A stated preference survey to explore patient preferences for novel preventive migraine treatments.

OBJECTIVE: The objective of this study was to explore patient preference for attributes of calcitonin gene-related peptide (CGRP) inhibitors for the preventive treatment of migraine and to describe differences in treatment preferences between patients. BACKGROUND: CGRP inhibitors are a novel class of migraine drugs specifically developed for the preventive treatment of migraine. Clinicians should understand patient preferences for CGRP inhibitors to inform and support prescribing choices. METHODS: Patients with migraine in the US and Germany were recruited to participate in an online discrete choice experiment (DCE) survey, which presented hypothetical treatment choices using five attributes: mode of administration, side effects, migraine frequency, migraine severity, and consistency of treatment effectiveness. Attribute selection was informed by a literature review and semi-structured patient interviews (n = 35), and evaluated using patient cognitive debriefing interviews (n = 5). RESULTS: Of 680 who consented to participate, 506 participants completed the survey and were included in the study (US = 257; Germany = 249). Overall, participants placed highest importance (preference weight, beta = 1.65, p < 0.001) on the treatment's ability to reduce the severity of migraine (mild vs. unchanged severity), followed by consistent treatment effectiveness (beta = 1.13, p < 0.001), and higher chance of reduced migraine frequency (beta = 1.00, p < 0.001). Participants preferred an oral tablet every other day (beta = 1.00, p < 0.001) over quarterly infusion, quarterly injections (p = 0.019), or monthly injection (p < 0.001). Preference for all treatment attributes were heterogeneous, and the subgroup analyses found that participants naïve to CGRP monoclonal antibody treatments had a stronger preference for oral therapy compared to those with such experience (p = 0.006). CONCLUSION: In this DCE assessing CGRP inhibitors attributes, the main driver of patient choice was treatment effectiveness, specifically reduced migraine severity, and consistent treatment effectiveness. Further, patients exhibited an overall preference for an oral tablet every other day over injectables. Patients' experience with previous treatments informs the value they place on treatment characteristics.

Real-World experience of interictal burden and treatment in migraine: a qualitative interview study.

BACKGROUND: The debilitating nature of migraine attacks is widely established; however, less is known about how the interictal burden (i.e., how patients are affected in-between migraine episodes) of migraine impacts on patients' health-related quality of life (HRQL). Acute and preventive treatments may lift the burden of the disease, but they often have unwanted side effects and limited effectiveness. The objective of this study was to understand the interictal burden of migraines, from the patient perspective, and to explore patient experience with migraine treatments. METHODS: Participants (n=35) with a self-reported diagnosis of migraine were recruited in the US, UK and Canada, including a subgroup of patients who had taken calcitonin gene-related peptide monoclonal antibody (CGRP mAb) treatment for at least three months. Participants completed a background questionnaire, followed by a semi-structured interview via telephone or video call. The interviews explored patients' migraine symptoms, perception of interictal burden and treatment experience. The interview transcripts were analysed using thematic analysis. RESULTS: The most reported migraine symptom was migraine pain, followed by aura, sensory sensitivity and nausea. Most participants reported interictal impact on HRQL, lifestyle changes they made to avoid triggers or in anticipation of an attack, impacts on work, career, daily activities and relationships. Emotional impacts were reported by all participants, including anger, depression, anxiety and hopelessness. Many participants who took preventive treatments reported improvements in HRQL and functioning but still experienced breakthrough attacks. Among patients who took CGRP mAbs, participants noted varying consistency of treatment effectiveness between treatment administrations. CONCLUSION: This study detailed the additional HRQL impact of migraine in-between migraine attacks and described the unmet need for effective treatment options to prevent and mitigate migraine attacks.

Measuring interictal burden among people affected by migraine: a descriptive survey study.

