7-day Recall Period Research Articles

Testing the Impact of Variations in Administration on the Kessler Psychological Distress Scale (K10).

Self-report measures are useful in psychological research and practice, but scores may be impacted by administration methods. This study investigated whether changing the recall period (from 30 to 7 days) and response option order (from ascending to descending) alters the score distribution of the Kessler Psychological Distress Scale (K10). Participants were presented with the K10 with either different recall periods or different response option orders. There was weak evidence of lower mean K10 scores when using a 7-day recall period than when using the 30-day recall period (B = 1.96, 95% CI [0.04-3.90]) but no evidence of a change in the estimated prevalence of very high psychological distress. Presenting the response options in ascending order did not affect mean scores, but there was weak evidence of reduced prevalence of very high distress relative to the descending order (incidence rate ratio [IRR] = 0.60, 95% CI [0.36-0.98]). These findings suggest that varying the administration method may result in minor differences in population estimates of very high psychological distress when using the K10.

Undergoing Lung Surgery (PSA-Lung) was appropriate for symptom assessment after discharge.

To examine whether a 7-day or 24-h recall period of Perioperative Symptom Assessment for Patients Undergoing Lung Surgery (PSA-Lung) was appropriate for symptom assessment after discharge. A total of 377 patients were recruited in a cohort study of patients who underwent lung surgery. We measured patient symptoms daily and weekly using the two recall period versions of the PSA-Lung scale, respectively. The psychometric properties of both versions were calculated. Spearman rank correlation coefficients and kappa (k) coefficients were used to measure the association between items score measured by the two version scales each week. Cohen's d effect size and mixed linear model were used to measure responsiveness to change over time. Spearman rank correlation coefficients between the symptom scores generated by the 7-day and 24-h versions (range 0.48-0.77; all P < 0.05). The correlations increased in patients in stable condition (weekly symptom change < 2). Cronbach's α coefficients for both ratings were > 0.87 and both had good test-retest reliability. The longitudinal analysis and Cohen's d effect sizes showed that both ratings had good ability to detect changes in all items. The 7-day retrospective scale was as effective as the 24-h retrospective scale in terms of psychometric performance. In the stage where the patient's symptoms change rapidly, it is recommended to use the 24-h retrospective scale for symptom monitoring. On the contrary, in a stable state, it can be considered to use the 7-day retrospective scale for monitoring to reduce the patient's burden.

Comparing estimates of psychological distress using 7-day and 30-day recall periods: Does it make a difference?

Self-report measures are widely used in mental health research and may use different recall periods depending on the purpose of the assessment. A range of studies aiming to monitor changes in mental health over the course of the COVID-19 pandemic opted to shorten recall periods to increase sensitivity to change over time compared to standard, longer recall periods. However, many of these studies lack pre-pandemic data using the same recall period and may rely on pre-existing data using standard recall periods as a reference point for assessing the impact of the pandemic on mental health. The aim of this study was to assess whether comparing scores on the same questionnaire with a different recall period is valid. A nationally representative sample of 327 participants in Australia completed a 7-day and 30-day version of the six-item Kessler Psychological Distress Scale (K6) and a single-item measure of psychological distress (TTPN item) developed for the Taking the Pulse of the Nation survey. Linear mixed models and mixed logistic regression models were used to assess whether altering the recall period systematically changed response patterns within subjects. No substantive recall period effects were found for the K6 or the TTPN, although there was a trend towards higher K6 scores when asked about the past 30 days compared to the past 7 days (b = 1.00, 95% CI: -0.18, 2.17). This may have been driven by the “feeling nervous” item which was rated higher using the 30-day compared to the 7-day recall period. Neither the K6 nor the TTPN item were significantly affected by the recall period when reduced to a binary variable of likely severe mental illness. The results indicate that altering the recall period of psychological distress measures does not substantively alter the score distribution in the general population of Australian adults.

Qualitative and psychometric evaluation of the PROMIS®-Fatigue SF-7a scale to assess fatigue in patients with moderately to severely active inflammatory bowel disease

