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Bradley E Chipps,1 Robert S Zeiger,2 David A Beuther,3 Robert A Wise,4 William McCann,5 Joan Reibman,6 Maureen George,7 Ileen Gilbert,8 James M Eudicone,8 Karin S Coyne,9 Gale Harding,9 Kevin R Murphy10 1Capital Allergy & Respiratory Disease Center, Sacramento, CA, USA; 2Department of Clinical Science, Kaiser Permanente Bernard J. Tyson School of Medicine, Pasadena, CA, USA; 3Department of Medicine, National Jewish Health, Denver, CO, USA; 4Division of Pulmonary and Critical Care Medicine, Johns Hopkins University School of Medicine, Baltimore, MD, USA; 5Allergy Partners, Asheville, NC, USA; 6Department of Medicine, New York University School of Medicine, New York, NY, USA; 7Columbia University School of Nursing, New York, NY, USA; 8BioPharmaceuticals Medical, AstraZeneca, Wilmington, DE, USA; 9Evidera, Bethesda, MD, USA; 10Boys Town National Research Hospital, Boys Town, NE, USACorrespondence: Bradley E Chipps, Capital Allergy & Respiratory Disease Center, 5609 J Street, Suite C, Sacramento, CA, 95819, USA, Tel +1 916-453-1454, Email bchipps@capitalallergy.com

Joan Reibman,1 Bradley E Chipps,2 Robert S Zeiger,3 David A Beuther,4 Robert A Wise,5 William McCann,6 Ileen Gilbert,7 James M Eudicone,7 Hitesh N Gandhi,7 Gale Harding,8 Katelyn Cutts,8 Karin S Coyne,8 Kevin R Murphy,9 Maureen George10 1New York University School of Medicine, New York, NY, USA; 2Capital Allergy & Respiratory Disease Center, Sacramento, CA, USA; 3Department of Clinical Science Kaiser Permanente Bernard J. Tyson School of Medicine, Pasadena, CA, USA; 4National Jewish Health, Denver, CO, USA; 5Division of Pulmonary and Critical Care Medicine, Johns Hopkins University School of Medicine, Baltimore, MD, USA; 6Allergy Partners, Asheville, NC, USA; 7BioPharmaceuticals Medical, AstraZeneca, Wilmington, DE, USA; 8Evidera, Bethesda, MD, USA; 9Boys Town National Research Hospital, Boys Town, NE, USA; 10Office of Research & Scholarship, Columbia University School of Nursing, New York, NY, USACorrespondence: Joan Reibman, New York University School of Medicine, 550 1st Avenue, Room NB7N24, New York, NY, 10016, USA, Tel +1 212-263-6479, Fax +1 212-263-8442, Email joan.reibman@nyumc.orgPurpose: Critical asthma outcomes highlighted in clinical guidelines include asthma-related quality of life, asthma exacerbations, and asthma control. An easy-to-implement measure of asthma control that assesses both symptom impairment and exacerbation risk and reflects the impact of asthma on patients’ lives is lacking. Hence, the objective of this study was to assess the Asthma Impairment and Risk Questionnaire (AIRQ®) construct validity relative to patient self-perception of asthma status and validated disease-specific patient-reported outcome (PRO) measures.Patients and methods: Baseline data were analyzed from patients (aged ≥ 12 years) with asthma participating in a 12-month observational study assessing the ability of AIRQ to predict exacerbations. At entry, patients completed a sociodemographic questionnaire, AIRQ, 3 questions addressing self-perceived asthma status, Saint George’s Respiratory Questionnaire (SGRQ), mini-Asthma Quality of Life Questionnaire (AQLQ), and Adult Asthma Adherence Questionnaire (AAAQ). Descriptive statistics were calculated for demographic and clinical characteristics. AIRQ construct validity was evaluated by assessing correlations between total AIRQ score and patient self-assessments, SGRQ, mini-AQLQ, and AAAQ scores. Comparisons of SGRQ, mini-AQLQ, and AAAQ total and component/domain scores by AIRQ control category were performed using general linear models and Scheffe’s post hoc adjustments for pairwise comparisons.Results: A total of 1112 patients were enrolled: 70% female, 78% White, mean (standard deviation) age 43.9 (19.5) years. There were highly significant correlations between AIRQ score and patient self-perception of overall control (r = 0.69; p < 0.001), total SGRQ (r = 0.74, p < 0.001), and mini-AQLQ (r = − 0.78, p < 0.001) scores. As AIRQ control category worsened, so did total and domain SGRQ, mini-AQLQ, and AAAQ impediment-to-inhaled-corticosteroid-adherence scores (all pairwise comparisons p < 0.001).Conclusion: Findings demonstrate the construct validity of AIRQ relative to patient self-perception of asthma status, disease-specific PRO measures, and treatment adherence barriers. AIRQ can be a useful instrument to raise awareness of the unrecognized impacts of asthma on patients’ lives.Keywords: exacerbation, patient-reported outcomes, construct validity, impact of asthma

