Real-world Survey Research Articles

Characteristics, clinical outcomes and patient-reported outcomes of patients with ulcerative colitis receiving tofacitinib: a real-world survey in the United States and five European countries

BackgroundTo describe variations in treatment patterns, clinical outcomes, patient-reported outcomes (PRO), and physician and patient satisfaction in patients with moderate-to-severe ulcerative colitis (UC) treated with tofacitinib in a real-world setting.MethodsData were drawn from the Adelphi UC Disease Specific Programme™, a point-in-time survey of physicians and their consulting patients in the US and Europe. For inclusion in this analysis, gastroenterologists completed medical record forms for the next seven consecutive consulting patients with confirmed UC, plus a further two patient record forms for patients treated with tofacitinib. Those same patients then completed a patient-reported questionnaire.ResultsGastroenterologists (n = 340) provided data for 2049 patients with UC, including 642 patients receiving tofacitinib. Physicians’ most frequent reason for choosing tofacitinib was overall efficacy (71.3% of patients). The proportion of patients in remission increased with length of treatment, from 13.7% at [0, 4) weeks to 68.3% at [52+] weeks. Both physicians and patients reported that the Mayo components of stool frequency and blood in stool were reduced with time on treatment. Improvement in symptoms (bloody diarrhea, abdominal pain/cramps, urgency, rectal bleeding, fatigue/tiredness) was reported in the first weeks of treatment, and increased with time. At week [52+], mean score reductions from treatment initiation to current in overall symptom severity, pain, and fatigue were 2.2 (to a current mean score of 1.1), 2.2 (to 0.9), and 2.1 (to 1.0), respectively. Comparing patients at weeks [0, 4) and [52+] (all PROs, p < 0.0001), the increase in EQ-5D-5L index total score was 0.29 points and in SIBDQ total score was 20.5 points; percent reductions in WPAI absenteeism was 34.4%, presenteeism 26.8%, overall work impairment 40.9% and activity impairment was 28.3%. These changes reached the thresholds for minimally clinically important differences. The majority of physicians (91.9%) and patients (93.5%) were satisfied with tofacitinib at week [52+].ConclusionPatients with moderate-to-severe UC treated with tofacitinib show considerable improvement in symptoms and quality of life from tofacitinib initiation to one year and beyond, with high rates of remission. Physicians and patients report satisfaction with UC control at recommended doses in a mostly biologic experienced population.

Relationships between body mass index and constipation, gastroesophageal reflux disease, stool forms based on the Bristol Stool Form Scale, and education level: results from an internet survey in Japan.

Detailed evaluations of body mass index (BMI) and stool form based on the Bristol Stool Form Scale (BSFS) in individuals with constipation, gastroesophageal reflux disease (GERD), and concomitant constipation and GERD have not been performed in Japan. This study was an internet survey conducted to examine the relationships between BMI and constipation, GERD, stool forms based on the BSFS, and education level. This internet-based survey recruited participants from general public survey panels. 10,000 individuals meeting the eligibility criteria were enrolled. Questions included demographics, medical data, and assessments based on validated measures for constipation and GERD. BMI was significantly lower in males with versus without constipation. BMI was significantly higher with GERD both males and females. Mean BMI increased from the BSFS-1/2 group through the BSFS-3/4/5 to the BSFS-6/7 groups in both sexes. BMI was highest in individuals with a maximum education level of junior high school and second highest in individuals completing high school. This is the first real-world survey that closely examines the relationship between BMI and stool forms of individuals in Japan. When the BMI increased, stool forms varied from hard to watery in Japanese people. BMI was related with education level in Japan. (Trial registration: UMIN000039688).

Modeling Semantic Correlation and Hierarchy for Real-World Wildlife Recognition

We explore the challenges of human-in-the-loop frameworks to label wildlife recognition datasets with a neural network. In wildlife imagery, the main challenges for a model to assist human annotation are two-fold: (1) the training dataset is usually imbalanced, which makes the model's suggestion biased, and (2) there are complex taxonomies in the classes. We establish a simple and efficient baseline, including the debiasing loss function and the hyperbolic network architecture, to address these issues. Moreover, we propose leveraging the semantic correlation to train the model more effectively by adding a co-occurrence layer to our model during training. We demonstrate the efficacy of our method in both a real-world wildlife areal survey recognition dataset and the public image classification dataset, CIFAR100-LT, CIFAR10-LT, and iNaturalist.