BACKGROUND: Previous research has extensively documented the impact of migraine episodes ('ictal') on patients' health-related quality of life. Few studies have looked at the impact of migraine on migraine-free days ('interictal'). This study was designed to describe interictal burden of migraine in a mixed group of people affected by migraine and to explore patient characteristics associated with interictal burden. METHODS: People with migraine in the United States (US) and Germany were recruited for a cross-sectional online survey, including a subgroup treated with calcitonin gene-related peptide (CGRP) monoclonal antibody (mAb). The survey included the Migraine Interictal Burden Scale (MIBS-4), Headache Impact Test (HIT-6), and items measuring patient demographics, clinical and treatment background. Data were analyzed using descriptive statistics and linear regression. RESULTS: Five hundred six people with migraine completed the survey (US: n = 257; Germany: n = 249), of whom 195 had taken a CGRP mAb for three or more months. Participants had a mean of 8.5 (SD = 6.4) Monthly Migraine Days (MMD) and 10.4 (SD = 7.1) Monthly Headache Days (MHD). The mean MIBS-4 score was 6.3 (SD = 3.4), with 67% reporting severe interictal burden (MIBS-4: ≥5). The mean HIT-6 score was 65.3 (SD = 6.0), with 86% reporting severe migraine impact (HIT-6: ≥60). MIBS-4 was correlated with the HIT-6 (r = 0.37), MMD and MHD (both r = 0.27). The HIT-6, MMD, MHD, CGRP mAb treatment, and depression all had an independent positive association with the MIBS-4. CONCLUSION: Two-thirds of the study sample reported substantial interictal burden. Whilst interictal burden was associated with migraine frequency and impact of migraine attacks, study results also show it represented a distinct aspect of the overall disease burden. Study findings further indicate unique associations between interictal burden and depression. A unique positive association between interictal burden and CGRP mAb treatment suggests a remaining unmet need among people affected by migraine treated with CGRP mAb.

Patient preferences for advanced hepatocellular carcinoma treatment: a multicountry stated preference study.

The present study aimed to explore patient preferences for attributes of advanced hepatocellular carcinoma (HCC) treatments. A stated preference survey was completed by 150 patients with HCC living in Europe. Overall survival (OS) was the most important attribute, closely followed by risk of diarrhea and hypertension, and other adverse event (AE) risks. Patients were willing to trade OS to reduce AE risks. While less important than OS and AEs, patients also preferred shorter waiting times, and one-off administration of selective internal radiation therapy and oral tablets over intravenous infusions. Although patients placed the most value on extending OS, they were willing to forego OS to avoid risk of treatment-related AEs, to maintain their quality of life.

Lay abstract This study aimed to understand patient preferences for characteristics of advanced hepatocellular carcinoma (HCC) treatments. A total of 150 people with HCC in Europe were presented a series of questions asking them to choose between two hypothetical treatments. Overall, length of life was the most important issue for patients, followed by avoiding diarrhea and hypertension, and then other side effects and treatment risks. Patients were willing to forego some months of life to avoid side effects or risks. Patients preferred to be given their treatment via a single minimally invasive hospital procedure or oral daily tablets compared with intravenous drips. In conclusion, although patients placed the most value on overall length of life, side effects and treatment risks were also important.

Colorectal cancer screening uptake over three biennial invitation rounds in the English bowel cancer screening programme.

OBJECTIVE: To examine patterns of colorectal cancer (CRC) screening uptake over three biennial invitation rounds in the National Health Service (NHS) Bowel Cancer Screening Programme (BCSP) in England. METHODS: We analysed data from the BCSP's Southern Hub for individuals (n=62,099) aged 60-64 years at the time of first invitation to screening with a follow-up period that allowed for two further biennial invitations. Data on sex, age and a neighbourhood-level measure of socioeconomic deprivation were used in the analysis. Outcomes included uptake of guaiac-based faecal occult blood (gFOB) test screening, inadequate gFOB screening (≥1 test kit(s) returned but failed to complete further gFOB tests needed to reach a conclusive test result), test positivity, compliance with follow-up examinations (usually colonoscopy) and diagnostic outcomes. RESULTS: Overall gFOB uptake was 57.4% in the first, 60.9% in the second and 66.2% in third biennial invitation round. This resulted in 70.1% of the initial cohort having responded at least once, 60.7% at least twice and 44.4% three times. Participation in the first round was strongly predictive of participation in the second round ('Previous Responders': 86.6% vs. 'Previous Non-Responders': 23.1%). Participation in the third round was highest among 'Consistent Screeners' (94.5%), followed by 'Late Entrants' (78.0%), 'Dropouts' (59.8%) and 'Consistent Non-Responders' (14.6%). Socioeconomic inequalities in uptake were observed across the three rounds, but sex inequalities decreased over rounds. Inadequate gFOB screening was influenced by screening history and socioeconomic deprivation. Screening history was the only significant predictor of follow-up compliance. CONCLUSIONS: Screening history is associated with overall gFOB uptake, inadequate gFOB screening and follow-up compliance. Socioeconomic deprivation is also consistently associated with lower gFOB uptake and inadequate gFOB screening. Improving regular screening among identified 'at-risk' groups is important for the effectiveness of CRC screening programmes.