Background: Asthma in children is a leading cause of missed school days, emergency department visits, and hospitalizations. Approximately 40% of children with asthma experience uncontrolled disease and annual exacerbations. There is a need for a validated composite tool for children, such as the Asthma Impairment and Risk Questionnaire (AIRQ), which was developed to assess current control and predict exacerbations in adolescents and adults with asthma., Objective: To obtain feedback from children with asthma and their parents/caregivers to inform development of a version of the AIRQ for pediatric use (Peds-AIRQ)., Methods: Children with asthma aged 5 to 11 years and their parents/caregivers participated in cognitive interviews to elicit language describing asthma symptoms and exacerbations and to assess understanding and relevance of draft Peds-AIRQ questions. Physicians and parents/caregivers provided clinical information and performed assessments relative to the children's asthma morbidity., Results: There were 60 dyads that participated: children's mean (SD) age = 7.9 (1.9) years; 68% male, 45% non-White, 32% Hispanic, and 40% with public health insurance. Overall, 53% had well-controlled, 30% partly controlled, and 17% uncontrolled asthma, based on the Global Initiative for Asthma symptom control questions. Oral or injected corticosteroids were used for asthma by 53% of the children in the previous year. Participants found draft Peds-AIRQ items understandable and relevant. Seven impairment and 3 risk questions were retained for validation, along with 5 additional items containing wording or control threshold variations., Conclusion: This study supports the need for developing a composite (impairment and risk) control tool to assess children aged 5 to 11 years with asthma and identified suitable questions for the validation of a Peds-AIRQ., Competing Interests: Disclosures Dr Lanz is a consultant for Amgen, AstraZeneca, Cogent, Genentech, Grifols, Novartis, and Sanofi/Regeneron; is a speaker for Amgen/AstraZeneca and Sanofi/Regeneron; and receives research grants from Regeneron, Sanofi, Genentech/Roche, and AstraZeneca. Dr Chipps has served as an advisor, consultant, and speaker for AstraZeneca, Boehringer Ingelheim, Circassia, Genentech, Novartis, Regeneron, and Sanofi. Dr Zeiger has received grants from Aerocrine, Genentech, MedImmune/AstraZeneca, Merck, GlaxoSmithKline, ALK Pharma, Teva, Sanofi, and the National Heart, Lung, and Blood Institute, has received consultant fees from AstraZeneca, Bayer, Genentech/Novartis, Merck, and Regeneron, and holds warrant shares in DBV Technologies. Dr Bacharier is a consultant for AstraZeneca, GlaxoSmithKline, Regeneron Pharmaceuticals Inc, Sanofi, Recludix, and Avillion; is a speaker for Sanofi and Regeneron; is a member of the data and safety monitoring board for AstraZeneca, Cystic Fibrosis Foundation/Vertex, Aravax, and DBV Technologies; and receives research support from the National Institutes of Health, AstraZeneca, and Sanofi. Dr Guilbert is a consultant for Amgen, AstraZeneca, Genentech, OM Pharma, Regeneron, and Sanofi; receives grants from Amgen, AstraZeneca, GlaxoSmithKline, OM Pharma, Regeneron, and Sanofi; and receives personal fees from Advent.com, AiCME, AstraZeneca, Genentech, OM Pharma, PlatformQ health, and Polarean. Dr Murphy has served as a consultant and is a speaker for AstraZeneca, Boehringer Ingelheim, Genentech, Greer, Merck, Mylan, Novartis, Regeneron, Sanofi, OptiNose, and