Impact of restricted access to, and low awareness of, mexiletine on people with myotonia: a real-world European survey

Although mexiletine effectively treats myotonia, supply disruptions affected Europe between 2008–2018. MyoPath was a mixed-methods, cross-sectional, market research survey conducted January–June 2018 to evaluate consequences of limited access to/awareness of mexiletine in people with myotonia. Part A: qualitative structured interviews (clinicians; advocates for adult patients); Part B: quantitative online questionnaire completed by people with self-reported history of myotonia. Part A: Interviews (clinicians, n=12; patient advocates, n=5; 12 countries) indicated poor mexiletine awareness among general neurologists. Patients chose between living with myotonia (other treatments were generally unsatisfactory) or importing mexiletine. Part B: Questionnaire respondents, myotonic dystrophy (DM)1, n=213; DM2, n=128; non-dystrophic myotonia (NDM), n=41; other n=8; (11 countries). Of the respondents, 76/390 (20%) people with awareness of/access to mexiletine described profound improvements in myotonia and health-related quality of life following treatment. Respondents with NDM had greatest mexiletine experience (n=28/41). Mexiletine was associated with fewer falls, less muscle stiffness, increased mobility. Treatment interruptions worsened myotonia and were associated with fatigue, pain, dysphagia, breathing difficulty, impaired digestion, poor sleep. However, 36/54 (67%) of currently treated people expressed anxiety about mexiletine's availability: this finding was expected (MyoPath was undertaken before mexiletine's approval in NDM). MyoPath provides the largest European exploration of patients’ views regarding impact of mexiletine on myotonia. Anticipated effects of mexiletine differ between people with different myotonic disorders: myotonia is the main symptom in NDM but one of many potential symptoms affecting those with DM. Nevertheless, findings indicate substantial harm caused to people with myotonia when mexiletine awareness/access is limited.

PCR200 Measuring the Burden of Alopecia Areata With the European Quality of Life-5 Dimensions (EQ-5D): Results From a Real-World Survey in 5 European Countries

PCR200 Measuring the Burden of Alopecia Areata With the European Quality of Life-5 Dimensions (EQ-5D): Results From a Real-World Survey in 5 European Countries

RWD105 Clinical Differences and Patient Reported Outcomes Between Insufficient Responders and Responders to Prescribed Acute Migraine Treatment: Results From a Real-World Patient Survey in China

RWD105 Clinical Differences and Patient Reported Outcomes Between Insufficient Responders and Responders to Prescribed Acute Migraine Treatment: Results From a Real-World Patient Survey in China

Use of prognostic gene expression profiling tests in primary breast cancer treatment: a German real-world patient survey.

Aims: In primary breast cancer, gene expression profiling tests can support adjuvant chemotherapy treatment decisions. Real-world test use in Germany was investigated in an online survey of female breast cancer patients (n=475). Materials & methods: Relationships between three groups were examined for clinical and statistical relevance: no test indication (n=353), test indication and tested (n=65) and test indication but not tested (n=57). Results: Atotal of 47% of participants with a test indication were not tested. Test rates increased by 23% from 2012-2018 (49%) to 2019-2021 (60%). Atotal of 65% of patients without testing received chemotherapy, whereas only 38% of tested patients received chemotherapy. Conclusion: The use of gene expression profiling tests correlates with a real-world chemotherapy reduction. Gene expression profiling testing may improve patient confidence in the decision for or against chemotherapy.