BackgroundThis study evaluated the content validity and psychometric properties of the Patient-Reported Outcomes Measurement Information System® (PROMIS)-Fatigue Short Form 7a (SF-7a) v1.0 scale to determine its suitability in clinical trials to assess fatigue in patients with moderately to severely active Crohn’s disease (CD) and ulcerative colitis (UC).MethodsA qualitative interview assessed patients’ experience living with CD (N = 20) and UC (N = 19). The contents of the SF-7a scale were cognitively debriefed to evaluate content validity. A psychometric evaluation was performed using data from clinical trials of patients with CD (N = 360) and UC (N = 214). Correlations with Inflammatory Bowel Disease Questionnaire (IBDQ), Crohn’s Disease Activity Index (CDAI; CD only), and Mayo score (UC only) determined validity. The Patient Global Impression of Change (PGIC) was used to evaluate reliability and responsiveness to change. Using PGIC as an anchor, a preliminary threshold for clinically meaningful change was identified to define fatigue response in both CD and UC patients.ResultsAll patients reported fatigue as a common symptom. Patients confirmed SF-7a items were relevant to assessing fatigue, instructions and response options were clear, and its 7-day recall period was appropriate. Higher SF-7a scores were associated with higher disease activity (CDAI and Mayo score) and lower health-related quality of life (IBDQ), confirming known groups validity. The correlation of the SF-7a scale was higher with fatigue-related items. (rs ≥ -0.70) than with items not directly associated with fatigue. Test-retest reliability was moderate to good (0.54–0.89) among patients with stable disease, and responsiveness to change in disease severity was demonstrated from baseline to Week 12. A ≥7point decrease was identified as a reasonable threshold to define clinically meaningful improvement.ConclusionThe SF-7a scale is a valid, reliable, and sensitive measure of fatigue in patients with moderately to severely active IBD and can be used to evaluate treatment response.

Qualitative exploration of the visual function impairments and impacts on vision-dependent activities of daily living in Retinitis Pigmentosa and Leber Congenital Amaurosis: content validation of the ViSIO-PRO and ViSIO-ObsRO measures

BackgroundRetinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) are rare inherited retinal degenerative disorders. The associated visual impairments have significant impacts on patients’ vision-dependent activities of daily living (ADL), mobility, and distal health-related quality of life (HRQoL). To adequately capture patient and caregiver perspectives in clinical trials, patient and observer-reported outcome instruments must demonstrate sufficient evidence of content validity in the target population. This study aimed to explore the patient experience of RP/LCA and assess the content validity of the Visual Symptom and Impact Outcomes PRO (ViSIO-PRO) and ObsRO (ViSIO-ObsRO) instruments in RP/LCA.MethodsA total of 66 qualitative, combined concept elicitation (CE) and cognitive debriefing (CD) interviews were conducted (33 adults, 10 adolescents, 8 children and 15 caregivers of children) in the US, France, Germany, and Canada. Patients had a clinical and genetic diagnosis of RP/LCA from a range of genotypes. CE results were used to further inform the development of a conceptual model and CD interviews assessed the relevance and understanding of the 44-item ViSIO-PRO and 26-item ViSIO-ObsRO instruments. Interviews were conducted across two iterative rounds to allow item modifications.ResultsFindings were consistent across RP/LCA genotypes. Night blindness, reduced peripheral vision, vision in very bright lighting and light/dark adaptation were the most frequently reported visual function symptoms impacting vision-dependent ADL and mobility. Impacts on distal HRQoL domains were also reported. The ViSIO-PRO and ObsRO items were well understood by participants and relevant across genotypes. The instructions, 7-day recall period and response scales were well understood and endorsed. Participant and expert clinician feedback supported modifications to item wording, the addition of six new ViSIO-PRO items and one new ViSIO-ObsRO item, and the removal of one ViSIO-PRO item due to lack of relevance.ConclusionsFindings support the content validity of the ViSIO-PRO and ViSIO-ObsRO instruments for use across RP/LCA genotypes. Ongoing research to evaluate the psychometric validity of the instruments will support future use of the instruments as efficacy endpoints in clinical trials and in general clinical practice to track disease severity and impact of disease on functioning.

Status of household dietary diversity and associated factors among rural and urban households of Northern Uganda

