Nieves Prior

Development of a disease-specific quality of life questionnaire for adult patients with hereditary angioedema due to C1 inhibitor deficiency (HAE-QoL): Spanish multi-centre research project

BackgroundThere is a need for a disease-specific instrument for assessing health-related quality of life in adults with hereditary angioedema due to C1 inhibitor deficiency, a rare, disabling and life-threatening disease. In this paper we report the protocol for the development and validation of a specific questionnaire, with details on the results of the process of item generation, domain selection, and the expert and patient rating phase.Methods/DesignSemi-structured interviews were completed by 45 patients with hereditary angioedema and 8 experts from 8 regions in Spain. A qualitative content analysis of the responses was carried out. Issues raised by respondents were grouped into categories. Content analysis identified 240 different responses, which were grouped into 10 conceptual domains. Sixty- four items were generated. A total of 8 experts and 16 patients assessed the items for clarity, relevance to the disease, and correct dimension assignment. The preliminary version of the specific health-related quality of life questionnaire for hereditary angioedema (HAE-QoL v 1.1) contained 44 items grouped into 9 domains.DiscussionTo the best of our knowledge, this is the first multi-centre research project that aims to develop a specific health-related quality of life questionnaire for adult patients with hereditary angioedema due to C1 inhibitor deficiency. A preliminary version of the specific HAE-QoL questionnaire was obtained. The qualitative analysis of interviews together with the expert and patient rating phase helped to ensure content validity. A pilot study will be performed to assess the psychometric properties of the questionnaire and to decide on the final version.

Management of angioedema without urticaria in the emergency department

Abstract Angioedema refers to a localized, transient swelling of the deep skin layers or the upper respiratory or gastrointestinal mucosa. It develops as a result of mainly two different vasoactive peptides, histamine or bradykinin. Pathophysiology, as well as treatment, is different in each case; nevertheless, the resulting signs and symptoms may be similar and difficult to distinguish. Angioedema may occur at any location. When the affected area involves the upper respiratory tract, both forms of angioedema can lead to an imminent upper airway obstruction and a life-threatening emergency. Emergency physicians must have a basic understanding of the pathophysiology underlying this process. Angioedema evaluation in the emergency department (ED) should aim to distinguish between histamine- and bradykinin-induced angioedema, in order to provide appropriate treatment to patients. However, diagnostic methods are not available at the ED setting, neither to confirm one mechanism or the other, nor to identify a cause. For this reason, the management of angioedema should rely on clinical data depending on the particular features of the episode and the patient in each case. The history-taking should be addressed to identify a possible etiology or triggering agent, recording complete information for an ulterior diagnostic study in the outpatient clinic. It is mandatory quickly to recognize and treat a potential life-threatening upper airway obstruction or anaphylaxis. This review focuses on the underlying mechanisms and management of histamine- and bradykinin-induced angioedema at the emergency department and provides an update on the currently available treatments.

Burden of Illness and Quality-of-Life Measures in Angioedema Conditions

Burden of illness studies and evaluation of health-related quality of life using validated questionnaires have become an important task in the comprehensive management of angioedema conditions, mainly angioedema associated with chronic spontaneous urticaria and hereditary angioedema caused by C1-inhibitor deficiency. A review of the principal tools and studies is presented. Both diseases present a higher proportion of psychiatric disorders, impair work and studies productivity, and produce high direct and indirect costs. These assessments also have been useful to evaluate the positive impact of new drugs and interventions. More studies are desirable, especially in other types of angioedema disorders, such as hereditary angioedema with normal C1 inhibitor.

Classification of angioedema without wheals

We read with interest the article by Azofra et al regarding off-label treatment of some patients with idiopathic nonhistaminergic acquired angioedema (InH-AAE) with omalizumab. 1 Besides describing the response to omalizumab of some patients with InHAAE, this article opens the discussion on the need to improve the classification of patients with angioedema without wheals. 2 Concerning InH-AAE, Cicardi et al in their consensus report claim that the term bradykinin mediated should be substituted for nonhistaminergic because, although bradykinin is assumed to mediate this type of angioedema, experimental evidence supporting this mechanism is limited. On the other hand, idiopathic acquired angioedema is classified into histaminergic and nonhistaminergic based on the prevention of angioedema by antihistamines. This way, InH-AAE comprises a heterogeneous group of patients with angioedema mediated by bradykinin but also by other mediators, such as cysteinyl leukotrienes, prostaglandins, or platelet-activating factor, and therefore with very different treatment approaches. This classification could lead to misunderstanding because the term nonhistaminergic is not synonymous of an assumed bradykinin-mediated angioedema. When reading the article by Azofra et al, physicians not very familiar with angioedema consensus and guidelines could interpret that bradykininmediated angioedema could be treated with omalizumab. InH-AAE has therefore been described as a heterogeneous group and should not be assumed to be bradykinin mediated. 1,2 The response to omalizumab of some patients with InH-AAE 1 supports that this type of angioedema is not synonymous with idiopathic bradykinergic angioedema. Moreover, it should be clarified which patients with InH-AAE should be treated with immunosuppressors or omalizumab and which ones with bradykinin-targeting drugs. In this regard, clinical characteristics of angioedema and response to corticosteroids or epinephrine in acute episodes should be considered. The patients described by

Diagnosis Of Aspirin-induced Asthma Combining The Bronchial And The Oral Challenge Tests

BACKGROUND We investigated the usefulness of the bronchial challenge (BC) with lysine-acetylsalicylate (L-ASA) in the diagnosis of aspirin-exacerbated respiratory disease (AERD) using a protocol that combined both the oral challenge (OC) and the BC tests. METHODS Adult asthmatic patients with suspected AERD who underwent BC with L-ASA were included in the study. If the BC result with L-ASA was negative, an OC was carried out to establish the diagnosis. AERD was ruled out if both the BC and the OC results were negative (nonresponders). Both responders and nonresponders were compared for age, gender, a personal or family history of atopy, underlying disease, current asthma treatment, and presence of nasal polyps. Six patients with asthma but no suggestive history of AERD were included as controls. RESULTS Twenty-two patients completed the study. Ten patients tested positive to the BC and/or OC (responders), whereas 12 did not (nonresponders). Seven out of the 10 responders had a positive BC result and 3 a positive OC result. After BC, 4 patients had an early asthmatic response, 1 had a dual response, and 2 had isolated late responses. No significant differences were observed in the aforementioned variables between responders and nonresponders. The results of both challenges were negative in the 6 controls. CONCLUSIONS The BC had a high positive predictive value, was safe, and when negative, the subsequent OC did not result in any severe adverse reactions. The BC elicited an isolated late asthmatic response that has not been previously described in the literature.