RWD150 Characteristics of Plaque Psoriasis Patients Initiating Guselkumab by Country: A Multi-National, Real-World Survey of Dermatology Practices

RWD150 Characteristics of Plaque Psoriasis Patients Initiating Guselkumab by Country: A Multi-National, Real-World Survey of Dermatology Practices

Evidence Gaps in the Management of Patients with Sickle Cell Disease (SCD) by Non-specialist Healthcare Professionals (HCPs): Results from the Real-World Assessment Survey for SCD in Saudi (ROARS)

The Kingdom of Saudi Arabia (KSA) exhibits an extremely high prevalence of SCD and is regarded as one of the worst-affected countries in the world. Up to 2.6% of the population diagnosed with SCD in parts of KSA, and the Real-World Assessment Survey for SCD in Saudi (ROARS) was conducted to obtain insights on the disease landscape in the country. Here, we present data on the SCD management strategies employed by both specialist and non-specialist HCPs in KSA. Between September and December 2021, HCPs (>2 years SCD experience, SCD load >50 patients, >50% of practice time spent treating SCD patients) completed the survey using the structured, bilingual ROARS questionnaires (English/Arabic) through personal quantitative web-assisted telephone interviews. These HCPs were asked a series of questions covering topics such as patient caseload, patient management strategies, and perceptions of SCD treatment. A total of 64 HCPs responded to the survey, including 31 specialists (adult hematologists, n=24; pediatric hematologists, n=7) and 33 non-specialists. Non-specialist professions included general practice/family medicine/primary care (n=16), internal medicine (n=7), pediatrics (n=9), and medical oncology (n=1). On average, the monthly patient caseload was greater with specialist HCPs than non-specialist HCPs (74 vs 59 patients, respectively). Specialist HCPs were more likely to treat patients with more severe SCD genotypes: 72.3% of patients treated by specialist HCPs were diagnosed with HbSS SCD compared to 57.6% of patients treated by non-specialist HCPs. On average, targeted family screening was performed for 82.1% of patients in the hospitals where specialist HCPs work compared to 58.6% of patients in the hospitals where non-specialist HCPs work. Patients treated by specialist HCPs were also followed-up more frequently than patients treated by non-specialist HCPs (55% vs 33% followed up with their patients three/four times a year, respectively). Differences in the treatments received by patients were evident between HCPs; 72% of patients treated by specialists were receiving hydroxyurea compared to 44% of patients treated by non-specialists. However, no substantial differences in the proportion of patients treated by innovative therapies (L-glutamine, voxelotor, or crizanlizumab) were evident between specialists (7% of patients) and non-specialists (8% of patients). Screening for common SCD complications occurred more frequently in patients treated by specialists than those treated by non-specialists. This was particularly evident for avascular necrosis (45% of patients vs 21% of patients), liver sequestration (29% vs 9%), pulmonary hypertension (42% vs 24%), and nephropathy/renal insufficiency (39% vs 27%). Alloimmunization was screened for in 13% of patients treated by specialist HCPs and 3% of patients treated by non-specialist HCPs; the corresponding screening rates for vision problems/retinopathy were 13% and 12%, respectively. Overall, 65% of specialist HCPs and 36% of non-specialist HCPs reported that uncontrolled vaso-occlusive crises (VOCs) had a high impact on their patients; the impact of other SCD burdens are presented in Figure 1. Both specialist and non-specialist HCPs noted the rarity of pediatric to adult transition programs for SCD patients within hospitals; 68% of specialist HCPs and 73% of non-specialist HCPs reported no program in their treatment center. Overall, 35% of specialist HCPs reported satisfaction with current treatment options for SCD compared to 60% of non-specialist HCPs (Figure 2). Reasons for specialist HCP dissatisfaction with current SCD treatment options included the limited number of treatment options (80%), inability to reach treatment goals set for patients (55%), and inability to provide long term symptomatic relief (35%). Differences in the management of SCD patients between specialist and non-specialist HCPs in KSA were evident in this study. Specialist HCPs perform SCD and SCD complication screening more frequently than non-specialist HCPs and follow up with their patients at more frequent intervals, highlighting the need for effective education and the implementation of clinical practice guidelines to improve patient management. These data also showcase the unmet need for transition programs, and the dissatisfaction with the capability of current treatment options to improve patients' outcomes. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Sickle Cell Disease (SCD) Burden on Patients Treated With Hydroxyurea (HU): Results from the Real-World Assessment Survey for SCD in Saudi (ROARS)

