STUDY OBJECTIVES:Most patient-reported outcome measures for narcolepsy focus on excessive daytime sleepiness or do not focus on specific disease impacts (e.g. cataplexy, cognitive difficulties, fatigue, daily function). The Functional Impacts of Narcolepsy Instrument (FINI) was developed to measure key functional impacts in people with narcolepsy type 1 and 2 (NT1/NT2). METHODS:The instrument was developed separately in NT1/NT2 populations with input from patients, clinical experts experienced in narcolepsy management, and clinical outcome scientists. Development included: patient experience interview studies informing item development; item development including de novo items, and modified/original items from the Patient-Reported Outcomes Measurement Information System (PROMIS) library; two waves of patient-debriefing interviews testing initial/modified item drafts; determination of scale structure using exploratory factor and Rasch analyses, and confirmatory factor analyses for NT2; consensus meetings between clinical sleep experts/clinical outcome experts to finalize the FINI draft items and structure with a 7-day recall period. RESULTS:The final FINI for NT1 had 28 items within six independent domains: Tiredness, Cognitive Functioning, Cataplexy, Social Activities, Everyday Activities, and Everyday Responsibilities. The final NT2 version (FINI-NT2) included the same items excluding the Cataplexy domain (23 items). Final FINI and FINI-NT2 items demonstrated good content validity and covered the main impacts of NT1 and NT2, respectively, as confirmed by patient interviews, exploratory/confirmatory factor analyses, and Rasch analysis. CONCLUSIONS:The novel FINI assesses key functional impacts of narcolepsy in people with NT1/NT2 for use across clinical settings, and adds to the number of existing clinical outcome assessments for central disorders of hypersomnolence. Statement of Significance People with narcolepsy type 1 (NT1) or narcolepsy type 2 (NT2) often experience impacts on their daily life, such as cognitive difficulties, fatigue, and impaired work/school responsibilities, leading to decreased quality of life. Despite these recognized challenges, there remains a need for an additional tool that measures functional outcomes and complements existing narcolepsy symptom, severity, and impacts assessments. The Functional Impacts of Narcolepsy Instrument (FINI) was developed to evaluate functional impacts of narcolepsy that are important and meaningful to people living with NT1 (FINI) and NT2 (FINI-NT2). The instrument offers a patient-centered outcome measure for assessing narcolepsy impacts and treatment benefits related to changes in functional outcomes that can be used in both the clinical trial setting and clinical practice.

Myelofibrosis (MF) is a bone marrow cancer causing impactful symptoms, notably excessive fatigue. While the Myelofibrosis Symptom Assessment Form (MFSAF) v4.0 and PROMIS Fatigue 7a are frequently used in clinical trials, they lack published evidence for content validity in MF. This study evaluated the content validity, psychometric properties, and patient-perceived meaningful change for both measures in an MF population. This observational, mixed-methods study involved qualitative concept elicitation (CE) and cognitive debriefing (CD) interviews (n = 17) with patients with primary or secondary MF. Psychometric properties, including test–retest reliability and construct validity, were assessed via online surveys administered at two timepoints (n = 39). Interviews confirmed fatigue as a universal symptom. CD results showed both instruments were well understood, relevant, and comprehensive, with no recommended changes. Participants identified a 1-to-2-point change on individual item ratings as a meaningful improvement. Psychometric analysis demonstrated excellent internal consistency (α > 0.80) and strong test–retest reliability (ICCs 0.85–0.93) for both measures. Construct validity was supported by expected large correlations with similar constructs (MSAS-SF) and lower correlations with distal concepts such as mental health (SF-12 v2). The MFSAF v4.0 and PROMIS Fatigue 7a effectively capture key MF symptoms and impacts. Both measures are well understood by patients and demonstrate sufficient reliability and validity for use as clinical trial endpoints. Patients can clearly conceptualize and describe meaningful changes in their symptom experience using these tools. This study evaluated how well two questionnaires, the MFSAF v4.0 and the PROMIS Fatigue 7a, measure symptoms and fatigue in people living with myelofibrosis. Myelofibrosis is a disease that often causes severe symptoms such as fatigue and weakness that can affect daily life and quality of life. The aim was to assess whether these patient-reported outcome measures accurately capture symptom and fatigue experiences reported by patients, and to explore what item-level changes patients considered meaningful. The study involved interviews with people with myelofibrosis, as well as surveys completed at two timepoints. Both the MFSAF v4.0 and the PROMIS Fatigue 7a showed evidence of reliability and consistency, were easy for patients to understand, and provided evidence of validity for measuring symptoms among patients with myelofibrosis. Patients generally felt that a change of 1 to 2 points on individual questionnaire items represented a meaningful improvement. Patients also reported that not all symptoms needed to improve for an overall change in their scores to feel meaningful. Overall, the results support considering the MFSAF v4.0 and the PROMIS Fatigue 7 questionnaires as outcome measures in myelofibrosis clinical trials. Further research is needed to confirm how well these measures detect change over time.

