Hereditary angioedema (HAE) is a rare but potentially life-threatening disease marked by spontaneous, recurrent attacks of swelling. The broad range of consequences of HAE on patients' lives is not well understood. The study objective was to comprehensively characterize the burden of illness and impact of HAE types I and II from the patient perspective. The HAE Burden of Illness Study in Europe was conducted in Spain, Germany, and Denmark to assess the real-world experience of HAE from the patient perspective via a one-time survey, which included items on clinical characteristics and physical and emotional impacts. One hundred eighty-six patients participated; 59% reported having an attack at least once a month, 67% reported moderate-to-severe pain during their last attack, and 74% reported moderate-to-severe swelling. The most common sites of the last attack were the abdomen and extremities; 24% experienced an attack in more than one site. The impact of HAE on daily activities was high during attacks and did not vary significantly by body site affected; patients also reported that HAE impacted their daily activities between attacks. Patients reported substantial anxiety about future attacks, traveling, and passing HAE to their children. Based on Hospital Anxiety and Depression Scale scores, 38 and 14% had clinically meaningful anxiety and depression, respectively. Despite standard of care, HAE patients still have frequent and painful attacks. Patients experience substantial impairment physically and emotionally both during and between attacks. A better understanding of these effects may help in the clinical management of HAE patients.

Background: Formative qualitative research is foundational to the methodological development process of quantitative health preference research (HPR). Despite its ability to improve the validity of the quantitative evidence, formative qualitative research is underreported.Objective: To improve the frequency and quality of reporting, we developed guidelines for reporting this type of research. The guidelines focus on formative qualitative research used to develop robust and acceptable quantitative study protocols and corresponding survey instruments in HPR.Methods: In December 2018, a steering committee was formed as a means to accumulate the expertise of the HPR community on the reporting guidelines (21 members, seven countries, multiple settings, and disciplines). Using existing guidelines and examples, the committee constructed, revised, and refined the guidelines. The guidelines underwent beta-testing by three researchers and further revision to the guidelines were made based on their feedback as well as from comments from members of the International Academy of Health Preference Research (IAHPR) and the editorial board of The Patient.Results: The guidelines have five components: introductory material (4 domains); methods (12); results/findings (2); discussion (2); and other (2). They are concordant with existing guidelines, published examples, beta testing results, and expert comments.Conclusions: Publishing formative qualitative research is a necessary step towards strengthening the foundation of any quantitative study, enhancing the relevance of its preference evidence. The guidelines should aid researchers, reviewers and regulatory agencies as well as promote transparency within HPR more broadly. Key Points for Decision Makers• These are the first guidelines on reporting formative qualitative research on patient experience that support the development of quantitative preference study protocols and corresponding instruments. • These guidelines focus on reporting techniques that enhance transparency and trustworthiness, thereby improving the likelihood that the scientific contributions of quantitative preference studies are well-founded, improving the validity of the quantitative evidence.

BackgroundHereditary angioedema (HAE) due to C1 inhibitor deficiency is a rare but serious and potentially life-threatening disease marked by spontaneous, recurrent attacks of swelling. The study objective was to characterize direct and indirect resource utilization associated with HAE from the patient perspective in Europe.MethodsThe study was conducted in Spain, Germany, and Denmark to assess the real-world experience of HAE via a cross-sectional survey of HAE patients, including direct and indirect resource utilization during and between attacks for patients and their caregivers over the past 6 months. A regression model examined predictors of medical resource utilization.ResultsOverall, 164 patients had an attack in the past 6 months and were included in the analysis. The most significant predictor of medical resource utilization was the severity of the last attack (OR 2.6; p < 0.001). Among patients who sought medical care during the last attack (23%), more than half utilized the emergency department. The last attack prevented patients from their normal activities an average of 4–12 hours. Patient and caregiver absenteeism increased with attack severity and frequency. Among patients who were working or in school (n = 120), 72 provided work/school absenteeism data, resulting in an estimated 20 days missing from work/school on average per year; 51% (n = 84) indicated that HAE has hindered their career/educational advancement.ConclusionHAE poses a considerable burden on patients and their families in terms of direct medical costs and indirect costs related to lost productivity. This burden is substantial at the time of attacks and in between attacks.

Venous stasis ulcers (VSU) account for approximately 80-90% of lower extremity ulcerations. Given their prevalence and chronic nature, VSU are thought to impose a significant economic burden on Medicare (the USA's largest health insurance program) and other third party payers. However, comprehensive studies on the costs of VSU treatment are lacking. The objective of this study therefore was to examine comprehensively the direct medical costs of treating patients with a VSU in routine clinical practice. A cohort of 78 patients who presented with a VSU to the Cleveland Clinic Foundation (CCF), a large primary and tertiary referral center, was studied retrospectively. All inpatient and outpatient costs related to VSU treatment that were incurred during the year following VSU presentation or until the ulcer healed, whichever occurred first, were quantified. A total of 71 (91%) patients healed during the study. The average duration of follow-up was 119 days (median: 84 days). The average number of visits per patient was seven (range: 2 to 57). A total of 14 (18%) patients underwent 18 hospitalizations for VSU care. The average total medical cost per patient was $9685 (median: $3036). Home health care, hospitalizations and home dressing changes accounted for 48%, 25% and 21% of total costs, respectively. Total costs were related to duration of active therapy, ulcer size and the presence of at least one comorbidity (p<0.05). VSU are costly to manage, especially when time to healing is prolonged. The present findings reflect an underestimate of VSU costs since indirect costs were not examined. Time absent from work, forced early retirement, loss of functional independence and unquantifiable suffering may be additional factors that contribute to the overall burden of VSU.

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