The prevalence of SCD in the Kingdom of Saudi Arabia (KSA) is one of the highest in the world with up to 2.6% of the population affected. The Real-World Assessment Survey for Sickle Cell Disease in Saudi (ROARS) was conducted to evaluate the impact of SCD on the quality of life (QoL) of this population. Here, we present data on the QoL burden experienced by patients with SCD in KSA treated with hydroxyurea (HU). Between September and December 2021, patients in KSA with SCD (age: 5-65 years) were surveyed (caregivers were surveyed for patients 5-14 years) using structured, bilingual ROARS questionnaires (English/Arabic) through personal quantitative web-assisted telephone interviews. The survey includes the Patient-Reported Outcomes Measurement Information System (PROMIS) global health domain, a validated tool for assessing QoL among SCD patients and caregivers. The survey evaluated topics such as the impact of SCD on QoL, symptoms, impact of SCD on productivity at work/school, and treatment goals. A total of 1011 responses to the survey were collected; of these, 32.3% (n=327) of patients were currently being treated with HU (47% of adult and 17% of pediatric patients surveyed) and are the focus of this analysis. Amongst these patients, the median age (range) was 25 (5-83) years. Patients were responsible for reporting their SCD genotype: 58% of respondents were diagnosed with HbSS (n=189), 26% with HbSC (n=86), 2% with HbSβ0 thalassemia (n=8), and 5% with HbSβ+ thalassemia (n=15). Overall, 35% (adults, n=84; pediatric n=25) of patients in the eastern province were treated with HU, 27% (adult, n=71; pediatric, n=17) in the western province, 24% (adult, n=55; pediatric, n=24) in the southern province, and 14% (adult, n=29; pediatric, n=22) in the central province. Among HU-treated patients, 64% reported never ceasing/stopping treatment, 21% rarely ceased/stopped treatment and 15% of patients cease/stop treatment sometimes, often, or all the time. The most common reason for patients ceasing/stopping treatment was forgetting to take their medication (79%). Of patients who cease/stop treatment sometimes, often, or all the time (n=48), 17% of these stated that their medication was not available while 4% could not afford their medication. The mean (SD) number of VOCs reported by HU-treated patients in the 12 months prior to survey completion was 4.6 (4.09), while 32% (n=104) of these patients reported experiencing ≥5 VOCs in the same period. The most common SCD symptoms (other than VOCs) experienced by HU-treated patients were bone ache (reported by 92% of respondents), fatigue/tiredness (85%), headache (79%), anxiety (61%), joint stiffness (56%) and numbness/tingling (51%). Most HU-treated patients reported poor/fair or good health for all QoL metrics assessed (Figure 1). Almost half of patients (49%) reported that their SCD was impactful/extremely impactful on their emotional wellbeing. Of those treated with HU, 71% of patients reported that HU is effective in treating their SCD symptoms. However, 84% of patients report a desire for alternatives to their current pain management medication while 72% report concerns about the long-term side effects of HU. The most important treatment goals for these patients were improvement in quality of life (82%), improvement in overall symptoms (79%), and reduction in the number of severe pain crises and increase in the number of days free from severe pain crises (79%). A total of 48% of HU-treated patients were bothered by limitations in performing everyday activities; 48% and 55% of patients also reported that their SCD has prevented them from progressing in their career or education, respectively. Patients treated with HU reported missing a mean (SD) of 6.9 (10.4) hours of work in the 7 days prior to survey completion because of their SCD. Access to HU treatment continues to be a challenge, with approximately 70% of patients surveyed not receiving HU. Despite the observed clinical benefit in symptom control, patients receiving HU continue to experience disease burden including substantial impact on QoL and productivity at work/school (as evident from the treatment goals identified by patients). These data highlight the necessity of optimizing HU accessibility and treatment strategies to improve patient outcomes. These data also illustrate the need for novel therapeutic options and management strategies for patients with SCD to target patient-reported treatment goals. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Helicobacter pylori Infection Combined with OLGA and OLGIM Staging Systems for Risk Assessment of Gastric Cancer: A Retrospective Study in Eastern China.