BackgroundIn Northern Uganda, 21 and 52.4% of children under five are underweight and stunted, respectively while 32.9% of pregnant women are anemic. This demographic situation suggests among other issues, a lack of dietary diversity among households. Good nutrition practices that confer dietary quality such as dietary diversity are known to depend on nutrition knowledge and attitude and are shaped by sociodemographic and cultural factors. However, there is a paucity of empirical evidence to support this assertion for the variably malnourished population of Northern Uganda.MethodsA cross-sectional nutrition survey was conducted among 364 household caregivers (182 from two locations in Northern Uganda; Gulu District (the rural) and Gulu City (the urban), selected through a multistage sampling approach. The aim was to determine the status of dietary diversity and its associated factors between rural and urban households of Northern Uganda. The household dietary diversity questionnaire and the food frequency questionnaire on a 7-day reference period were used to collect data on household dietary diversity whereas multiple choice questions and the five points Likert Scale were used to determine knowledge and attitude toward dietary diversity. Consumption of ≤ 5 food groups were regarded as low in dietary diversity, 6–8 food groups as medium and ≥ 9 as high dietary diversity score using the FAO 12 food groups. An Independent two-sample t-test was used to differentiate the status of dietary diversity between the urban and rural areas. The Pearson Chi-square Test was used to determine the status of knowledge and attitude while Poisson regression was used to predict dietary diversity based on caregivers’ nutritional knowledge and attitude and their associated factors.ResultsThe 7-day dietary recall period revealed that dietary diversity was 22% higher in urban (Gulu City) than in the rural area (Gulu District) with rural and urban households achieving medium (score of 8.76 ± 1.37) and high (score of 9.57 ± 1.44) dietary diversity status, respectively. Diets in both locations were dominated by starchy cereals and tubers while animal-source foods and fruits and vegetables were the least consumed. A higher proportion (51.65%) of urban respondents had good nutrition knowledge toward dietary diversity compared to their rural counterparts (23.08%) and a significantly higher proportion (87.91%) of the former exhibited positive attitude towards dietary diversity than the rural counterparts (72.53%). Application of the Poisson regression shows that nutritional knowledge was a positive predictor of dietary diversity in the rural (β = 0.114; ρ = 0.000) than in the urban areas (β = -0.008; ρ = 0.551). Caregivers attitude had no significant effect across locations. In terms of associated factors, marital status is a positive predictor of dietary diversity in the urban (β = 1.700; ρ = 0.001) than the other location (β = -2.541; ρ = 0.008). Whereas education level of household caregiver and household food expenditure show negative effects across the two locations, the educational level of the household head is an outlier as it positively predicted dietary diversity in the rural (β = 0.003; ρ = 0.002) when compared to urban area (β = -0.002; ρ = -0.011).ConclusionRural households in Northern Uganda have medium-level dietary diversity with urban households having high dietary diversity. Diets in both locations are dominated by starchy cereals and roots and tubers. The urban–rural food divide can be harmonized through nutrition education and outreach, specifically focusing on the FAO 12 food groups. Attitude toward consumption of fruits and vegetables which are seasonally abundant would improve dietary diversity and nutritional outcomes in the study area.

Speed of clinical improvement in the real-world setting from patient-reported Psoriasis Symptoms and Signs Diary: Secondary outcomes from the Psoriasis Study of Health Outcomes through 12 weeks.

Rapid skin improvement is a key treatment goal of patients with moderate-to-severe psoriasis (PsO). To compare the speed of clinical improvement of approved biologics on the symptoms and signs of psoriasis assessed by patients using the validated Psoriasis Symptoms and Signs Diary (PSSD) through 12 weeks. Psoriasis Study of Health Outcomes (PSoHO) is an international, prospective, non-interventional study that compares the effectiveness of anti-interleukin (IL)-17A biologics versus other biologics, together with pairwise comparisons of ixekizumab versus five individual biologics in patients with PsO. Using the PSSD 7-day recall period, patients assessed the symptoms (itch, skin tightness, burning, stinging and pain) and signs (dryness, cracking, scaling, shedding/flaking, redness and bleeding) of their psoriasis (0-10). Symptom and sign summary scores (0-100) are derived from the average of individual scores. Percentage change in summary scores and proportion of patients with clinically meaningful improvements (CMI) in PSSD summary and individual scores are evaluated weekly. Longitudinal PSSD data are reported as observed with treatment comparisons analysed using mixed model for repeated measures (MMRM) and generalized linear mixed models (GLMM). Across cohorts and treatments, eligible patients (n = 1654) had comparable baseline PSSD scores. From Week 1, the anti-IL-17A cohort achieved significantly larger score improvements in PSSD summary scores and a higher proportion of patients showed CMIs compared to the other biologics cohort through 12 weeks. Lower PSSD scores were associated with a greater proportion of patients reporting their psoriasis as no longer impacting their quality-of-life (DLQI 0,1) and a high level of clinical response (PASI100). Results also indicate a relationship between an early CMI in PSSD score at Week 2 and PASI100 score at Week 12. Treatment with anti-IL-17A biologics, particularly ixekizumab, resulted in rapid and sustained patient-reported improvements in psoriasis symptoms and signs compared with other biologics in a real-world setting.

The 7-day recall period version of the Urticaria Control Test—UCT7

The Urticaria Control Test (UCT) is a well-established, very easy to use and calculate 4-item patient-reported outcome measure (PROM) to assess chronic urticaria (CU) disease control during the previous 4 weeks. Clinical trials and practice may benefit from the use of a UCT version with a shorter recall period, but this does not exist. To develop and validate a UCT version with a 7-day recall period, the UCT7. The UCT7 was developed, based on the UCT, and tested, in 152 patients with CU (spontaneous: n=101, inducible: n=51) for its reliability, validity and screening accuracy, and clinimetric properties, i.e. the cutoff for well-controlled disease and the minimal clinically important difference (MCID). The UCT7 showed excellent internal consistency reliability with a Cronbach alpha value of 0.91 and test-retest reliability with an intraclass correlation coefficient of 0.83. Convergent validity was high and strongly correlated with anchors of disease control, wheal and angioedema frequency, and urticaria-related quality of life impairment. The UCT7 showed excellent sensitivity to change, however, changes in angioedema activity and impact did not correlate well with changes in UCT7. Based on receiver-operating characteristic curve analysis, the proportion of correctly classified patients, and patients' assessment of treatment efficacy, we recommend a cutoff value of 12 points for identifying patients with well-controlled disease. The UCT7 MCID for improvement was estimated to be 2 points. The UCT7 is a validated 7-day recall period version of the UCT. It is ideal for the assessment of disease control at short intervals in CU patients in clinical studies and practice.