Teva. Ms Winders is a consultant/speaker for ALK, AstraZeneca, Chiesi, Genentech, GlaxoSmithKline, Roche, Sanofi, and Regeneron. Dr Mohammad has participated on an advisory board for AstraZeneca. Ms LeNoir has participated on an advisory board for AstraZeneca and is a board member at Digital Health Equitable Alliance and African American Wellness Project. Dr Gilbert and Mr Eudicone are employees of AstraZeneca, the sponsor of this study, and may hold stock or stock options. Dr Coyne and Ms Harding are employees of Evidera, which was contracted by AstraZeneca for study design support, to collect and analyze data for this study, and to provide medical writing support. Dr George has served as a consultant to AstraZeneca, Insmed, and Teva and has served as a speaker for AstraZeneca and Regeneron., (Copyright © 2024 The Authors. Published by Elsevier Inc. All rights reserved.)

Background: Bowel urgency, the sudden and immediate need to have a bowel movement, is common in patients with ulcerative colitis (UC) and Crohn's disease (CD). While its impact in UC is well established, less is known about its importance in CD. Further, what level of bowel urgency control patients with UC or CD would consider to be acceptable or in remission has not been established. This qualitative study aimed to understand perceptions of bowel urgency and remission in these patients., Methods: Semi-structured combined concept elicitation and cognitive interviews were conducted to explore how adults with moderate-to-severe UC or CD and current or recent bowel urgency think about the concept of bowel urgency and its remission. The Urgency Numeric Rating Scale (UNRS) was used to examine different levels of bowel urgency severity and to investigate what level of bowel urgency patients would consider as representing remission., Results: Forty adults (n = 19 UC, n = 21 CD) recruited from six US sites completed the study. Sociodemographic and clinical characteristics were similar in both groups. Both groups reported impacts of bowel urgency on physical, social, professional, and emotional aspects of their lives. Most participants (n = 15 UC, n = 18 CD) reported having experienced one or more incidents of urgency-related fecal incontinence. Most participants considered remission to be a state with a normal or reduced number of bowel movements and no or less frequent bowel urgency, and they noted that remission would increase their ability to engage in daily activities without fear of fecal incontinence. Participants were able to map different levels of severity of bowel urgency to UNRS score ranges (scale: 0-10), and they indicated that a mean UNRS score of 5 (UC) or 4 (CD) would be the highest point on the NRS at which they would still consider their bowel urgency to be in remission., Conclusions: Bowel urgency is an important issue for patients with either moderate-to-severe UC or CD, and its remission would improve their lives. Further, these patients may still consider bowel urgency to be in remission even at UNRS scores as high as 4 or 5., Competing Interests: Declarations Ethics approval and consent to participate All participants provided electronic or written informed consent prior to their interview. The study protocol and recruiting clinical sites were approved by Advarra (Columbia, MD), a central institutional review board (study reference number: Pro00069827). Consent for publication Not applicable. Competing interests T.H.G., A.P.B., and R.M. are employees and shareholders of Eli Lilly and Company. L.S., G.H., M.S., and T.F. are employees of Evidera, which received funding from Eli Lilly and Company to conduct this research. M.D. is a consultant/advisor for Abbvie, Abivax, Arena Pharmaceuticals, Astra Zeneca, Boehringer Ingelheim International GmbH, Bristol-Meyer Squibb, Eli Lilly and Company, F. Hoffmann-La Roche Ltd, Genentech Inc, Gilead, Janssen Pharmaceuticals, Merck, Pfizer Inc, Prometheus Biosciences, and Takeda Pharmaceuticals., (© 2024. The Author(s).)

