Clinical characteristics and burden of illness in patients with hereditary angioedema: findings from a multinational patient survey.

BACKGROUND: Hereditary angioedema (HAE) is a rare, debilitating, genetic disease characterized by unpredictable, recurrent, and potentially fatal swelling of the skin and mucous membranes. We conducted a noninterventional, cross-sectional, web-based survey of patients with a self-reported diagnosis of HAE type 1/2 in Australia, Austria, Canada, France, Germany, Spain, Switzerland, and the United Kingdom to gain a comprehensive real-world understanding of the characteristics of HAE and its burden from the perspective of the patient. The survey included questions on clinical and demographic characteristics, burden of disease, and treatment. Instruments used to measure patient-reported outcomes included the Angioedema Quality of Life questionnaire (AE-QoL), 12-Item Short-Form Health Survey (SF-12v2), Angioedema Control Test (AECT), Hospital Anxiety and Depression Scale (HADS), and Work Productivity and Impairment questionnaire (WPAI). Data were analyzed with descriptive statistics. RESULTS: A total of 242 patients (67.4% female; mean [range] age 43.8 [18-92] years) completed the survey. The mean (SD) age at first symptoms was 11.5 (8.9) years, while diagnosis occurred at 20.8 (13.2) years. Patients reported a mean (SD) of 12.5 (14.1) attacks in the past 6 months. The most recent attack occurred within the past month in 79.7% of patients; most were of moderate severity, 6.6% affected the larynx, 21.9% lasted ≥ 3 days, and 76.4% were treated with on-demand medication. Hospitalizations and emergency/urgent care visits were highest for patients with more attacks. At the time of the survey, 62.4% of patients were using long-term prophylaxis, including 34.4% using androgens. Moderate to severe anxiety and depression were reported in 38.0% and 17.4% of patients, respectively, as measured using the HADS. The severity of anxiety and depression was associated with poorer quality of life and productivity, measured using the AECT (mean overall score 8.00 [moderate perceived disease control]), AE-QoL, WPAI, and SF-12v2. Scores for AECT, AE-QoL, and WPAI were also worse with a higher number of attacks. CONCLUSIONS: This survey study of a broad international sample of patients with HAE showed that despite the availability of on-demand treatment and long-term prophylaxis for the prevention of attacks, patients across a wide geographical area continue to have high disease activity, likely due to restrictions in the availability of medications or incorrect use. Subsequently, significant disease burden, including impaired quality of life and mental health and decreased productivity, was evident. Increased patient education and access to newer, more effective therapies are needed.

Does scrolling affect measurement equivalence of electronic patient-reported outcome measures (ePROM)? Results of a quantitative equivalence study.

BACKGROUND: Scrolling is a perceived barrier in the use of bring your own device (BYOD) to capture electronic patient reported outcomes (ePROs). This study explored the impact of scrolling on the measurement equivalence of electronic patient-reported outcome measures (ePROMs) in the presence and absence of scrolling. METHODS: Adult participants with a chronic condition involving daily pain completed ePROMs on four devices with different scrolling properties: a large provisioned device not requiring scrolling; two provisioned devices requiring scrolling - one with a "smart-scrolling" feature that disabled the "next" button until all information was viewed, and a second without this feature; and BYOD with smart-scrolling. The ePROMs included were the SF-12, EQ-5D-5L, and three pain measures: a visual analogue scale, a numeric response scale and a Likert scale. Participants completed English or Spanish versions according to their first language. Associations between ePROM scores were assessed using intraclass correlation coefficients (ICCs), with lower bound of 95% confidence interval (CI) > 0.7 indicating comparability. RESULTS: One hundred fifteen English- or Spanish-speaking participants (21-75y) completed all four administrations. High associations between scrolling and non-scrolling were observed (ICCs: 0.71-0.96). The equivalence threshold was met for all but one SF-12 domain score (bodily pain; lower 95% CI: 0.65) and two EQ-5D-5L item scores (pain/discomfort, usual activities; lower 95% CI: 0.64/0.67). Age, language, and device size produced insignificant differences in scores. CONCLUSIONS: The measurement properties of PROMs are preserved even in the presence of scrolling on a handheld device. Further studies that assess scrolling impact over long-term, repeated use are recommended.