The Amblyopia Quality of Life (AmbQoL): Development and Content Validation of a Novel Health-Related Quality of Life Instrument for Use in Adult and Pediatric Amblyopia Populations.

Amblyopia is a neurodevelopmental vision disorder, characterized by poor vision in one or both eyes. Given the lack of existing clinical outcome assessments (COA) considered fit-for-purpose for amblyopia clinical trials, this study developed new COAs to assess amblyopia symptoms and health-related quality of life (HRQoL) impacts in adult and pediatric amblyopia populations that conform with best practice standards and regulatory guidelines. Findings from a targeted qualitative literature review informed the development of three versions of the new Amblyopia Quality of Life Questionnaire (AmbQoL): a patient-reported outcome measure (PRO) for individuals aged 13years and older, a PRO for children aged 9-12years, and an observer-reported outcome measure (ObsRO) for caregivers of children aged 4-8years. Qualitative interviews were conducted with the target populations, and with ophthalmologists experienced in treating amblyopia patients to evaluate the content validity for further development of the AmbQoL. A translatability assessment was conducted to ensure cultural appropriateness and usability across multiple languages. Feedback from Food and Drug Administration (FDA) was also sought on the instruments, and clinical experts provided input at key stages. Interviews were conducted with 112 patients/caregivers and ten ophthalmologists from the USA, France, and Germany. The instructions, items, and response options were well understood across all AmbQoL versions. Feedback from the patients, caregivers, ophthalmologists, the translatability assessment, the FDA, and the expert clinicians informed minor wording modifications to enhance clarity and translatability. Some items were removed due to low relevance. The study resulted in a 23-item adult/adolescent PRO, 24-item child PRO, and 12-item ObsRO, each employing a 7-day recall period. Each AmbQoL version has documented support for its face and content validity for use in amblyopia populations aged ≥ 4years. Further research is necessary to evaluate the psychometric measurement properties of the AmbQoL instruments to enable their use in amblyopia treatment trials.

The Oxford Elbow Score demonstrated good measurement properties when used with a shortened 7-day recall period

The Oxford Elbow Score (OES) is a well-validated, elbow-specific, patient-reported outcome measure (PROM), originally assigned a 4-week recall period. For PROMs, short recall periods could have some advantages, such as optimizing validity by minimizing the negative effects of inaccurate recollection and temporal trends (increase or decrease) in symptoms over the course of the recall period. Temporal trends in elbow function can, for example, be expected to occur over 4 weeks in patients recovering from an injury or surgery. The purpose of this study was to evaluate the measurement properties of the OES using a shortened, 7-day, recall period (OES-7d). The inclusion criteria were fracture, tendon rupture or dislocation affecting the elbow, and age ≥18 years. Patients with Quick Disabilities of the Arm, Shoulder and Hand (QuickDASH) scores of ≥10 points preinjury (pre-existing upper extremity condition) or concurrent upper extremity injuries were excluded. Patients completed the OES-7d, QuickDASH, and Single Assessment Numeric Evaluation-Function for the last 7 days preinjury (T1), the first 7 days postinjury (T2) and a 7-day period 3-5 months postinjury (T3). Correlations were assessed with Spearman's rho. Analyses of construct validity (correlation between scores) and internal consistency (Cronbach's alpha) were based on T3 data. Responsiveness was assessed by correlating changes in scores (change scores) between time points. Intra-rater reliability was assessed by calculating intraclass correlation coefficients based on 2 administrations (1- to 3-week interval) of PROMs in a separate group of patients who had sustained an elbow injury 1-2 years previously. Seventy-five patients (45 women) were included between May 2020 and July 2021. Their mean age was 51.7 years. At T3, Spearman's rho was-0.91 for the correlation between OES total and QuickDASH scores and 0.76 for the correlation between OES total scores and Single Assessment Numeric Evaluation-Function values (construct validity). Spearman's rho for correlation between OES total and QuickDASH change scores from T2 to T3 (T3 minus T2) was-0.85 (responsiveness for improvement) and-0.88 for change scores from T1 to T2 (T2 minus T1, responsiveness for deterioration). For the OES domains, Cronbach's alpha was 0.83 for elbow function, 0.91 for pain and 0.90 for social-psychological domains. The intraclass correlation coefficient for the OES total score was 0.96. The OES demonstrated good measurement properties when used with a 7-day recall period (OES-7d). These results further establish the OES as a well-validated, elbow-specific PROM and support using a 7-day recall period.