Background: National and international asthma guidelines and reports do not include control tools that combine impairment assessment with exacerbation history in one instrument., Objective: To analyze the performance of the composite Asthma Impairment and Risk Questionnaire (AIRQ) in assessing both domains of control and predicting exacerbation risk compared with the Global Initiative for Asthma (GINA) 4-question symptom control tool (GINA SCT), Asthma Control Test (ACT), and physician expert opinion (EO) informed by GINA SCT responses and appraisal of GINA-identified risk factors for poor asthma outcomes., Methods: Multivariable logistic regressions evaluated AIRQ and GINA SCT as predictors of ACT. McNemar's test compared the proportion of patients categorized at baseline as completely or well-controlled by each assessment but with current impairment or previous-year and subsequent-year exacerbations., Results: The analysis included 1064 patients aged 12 years or older; mean (SD) age 43.8 years (19.3); 70% female; 79% White; and 6% Hispanic or Latino. AIRQ and GINA SCT were highly predictive of ACT well-controlled vs not well-controlled and very poorly controlled (receiver operator characteristic area under curve AIRQ = 0.90, GINA SCT = 0.86, P = .03 AIRQ vs GINA SCT) and ACT very poorly controlled vs well-controlled and not well-controlled asthma (receiver operator characteristic area under curve AIRQ = 0.91, GINA SCT = 0.87, P = .01 AIRQ vs GINA SCT). AIRQ rated fewer patients as having completely or well-controlled asthma who had current impairment (P < .01) or with previous-year and subsequent-year exacerbations (P < .001) than did GINA SCT, ACT, and EO., Conclusion: AIRQ performs better in assessing both domains of current control and predicting exacerbation risk than do control tools and EO informed by GINA SCT and risk factors for poor asthma outcomes., (Copyright © 2024 The Authors. Published by Elsevier Inc. All rights reserved.)

Background: Patient perception of medication onset of effect is important for adherence. Although the Onset of Effect Questionnaire (OEQ) has been validated in patients with asthma, it has not been evaluated in patients with chronic obstructive pulmonary disease (COPD). This study evaluated the COPD-OEQ in patients with COPD., Methods: Two analyses (qualitative and quantitative) were conducted to assess the content validity and psychometric properties of the COPD-OEQ in participants with COPD. In the qualitative analysis, interviews assessed content validity by concept elicitation (CE) and cognitive interviewing (CI). CE included questions to understand patient experience related to onset of medication effect. CI included completion of the COPD-OEQ and assessment of the COPD-OEQ items, response options, and instructions. During the 2-week quantitative analysis, 2 versions of the COPD-OEQ (Weekly and Daily) were administered to assess test-retest reliability, construct validity, and known-groups validity., Results: The qualitative analysis demonstrated that 3 of the 5 COPD-OEQ items were relevant and understood as intended. Qualitative findings demonstrated inconsistent evidence that the COPD-OEQ Weekly and Daily were reliable and valid measures in participants with COPD. Test-retest reliability was observed for the COPD-OEQ Weekly and Daily; however, construct validitywas weak and demonstrated inconsistent correlations among COPD-OEQ items. Overall, known-groups validity was not demonstrated., Conclusion: The weak evidence from the quantitative analysis of the COPD-OEQ Weekly and Daily tools does not support use of the OEQ in general COPD. The study supports the content validity for the assessment of perceived onset of effect in patients with COPD., (JCOPDF © 2024.)