Micro actions in colorectal cancer screening participation: a population-based survey study.

BACKGROUND: Low uptake of colorectal cancer (CRC) screening is a cause for concern. This study explored people's anticipated response to receiving the test kit to shed light on past screening uptake and help inform future interventions to increase participation. METHODS: Face-to-face interviews were conducted with respondents living in England who were eligible for CRC screening as part of a population-based 'omnibus' survey. Respondents were asked what they would do ('micro actions') if they received a CRC screening test kit through the mail (apart from completing it or not), and their unprompted responses were coded (multiple codes allowed). Past 'ever' uptake and screening intention were also recorded. The final analysis included 1237 respondents aged 60-70. RESULTS: Respondents who said that they would decide after some thought' (p < .001), 'put [it] aside to deal with later' (p < .001), 'put it on the "to do list/ pile"' (p < .05) or 'discuss it with a health care professional' (p < .01) had decreased odds of having participated. Those who said they would 'read the instruction leaflet' (p < .001), 'put the kit near the toilet' (p < .001) or 'decide when to do the test' (p < .05) were more likely to have taken part in CRC screening. With the exception of 'decide when to do the test' and 'discuss it with a health care professional', all associations with past uptake remained significant after adjusting for other micro actions and screening intention. 'Make a note somewhere (to remind myself)' was mentioned by less than 1 % of respondents. CONCLUSIONS: Delay-causing and preparatory micro actions were associated with past CRC screening uptake. Self-regulatory micro actions (e.g. making a note to remind oneself) were rarely mentioned as responses to receiving a screening invitation. Interventions aimed at reducing delay and facilitating preparatory and self-regulatory behaviours might help increase uptake. The behaviour-focused survey method is a promising avenue for future health behaviour research.

A survey exploring caregiver burden and health-related quality of life in hereditary transthyretin amyloidosis.

BACKGROUND: Hereditary transthyretin amyloidosis (ATTRv) is an ultra-rare, life-shortening disease with a high unmet need. This study examined ATTRv caregiver health-related quality of life (HRQoL) and productivity. METHODS: A cross-sectional online survey, including EQ-5D-3L, Hospital anxiety and depression scale (HADS), and caregiver and patient characteristics questions, was developed to assess ATTRv caregiver burden. A companion general population survey collected EQ-5D-3L, HADS and chronic health conditions data. Caregiver-control group differences in HRQoL were assessed using t-tests and chi-square tests. Ordinary Least Squares regression was used to estimate the disutility of being a caregiver compared to controls stratified by patient ambulatory status. RESULTS: Thirty-six caregivers and matched controls completed the survey (n = 72). The disease severity of patients they cared for was varied: 33% required no assistance walking, 58% required assistance with walking and 9% required a wheelchair/were bedridden. On average, caregivers spent 6 h daily on practical care and 4 h daily on emotional support. Fifty-six percent indicated that they had changed their employment due to providing ATTRv care. Caregivers reported lower HRQoL, as indicated by lower EQ-5D 3L utility scores (M = 0.772, SD = 0.178 vs. M = 0.849, SD = 0.218) and higher HADS anxiety (9.3 vs. 6.1, p < 0.01) and depression (7.6 vs. 4.4, p < 0.01) scores, compared with matched controls. Caregivers were also more likely to report sleep problems (33% vs. 8%, p < 0.01) and stress (42% vs. 0%, p < 0.001) as chronic conditions than controls. CONCLUSIONS: The study results indicate that caring for a person with ATTRv can have a considerable negative impact on caregivers' HRQoL and productivity. The study findings provide important information for economic evaluations of ATTRv treatments.

The Burden of Caring for Individuals with Tuberous Sclerosis Complex (TSC) Who Experience Epileptic Seizures: A Descriptive UK Survey.