Abstract Introduction Narcolepsy, a rare, chronic neurologic disorder that affects sleep-wake stability, is associated with detrimental effects on functioning, quality of life, and productivity. There is a need for a fit-for-purpose clinical outcome assessment that captures the functional impacts of narcolepsy in various clinical settings to complement the Narcolepsy Severity Scale. The objective was to develop a new patient-reported outcome (PRO) measure, the Functional Impacts of Narcolepsy Instrument (FINI), for evaluating treatment response in patients with narcolepsy type 1 (NT1) or type 2 (NT2). Methods Initial item generation was derived using concept elicitation (patients with NT1, n=21), cognitive debriefing (NT1, n=20; NT2, n=15), and item debriefing with medical experts. A mixed-methods approach was used to determine the final PRO using two independent datasets: a cross-sectional, US observational NT1 study (n=126) and a global Phase 2 study (NT1 and NT2, n=125). Results Concept elicitation interviews were conducted with patients with NT1 to understand meaningful symptoms and impacts and generate items deemed relevant by patients and medical experts. The original instrument version was then refined and cognitively debriefed with patients in two stand-alone studies resulting in a 48-item pilot version. All items have a 7-day recall period on a 5-point Likert scale of frequency from “never” to “always” or severity from “not at all” to “very much.” The four a priori factors were expanded to a six-factor structure based on exploratory factor analysis results and confirmed to be multidimensional on Rasch analysis, resulting in a final 28-item version with 6-domains (tiredness, cognitive functioning, cataplexy [for NT1], social activities, everyday activities, and everyday responsibilities) (FINI v1.0). Summary scores for each of 6 domains for NT1 and 5 domains for NT2 were calculated and transformed to a standardized scale from 0 (best health) to 100 (worst health). Conclusion The FINI is a modular, domain-specific assessment of functional impacts that is fit-for-purpose for patients with NT1 (FINI) and NT2 (FINI-NT2). It has demonstrated good content validity and psychometric methods support scoring and interpretation. The FINI can be used to generate evidence in clinical trials or practice for functional recovery in patients with NT1 and NT2. Support (if any) Funded by Takeda

Abstract Introduction The Functional Impacts of Narcolepsy Instrument (FINI) is a fit-for-purpose patient-reported outcome (PRO) measure developed for the assessment of patients with narcolepsy type 1 (NT1) or type 2 (NT2). To provide a baseline for interpretation in future interventional trials, comparative scores for the FINI domains were obtained from a cohort of healthy volunteers included in a United States observational study. Methods Healthy volunteers without excessive daytime sleepiness (EDS; ≤10 on the Epworth Sleepiness Scale [ESS] at screening) or any associated medical disorder were eligible for inclusion. Participants received a link to complete a cross-sectional online survey that included a 23-item, 5-domain form of the FINI (excluding cataplexy items) and additional selected quality of life reference questionnaires (SF-36 and EQ-5D). FINI items evaluate narcolepsy/EDS symptoms and impacts on daily functioning. All items have a 7-day recall period on a 5-point Likert scale of frequency from “never” to “always” or severity from “not at all” to “very much.” For each domain, an average item score is calculated and standardized to a 0–100 scale. Lower scores indicate better health/functioning. Responses were compared to patients with NT1 and NT2 from an observational study (n=126) and a phase 2 trial (NCT04096560; n=125). Results Thirty-one healthy volunteers (45.2% female) completed the survey. Participants had a mean age of 44.8 years and a median (range) ESS score of 4.0 (0–10.0) at baseline. FINI domain scores ranged from 10.0 to 22.2 in healthy volunteers, significantly lower than values reported for patients with narcolepsy in observational and phase 2 studies (46.2 to 67.9). Similar patterns were observed for reference PROs, with better quality of life/health status reported for healthy controls than for narcolepsy samples. Mean (SD) FINI domain values for healthy volunteers were: Tiredness: 22.2 (19.8); Cognitive Functioning: 10.0 (10.5); Social Activities: 16.1 (17.1); Everyday Activities: 12.9 (10.7); and Everyday Responsibilities: 11.6 (15.8). Conclusion Mean FINI domain scores from healthy volunteers can serve as normative thresholds and provide a benchmark for comparison of FINI responses reported by patients with narcolepsy, allowing for the evaluation of treatment effects in clinical trials. Support (if any) Funded by Takeda