Helicobacter pylori (H. pylori) infection is a high-risk factor for gastric cancer (GC). The main aim of this study was to evaluate the effect of H. pylori on gastritis staging systems and the value of H. pylori combined with gastritis staging systems in predicting GC risk. This study enrolled 609 patients with gastric atrophy (GA) and 527 patients with gastric intestinal metaplasia (GIM), who were graded by the OLGA and OLGIM staging systems, respectively. Each individual underwent serum pepsinogen (PG) test, H. pylori detection and questionnaire investigation. We did a real-world retrospective follow-up survey for them in April 2022. Compared with H. pylori-negative patients, H. pylori-positive patients had higher serum PGs/gastrin-17 (G-17) levels and lower PGR levels, regardless of OLGA/OLGIM stages I-II or III-IV. Furthermore, eight patients with atrophic gastritis who progressed to GC were previously in OLGA stages III-IV and OLGIM stages II-IV. The average duration of this process was 2.19±1.03 years. Logistic regression analysis indicated that PGI and H. pylori infection were independent risk factors of individuals with OLGA stages III-IV. Age and PGR were independent risk factors of patients with OLGIM stages III-IV. PGI and PGR had good clinical diagnostic values for OLGA stages III-IV and OLGIM stages III-IV, respectively. Patients with OLGA/OLGIM stages III-IV should undergo endoscopic surveillance regardless of H. pylori infection. H. pylori-positive patients with OLGIM stage II also have a high risk of GC. H. pylori combined with PGI and PGR is helpful to evaluate the severity of chronic gastritis.

The current status of chronic kidney disease-mineral and bone disorder management in China

Chronic kidney disease-mineral and bone disorder (CKD-MBD) is one of the most common complications of patients with chronic kidney disease (CKD). After considering updated international and domestic CKD-MBD management guidelines, the current management status of CKD-MBD in China remains unclear. We aimed to investigate the management status of CKD-MBD in China. A nationwide survey was combined with a real-world study from Sichuan. Targets recommended in KDIGO 2017 and Chinese 2019 guidelines were used as targets. We compared the data between Sichuan from western China and the eastern developed areas of China, and also compared the results of the real-world data from Sichuan with those of DOPPS5. In the questionnaire, a total of 51,039 maintenance hemodialysis (MHD) patients from 272 centers were involved. Estimated achievement rates for Ca (2.1–2.5 mmol/L), P (1.13–1.78 mmol/L), and iPTH (150–600 pg/mL) levels were 57.1%, 41.1% and 52.0%, respectively. Differences in MBD management between Sichuan from questionnaire and central region. In the real-world survey, a total of 7,053 patients were enrolled. Among them, 57.6%, 24.3%, and 55.0% of patients met corrected Ca, serum P, and iPTH targets specified in Chinese 2019 guidelines, respectively. The comprehensive achievement rate was 7.5%. There are differences in MBD management between Sichuan and DOPPS5 regions, with Sichuan being relatively poorer. The level of the above parameters varies among different genders, age groups, and hospital grades. The achievement rate of serum P was higher in tertiary hospitals and elderly patients (P < 0.05). Current MBD management is poor. Phosphate levels in patients treated in secondary and lower hospitals and young dialysis patients should be strengthened.

Investigating the effects of gradual deployment of market penetration rates (MPR) of connected vehicles on delay time and fuel consumption

Purpose This research paper aims to investigate the effects of gradual deployment of market penetration rates (MPR) of connected vehicles (MPR of CVs) on delay time and fuel consumption. Design/methodology/approach A real-world origin-destination demand matrix survey was conducted in Boston, MA to identify the number of peak hour passing vehicles in the case study. Findings The results showed that as the number of CVs (MPR) in the network increases, the total delay time decreases by an average of 14% and the fuel consumption decreases by an average of 56%, respectively, from scenarios 3 to 15 compared to scenario 2. Research limitations/implications The first limitation of this study was considering a small network. The considered network shows a small part of the case study. Originality/value This study can be a milestone for future research regarding gradual deployment of CVs’ effects on transport networks. Efficient policy(s) may define based on the results of this network for Brockton transport network.