INTRODUCTION: Tuberous sclerosis complex (TSC) is a rare multisystem genetic condition characterised by benign tumours; prevalent manifestations include epilepsy and neuropsychiatric disorders. This study examined the burden of TSC for primary caregivers and families, exploring the impact of characteristics such as seizures. METHODS: Primary caregivers of individuals with TSC in the United Kingdom participated in an online survey, comprising the Pediatric Quality of Life Inventory™ Family Impact Module, Hospital Anxiety and Depression Scale (HADS), and TSC-specific items. Responses were analysed using descriptive and regression analysis statistics (closed-ended) or qualitative content analysis (open-ended). RESULTS: Seventy-three participants partially completed and 59 fully completed the survey; 95% were female, and 90% were parents of an individual with TSC. A median (range) of 2 (1-11) household members were carers. Primary caregivers spent a mean (standard deviation [SD]) of 104.3 (51.7) hours caring in the previous week, reporting high mean (SD) HADS scores of 11.2 (4.8) (anxiety) and 7.9 (4.4) (depression) and considerable family burden. Increased seizure frequency increased hours spent caring by primary caregivers (p = 0.01) and was associated with a decreased mean (SD) family functioning score of 46.2 (23.0) and parent health-related quality of life (HRQL) score of 45.4 (20.3) (both p = 0.03). Multivariable models predicted intellectual disability increased hours spent caring by primary caregivers (p = 0.01-0.04), and neuropsychiatric comorbidities decreased family functioning (p = 0.02) and caregiver HRQL (p < 0.01). CONCLUSION: These findings highlight the role of epileptic seizures and neuropsychiatric disorders in the considerable burden of TSC on primary caregivers and families.

Shared decision-making and the caregiver experience in tuberous sclerosis complex: results from a UK survey.

BACKGROUND: Tuberous sclerosis complex (TSC) is a rare genetic condition commonly accompanied by neurological and neuropsychological disorders, resulting in a high burden of illness for individuals and a substantial impact on their caregivers. Due to the diversity and complexity of clinical manifestations, patients with TSC need aligned multidisciplinary healthcare services starting in childhood through to adulthood. However, patients and caregivers are sometimes dissatisfied with the care provided, for which one of the most common reasons is a lack of involvement in clinical decision-making. Shared decision-making, whereby clinicians make clinical management decisions together with patients and their caregivers, is advocated for in the management of epilepsy, but evidence of its benefit in managing TSC is currently lacking. In this cross-sectional, UK-based analysis we used an online survey to capture the experiences of primary caregivers for individuals with TSC, including the impact on work productivity, clinical shared decision-making, satisfaction with care, and the impact of the coronavirus disease 2019 (COVID-19) pandemic. RESULTS: In total, 73 eligible caregivers provided consent (analysis set), with 14 completing the survey partially and 59 completing the full survey. Many caregivers (72%) reported receiving recommendations about new treatments from their doctor and discussing the treatment together, with a high proportion (89%) preferring that treatment was initiated at a low dose. Most caregivers (69%) were satisfied or extremely satisfied with pediatric TSC healthcare services, but only 25% were satisfied or extremely satisfied with the transition to adult TSC healthcare services. Several (n = 30) caregivers specified the impact of caring on their work productivity and career in optional open-ended survey responses. Finally, 80% of caregivers indicated that the COVID-19 pandemic had a "large" or "very large" impact on their caring activities, negatively affecting the emotional wellbeing and behavior of individuals with TSC, and caregivers' ability to work and arrange medical appointments. CONCLUSIONS: Caregivers largely feel involved in treatment decisions, and the majority were satisfied with healthcare services for children with TSC. However, many highlighted the need for an improved transition from pediatric to adult healthcare services. The survey also showed that COVID-19 has considerably affected caregivers and individuals with TSC.

Patient and Caregiver Health State Utilities in Tuberous Sclerosis Complex.

BACKGROUND: Tuberous sclerosis complex (TSC) is a rare multisystem disorder often associated with treatment-resistant epilepsy. Cost-effectiveness analysis for new antiseizure medications typically requires health state utilities (HSUs) that reflect the burden of a given condition. OBJECTIVE: This study aimed to estimate HSUs, with a focus on valuing the impact of seizure type and seizure frequency on health-related quality of life (HRQL) for patients with TSC and their caregivers. METHODS: A targeted literature review and qualitative research with healthcare professionals and caregivers informed the development of health state vignettes describing the experience of living with TSC or caring for a child with TSC. Vignettes were evaluated in interviews with the UK general population using the time trade-off (TTO) method. RESULTS: Sixteen vignettes were developed describing patient HRQL (n = 8) and caregiver HRQL (n = 8). Two hundred interviews were conducted via online video calls due to COVID-19 pandemic restrictions. Two hundred participants evaluated the patient (n = 100) and caregiver (n = 100) health state vignettes. Estimated utility scores varied consistently according to seizure type and seizure frequency. Patient TTO utility scores ranged between -0.234 (highest seizure frequency and multiple seizure types) and 0.725 (seizure-free state). Caregiver TTO utility scores ranged from 0.221 to 0.905. CONCLUSIONS: Findings highlight the substantial burden of living with TSC and caring for a child with TSC. Patient and caregiver burden was greater for generalised versus focal seizures. The burden was greatest for a combination of both seizure types and worsened with increasing seizure frequency.