Patient perspectives on the pathway to psoriatic arthritis diagnosis: results from a web-based survey of patients in the United States.

BACKGROUND: There are limited real-world data on the diagnostic experiences of patients with psoriatic arthritis (PsA), including medical care sought and potential barriers to diagnosis. We aim to describe patient experiences related to receiving a PsA diagnosis. METHODS: Ours was a mixed-method, 2-phase study. Phase 1 comprised concept elicitation and cognitive interviews with clinical experts and adults diagnosed with PsA to develop a cross sectional, web-based survey. US adults with a self-reported PsA diagnosis were recruited through a patient support community (CreakyJoints), an online patient research registry (ArthritisPower), and social media outreach. In Phase 2, the online survey collected data on sociodemographics, clinical symptoms, disease burden, and diagnosis history of survey respondents with PsA. RESULTS: Of the 203 respondents included, 172 (84.7%) were female, and the mean (SD) age was 51.6 (10.8) years. The time between seeking medical attention and receiving a diagnosis was < 6 months for 69 respondents, 6 months to 4 years for 68 respondents, and ≥ 5 years for 66 respondents. Most respondents sought care from general practitioners (79.8%) and rheumatologists (66.5%). Common initial symptoms that led respondents to seek medical attention were joint pain (70.0%) and stiffness (53.7%). Among the initial symptoms that led respondents to seek care, joint pain, swollen joints, and sausage-like fingers or toes (indicating dactylitis) were more common among respondents with shorter time to diagnosis, whereas stiffness, fatigue, enthesitis (indicated by foot problems, tendon and ligament pain), and back pain were more common among respondents with longer time to diagnosis. Common misdiagnoses were psychosomatic issues (26.6%) and osteoarthritis (21.7%). Respondents with shorter times to diagnosis had lower frequencies of misdiagnosis. CONCLUSIONS: Respondents with PsA reported delays in diagnosis and misdiagnoses on their journey to a PsA diagnosis. Symptom differences, such as enthesitis and stiffness, were noted among respondents with shorter vs longer time to diagnosis. Increased understanding of diagnostic barriers may lead to earlier diagnosis and appropriate management to improve outcomes.

Content validity of a novel patient-reported and observer-reported outcomes assessment to evaluate ocular symptoms associated with infectious conjunctivitis in both adult and pediatric populations.

BACKGROUND: Acute infectious conjunctivitis is a common condition most frequently caused by viruses or bacteria. Clinical outcome assessments have been used to assess signs and symptoms of bacterial and viral conjunctivitis, but have not been evaluated for content validity. We aimed to develop content-valid patient- (PRO) and observer-reported outcome (ObsRO) instruments to assess symptoms of ocular discomfort associated with viral or bacterial conjunctivitis in adult and pediatric patients. METHODS: Draft items were developed from a previous review of published studies from 2001 to 2015. Patients and caregivers of patients with a diagnosis of viral or bacterial conjunctivitis within the past 6 months were recruited. Concept elicitation with open-ended questions explored signs and symptoms, followed by cognitive interviewing to assess clarity and relevance of the draft items. Patients aged ≥8 years were interviewed for the PRO; parents/caregivers of children aged 1-10 years were interviewed for the ObsRO. Interviews were conducted in three rounds to allow changes. Concept saturation was documented using a saturation grid. Cognitive interview data were analyzed iteratively and focused on clarity, relevance and inconsistent interpretation of the instrument's content. RESULTS: Overall, 23 patients or parents/caregivers participated (round 1, n = 10; round 2, n = 6; round 3, n = 7). Data saturation was reached by the 16th interview. The most frequent spontaneously reported signs/symptoms were: discharge, red/pink eyes, itchiness, swelling/puffiness, watery eyes, pain, burning and foreign body sensation. Itching, pain/burning/stinging and foreign body sensation were most commonly reported as the top three most bothersome symptoms. Interview results indicated that items on pain, itching and foreign body sensation for the PRO and pain or discomfort for the ObsRO were relevant to the patients' experience of conjunctivitis and were clear and easy to understand. CONCLUSIONS: PRO and ObsRO items were found to be clear, relevant and appropriate in assessing key viral and bacterial conjunctivitis symptoms in adult and pediatric patients.