Rationale: A COPD Foundation working group sought to identify measures of exercise endurance, a meaningful aspect of physical functioning in everyday life among patients with chronic obstructive pulmonary disease (COPD) that is not fully accepted in regulatory decision making, hampering drug development. Objectives: To demonstrate, as we previously asserted (Casaburi COPD 2022;9:252), that constant work rate cycling endurance time is an appropriate exercise endurance measure in patients with COPD. Methods: To validate this assertion, we assembled an integrated database of endurance time responses, including 8 bronchodilator (2,166 subjects) and 15 exercise training (3,488 subjects) studies (Casaburi COPD 2022;9:520). Results: Construct validity was demonstrated: 1 ) peak physiologic and perceptual responses were similar for constant work rate and incremental cycling; 2 ) after bronchodilator therapy, there were greater increases in endurance time in patients with more severe airflow limitation; 3 ) after exercise training, endurance time increases were similar across airflow limitation severities; and 4 ) there were correlations between changes in endurance time and changes in mechanistically related physiologic and perceptual variables. Test-retest reliability was demonstrated, with consistency of changes in endurance time at two time points after the intervention. Responsiveness was confirmed, with significant increases in endurance time after active (but not placebo) bronchodilator therapy, with greater increases seen with more severe airflow limitation and after exercise training. On the basis of regression analysis using multiple anchor variables, the minimum important difference for endurance time increase is estimated to be approximately 1 minute. Conclusions: Constant work rate cycling endurance time is a valid exercise endurance measure in COPD, suitable for contributing to the evaluation of treatment benefit supporting regulatory decision making and evidence-based therapeutic recommendations.

Background: Rotator cuff disorders include a broad spectrum of pathological conditions including partial-thickness and full-thickness tears. Studies have shown partial-thickness rotator cuff tear (PTRCT) prevalence to be twice that of full-thickness tears. In the working population, PTRCTs are one of the most common causes of shoulder pain and often result in occupational disability due to pain, stiffness, and loss of shoulder function. Treatment of PTRCTs remains controversial. The purpose of this study was to consolidate the existing high-quality evidence on best management approaches in treating PTRCTs using both nonoperative and operative approaches., Methods: A scoping review with best evidence synthesis was performed as per the Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews. MEDLINE (OVID), EMBASE (OVID), Cochrane Library (Wiley), SCOPUS, Web of Science Core Collection, CINAHL Plus with Full Text (EBSCOhost), PubMed Central, and Science Direct were searched from 2000 to March 3, 2023. Level 1 studies, and systematic reviews and meta-analyses that included level 1 and 2 studies, were included., Results: The search yielded 8276 articles. A total of 3930 articles were screened after removing 4346 duplicates. Application of inclusion criteria resulted in 662 articles that were selected for full-text review. Twenty-eight level 1 studies, 1 systematic review, 4 meta-analyses, and 1 network meta-analyses were included in the best evidence synthesis. Nonoperative strategies included injections (ie, platelet-rich plasma, corticosteroid, prolotherapy, sodium hyaluronate, anesthetic, and atelocollagen), exercise therapy, and physical agents. Operative interventions consisted of débridement, shaving of the tendon and footprint, transtendon repair, and traditional suture anchor repair techniques with and without tear completion. Both nonoperative and operative strategies demonstrated effectiveness at managing pain and functional outcome for PTRCTs. The evidence supports the effectiveness of surgical intervention in treating PTRCTs regardless of arthroscopic technique., Conclusion: The results of this scoping review do not support superiority of operative over nonoperative management and suggest that both strategies can be effective at managing pain and functional outcome for PTRCTs. Surgery, however, is the most invasive and costly approach, with the highest risk of complications such as infection. Other variables such as patient expectation, treating practitioner bias, or preference may change which modalities are offered and in what sequence., (Copyright © 2023 Journal of Shoulder and Elbow Surgery Board of Trustees. Published by Elsevier Inc. All rights reserved.)