Time Trade-Off Utilities for Hereditary Angioedema Health and Caregiver States.

BACKGROUND: Hereditary angioedema (HAE) is an inherited chronic rare disease characterised by recurrent swelling attacks that are associated with significant physical and psychological burden. There is limited understanding of the effect of attack location on this burden and of caregiver burden. OBJECTIVE: Our objective was to capture the relative burden of HAE health and caregiver states, including different attack locations, through a time trade-off (TTO) analysis involving participants from the general public. METHODS: Qualitative interviews were undertaken to inform vignette development for the TTO study, including vignettes for abdominal, facial, hand and laryngeal attack health states, and an attack-free and caregiver state. Members of the general public in England rated vignettes in TTO interviews, which included a visual analogue scale (VAS) component. For the development of the health state vignettes, qualitative interviews with 15 patients, 5 caregivers and 1 clinical expert were performed. TTO analysis was based on vignette valuation completed by 100 members of the general public. RESULTS: The TTO values were as follows: attack-free, 0.783 (standard deviation [SD] 0.316); hand: 0.582 (SD 0.380); facial: 0.483 (SD 0.448); abdominal: 0.345 (SD 0.458); and laryngeal: 0.128 (SD 0.529). The caregiver rating was 0.762 (SD 0.303). V' scores were similar and consistent with TTO values. CONCLUSION: TTO utility values demonstrate that HAE places a significant burden on patients, which is influenced by attack location, and on caregivers. These utility weights can provide important information on quality of life for future economic evaluations of treatments.

Preferences and Utilities for Treatment Attributes in Type 2 and Non-ambulatory Type 3 Spinal Muscular Atrophy in the United Kingdom.

BACKGROUND: Spinal muscular atrophy (SMA) is a rare neuromuscular disease that affects motor neurons, resulting in progressive skeletal muscle weakness and atrophy. OBJECTIVE: The aim was to understand the value patients with SMA and caregivers place on treatment attributes and to estimate health utilities for SMA treatment outcomes from a general public sample. METHODS: Two discrete choice experiments were designed to elicit treatment preferences and health utilities, respectively. Patients with Type 2 and non-ambulatory Type 3 SMA, caregivers of patients with SMA and a general public sample in the UK completed the surveys. Patients and caregiver participants were recruited through patient associations. General public participants were recruited via a survey recruitment panel. Attributes included motor function, breathing function, treatment administration, treatment reactions, eyesight monitoring, contraception (patients only) and overall survival (general public only). Clustered conditional logit models were used to estimate treatment preferences, and marginal rates of substitution were used to estimate disutilities. RESULTS: Adult patients (n = 84) were twice as likely to choose a treatment with improved (vs. stable) motor and breathing function and four to five times less likely to choose a treatment with deteriorated (vs. stable) motor and breathing function as a treatment outcome. Caregivers (n = 83) were three to nine times more likely to choose improved and two to four times less likely to choose deteriorated (vs. stable) motor and breathing function. Both patients and caregivers preferred oral over intrathecal treatment. Treatment reactions, eyesight monitoring or contraception had no significant effect on patient choices. Conversely, caregivers preferred avoidance of treatment reactions. General public data (n = 506) yielded disutilities for unable to sit (- 0.408), need for > 16 h daily mechanical breathing support (- 0.304) and intrathecal therapy (- 0.071). CONCLUSIONS: Study results show the importance of motor and breathing function to patients and caregivers, and an oral treatment preference. Disutilities (decrements to utility) were substantial for SMA disease outcomes and care aspects.

Patient and Caregiver Treatment Preferences in Type 2 and Non-ambulatory Type 3 Spinal Muscular Atrophy: A Discrete Choice Experiment Survey in Five European Countries.