Real-World Patient Experience on the Path to Diagnosis of Ankylosing Spondylitis.

INTRODUCTION: We describe the journey to diagnosis of ankylosing spondylitis (AS) from the patient perspective and examine differences in this journey by sex. METHODS: US adults aged ≥ 18 years with a self-reported AS diagnosis were recruited online through CreakyJoints, a patient support community, and ArthritisPower, a patient research registry. Respondents completed a web-based survey on sociodemographics, disease burden, and diagnosis history. Results were stratified by sex and time to diagnosis using two-sample t tests and χ2 tests, respectively, to observe differences across the groups; P < 0.05 was considered statistically significant. RESULTS: Among 235 respondents, 174 (74.0%) were female. Mean (SD) ages of female and male respondents were 48.6 (10.6) and 53.1 (10.3) years, respectively. From the time respondents began seeking medical attention, 87 were diagnosed within ≤ 1 year, 71 in 2-9 years, and 77 after ≥ 10 years. Symptoms that led respondents to seek treatment were back pain (73.2%) and joint pain (63.8%); fatigue and difficulty sleeping were more common among respondents with longer times to diagnosis. During the diagnosis process, men with AS tended to receive quicker AS diagnosis compared with women. Overall, commonly reported initial diagnoses among respondents with longer time to AS diagnosis included back problems and psychosomatic disorders. Significantly more women reported misdiagnoses of fibromyalgia (20.7 vs. 6.6%) and psychosomatic disorders (40.8 vs. 23.0%) compared with men. CONCLUSIONS: Diagnosis delays and misdiagnoses were common among respondents with AS. Increasing awareness about AS among referring providers may minimize diagnosis delay. FUNDING: Novartis Pharmaceuticals Corporation. Plain language summary available for this article.

Glucagon-Like Peptide-1 Receptor Agonist Treatment Attributes Important to Injection-Experienced Patients with Type 2 Diabetes Mellitus: A Preference Study in Germany and the United Kingdom.

INTRODUCTION: This study assessed the relative importance of treatment-related attributes in influencing patient preferences for glucagon-like peptide-1 receptor agonists (GLP-1RAs) among injection-experienced type 2 diabetes mellitus (T2DM) patients in Germany and the United Kingdom. METHODS: T2DM patients experienced with injecting once-weekly (QW) exenatide or once-daily (QD) liraglutide completed an online discrete-choice experiment (DCE) survey. Patients chose between hypothetical blinded treatment profiles reflecting attributes of GLP-1RAs. The DCE survey included eight attributes: efficacy, side effects, device size, needle size, titration, injection preparation, long-term efficacy/safety, and dosing frequency. Odds ratios (ORs) and 95% confidence intervals (CIs) were calculated using a conditional logit model indicating the likelihood of choosing a treatment with a given attribute level versus a reference attribute level. RESULTS: 510 GLP-1RA injection-experienced patients completed the survey; 45.3% respondents were being treated with exenatide QW and 54.7% respondents were being treated with liraglutide QD. In terms of GLP-1RA attributes, patients indicated a preference for a treatment with greater efficacy (i.e., a 1.5-point improvement in HbA1c) (OR 2.58; 95% CI 2.37, 2.80; p < 0.001), fewer side effects (OR 2.67; 95% CI 2.52, 2.82; p < 0.001), once-weekly rather than once-daily administration (OR 2.26; 95% CI 2.13, 2.39; p < 0.001), and the preparation required for a multi-use pen (OR 1.71; 95% CI 1.55, 1.88; p < 0.001). Needle size, device size, and titration were not significant drivers of patient preference. CONCLUSIONS: Among GLP-1RA injection-experienced patients, key drivers of treatment preference for a hypothetical GLP-RA profile were side effects, efficacy, dosing frequency, and required preparation. Understanding patient preferences is important for optimizing treatment decision-making and improving treatment adherence. FUNDING: AstraZeneca.