The role of recall periods when predicting food insecurity: A machine learning application in Nigeria

Defining and measuring food insecurity at the household level is critical for policymakers, aid agencies, and international organizations. Food insecurity indicators, such as the Food Consumption Score, are based on a given recall period, usually 7-days. Still, using other indicators or methodologies makes surveys use different recall periods (e.g., 30-days or 12 months). This study uses machine learning methods and four waves of the Nigeria LSMS-ISA datasets to assess the implications of using different recall periods when predicting food insecurity measures. In addition to machine learning methods, the novelty of this study is the use of big data and relevant weather data to predict the food insecurity status of Nigerian farming families. Our results show that experience-based food insecurity indicators, measured using a 7-days recall period, have a high predictability accuracy (78%–90%). More importantly, we find that predictors computed using a 7-day recall period can detect about seven out of ten households considered food-insecure by indicators measured using a recall period of 30-days.

Validating a Self-Reported Medication Nonadherence Measure in the Context of Multiple Chronic Diseases and Routes of Medication Administration Among Patients with Type 2 Diabetes.

Patients with diabetes may take oral and injectable medications and often have comorbid chronic diseases. It is unclear whether to assess nonadherence for oral and injectable medications separately or combined and for comorbid conditions separately or combined. We conducted two cognitive interview studies among patients with type 2 diabetes who were prescribed medications for oral or injectable diabetes medications (Study 1) or at least one diabetes, blood pressure, and cholesterol medication (Study 2). Participants completed the two-domain DOSE-Nonadherence measure, which assesses extent of nonadherence and reasons for nonadherence. We asked about interpretation of instructions and items, recall period, ability to respond accurately with separate versus combined versions, and comprehensiveness of reasons for nonadherence to injectable medications. Based on Study 1 (n=14), nonadherence to injectable and oral medications should be assessed separately. Participants believe they can respond accurately to 7-day recall period for daily medications and a one-month recall period for weekly injectable medications. New reasons for nonadherence to injectable medications were perceived as relevant. Based on Study 2 (n-12), nonadherence to medications for diabetes, blood pressure, and cholesterol should be assessed separately. Although separate versions increase response time, it may improve accuracy. Responses to the measure can facilitate conversations about nonadherence between providers and patients to inform clinical decision-making.

S403 Dupilumab Reduces the Emotional and Dysphagia-Related Impacts of Eosinophilic Esophagitis to Improve Health-Related Quality of Life

Introduction: Eosinophilic esophagitis (EoE) is a chronic, type 2 inflammatory disease of the esophagus which substantially impairs quality of life (QoL). Dupilumab, a fully human mAb, blocks the shared receptor component for IL-4/IL-13, key drivers of type 2 inflammation in EoE. In the 3-part LIBERTY-EoE-TREET phase 3 trial (NCT03633617), Parts A and B demonstrated that weekly (qw) dupilumab 300mg improved clinical, symptomatic, histologic, and endoscopic aspects of EoE and was generally well tolerated in patients (pts) ≥12 years at Wk 24. The objective of this post-hoc analysis was to assess the effect of dupilumab vs placebo on improving aspects of QoL using the EoE Impact Questionnaire (EoE-IQ), a novel disease-specific measure of health-related QoL in EoE patients, during the 24-wk double-blind treatment period in Parts A and B. Methods: In Part A, 42 pts received dupilumab 300mg qw, and 39 received placebo. In Part B, 80 pts received dupilumab 300mg qw, and 79 received placebo for 24 wks. The EoE-IQ is a patient-reported 11-item questionnaire that measures the impact of EoE on emotional, social, work and school, and sleep aspects of a patient (over a 7-day recall period). It was developed in line with best practices in the field of clinical outcomes assessment and includes the most relevant QoL impacts of EoE as identified through literature review and discussion with therapeutic area experts and confirmed via patient interviews. Response to each item is on a 5-point scale ranging from 1-5 with a higher score indicating a more negative impact on QoL. Results: The most burdensome impacts of EoE at baseline were related to overall emotional impact and anxiety around dysphagia (Table). Dupilumab showed a nominally significant reduction vs placebo in 6 items in Parts A and B: ‘bothered’, ‘worried about swallowing’, ‘worried about choking’, ‘worried about swallowing in public’, ‘social activities’, ‘sleep disruption’ (all nominal p< 0.05), and in 1 additional item in Part B (‘embarrassed’, nominal p< 0.05). The largest placebo adjusted change from baseline to Wk 24 was observed in items relating to overall emotional impact and anxiety, including ‘worried about swallowing in public’ (Part A: –0.90; Part B: –0.45), ‘bothered’ (–0.64; –0.53), ‘worried about swallowing’ (–0.73; –0.65), and ‘worried about choking’ (–0.61; –0.57). Conclusion: EoE QoL was improved among pts on dupilumab 300mg qw, with improvement driven by emotional and social impacts, and sleep. Table 1. - EoE-IQ at baseline and after 24 weeks treatment with weekly dupilumab 300mg or placebo. Values after first rescue treatment use were set to missing (censoring), then multiple imputation was used to impute missing values. Mean absolute value at baseline Mean absolute value at Week 24 EoE-IQ Item Dupilumab300 mg qw Dupilumab 300 mg qw LS Mean Difference vs placebo P value Part A 1. Bothered 3.08 1.80 –0.64 0.0043 2. Worried swallowing 2.88 1.69 –0.73 0.0011 3. Worried choking 2.42 1.60 –0.61 0.0040 4. Embarrassed 1.91 1.34 –0.21 0.2336 5. Worried swallowing public 2.80 1.46 –0.90 0.0001 6. Social activities 2.06 1.40 –0.43 0.0311 7. Family 1.44 1.29 –0.20 0.2560 8. Friends 1.39 1.28 –0.31 0.0813 9. Keep up work/school 1.48 1.30 –0.28 0.1379 10. Miss work/school 1.37 1.34 –0.09 0.5504 11. Sleep disruption 1.82 1.21 –0.42 0.0027 Part B 1. Bothered 3.50 1.97 –0.53 0.0004 2. Worried swallowing 3.10 1.60 –0.65 < 0.0001 3. Worried choking 2.72 1.45 –0.57 < 0.0001 4. Embarrassed 2.21 1.28 –0.28 0.0141 5. Worried swallowing public 2.96 1.53 –0.45 0.0020 6. Social activities 2.34 1.35 –0.30 0.0117 7. Family 1.72 1.22 0.01 0.8765 8. Friends 1.68 1.19 –0.06 0.4409 9. Keep up work/school 1.81 1.32 –0.08 0.4170 10. Miss work/school 1.32 1.18 –0.09 0.3390 11. Sleep disruption 1.94 1.36 –0.22 0.0409 EoE-IQ, Eosinophilic Esophagitis Impact Questionnaire; LS, least squares; qw, weekly.