BACKGROUND: Spinal muscular atrophy (SMA) is a rare neuromuscular disease that affects motor neurons, resulting in progressive skeletal muscle weakness and atrophy. OBJECTIVE: The aim of this study was to examine treatment preferences of patients and caregivers of patients with Type 2 and non-ambulatory Type 3 SMA in the Netherlands, Belgium, Finland, Ireland and Portugal. METHODS: A discrete choice experiment (DCE) survey was developed to elicit the preferences of adult patients and caregivers regarding different treatment aspects of SMA. This survey built on the design of a similar study undertaken in the UK. The DCE described choice questions in terms of attributes and levels combined using a D-efficient design. The attributes described improvements or worsening in motor and breathing function. The mode of treatment administration (intrathecal injection, single intravenous infusion or regular oral therapy) was described. Treatment risks and side effects related to currently available treatments including risk of liver injury, fatigue, headache, nausea, diarrhoea and rash were described. Lastly, an attribute described whether a treatment had evidence of treatment effectiveness in different SMA types. Participants were recruited via patient advocacy associations to complete an online survey. A clustered conditional logit model was used to estimate treatment preferences. RESULTS: Participants (n = 65) were 4.8 times and 8.1 times more likely to choose a treatment with stable or improved (vs worse) motor function, respectively. Similarly, participants were 4.3 times and 5.8 times more likely to choose stable or improved (vs worse) breathing function, respectively. Treatments with a risk of liver injury, fatigue, headache and nausea were 1.6 times less likely to be chosen than treatments with a risk of diarrhoea and rash. Treatments with demonstrated effectiveness in Type 1 SMA only were 2.3 times less likely to be chosen than those with demonstrated effectiveness in Types 1-3 SMA. Treatments administered via intrathecal injections were also 1.8 times less likely to be chosen than daily oral treatments. DISCUSSION: Study results show the importance of improvement as well as stabilisation of motor and breathing function to patients and caregivers, and a preference for oral treatments, treatments with demonstrated effectiveness in Types 2-3 SMA, and avoidance of liver injury risk.

Patient and Caregiver Health State Utilities in Lennox-Gastaut Syndrome and Dravet Syndrome.

PURPOSE: Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS) are rare, treatment-resistant forms of epilepsy characterized by childhood onset of seizures. Cost-effectiveness analysis for new antiseizure medications typically requires health state utilities (HSUs) that reflect the patient and caregiver burden of the relevant conditions. This study aimed to estimate HSUs for LGS and DS. Focus was placed on valuing the impact of seizure frequency and seizure-free days on health-related quality of life (HRQL) for patients and caregivers. METHODS: Health state vignettes that described the experience of living with and caring for a child with LGS or DS were developed based on a targeted literature review and feedback from interviews with LGS and DS clinical experts and DS caregivers. Vignettes varied by the number of seizures and seizure-free days per month. Twenty-four vignettes were developed that described patient HRQL (6 per condition) and caregiver HRQL (6 per condition) for LGS or DS. Vignettes were evaluated via interviews from the general population in the United Kingdom and Sweden using a visual analog scale and time trade-off (TTO) method. Participants were recruited by interviewers based in different regions of the United Kingdom and Sweden using convenience sampling. FINDINGS: Two hundred interviews were conducted by video call from March to April 2020. One hundred participants evaluated each of the 6 patient and 6 caregiver vignettes for LGS; a different 100 participants evaluated each of the DS vignettes. The estimated utility values vary consistently according to seizure frequency and number of seizure-free days. Patient TTO utility values range from -0.186 (highest seizure frequency and fewest seizure-free days) to 0.754 (seizure-free state) for LGS and from 0.171 to 0.778 for DS. The caregiver TTO utility values range from 0.032 to 0.810 and 0.510 to 0.881 for LGS and DS, respectively. Fewer seizures and additional seizure-free days are associated with better patient and caregiver HRQL. IMPLICATIONS: This study estimated utility values for patients with LGS or DS and their caregivers using visual analog scale and TTO valuation among a general public sample. These utility values can be used in cost-effectiveness assessments of new antiseizure medications. The findings indicate the importance of seizure-free days as well as seizure frequency in patient and caregiver HRQL, which may inform the design of future clinical trials.

Burden of allergic rhinitis and impact of MP-AzeFlu from the patient perspective: pan European patient survey.