Background Psoriasis is a common autoimmune dermatologic condition which has a pronounced negative impact on patient quality of life and disease burden. Currently, there are a number of treatments available for psoriasis, with differences in efficacy, mechanism of action, mode of administration, adverse effects, and tolerability. However, a reliable, validated patient-reported instrument to address patient expectations and of psoriasis treatment has not been developed. This project was undertaken with the aim of developing a fit-for-purpose self-reported instrument to inform patient expectations and preferences of psoriasis treatments. Methods Two studies, both utilizing qualitative and quantitative methods, were conducted in patients within the entire spectrum of psoriasis severity. In Study 1, a group concept mapping (GCM) exercise was conducted with dermatologists and moderate-to-severe psoriasis patients to identify concepts important in the treatment of psoriasis. In Study 2, a preliminary Treatment Acceptability Questionnaire (TAQ) was developed using GCM-derived concepts from Studies 1 and 2, followed by cognitive debriefing (CD) telephone interviews of the preliminary TAQ. In Study 2, another GCM exercise was conducted with mild and newly diagnosed psoriasis patients. Psychometric analyses were performed on the TAQ to evaluate validity and reliability. Results The Study 1 GCM exercise generated 43 concepts from moderate-to-severe psoriasis patients (n = 20) and dermatologists (n = 10). In Study 2, 37 GCM concepts were generated from mild and newly diagnosed psoriasis patients (n = 20). From the 2 GCM exercises, 28 concepts were selected to form the preliminary TAQ; CD interviews indicated strong understanding and relevance of TAQ items among patients with disease ranging from mild to severe. The final TAQ consisted of 20 items; psychometric analysis demonstrated strong validity and reliability of the TAQ. Conclusions The TAQ is a novel psychometrically validated patient-reported instrument to inform healthcare providers of patients’ expectations of and preferences for treatment of their psoriasis and can help in shared decision making between patients and physicians.

The effect of group medical visits (GMV) compared to individual medical visits (IMV), on weight and blood pressure in a large primary care practice serving a predominantly underserved population, was assessed. The records of 304 patients attending a weight-loss program were analyzed using mixed-effects regression models. Patients in GMV lost an average of 11.63 lbs, whereas patients in IMV lost an average of 3.99 lbs (p < 0.001). A total of 55% of patients lost ≥7% in GMV compared to 11% of patients in IMV (p ≤ 0.001). Individuals who lost >5% of their baseline weight had a higher reduction in overall blood pressure. For systolic and diastolic blood pressure, the differences between baseline and three months for GMV and IMV were −7.4 vs. 4.1 mm of Hg (p = 0.002) and −4.6 vs. 4.2 mm of Hg (p = 0.003), respectively. Results from this study demonstrate that GMV may be a potentially useful modality for addressing weight and blood pressure in an underserved population.