642P Frequency and impact of retreatment in relapsed refractory multiple myeloma (RRMM): Real-world survey conducted in 5 European countries (United Kingdom, France, Germany, Italy, Spain)

To better understand the frequency and impact of retreatment (ReTx) on treatment (Tx) outcomes in patients (pts) with RRMM, we analysed the results from a real-world (RW) survey conducted in Europe. Data were derived from the Adelphi MM Disease Specific Programme™, a point-in-time survey of haematologists and haemato-oncologists conducted from May to Nov 2021. Descriptive information on frequency, and impact of ReTx, including reasons for prescribing and stopping ReTx were analyzed across line of Tx (LOT). Pt record forms were completed by physicians for pts with confirmed MM who were actively receiving Tx (ie, a quota of ≥2 pts on each LOT and triple-class exposed). A total of 256 physicians provided data on 1778 pts with RRMM who received ≥2 LOT. ReTx with IMiDs® or proteasome inhibitors (PIs) was seen in 70% (lenalidomide [LEN] regimen ReTx only, 21%) and 62% (bortezomib [BORT] regimen ReTx only, 29%) of pts, respectively, and most often occurred in 2L or 3L (Table). In contrast, CD38- antibodies were previously used in 5% of pts (daratumumab [DARA] ReTx only, 3%). Overall, ReTx with a prior agent and/or class was similar across countries; however, LEN ReTx rates were numerically lower in the UK (10%) and Spain (11%) than Germany (35%), France (27%), and Italy (22%). Following Tx guidelines and disease progression were the primary reasons for choosing and stopping ReTx, respectively, regardless of LOT. Table shows median duration of Tx (mDOT) and median time to next Tx (mTTNT) for pts with BORT, LEN, or DARA ReTx in 2L-4L.Table: 642PBORTLENDARAReTxNo ReTxReTxNo ReTxReTxNo ReTxOverall population (≥2L; n=1778), %297121793972L (n=1778), %178310901993L (n=1270), %138710901994L (n=633), %793991397mDOTa2L (n=1067)%1783892<1100months7.211.310.810.76.610.83L (n=551)%15851090298months10.810.510.010.84.010.84L (n=92)%13879910100months7.97.09.76.6–7.0mTTNT (start of listed line to start of next line)b2L (n=1069)%1882991<1100months16.116.415.016.54.416.43L (n=540)%15851189298months16.014.316.814.04.014.64L (n=68)%16849910100months13.311.712.911.8–11.9aIncludes pts with known LOT start and end date. bIncludes pts with known start date of listed line and next line. Open table in a new tab aIncludes pts with known LOT start and end date. bIncludes pts with known start date of listed line and next line. Consistent with ESMO guidelines, ReTx with PIs or IMiDs® was common in 2L and 3L (primarily BORT and LEN), whereas rates of DARA ReTx were low. Trends were observed with mDOT and mTTNT, suggesting some retreatment options may be suboptimal in a RW setting. The variation in ReTx patterns between countries was more prominent with LEN-based regimens compared with others.

34647 Exploring differences in treatment patterns between adult and pediatric psoriasis patients: Results from a real-world survey

Real-world data comparing psoriasis treatment use in children and adults is limited. Understanding disease and treatment differences amongst these populations helps to identify potential opportunities for optimization of care. Data were drawn from the Adelphi pediatric (ages ≤17) and adult (ages ≥18) Psoriasis Disease Specific Programmes (DSP): point-in-time surveys of physicians and their psoriasis patients in the US collected in August-November 2018 (adults) and December 2019-April 2020 (pediatric).