OBJECTIVE: The aims of this survey were to (1) assess the burden of allergic rhinitis (AR) from the patient perspective, (2) investigate MP-AzeFlu use in real life and its impact on patients' lives and (3) explore factors associated with treatment satisfaction. METHODS: A cross-sectional, quantitative, online, questionnaire-based survey was conducted in seven European countries (March-June 2019). Questions explored AR burden and treatment satisfaction. Satisfaction was assessed using the Treatment Satisfaction Questionnaire for Medication 9-item (TSQM-9; max score = 100). Participants (aged ≥18 years) had a doctor/healthcare provider confirmed AR diagnosis and used MP-AzeFlu within the last year. RESULTS: Pre-MP-AzeFlu treatment, participants (n = 1004) reported an average of 3.3 (SD:3.5) doctor visits/year, 8.1 (SD:11.0) days/year absenteeism and 15.8 (SD:18.9) days/year presenteeism due to AR. Only 48% of participants used MP-AzeFlu twice/day as recommended. Post-MP-AzeFlu 57% of participants reported better QoL, 47% reported fewer doctor visits and 52% discontinued polypharmacy. Absenteeism and presenteeism were reduced by 2.5 (SD 10.0) and 7.3 (SD:16.0) days/year, respectively. 70% of participants were more/much more satisfied with MP-AzeFlu versus previous AR treatment(s), and ≥70% were satisfied/extremely satisfied with its ability to prevent/treat AR, relieve symptoms and with its onset of action. Mean global, effectiveness and convenience TSQM-9 scores were 70.0 (SD:19.8), 68.3 (SD:21.6) and 72.7 (SD:20.4), respectively. Treatment satisfaction and effectiveness were significantly improved when MP-AzeFlu was taken as recommended. CONCLUSIONS: The impact of AR on patients' lives remains high. Real-life use of MP-AzeFlu reduces that impact and is associated with a high level of effectiveness, convenience and global satisfaction.

Does psychosocial stress exacerbate avoidant responses to cancer information in those who are afraid of cancer? A population-based survey among older adults in England.

OBJECTIVE: Communication of cancer information is an important element of cancer control, but cancer fear may lead to information avoidance, especially when coping is low. We examined the association between cancer fear and cancer information avoidance, and tested whether this was exacerbated by psychosocial stress. DESIGN: Cross-sectional survey of 1258 population-based adults (58-70 years) in England. MAIN OUTCOME MEASURES: Cancer fear (intensity and frequency), perceived psychosocial stress and cancer information avoidance. Control variables were age, gender, ethnicity, marital status and education. RESULTS: A quarter (24%) of respondents avoided cancer information. Ordinal logistic regression analyses showed main effects of psychosocial stress (OR = 1.17, 95% CI 1.07-1.29) and cancer fear: cancer information avoidance was lowest in those with no cancer fear (13%), followed by those with moderate (24%; OR = 2.15, 95% CI: 1.49-3.12), and high cancer fear (35%; OR = 3.90, 95% CI: 2.65-5.73). In the adjusted model, the interaction between cancer fear and stress was significant (OR = 1.14, 95% CI 1.004-1.29, p < .05): 40% of those with high fear/high stress avoided cancer information compared with 29% with high fear/low stress. CONCLUSION: Cancer fear and psychosocial stress interact to produce disengagement with cancer-related information, highlighting the importance of affective processes to cancer control efforts.

Self-Reported And Objectively Recorded Colorectal Cancer Screening Participation In England.

OBJECTIVE: To compare self-reported with objectively recorded participation in Faecal Occult Blood testing (FOBt) colorectal cancer (CRC) screening in a national programme. METHODS: Survey respondents living in England who were eligible for screening were asked in face-to-face interviews if they had ever been invited to do a CRC screening test, how many times they had been invited, and how many times they had participated. National Health Service (NHS) Bowel Cancer Screening Programme (BCSP) records were consulted for respondents who had consented to a record check. The outcome measures were 'ever uptake' (responded to ≥1 invitation), 'repeat uptake' (responded to ≥2 invitations), and 'consistent uptake' (responded to all invitations). RESULTS: In the verified group, self-reported ever uptake was highly consistent with recorded ever uptake (87.0% vs. 87.8%). Among those who indicated that they had been invited more than once, self-reported repeat uptake was 89.8% compared with 84.8% recorded repeat uptake. Among those with more than one recorded invitation, self-reported repeat uptake was 72.7% compared with 77.2% recorded repeat uptake, and self-reported consistent uptake was 81.6% compared with 65.6% recorded consistent uptake. CONCLUSION: Our results suggest that people can accurately report whether they have ever taken part in CRC screening. The vast majority of those whose records were verified could also accurately report whether they had taken part in screening at least twice. They were somewhat less accurate in reporting whether they had responded to all screening invitations.