Purpose Psychometric evaluation of the Nocturia Impact (NI) Diary was conducted to support its use as a trial endpoint. Methods As part of a randomized, controlled Phase 2 clinical trial investigating a novel drug candidate for nocturnal polyuria, adult nocturia patients completed the NI Diary and a voiding diary for three nights preceding their clinic visit at Baseline and Weeks 1, 4, 8, and 12 (end of treatment). Exit interviews were conducted to obtain patient impressions of the NI Diary. Results A total of N = 302 participants were included. Confirmatory factor analysis (CFA) indicated that the 11-item measure is unidimensional with values of CFI, TLI, and RMSEA meeting relevant thresholds. Good internal consistency (Cronbach’s α 0.941) and test–retest reliability (intra-class correlation coefficients 0.730–0.880). Convergent validity with two reference measures was demonstrated with strong correlations of 0.573–0.730 were shown. Significant differences ( P = 0.0018, standardized effect size = 0.372) between groups defined by number of night-time voids supported known-groups validity. Exit interviews in 66 patients indicated all participants experienced improvement in at least 1 NI Diary item and that a 1-point improvement on the item response scale and 1-void reduction per night (associated with an average best cut point on ROC analysis of − 11.6) constituted meaningful improvement. Anchor and distribution-based analyses identified a meaningful change threshold of − 15 to − 18 points on the NI Diary. Conclusion The NI Diary is a reliable and valid patient-reported psychometric instrument which is fit-for-purpose to evaluate the impact of nocturia on patient quality of life in the clinical trial setting. Trial registration number and registration date NCT03201419; June 28, 2017.

Background: Clinicians treating multiple sclerosis (MS) should consider patient preferences when making treatment decisions. An online mixed-methods approach to elicit patient-centered concepts, group concept mapping (GCM), was used to generate statements reflecting the patient experience in relapsing-remitting MS and identify the most important patient-centered outcomes from patient and clinician perspectives. Patients and Methods: Twenty patients and 12 MS specialists in the United States provided statements describing what an ideal treatment would do to improve symptoms and daily functioning. Statements were sorted by participants into meaningful domains and rated on importance on an 11-point scale. Results: Sixty-four unique statements supporting 6 domains of clustered concepts were generated. Patient and clinician ratings of importance were highly correlated (r=0.82); however, patients rated the domains of Activities of Daily Living, Prevent & Cure, and Address Symptoms as highest in importance, whereas clinicians rated Prevent & Cure, Safe & Effective, and Activities of Daily Living as highest in importance. Statements rated above the domain mean by both patients and clinicians included "Improve cognitive function" and "Improve motor function" in the Activities of Daily Living domain and "Help with memory issues" and "Help preserve cognition" in the Address Symptoms domain. The statement "Improve short term memory" was 1 of 3 statements rated above the domain mean by patients but below the domain mean by clinicians. Conclusion: High levels of agreement of concept importance were found between patients and MS specialists, although certain domains and statements were rated more highly by one group. Overall, concepts such as cognitive function, physical and emotional functioning, and activities of daily living were perceived as having great importance for treatment outcomes versus symptom-focused outcomes like gait or tingling sensations. This comprehensive concept model for the MS patient experience can be used for further development of patient-centered outcome measures in MS treatment.

OBJECTIVES To determine the feasibility and acceptability of an Ayurveda/Yoga intervention for weight loss, using dual-diagnosis inclusion criteria, dual-paradigm outcomes, and a semistandardized protocol with tailoring according to the Ayurvedic constitution/imbalance profile of each participant. DESIGN Seventeen participants enrolled in a weekly intervention for 3 months. Outcome measurements were performed at baseline, postintervention, and 3 and 6 months follow-up. SETTING The intervention was conducted through the University of Arizona, Department of Family and Community Medicine from April through December 2012. SUBJECTS Participants included 2 men and 15 women recruited from the community of Tucson, AZ using flyers and hospital message boards. Seventeen enrolled and 12 participants provided complete follow-up data. INTERVENTION Participants met with an Ayurvedic practitioner twice monthly (six times) and followed semistandardized dietary guidelines with individual tailoring to address relevant psychophysiological imbalances obstructing weight loss and a standardized protocol of therapeutic yoga classes three times weekly with recommended home practice of two to four additional sessions. OUTCOME MEASURES Primary outcome was weight loss. Other biomedical outcomes included body mass index, body fat percentage, waist and hip circumference, waist to hip ratio, and blood pressure. Unique instruments were designed to collect data on outcomes associated with the Ayurvedic medical paradigm, including dietary changes by food qualities, mood/affect, relationships, and changes in Ayurvedic imbalance profiles. RESULTS Participants lost an average of 3.5 kg during the 3-month intervention. Weight loss at 3 and 6 months postintervention increased to an average of 5.6 kg and 5.9 kg, respectively. Participants who lost 3% of their body weight during the 12 week intervention, lost on average an additional 3% during the follow-up period. Psychosocial outcomes also improved. No additional services were provided to participants during the follow-up period. CONCLUSIONS A whole-systems Ayurvedic medicine and Yoga therapy approach provides a feasible promising noninvasive low-cost alternative to traditional weight loss interventions with potential added benefits associated with sustainable holistic lifestyle modification and positive psychosocial changes.