Glucagon-like Peptide-1 Receptor Agonist Treatment Attributes Important to Injection-Naïve Patients with Type 2 Diabetes Mellitus: A Multinational Preference Study.

INTRODUCTION: Glucagon-like peptide-1 receptor agonists (GLP-1RAs) differ in efficacy, side effects, dosing frequency, and device-related attributes. This study assessed the relative importance of treatment-related attributes in influencing preferences for GLP-1RAs among injection-naïve patients with type 2 diabetes mellitus (T2DM). METHODS: Injection-naïve T2DM patients from five countries completed a Web-based discrete choice experiment (DCE) survey. Patients chose between hypothetical treatment profiles reflecting important and differentiating attributes of GLP-1RAs. Eight attributes were included: efficacy, side effects, device size, needle size, titration, preparation, evidence of long-term efficacy/safety, and dosing frequency. Odds ratios (ORs) and 95% confidence intervals were calculated using a conditional logit model to indicate the likelihood of choosing a treatment with a given attribute level versus a reference attribute level. The influence of individual attributes when considering full treatment profiles was examined using exenatide once weekly (QW) and liraglutide once daily (QD) as case examples. RESULTS: A total of 1482 patients with T2DM completed the DCE survey. Side effects, efficacy, and dosing frequency were the three most important attributes influencing preferences; needle size, device size, and required preparation were least important. Total sample analysis indicated that a profile of GLP-1RA approximating exenatide QW (single pen) was preferred over a profile approximating liraglutide QD (OR 3.36; p < 0.001), when efficacy was assumed to be equal. CONCLUSION: The most influential drivers of treatment preferences for a hypothetical GLP-RA profile were side effects, efficacy, and dosing frequency among injection-naïve T2DM patients. Preference elicitation can promote patient-centered care and inform new generations of T2DM treatments, which can lead to improved adherence and health outcomes.

Patient preferences for diabetes treatment attributes and drug classes.

OBJECTIVE: To identify which treatment attributes are most influential in determining patient preferences for diabetes treatments and explore patient preferences for diabetes drug classes. RESEARCH DESIGN AND METHODS: US adults with type 1 or type 2 diabetes completed an online adaptive conjoint analysis survey. The survey examined 14 attributes, including efficacy, regimen, and risk of common side effects and rare but serious adverse events. Respondents selected between hypothetical treatments with different attributes. Sawtooth Software, ordinary least-squares regression, and hierarchical Bayes regression were used to calculate utilities (i.e. preference weights), importance ratings, and shares of preference across 13 diabetes drug classes or combination products. RESULTS: A total of 167 adults (mean age 58 years; 55% female) with type 1 or type 2 diabetes completed the survey. Based on importance ratings, the most influential attributes driving preferences were regimen, risk of diarrhea, weight change, risk of hypoglycemia, and efficacy. Sodium-glucose co-transporter-2 inhibitors (SGLT-2is) were highly preferred in direct comparison to each of the other classes (range: 84.2-99.9%), with the exception of dipeptidyl peptidase-4 inhibitors (DPP-4is); DPP-4is (52.9%) were preferred over SGLT-2is (47.1%). CONCLUSIONS: Although preferences varied across participants, attributes with the greatest likelihood of affecting daily life and routine were generally more influential in determining patient preferences. DPP-4is and SGLT-2is were overwhelmingly preferred over other drug classes, primarily due to favorable regimen and side effect profiles. Understanding patient preferences can help optimize patient-centered treatment and may lead to improved patient satisfaction, adherence, and outcomes. LIMITATIONS: The primary limitations of this study are that a small sample size of type 1 diabetes patients were included, which may reduce the reliability of the preference estimates, and patients were recruited from a patient panel and may not be representative of patients with diabetes in the US.