Women With Psoriatic Arthritis Experience Higher Disease Burden Than Men: Findings From a Real-World Survey in the United States and Europe.

Although psoriatic arthritis (PsA) is equally present in men and women, sex may influence clinical manifestations and the impact of disease on patients' lives. This study assessed differences in clinical characteristics, disability, quality of life (QOL), and work productivity by sex in real-world practice. A cross-sectional survey of rheumatologists/dermatologists and their patients with PsA was conducted in France, Germany, Italy, Spain, the United Kingdom, and the United States between June and August 2018. Data collected included demographics, treatment use, clinical characteristics (tender joint count, swollen joint count, body surface area affected by psoriasis), QOL (EuroQoL 5-Dimension questionnaire [EQ-5D], Psoriatic Arthritis Impact of Disease [PsAID12]), disability (Health Assessment Questionnaire-Disability Index [HAQ-DI]), and work productivity (Work Productivity and Impairment Index [WPAI]). Outcomes were compared between men and women using parametric and nonparametric tests, as appropriate. Of 2270 patients (mean age 48.6 [SD 13.3] yrs, mean disease duration 4.9 [SD 6.0] yrs), 1047 (46.1%) were women. Disease duration, disease presentation, and biologic use (mean 54.2%) were comparable between women and men. Women reported worse QOL (EQ-5D: 0.80 [SD 0.2] vs 0.82 [SD 0.2]; P = 0.02), greater disability (HAQ-DI: 0.56 [SD 0.6] vs 0.41 [SD 0.5]; P < 0.01) and work activity impairment (WPAI: 27.9% [SD 22.0] vs 24.6% [SD 22.4]; P < 0.01) than men. However, women had a lower burden of comorbidities (Charlson Comorbidity Index: 1.10 [SD 0.5] vs 1.15 [SD 0.6]; P < 0.01). In patients with similar PsA disease activity and treatment, women experienced greater disease impact than men. This represents a significant consideration for the therapeutic management of PsA.

The Burden of Nocturnal Symptoms in Patients with Chronic Obstructive Pulmonary Disease: Results of a Real-World Survey in the USA.

IntroductionPatients with chronic obstructive pulmonary disease (COPD) often have poor sleep quality and report a worsening of respiratory symptoms during night-time. However, current clinical guidelines for COPD management do not specifically consider nocturnal symptoms when recommending pharmacological treatment. This study aimed to better understand the burden of nocturnal symptoms in patients with COPD, and to evaluate the importance of nocturnal symptom control compared with daytime and overall symptom control.MethodsData were analyzed from the Adelphi Respiratory Disease Specific Programme, a point-in-time survey of physicians and their patients, conducted in the USA in 2019. Primary care physicians and pulmonologists who managed three or more patients with COPD per month were eligible for inclusion; eligible patients were ≥ 18 years old, with a physician-confirmed diagnosis of COPD.ResultsSurveys from 171 physicians and 800 patients were analyzed. Everyday symptoms were reported in 14% of patients. In total, 88% of patients reported daytime symptoms, and 74% of patients experienced nocturnal symptoms, with 7% reporting daily nocturnal symptoms. Patients experiencing nocturnal symptoms every day had the greatest impairment in their activity as per the Work Productivity and Activity Impairment questionnaire (mean total activity impairment, 66.9%; nocturnal symptoms once or twice a week, 41.1%; no nocturnal symptoms, 26.4%). Patients experiencing daily nocturnal symptoms also had the lowest quality of life (QoL) as per the EuroQoL 5-Dimension 3-Level score. Physicians reported prescribing therapy based on sustained 24-h symptomatic relief for the majority of patients (78%). They reported nocturnal symptom control as a factor in their choice of therapy for 38% of patients, and daytime symptom control as a reason for 61% of patients.ConclusionDaytime and nocturnal symptoms are common among patients with COPD. Frequency of nocturnal symptoms is related to a significant impairment in activity and health-related QoL.Supplementary InformationThe online version contains supplementary material available at 10.1007/s41030-022-00196-7.