Background Skin cancer is the most common cancer in the US. Training massage therapists (MTs) in skin cancer prevention and detection creates opportunity for reducing skin cancer burden. Little is known about MTs’ perceptions of skin cancer prevention and detection, their discussions of these topics with clients, or their referral recommendations for suspicious skin lesions. Purpose We surveyed MTs’ perceptions of their role in engaging in conversations about skin cancer prevention, viewing the skin for suspicious lesions, and referring clients with such lesions to health care providers. Setting, Participants, Research Design We administered an online survey from 2015–2017 of licensed MTs practicing in the US and at least age 21 years (n = 102); quantitative and qualitative data were analyzed in 2017. Main Outcome Measures The main variables assessed were MTs perceptions of (a) appropriateness for asking clients about skin cancer history, skin cancer prevention, suspicious lesion referral and follow-up; and (b) comfort with recognizing and discussing suspicious lesions, recommending a client see a doctor for suspicious lesion, and discussing skin cancer prevention. Results Quantitative data revealed that most MTs were amenable to discussing skin cancer prevention during appointments; few were engaging in these conversations. MTs were more comfortable discussing suspicious lesions and recommending that a client see a doctor than they were sharing knowledge about skin cancer and sun safety. Categories based on qualitative content analysis were: sharing information for the client’s benefit, and concerns about remaining within scope of practice. Conclusions MTs have boundaries for skin cancer risk-reduction content to include in a client discussion and remain in their scope of practice. These findings will help support a future educational intervention for MTs to learn about and incorporate skin cancer risk-reduction messages and activities into their practice.

Purpose: To determine whether increasing the proportion of healthier options in vending machines decreases the amount of calories, fat, sugar, and sodium vended, while maintaining total sales revenue. Design: This study evaluated the impact of altering nutritious options to vending machines throughout the Banner Health organization by comparing vended items’ sales and nutrition information over 6 months compared to the same 6 months of the previous year. Setting: Twenty-three locations including corporate and patient-care centers. Intervention: Changing vending machine composition toward more nutritious options. Measures: Comparisons of monthly aggregates of sales, units vended, calories, fat, sodium, and sugar vended by site. Analysis: A pre–post analysis using paired t tests comparing 6 months before implementation to the equivalent 6 months postimplementation. Results: Significant average monthly decreases were seen for calories (16.7%, P = .002), fat (27.4%, P ≤ .0001), sodium (25.9%, P ≤ .0001), and sugar (11.8%, P = .045) vended from 2014 to 2015. Changes in revenue and units vended did not change from 2014 to 2015 ( P = .58 and P = .45, respectively). Conclusion: Increasing the proportion of healthier options in vending machines from 20% to 80% significantly lowered the amount of calories, sodium, fat, and sugar vended, while not reducing units vended or having a negative financial impact.

Background This article presents findings from qualitative interviews conducted as part of a research study that trained Acupuncture, Massage, and Chiropractic practitioners’ in Arizona, US, to implement evidence-based tobacco cessation brief interventions (BI) in their routine practice. The qualitative phase of the overall study aimed to assess: the impact of tailored training in evidence-based tobacco cessation BI on complementary and alternative medicine (CAM) practitioners’ knowledge and willingness to implement BIs in their routine practice; and their patients’ responses to cessation intervention in CAM context. Methods To evaluate the implementation of skills learned from a tailored training program, we conducted semi-structured qualitative interviews with 54 CAM practitioners in Southern Arizona and 38 of their patients. Interview questions focused on reactions to the implementation of tobacco cessation BIs in CAM practice. Results After participating in a tailored BI training, CAM practitioners reported increased confidence, knowledge, and motivation to address tobacco in their routine practice. Patients were open to being approached by CAM practitioners about tobacco use and viewed BIs as an expected part of wellness care. Conclusions Tailored training motivated CAM practitioners in this study to implement evidence-based tobacco cessation BIs in their routine practice. Results suggest that CAM practitioners can be a valuable point of contact and should be included in tobacco cessation efforts.