Generation of evidence supporting the content validity of SF-36, FACIT-F, and LupusQoL, and novel patient-reported symptom items for use in patients with systemic lupus erythematosus (SLE) and SLE with lupus nephritis (LN)

ObjectiveSLE and lupus nephritis (LN) have significant impacts on the health-related quality of life of patients living with the condition, which are important to capture from the patient’s perspective using...

A Qualitative Study to Develop and Evaluate the Content Validity of the Vitiligo Patient Priority Outcome (ViPPO) Measures.

IntroductionVitiligo can be associated with a psychological burden, stigmatization and impaired quality of life. Tools to assess the impact of vitiligo exist; however, none were developed in line with the FDA’s patient-reported outcome (PRO) Guidance for Industry. This study aimed to explore the content validity of two newly developed PRO measures to assess the impact of facial and total body vitiligo on how patients feel and function.MethodsDraft PRO measures were developed from existing literature and input from PRO experts, a patient advocate and a clinical expert. Qualitative interviews were conducted with US participants living with vitiligo and international dermatologists with vitiligo expertise. Concept elicitation methodology explored the relevance of concepts in the draft PRO, while cognitive debriefing assessed conceptual relevance and understanding/interpretation. Items were iteratively amended/added throughout the interview study.ResultsThe 60 participants included adults (n = 48, 63% female, 18–62 years old) and adolescents (n = 12, 67% female, 12–17 years old) with Fitzpatrick Skin Types I–VI. Expert dermatologists from the US (n = 8), EU (n = 4), India (n = 1) and Egypt (n = 1) participated. Concept elicitation was utilized to confirm the signs/symptoms of vitiligo and the associated impact on emotional/psychological wellbeing, social functioning, daily life and work/school. Conceptual saturation was achieved. Most participants reported impacts on their emotional/psychological wellbeing (n = 57, 95%), e.g. feeling self-conscious (n = 35, 58%). Participants reported impacts on social functioning (n = 53, 88%), e.g. vitiligo being noticed by others (n = 42, 70%). There was general consensus between participants and expert dermatologists. Cognitive debriefing confirmed that the items were well understood. Most items were conceptually relevant; feeling self-conscious and feeling frustrated were highly endorsed. Items were removed based on low conceptual relevance (feeling abandoned, skin roughness) and expected redundancy (four items), resulting in two measures with three proposed domain scores: Emotional/Psychological Wellbeing; Social Functioning; and Physical Sensation. No comprehension concerns were observed in relation to the 7-day recall period or the item response scale/options. Eight dermatologists reviewed the PRO measures, confirming comprehensiveness and relevance.ConclusionThe draft Vitiligo Patient Priority Outcomes (ViPPO) measures evaluate the impact of facial (ViPPO-F) and total body (ViPPO-T) vitiligo on emotional/psychological and social functioning. The ViPPO measures are well understood, comprehensive and content valid for adults and adolescents with vitiligo.Supplementary InformationThe online version contains supplementary material available at 10.1007/s13555-022-00772-4.

