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A cross-sectional questionnaire assessing patient and physician use of short-term prophylaxis for hereditary angioedema.

ABSTRACT: Current guidelines recommend short-term prophylaxis (STP) before invasive procedures to prevent hereditary angioedema (HAE) attacks; however, adherence to these guidelines may be variable because this indication lacks Food and Drug Administration approval in the United States.To ascertain the STP experiences of patients with HAE and HAE-treating physicians.Online questionnaires focusing on STP experiences were distributed by the US Hereditary Angioedema Association to the first 250 patients with HAE and to registered HAE-treating physicians. SAS 9.3 was used to perform descriptive statistics and to test the difference between patients who underwent procedures and those who did not using Pearson ?(2) test, Fisher exact test, and 2-sample t test.For the patient survey, 219 respondents met the criteria for HAE type 1 and 2; 37 (17%) underwent 66 invasive procedures, and all reported receiving STP. Eight patients (22%) reported failed STP, but only 3 required on-demand therapy. For STP, anabolic steroids and plasma-derived C1 inhibitor were the most and second-most commonly used, respectively. For the physician survey, 37 physicians reported caring for 433 patients with HAE. Depending on the procedure, 19% to 54% of physicians used STP and 30% to 86% prescribed on-demand therapy; 69% and 78% of physicians prescribed plasma-derived C1 inhibitor as STP for minimally invasive and invasive procedures, respectively. Physicians reported excellent efficacy for the STP treatments used.Physicians reported excellent outcomes using primarily newer STP therapies, namely plasma-derived C1 inhibitor, which was discordant to patient-reported outcomes using older STP therapies, namely anabolic steroids. Well-controlled STP studies are needed to clarify use for patients with HAE in the United States.

SUBMITTER: Nanda MK

PROVIDER: S-EPMC4141470 | biostudies-literature | 2014 Aug

REPOSITORIES: biostudies-literature

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A cross-sectional questionnaire assessing patient and physician use of short-term prophylaxis for hereditary angioedema.

Nanda Maya K MK Singh Umesh U Wilmot Joyce J Bernstein Jonathan A JA

Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology 20140611 2

<h4>Background</h4>Current guidelines recommend short-term prophylaxis (STP) before invasive procedures to prevent hereditary angioedema (HAE) attacks; however, adherence to these guidelines may be variable because this indication lacks Food and Drug Administration approval in the United States.<h4>Objective</h4>To ascertain the STP experiences of patients with HAE and HAE-treating physicians.<h4>Methods</h4>Online questionnaires focusing on STP experiences were distributed by the US Hereditary ...[more]

PMID: 24928306

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Project description:BackgroundHereditary angioedema (HAE) is a rare disease characterized by unpredictable, recurring subcutaneous or submucosal swelling. Without effective therapy, HAE can negatively impact patients' quality of life. Management of HAE includes on-demand treatment of attacks and short- and long-term prophylaxis (LTP) to prevent attacks. Newer therapies may be more tolerable and effective in managing HAE; however, therapies such as androgens are still widely used in some countries owing to their relative ease of access and adequate disease control for some patients. This study evaluated the characteristics, treatment patterns, clinical outcomes, and healthcare resource utilization of a multinational cohort of patients with HAE, with a focus on understanding reasons for recommending or discontinuing available therapies.MethodsA retrospective chart review was conducted at 12 centers in six countries and included data from patients with HAE type 1 or 2 who were ≥ 12 years of age at their first clinical visit. The relationship between LTP use and attack rates was evaluated using a multivariable Poisson regression model. Data were collected between March 2018 and July 2019.ResultsData from 225 patients were collected (62.7% female, 86.2% White, 90.2% type 1); 64.4% of patients had their first HAE-related visit to the center prior to or during 2014. Treatment patterns varied between countries. Overall, 85.8% of patients were prescribed on-demand treatment and 53.8% were prescribed LTP, most commonly the androgen danazol (53.7% of patients who used LTP). Plasma-derived C1 inhibitor (Cinryze®) was used by 29.8% of patients for LTP. Patients who received LTP had a significantly lower rate of HAE attacks than patients who did not receive any LTP (incidence rate ratio (95% confidence interval) 0.90 (0.84-0.96)). Androgens were the most commonly discontinued therapy (51.3%), with low tolerability cited as the most frequent reason for discontinuation (50.0%).ConclusionsOverall, findings from this study support the use of LTP in the prevention of HAE attacks; a lower rate of attacks was observed with LTP compared with no LTP. However, the type of LTP used varied between countries, with tolerability and accessibility to specific treatments playing important roles in management decision-making.

