Objectives: To qualitatively explore patient experiences of severe, recurrent, bilateral nasal polyps (NP). Methods: A targeted literature review of published qualitative studies and online blogs describing patient experiences of NP was conducted. Semistructured concept elicitation interviews were conducted in the United States and Germany with participants $18 years with severe, recurrent, bilateral NP to explore their symptom experience and impacts on health-related quality of life (HRQoL; NCT03221192). A subset of 10 participants reported symptoms and impacts using a smartphone or tablet application (app) over a 10-day period. Results: A paucity of qualitative evidence regarding patient experience of NP was identified from the literature or blog review. Twenty-seven participant interviews were conducted. Thirty-six symptoms were identified, including 7 primary symptoms (nasal congestion [n = 27 of 27], breathing difficulties [n = 27 of 27], postnasal drip [n = 25 of 27], runny nose [n = 24 of 27], head/facial pressure [n = 23 of 27], loss of smell [n = 23 of 27], loss of taste [n = 22 of 27]) and 29 secondary symptoms (the most common were mucus/catarrh and nose bleeds [both n = 20 of 27]). Most symptoms were reported to vary both within and between days. Sixty impacts of severe NP were reported, including impacts on sleep (n = 22 of 27), physical functioning (n = 21 of 27), activities of daily living (n = 21 of 27), emotional well-being (n = 27 of 27), treatment (n = 23 of 27), social life (n = 26 of 27), and work (n = 19 of 27). Symptoms/impacts reported using the app were consistent with interview findings, although new symptoms were identified (ear pain, throat pain, nasal scabs, and nasal burning). These results supported the development of a conceptual model outlining concepts related to symptoms, impacts, and treatment of NP. Conclusions: Severe, recurrent, bilateral NP are associated with a range of symptoms that have significant detrimental impact on HRQoL.

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Background:Lupus nephritis (LN) is an autoimmune disease characterized by inflammation of the kidneys as a result of systemic lupus erythematosus (SLE). Approximately 50% of SLE patients will develop LN, which is considered to be one of the most severe manifestations of SLE and the leading cause of morbidity and mortality in SLE. While there is ample existing evidence on disease experience and PROs used in extra-renal SLE, little research has been done in LN. Qualitative interviews with patients can help identify concepts that are both important and relevant to the patient. In order to effectively evaluate treatment benefit, it is critical that PRO measures used to assess such concepts and define clinical trial endpoints are fit for purpose and have strong evidence of content validity in the specific context of use.Objectives:The objective of this study was to understand the patient experience of LN and to identify and characterize the signs and symptoms of LN and their impact on health-related quality of life (HRQoL) through the development of a disease-specific conceptual model. This model was then used to evaluate the content validity of existing PRO measures available for use in LN.Methods:A structured literature search was conducted in Medline, Embase and PsycINFO to identify qualitative research articles documenting the patient experience of LN. PRO measures developed or commonly used to assess patient experiences of LN were also identified. Semi-structured concept elicitation interviews were conducted with 15 adult patients in the US with a clinician-confirmed diagnosis of LN (defined in accordance with established clinical guidelines). Supplementary qualitative data were also collected from a review of publicly available online blogs/forums. Findings were used to inform the development of a conceptual model detailing the impact of LN signs, symptoms and HRQoL and evaluate the validity of existing measures used within LN.Results:Searches revealed a paucity of qualitative research conducted with LN patients, supporting the need for prospective research in LN. Consistent with existing literature in SLE, the core signs and symptoms identified from the qualitative literature review, interviews and blog/forum review included joint pain, fatigue, joint stiffness, swelling (particularly in the extremities) and skin rashes. LN patients also reported urinary frequency, urgency, foamy urine and blood in their urine. Disease impact on physical functioning, activities of daily living, emotions, social life, work/finances and sleep were reported. PRO measures commonly used to evaluate patient experiences in LN included the SF-36, LupusQOL, LupusPRO, SLE Symptom Checklist, KDQoL and KSQ. Conceptual mapping of instruments against the newly developed conceptual model (Figure 1) highlighted that no single measure provides a comprehensive assessment of all symptoms/impact important to LN patients. Furthermore, items are largely focused on impact of symptoms with few items on symptom severity.Figure 1.Conceptual model of lupus nephritis symptoms and associated impactsConclusion:The presentation of signs and symptoms in LN patients appears similar to those reported in extra-renal SLE populations, with the addition of swelling and urinary symptoms. Qualitative research with LN patients guided the development of a comprehensive LN conceptual model outlining the disease experience from the patients’ perspective. These insights can be useful to inform PRO measurement strategies for clinical trials in LN.Acknowledgments:With thanks to Dr. Betty Diamond and Dr. David Wofsy for their collaboration and helpful insightsDisclosure of Interests:Pamela Berry Employee of: Janssen, Kate Burrows Consultant of: Adelphi Values a health outcomes research company commissioned by Janssen to conduct the research reported in this abstract, Rebecca Hall Consultant of: Adelphi Values a health outcomes research company commissioned by Janssen to conduct the research reported in this abstract., Adam Gater Consultant of: Adelphi Values a health outcomes research company commissioned by Janssen to conduct the research reported in this abstract, Helena Bradley Consultant of: Adelphi Values a health outcomes research company commissioned by Janssen to conduct the research reported in this abstract, Amy Ward Consultant of: Adelphi Values a health outcomes research company commissioned by Janssen to conduct the research reported in this abstract, Chloe Tolley Consultant of: Adelphi Values a health outcomes research company commissioned by Janssen to conduct the research reported in this abstract, Patricia Delong Employee of: Janssen, Elizabeth C Hsia Shareholder of: Johnson & Johnson, Employee of: Janssen Research & Development, LLC