PCR46 A Mixed-Methods Study Using Cognitive Debriefing to Assess the Content Validity of a New Health and Functioning Measure for Tobacco and/or Nicotine Product Users

To inform tobacco harm reduction strategies, a new consumer reported outcome measure (CROM) was developed to assess perceived Health and Functioning (H&F) among users of tobacco and/or nicotine products (TNP). This study assessed the face, content validity, and early measurement properties of the draft H&F CROM. Cognitive debriefing interviews were conducted in two waves in the UK and US with adult users of: cigarettes (n=20), e-cigarettes (n=20), heated tobacco products (IQOSTM) (n=20), smokeless tobacco products (n=10), and nicotine replacement therapy products (n=10). Participants completed the draft measure (195 items) electronically through a “think-aloud” process. Different response options were tested. Transcripts were analyzed thematically using Atlas.ti. Exploratory psychometric analysis was conducted by Rasch Measurement Theory (RMT) analysis. Expert panel meetings were held to inform result interpretation and updating the CROM after each wave. The participants generally endorsed the content of the CROM, while the interim analysis allowed for some early item and scale refinements. The RMT analysis largely supported the scales’ reliability, sample-to-scale targeting, and discrimination potential across the TNP user groups. Conceptual overlap between some items was identified through both qualitative and quantitative evidence. Mixed-methods evidence was used to guide the selection of response scale, e.g., severity difficulty or frequency that could better match the underlying concept of each scale, while the 7-day recall period was found to have less of a ceiling effect. This study provides early supporting evidence of the new H&F CROM’s content relevance, clarity, and ease of completion. It comprises ten scales (146 items) covering different health concepts (including oral, lung, and general health and breathing difficulty), appearance, bother with TNP smell, as well as different functioning concepts (including work, social, and well-being). A psychometric validation study is being conducted to inform the item content, scoring structure, and measurement properties of the CROM.

Key measurement concepts and appropriate clinical outcome assessments in pediatric achondroplasia clinical trials

BackgroundThis study aimed to identify fit-for-purpose clinical outcome assessments (COAs) to evaluate physical function, as well as social and emotional well-being in clinical trials enrolling a pediatric population with achondroplasia. Qualitative interviews lasting up to 90 min were conducted in the US with children/adolescents with achondroplasia and/or their caregivers. Interviews utilized concept elicitation methodology to explore experiences and priorities for treatment outcomes. Cognitive debriefing methodology explored relevance and understanding of selected COAs.ResultsInterviews (N = 36) were conducted with caregivers of children age 0–2 years (n = 8) and 3–7 years (n = 7) and child/caregiver dyads with children age 8–11 years (n = 15) and 12–17 years (n = 6). Children/caregivers identified pain, short stature, impacts on physical functioning, and impacts on well-being (e.g. negative attention/comments) as key bothersome aspects of achondroplasia. Caregivers considered an increase in height (n = 9/14, 64%) and an improvement in limb proportion (n = 11/14, 71%) as successful treatment outcomes. The Childhood Health Assessment Questionnaire (CHAQ) and Quality of Life in Short Stature Youth (QoLISSY-Brief) were cognitively debriefed. CHAQ items evaluating activities, reaching, and hygiene were most relevant. QoLISSY-Brief items evaluating reaching, height bother, being treated differently, and height preventing doing things others could were most relevant. The CHAQ and QoLISSY-Brief instructions, item wording, response scales/options and recall period were well understood by caregivers and adolescents age 12–17. Some children aged 8–11 had difficulty reading, understanding, or required caregiver input. Feedback informed minor amendments to the CHAQ and the addition of a 7-day recall period to the QoLISSY-Brief. These amendments were subsequently reviewed and confirmed in N = 12 interviews with caregivers of children age 0–11 (n = 9) and adolescents age 12–17 (n = 3).ConclusionsAchondroplasia impacts physical functioning and emotional/social well-being. An increase in height and improvement in limb proportion are considered to be important treatment outcomes, but children/adolescents and their caregivers expect that a successful treatment should also improve important functional outcomes such as reach. The CHAQ (adapted for achondroplasia) and QoLISSY-Brief are relevant and appropriate measures of physical function and emotional/social well-being for pediatric achondroplasia trials; patient-report is recommended for age 12–17 years and caregiver-report is recommended for age 0–11 years.

