatment of hereditary angioedema attacks.

Avapritinib versus Placebo in Indolent Systemic Mastocytosis.

BACKGROUND: Indolent systemic mastocytosis (ISM) is a clonal mast-cell disease driven by the KIT D816V mutation. We assessed the efficacy and safety of avapritinib versus placebo, both with best supportive care, in patients with ISM. METHODS: We randomized patients with moderate to severe ISM (total symptom score [TSS] of ≥28; scores range from 0 to 110, with higher numbers indicating more severe symptoms) two to one to avapritinib 25 mg once daily (n=141) or placebo (n=71).

Expert Review and Consensus on the Treat-to-Target Management of Hereditary Angioedema: From Scientific Evidence to Clinical Practice.

Background: Hereditary angioedema with C1 inhibitor deficiency (HAE-C1INH) is a rare disease characterized by swelling episodes. It affects quality of life (QOL) and can be fatal when the upper airways are involved. Treatment is individualized, with therapeutic options including on-demand treatment (ODT) and short- and long-term prophylaxis (STP, LTP).

Classification and Treatment of Angioedema without Wheals: A Spanish Delphi Consensus.

Introduction: Problems in the definition and classification of angioedema, leading to difficulties in its diagnosis and treatment, have been identified; therefore, an improvement in the current classification of angioedema is required.

Objective: The aim of this study was to propose a practical classification of angioedema without wheals that helps to establish a differential diagnosis and take appropriate therapeutic decisions.

Methods: An initial proposal of classification of angioedema without wheals was agreed by a scientific committee of experts and was subsequently validated by a panel of experts by means of consensus based on the Delphi methodology.

Hereditary angioedema due to C1 inhibitor deficiency: real-world experience from the Icatibant Outcome Survey in Spain.

Background: The Icatibant Outcome Survey (IOS) is an international registry monitoring the use of icatibant, a bradykinin B receptor antagonist indicated for the acute treatment of hereditary angioedema (HAE) attacks. Our goal was to assess disease characteristics and icatibant treatment outcomes in patients with HAE due to C1 inhibitor deficiency (HAE type 1 or 2 (HAE-1/2)) from Spain relative to other countries participating in IOS.

Methods: Descriptive retrospective analyses of data are reported from 10 centers in Spain vs 51 centers in 12 other participating countries (July 2009 to January 2019).

Angioedema Due to Acquired Deficiency of C1-Inhibitor: A Cohort Study in Spain and a Comparison With Other Series.

Background: Data on acquired angioedema due to C1-inhibitor deficiency (C1-INH-AAE) from 4 European countries (France, Italy, Germany, and Hungary) were recently published.

Objective: To report data from a group of 50 patients with acquired C1-INH deficiency from Spain, of whom 46 had angioedema, and compare them with other European series.

Methods: We performed a retrospective observational study of 46 patients with C1-INH-AAE and 4 asymptomatic patients.

One-Dilution Rapid Desensitization Protocol to Chemotherapeutic and Biological Agents: A Five-Year Experience.

Background: Rapid drug desensitization (RDD) becomes a crucial procedure to allow treatment continuation in patients who suffer drug hypersensitivity reactions (DHRs) to chemotherapeutic (CMT) and biological agents (BA).

Objective: The aim of the study was to compare the efficacy and safety of a one-bag dilution protocol (1DP) with a conventional three-bag dilution protocol (3DP) for desensitization of patients with CMT or BA hypersensitivity.

Methods: Retrospective analysis of patients with immediate DHRs to CMT or BA who underwent at least 1 RDD procedure in our department between 2014 and 2019 was performed.

A Comparative Study of Sex Distribution, Autoimmunity, Blood, and Inflammatory Parameters in Chronic Spontaneous Urticaria with Angioedema and Chronic Histaminergic Angioedema.

Background: Recurrent idiopathic histaminergic angioedema is currently classified as a subtype of angioedema, as well as a subtype of chronic spontaneous urticaria (CSU), based on the fact that both are mast cell-mediated and respond to the same treatments.

Objective: In the present work, we sought to verify whether chronic histaminergic angioedema (CHA) is an entity distinct from CSU or represents a CSU subtype that lacks hives.

Methods: We performed a prospective study comparing 68 CHA patients, angioedema without hives, with 63 CSU patients, with hives and angioedema, from whom we collected demographic and clinical data, as well as blood and serum markers.

Food-dependent NSAID-induced hypersensitivity (FDNIH) reactions: Unraveling the clinical features and risk factors.

Background: In up to 70%-80% of patients with a suspected non-steroidal anti-inflammatory drug hypersensitivity (NSAIDH), challenge tests with the culprit drug yield negative results. On the other hand, there could be a NSAIDH overdiagnosis when anaphylaxis is the clinical manifestation. We hypothesize that some negative NSAID challenge tests and an overdiagnosis of NSAIDH occur in patients with food-dependent NSAID-induced hypersensitivity (FDNIH).

Digital technology for anaphylaxis management impact on patient behaviour: A randomized clinical trial.

Background: Epinephrine is the first-line treatment for anaphylaxis. Patients at risk should always carry an epinephrine autoinjector (EAI). Several EAI gaps have been identified.

Peripheral Corticotropin-Releasing Factor Triggers Jejunal Mast Cell Activation and Abdominal Pain in Patients With Diarrhea-Predominant Irritable Bowel Syndrome.

Introduction: To determine the effect of peripheral CRF on intestinal barrier function in diarrhea-predominant IBS (IBS-D). Irritable bowel syndrome (IBS) pathophysiology has been linked to life stress, epithelial barrier dysfunction, and mast cell activation. Corticotropin-releasing factor (CRF) is a major mediator of stress responses in the gastrointestinal tract, yet its role on IBS mucosal function remains largely unknown.

Molecular diagnosis usefulness for idiopathic anaphylaxis.

Purpose Of Review: Molecular diagnosis has become an indispensable tool in allergy. In suspected idiopathic anaphylaxis, it is mandatory to extend the diagnostic search to its limits. The current review evaluates how molecular diagnosis allows to identify a number of difficult to prove potential culprits.

Genetic Variation of Kallikrein-Kinin System and Related Genes in Patients With Hereditary Angioedema.

Hereditary angioedema (HAE) is an autosomal dominant disease caused by C1-INH deficiency due to mutations in (C1-INH-HAE) in most of the cases, or by specific mutations in factor XII gene, (F12-HAE). Identification of polymorphisms in the genes encoding proteins from key pathways driving HAE can help to understand how genetic diversity contributes to its phenotypic variability. Here, 15 genes related to the Kallikrein-Kinin System (KKS) were analyzed by next generation sequencing in 59 patients with C1-INH-HAE or F12-HAE from Brazil, Denmark and Spain, and 19 healthy relatives in a total of 31 families.