the condition available?" Scoring was 0/200 for No/Yes. With a mean less than 100, the condition was not recommended. We sought to assess the importance of uncertainty around the SS score (score reported, "65% of the maximum" (200)). Methods: We examined uncertainty with missing data and sampling variability, both ignored by ACMG. As missing responses (MR) were not reported for SS, we tested possible score proportions consistent with 65% to determine potential MR and score boundary estimates without making assumptions on the nature of the MR (Manski, 1989). ACMG treated its MCKAT SS score as a population parameter, rather than the sample estimate that it was. Bootstrapping estimated sampling variability around the estimate based on the sample size and the possible MR. Results: A range of [64.5, 65.5) was consistent with 65% reported (actual score [129, 131)). This was consistent with MR = 0, 3 or 6 out of the 23 responses to any MCKAT question. Boundary estimates were, respectively, [130.4, 130.4], [113,139.1] and [95.7, 147.8]. The last implies the identification region includes a score that would imply a recommendation against screening. Bootstrapping around the original mean showed 5.9 % of bootstraps below the 100 score cutoff. Bootstrapping around the two lower bounds corresponding to MR = 3 and 6 implied 27.9 % and 57.3 % of means below 100. Conclusions: ACMG should have completely reported MR and respondent scores (or variances) for all survey questions. Then readers and policy makers could fully assess the confidence they can have in recommendations. Accounting for uncertainty in this one question indicates a lack of confidence in the recommendation for MCKAT.

Objectives: Asthma affects more than 300 million people worldwide and over 26 million people in the US. Incidence of psychological distress (PD) in asthma, a consequence of coexisting psychological comorbidities like anxiety and/or depression, is on the rise. The study aims to identify sociodemographic and other factors that are associated with PD and assess its impact specifically on reliever medication use in Asthma. Methods: A retrospective, cross-sectional research data, obtained from the Medical Expenditure Panel Survey (MEPS, 2013(MEPS, -2015 and consisted of a sample of 5307 community dwelling asthmatics. PD was identified by using Kessler-6 scale (a score of $ 13), a self-administered written questionnaire embedded in MEPS. Use of three or more canisters of quick relief inhalers was defined as overuse of relievers. A Chi-square analyses was performed to test association between PD and reliever use. A set of logistic regression (LR) analyses was performed to determine the predictors of PD as well as reliever overuse in asthmatics with PD. Results: Compared to those without distress, a higher proportion of psychologically distressed asthmatics reported reliever overuse (2=310956.703, 23.3% vs 12.2%, p,0.0001). Female asthmatics were 32.3% more likely to have PD than males (OR=1.323, C.I=1.320-1.326, p,0.0001). The LR results revealed that female asthmatics within the PD group were more likely to overuse relievers than males (OR= 2.263, C.I= 2.255-2.2343, p,0.0001), and those with 12 years or more of education were less likely to overuse relievers compared to those with less education (OR=0.555, C.I=0.553-0.556, p,0.0001). A Phi correlation coefficient (r4=-0.103) revealed a significant negative correlation between PD scores and reliever overuse. Conclusions: Comorbidity of PD in asthma is associated with over-utilization of reliever medications. Inappropriate medication use can affect treatment plan, compromise patient's quality of life, and increase the overall cost of care in asthma.