Consumption of High-Risk Foods in the Canadian Population, Foodbook Study, 2014 to 2015

Many foods have the potential to cause foodborne illness; however, some pose a higher risk. Data were collected through the Foodbook study, a population-based telephone survey conducted between 2014 and 2015 that assessed 10,942 Canadians' food exposures using a 7-day recall period. The 19 foods included in the survey were identified as high risk for common foodborne pathogens in Canada. Results were analyzed by age group, gender, region of residence, income, and education. Consumption proportions of high-risk foods ranged from 0.4% (raw oysters) to 49.3% (deli meats). Roughly 94% of the population reported consuming one or more high-risk food in the past week. Certain high-risk food behaviors were associated with demographic characteristics. High-risk adults such as those 65 years or older still report consuming high-risk foods of concern, including deli meats (41.8%), soft cheeses (13.7%), and smoked fish (6.3%). Consumption of certain foods differed between genders, with males consuming significantly more deli meats, hot dogs, and raw or undercooked eggs and females consuming significantly more prebagged mixed salad greens. The overall number of high-risk foods consumed was similar, with both genders most frequently consuming three to five high-risk foods. High-risk food consumption was seen to increase with increasing household income, with 14.2% of the highest income level consuming six-plus high-risk foods in the past week, compared with 7.1% of the lowest income level. If a respondent had heard of a risk of foodborne illness associated with a food, it did not affect whether it was consumed. Additional consumer food safety efforts put in place alongside current messaging may improve high-risk food consumption behaviors. Enhancing current messaging by using multifaceted communications (e.g., social media and information pamphlets) and highlighting the large incidence and severity of foodborne illnesses in Canada are important strategies to improve behavior change.

Do You Recall?: Results From a Within-Person Recall Study of the Patient-Reported Outcomes Measurement Information System (PROMIS) Short Form v2.0 – Physical Function 8c

ObjectivesThis study aimed to determine whether responses to Patient-Reported Outcomes Measurement Information System Short Form v2.0 – Physical Function 8c (PROMIS PF8c) items differed when the use of a 7-day recall period was compared with no specified recall period. MethodsUsing a within-subject design, we surveyed 1810 individuals from the US general population, administering PROMIS PF8c at survey beginning and end. The order of measure presentation was randomly assigned. We calculated recall difference scores (RDSs) as no recall score minus 7-day recall score using both item response theory-based T scores and raw summed scores. We examined the distribution and created Bland-Altman plots for both RDSTscore and RDSRaw. We also calculated correlations between no recall versus 7-day recall T score and raw scores. Finally, we determined whether differences in no recall versus 7-day recall scores were associated with patient-reported PF. ResultsRDSTscore and RDSRaw had means (root mean square differences) of 0.00 (5.43) and −0.04 (3.79), respectively. The vast majority (%) of RDSTscore and RDSRaw values fell between the Bland-Altman limits of agreement (−10.65 to 10.66 and −7.46 to 7.38, respectively). Pearson’s correlations between no recall and 7-day recall for T scores and raw scores were 0.88 and 0.87, respectively. Effect sizes for mean RDSTscore and RDSRaw compared across level of Eastern Oncology Cooperative Group performance status, patient global impression of PF severity, and single PF items were near 0. ConclusionsWe did not find any significant recall period effect on PF8c responses. Therefore, we recommend the use of the PROMIS physical function standard, with no specified recall time period.

Development of the Multiple Myeloma Symptom and Impact Questionnaire: A New Patient-Reported Outcome Instrument to Assess Symptom and Impacts in Patients With Multiple Myeloma

ObjectivesThis study aimed to develop and assess the content validity of a patient-reported outcomes (PROs) instrument to measure symptoms and impacts experienced by patients with active multiple myeloma (MM). MethodsThe PRO instrument was developed using an iterative, mixed-methods approach. The list of concepts was generated based on a review of existing evidence (qualitative studies and literature) and post hoc psychometric evaluations of 2 PRO instruments in 3 clinical trials. A total of 30 adult patients with MM from the United States participated in hybrid concept elicitation/cognitive debriefing interviews to validate the content validity of the newly developed PRO instrument. Translatability assessment was completed in 8 languages. ResultsThe item generation process resulted in 17 symptom and 9 impact concepts for evaluation. The concept elicitation interviews and analysis were based on the first 25 participants; evidence of saturation was observed. The cognitive debriefing interviews and analysis were based on the last 23 participants across 4 waves of interviews. On the basis patient feedback, 10 items were removed, and 1 item was added to the PRO instrument. The translatability assessment resulted in 1 minor revision. The multiple myeloma symptom and impact questionnaire (MySIm-Q) includes 11 symptom and 6 impact concepts, organized within 8 hypothesized subdomains, with each concept measured using a 5-point verbal rating scale and a 7-day recall period. ConclusionsThe MySIm-Q instrument was developed using rigorous and mixed methodology and with direct input from patients who received a diagnosis of MM. The MySIm-Q has good content validity and is culturally relevant for use in global clinical trials.