Social Cognitive Mediators of Sociodemographic Differences in Colorectal Cancer Screening Uptake.

BACKGROUND: This study examined if and how sociodemographic differences in colorectal cancer (CRC) screening uptake can be explained by social cognitive factors. METHODS: Face-to-face interviews were conducted with individuals aged 60-70 years (n = 1309) living in England as part of a population-based omnibus survey. RESULTS: There were differences in screening uptake by SES, marital status, ethnicity, and age but not by gender. Perceived barriers (stand. b = -0.40, p < 0.001), social norms (stand. b = 0.33, p < 0.001), and screening knowledge (stand. b = 0.17, p < 0.001) had independent associations with uptake. SES differences in uptake were mediated through knowledge, social norms, and perceived barriers. Ethnic differences were mediated through knowledge. Differences in uptake by marital status were primarily mediated through social norms and to a lesser extent through knowledge. Age differences were largely unmediated, except for a small mediated effect via social norms. CONCLUSIONS: Sociodemographic differences in CRC screening uptake were largely mediated through social cognitive factors. Impact. Our findings suggest that multifaceted interventions might be needed to reduce socioeconomic inequalities. Ethnic differences might be reduced through improved screening knowledge. Normative interventions could emphasise screening as an activity endorsed by important others outside the immediate family to appeal to a wider audience.

Piloting the Impact of Three Interventions on Guaiac Faecal Occult Blood Test Uptake within the NHS Bowel Cancer Screening Programme.

This study evaluated the impact of three interventions on uptake of the guaiac faecal occult blood test (gFOBT) in Greater London. The interventions were designed to improve awareness and understanding of the NHS Bowel Cancer Screening Programme (BCSP) and assist stool sampling. Logistic regression analysis of BCSP London data (N = 205,541 invitees aged 60-74) compared uptake at 12 weeks between intervention groups and a control group, sent kits as usual between January-April 2013 and January-April 2014. An endorsement flyer, included with gFOBT kits, had no impact on uptake (P = 0.68). In 60-69-year-olds, there was a small but significant increase in modelled uptake amongst invitees sent both the flyer and a kit enhancement pack compared with controls (45.1% versus 43.4%, OR = 1.07, P = 0.047). In North East London, the flyer together with outdoor advertising was associated with a small but significant increase (45.6% versus 43.4%, OR = 1.09, P = 0.027). The largest increases were seen when all three interventions (flyer, pack, and advertising) were combined (49.5% versus 43.4%, OR = 1.28, P < 0.001). The increased uptake in the intervention groups was largest in "first-timers" and smaller amongst previous nonresponders and previously screened invitees.

Preformulated implementation intentions to promote colorectal cancer screening: a cluster-randomized trial.

OBJECTIVE: To evaluate an intervention based on implementation intention principles designed to increase uptake of colorectal cancer screening, and to examine differential efficacy by socioeconomic deprivation. METHOD: In England, adults aged between 60 and 69 years are invited for biennial fecal occult blood testing. A test kit and an information leaflet are mailed to each individual by the "Hubs" that deliver the national screening program. In the intervention group, three preformulated implementation intentions, based on known barriers to carrying out the test, were added to the information leaflet. Over a 12-week period, each week was randomly allocated to either the intervention (n = 12,414 invitations) or the control condition (n = 10,768), with uptake recorded at the Hub. Socioeconomic deprivation of each individual's area of residence was categorized into tertiles. RESULTS: There was no overall difference in uptake between control (40.4%) and intervention (39.7%) conditions, odds ratio (OR) = 0.97, 95% confidence interval (CI) [0.91, 1.04]. There was an interaction with deprivation, OR = 1.11, 95% CI [1.04, 1.18], but the positive effect observed in the lowest socioeconomic status (SES) tertile was small (35.2% vs. 33.0%), OR = 1.103, 95% CI [1.01, 1.21], and offset by a negative effect in the least deprived tertile (45.6% vs. 48.2%), OR = 0.90, 95% CI [0.82, 0.99]. The intervention had no significant effect in the middle tertile (38.9% vs. 40.8%), OR = 0.92, 95% CI [0.81, 1.04]. CONCLUSION: Preformulated implementation intentions did not increase overall colorectal cancer screening uptake and failed to make a sufficiently large impact on uptake among lower SES groups to merit their future use in this context.