An innovative improvement in Warner's randomized response device for evasive answer bias

We propose an innovative randomized response estimator that is free from the probabilities of asking the sensitive question and its complement. To utilize the proposed estimator, every respondent uses an independent electronic randomization device, unobserved by the interviewer, which encourages participation by guaranteeing the protection of their privacy. In this paper, such an electronic randomization device is developed, tested through simulation studies, and implemented in a real-world survey.

Physician-Reported Perspectives on Myasthenia Gravis in the United States: A Real-World Survey.

IntroductionMyasthenia gravis (MG) is a rare, debilitating, chronic disorder caused by the production of pathogenic immunoglobulin G autoantibodies against the neuromuscular junction. A lack of real-world studies in rare diseases reflects a relatively limited understanding of the significant unmet needs and burden of disease for patients. We aimed to provide comprehensive real-world insights into the management and burden of MG from treating physicians in the United States (US).MethodsData were collected using the Adelphi Real World MG Disease Specific Programme™, a point-in-time survey of physicians and their patients with MG, in the US between March and July 2020. Physician-reported clinical data, including demographics, comorbidities, symptoms, disease history, treatments, and healthcare resource utilization, were collected.ResultsIn total, 456 patient record forms were completed by 78 physicians based in the US. At time of survey completion, patient mean age was 54.5 years. Mean time from symptom onset to diagnosis was 9.0 months (n = 357). Ocular symptoms were reported in 71.7% of patients. General fatigue affected 47.1% of patients and over half of those reported the severity as moderate or severe (59.5%, n = 128). Acetylcholinesterase inhibitors and/or steroids were the most frequently prescribed first-line treatment type among patients receiving treatment at time of survey completion and with moderate-to-severe symptoms (77.9%, n = 159/204). High-dose steroids (n = 14) and intravenous immunoglobulin (n = 13) were the most prescribed acute treatments among those receiving an acute treatment at time of survey completion (n = 36), with symptom exacerbations or myasthenic crises being the most common reasons for acute treatment. On average, 2.5 healthcare professionals were involved in patient management and 5.0 consultations were made per patient over the last 12 months.ConclusionsOur findings indicated that, despite treatment, there is a proportion of patients with MG in the US who had a significant need for improved disease management.Supplementary InformationThe online version contains supplementary material available at 10.1007/s40120-022-00383-3.

Effect of Fatigue on Health-Related Quality of Life and Work Productivity in Psoriatic Arthritis: Findings From a Real-World Survey.

To evaluate fatigue frequency and severity among patients with psoriatic arthritis (PsA) and assess the effect of fatigue severity on patient-reported outcome measures (PROMs) assessing quality of life, function, and work productivity. Data were derived from the Adelphi Disease Specific Programme, a cross-sectional survey conducted in 2018 in the United States and Europe. Patients had physician-confirmed PsA. Fatigue was collected as a binary variable and through its severity (0-10 scale, using the 12-item Psoriatic Arthritis Impact of Disease fatigue question) from patients; physicians also reported patient fatigue (yes/no). Other PROMs included the 5-level EuroQol 5-dimension questionnaire (EQ-5D-5L) for health-related quality of life (HRQOL), Health Assessment Questionnaire-Disability Index, and Work Productivity and Activity Impairment Questionnaire. Multivariate linear regression was used to evaluate the association between fatigue severity and other PROMs. Among the 831 included patients (mean age 47.5 yrs, mean disease duration 5.3 yrs, 46.9% female, 48.1% receiving a biologic), fatigue was reported by 78.3% of patients. Patients with greater fatigue severity had greater disease duration, PsA severity, pain levels, body surface area affected by psoriasis, and swollen and tender joint counts (all P < 0.05). In multivariate analyses, patients with greater fatigue severity experienced worse physical functioning, HRQOL, and work productivity (all P < 0.001). Presence of fatigue was underreported by physicians (reported in only 32% of patients who self-reported fatigue). Prevalence of patient-reported fatigue was high among patients with PsA and underrecognized by physicians. Fatigue severity was associated with altered physical functioning, work productivity, and HRQOL.