Community outreach and engagement (COE) is a fundamental activity of cancer centers as they aim to reduce cancer disparities in their geographic catchment areas. As part of COE, NCI-Designated Cancer Centers must monitor the burden of cancer in their catchment area, implement and evaluate evidence-based strategies, stimulate catchment area relevant research, support clinical trial enrollment, and participate in policy and advocacy initiatives, in addition to other responsibilities. The Cancer Center Community Impact Forum (CCCIF) is a national annual meeting of COE professionals who work at or with cancer centers across the country. CCCIF grew out of earlier discussions at American Society of Preventive Oncology (ASPO) annual meetings, where COE was often discussed, but not exclusively. The third annual CCCIF meeting-hosted by the Sidney Kimmel Cancer Center at Thomas Jefferson University-was held in June 2022 in Philadelphia, PA, where more than 200 participants listened to dynamic presentations across 12 COE-related panel sessions. CCCIF leadership and ASPO AD/PL Workshop Planners worked together on the agenda. The 12 sessions used a COE lens to focus on: Diversity, Equity, and Inclusion; Policy; State Cancer Coalitions; Evaluation and Metrics; Implementation Science; In-reach; Outreach; Training and Education; Funding, Personnel and Resources; Clinical Trials; Innovative Methods; and Lessons from the COVID-19 pandemic. This article is a summary of main points and key lessons from each session, as well as a summary of overarching themes that were evident across the sessions., (©2023 American Association for Cancer Research.)

Purpose: Bowel urgency (BU) is an important symptom of Crohn's disease (CD), however there is no patient-reported outcome (PRO) scale validated in this population to assess BU severity. Here we evaluated the content validity and psychometric properties of the Urgency Numeric Rating Scale (NRS)., Method: Qualitative interviews were conducted with moderate-to-severe CD participants to confirm importance and relevance of BU in this population, cognitively debrief the Urgency NRS, and explore score interpretation and CD remission. A quantitative web survey study was conducted to explore the measurement properties of the urgency NRS., Results: Qualitative Interview: 34 of 35 participants reported BU. It was most bothersome for 44%, 47% reported it daily, 18% with every bowel movement. BU had a severe impact on daily activities, causing many participants to stay home more than preferred. Patients confirmed the relevance, appropriateness, comprehensibility of the item, recall period, response options, and instructions of the Urgency NRS. Small reductions on the Urgency NRS score reflected meaningful improvements. Quantitative survey: The study sample comprised 76 participants (65.8% female). Mean Urgency NRS score was 4.7 (SD 2.26; N = 76) at Week 1, with no floor/ceiling effect. Test-retest reliability was acceptable. Construct and known-groups validity against selected PROs were overall strong and within ranges hypothesized a priori., Conclusion: The Urgency NRS is a valid and reliable instrument to assess BU severity in CD., (© 2023. The Author(s).)

Background: Asthma control is often overestimated in routine practice, and despite advances in the understanding of immunopathology and the availability of new precision therapies, the burden of disease remains unacceptably high., Objective: To compare the performance of the Asthma Impairment and Risk Questionnaire (AIRQ) with patient and physician assessments and the Asthma Control Test (ACT) in identifying asthma control., Methods: Baseline data from a longitudinal study of the AIRQ were analyzed. Patients with asthma in the United States aged 12 years and older followed in 24 specialty practices and 1 specialty-affiliated primary care clinic were enrolled between May and November 2019. At entry, participants completed AIRQ and ACT, and participants and physicians completed 5-point Likert scale assessments of control., Results: A total of 1112 participants were enrolled (mean [SD] age = 43.9 [19.3] years, 70% of the female sex, 78% White). Overall, 62% of participants rated themselves as well- or completely controlled, and 54% were rated comparably by physicians. The ACT classified 49% of participants as well-controlled, with 35% similarly categorized by AIRQ. Previous-year exacerbations were experienced by 32% of participants who self-rated as well- or completely controlled, 30% who were rated as well- or completely controlled by physicians, and 29% assessed as well-controlled by ACT, but only 15% of those classified as well-controlled by AIRQ., Conclusion: The burden of asthma is substantial in patients cared for by asthma specialists, and asthma control is overestimated by patients, physicians, and the symptom-based ACT. The AIRQ assesses risk in addition to symptom control and may serve to improve asthma control determination by assessing previous exacerbations., (Copyright © 2023 The Authors. Published by Elsevier Inc. All rights reserved.)