Project description:BackgroundThere is considerable burden of illness in hereditary angioedema (HAE). However, instruments to assess health-related quality of life (HRQoL) in HAE are limited. The Angioedema Quality of Life Questionnaire (AE-QoL) was developed to measure HRQoL in patients with recurrent angioedema; the validity of the AE-QoL in patients with HAE is described.MethodsTo identify disease-related experiences with a focus on the impact of HAE on HRQoL, interviews were conducted with a group of clinician experts and patients with HAE from Canada, France, Germany, Spain, the United Kingdom, and the United States, along with a targeted literature review. Concepts were mapped to the AE-QoL to assess item relevance, interpretation, and conceptual coverage. Cognitive interviews assessed item clarity and relevance. A psychometric validation was performed using data from a phase 3 trial.ResultsInterviews were conducted with seven clinicians and 40 adult patients. Patients reported 35 unique impacts of HAE on their lives, the most frequent being on work/school, social relationships, physical activities, and emotions, particularly fear/worrying and anxiety. Saturation for these impacts was reached, and all concepts covered in the AE-QoL were reported during the interviews. Patients agreed that the questionnaire items and response options were clear and relevant, and the 4-week recall period was appropriate. The psychometric validation included data from 64 patients. For AE-QoL total scores, excellent internal consistency (Cronbach's alpha > 0.90), test-retest reliability (intraclass coefficient > 0.80), convergent validity with the Sheehan Disability Scale (r = 0.663), divergent validity with the EQ-5D-5L index (r = 0.292) and EQ-VAS (r = 0.337), and known-groups validity (p < 0.0001; ɳ2 = 0.56) were demonstrated.ConclusionsQualitative and psychometric analyses showed that the AE-QoL is a reliable and valid instrument for measuring HRQoL in adult patients with HAE from six countries.

Project description:BackgroundIn China, the utilization of medical resources is tense, and most hospitals are highly congested because of the large population and uneven distribution of medical resources. Online health communities (OHCs) play an important role in alleviating hospital congestions, thereby improving the utilization of medical resources and relieving medical resource shortages. OHCs have positive effects on physician-patient relationships and health outcomes. Moreover, as one of the main ways for patients to seek health-related information in OHCs, physician-patient communication may affect patient compliance in various ways. In consideration of the inevitable development of OHCs, although they have several shortcomings, identifying how physician-patient communication can impact patient compliance is important to improve patients' health outcomes through OHCs.ObjectiveThis study aimed to investigate the impact of physician-patient communication on patient compliance in OHCs through the mediation of the perceived quality of internet health information, decision-making preference, and physician-patient concordance, using an empirical study based on the self-determination theory.MethodsA research model was established, including 1 independent variable (physician-patient communication), 3 mediators (perceived quality of internet health information, decision-making preference, and physician-patient concordance), 1 dependent variable (patient compliance), and 4 control variables (age, gender, living area, and education level). Furthermore, a Web-based survey involving 423 valid responses was conducted in China to collect data, and structural equation modeling and partial least squares were adopted to analyze data and test the hypotheses.ResultsThe questionnaire response rate was 79.2% (487/615) and the validity rate was 86.9% (423/487); reliability and validity are acceptable. The communication between physicians and patients in OHCs positively affects patient compliance through the mediation of the perceived quality of internet health information, decision-making preference, and physician-patient concordance. Moreover, physician-patient communication exhibits similar impacts on the perceived quality of internet health information, decision-making preference, and physician-patient concordance. Patients' decision-making preference shows the weakest impact on patient compliance compared with the other 2 mediators. Ultimately, all 3 mediators play a partially mediating role between physician-patient communication and patient compliance.ConclusionsWe conclude that physician-patient communication in OHCs exhibits a positive impact on patient compliance; thus, patient compliance can be improved by guiding physician-patient communication in OHCs. Furthermore, our findings suggest that physicians can share high-quality health information with patients, discuss benefits, risks, and costs of treatment options with patients, encourage patients to express their attitudes and participate in health-related decision making, and strengthen the emotional connection with patients in OHCs, thereby decreasing patients' misunderstanding of information and increasing concordance between physicians and patients. OHCs are required to not only strengthen the management of their published health information quality but also understand users' actual attitudes toward information quality and then try to reduce the gap between the perceived and actual quality of information.

Dataset Information

A cross-sectional questionnaire assessing patient and physician use of short-term prophylaxis for hereditary angioedema.

Publications

A cross-sectional questionnaire assessing patient and physician use of short-term prophylaxis for hereditary angioedema.

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