Objective Anifrolumab is a new therapeutic approach for individuals with systemic lupus erythematosus (SLE) directed at blocking the type 1 interferon pathway. Despite the expanding body of literature on Anifrolumab, an essential aspect remains absent: the subjective patient experience of treatment effects and implications for daily life. The present study aimed to fill this void by elucidating the nuanced perspectives of SLE patients receiving Anifrolumab treatment by conducting qualitative in-depth interviews (IDIs). Methods SLE patients at Aarhus University Hospital who had received at least 3 infusions of Anifrolumab were approached for inclusion in a study comprising two main elements: 1) Qualitative IDIs and 2) Collection of patient data from electronic medical records (EMRs). The 60-minute IDIs were semi-structured and based on a discussion guide that included both open-ended and close-ended questions. Verbatim transcripts were coded and analyzed using qualitative software to understand concepts important to patients and to understand patients' own experiences before and after Anifrolumab therapy. A clinical chart review was then conducted using EMR data at baseline, 3 months, and 6 months (where available) after Anifrolumab initiation. Results IDIs were successfully conducted with 14 patients and EMR data was collected from 16 patients (treatment days range: 62–474). The symptoms reported most prior to Anifrolumab treatment were fatigue (93%), joint pain (93%), sun sensitivity (86%), skin rashes (79%), and hair loss (71%) (figure 1a). All symptoms improved, although still present, and none worsened during the treatment (figure 1b). Patients reported significant impact of disease on daily life before treatment: day-to-day activities (79%), emotional aspects (71%), social life (71%), physical activity (64%), concentration/memory (57%), work (50%) and family/romantic relationships (50%) (figure 2a). Patients reported improvements on all aspects after treatment but were still impacted (figure 2b). From the EMR data, we observed a fall in disease activity after treatment initiation with a concomitant reduction in the use of corticosteroids. Conclusions This study provides valuable insights into the subjective experiences of SLE patients treated with Anifrolumab. These findings collectively contribute to a comprehensive understanding of the treatment's efficacy and its tangible effects on both subjective and objective parameters in SLE patients. Acknowledgements This study was sponsored by AstraZeneca.

Anifrolumab is a new therapeutic approach for individuals with systemic lupus erythematosus (SLE) directed at blocking the type 1 interferon pathway. Despite the expanding body of literature on Anifrolumab, an essential aspect remains absent: the subjective patient experience of treatment effects and implications on patients' health-related quality of life (HRQoL). The present study aimed to fill this void by elucidating the nuanced perspectives of SLE patients receiving Anifrolumab treatment by conducting qualitative in-depth interviews (IDIs). SLE patients at Aarhus University Hospital who had received at least three infusions of Anifrolumab were approached for inclusion in the study, which comprised two main elements: (1) qualitative IDIs and (2) collection of patient data from electronic medical records (EMRs). The IDIs were semi-structured and based on a discussion guide that included open-ended and close-ended questions. Verbatim transcripts were coded and analysed using qualitative software to understand concepts important to patients and to understand patients' own experiences before and after Anifrolumab therapy. A clinical chart review was conducted using EMR data at baseline, 3 months, and 6 months after Anifrolumab initiation. IDIs were completed with 14 patients, and EMR data was collected from 16 patients (treatment days range: 62-474). Of the 23 symptoms spontaneously reported by patients prior to Anifrolumab treatment, fatigue, joint pain, sun sensitivity, joint stiffness, skin rashes, and hair loss were the most common. Most symptoms improved, and none worsened during treatment. Patients reported significant impacts of disease on daily life before treatment: day-to-day activities, social life, emotional aspects, physical activity, concentration/memory, work/employment, and family/romantic relationships. Patients reported improvements in all aspects after treatment but were still impacted. From the EMR data, we observed a fall in disease activity after treatment initiation with a concomitant reduction in the use of corticosteroids. This study provides valuable insights into the subjective experiences of SLE patients treated with Anifrolumab, and the findings collectively contribute to a comprehensive understanding of the treatment's efficacy from the patients' perspective and its tangible effects on both subjective and objective parameters in SLE patients.

Objectives: Beyond prevention of organ damage, treatment goals in SLE include optimization of health-related quality of life (HRQoL). The Lupus Low Disease Activity State (LLDAS) has received increasing attention as a goal whenever remission cannot be achieved. How SLE disease activity, organ damage and LLDAS attainment relate to patient-reported outcomes (PROs) is not fully explored, which formed the scope of this investigation. Methods: We included 327 patients with SLE from a tertiary referral centre. Longitudinal registrations of disease activity using SLEDAI-2K and physician global assessment (PhGA), organ damage using the SLICC/ACR damage index (SDI), pharmacotherapies, EQ-5D-3L data, as well as visual analogue scale (VAS) scores for fatigue, pain and overall SLE-related health state over a median follow-up time of 8.5years were analysed. Results: In the overall population, as well as subgroups of patients with recent-onset SLE and those with clinically active, autoantibody-positive disease, LLDAS attainment, lower PhGA and lower clinical SLEDAI-2K scores were associated with favourable HRQoL by EQ-5D-3L and VAS assessments, while increasing SDI scores were associated with poor PROs except for fatigue in the overall population. PROs were further enhanced by being in LLDAS sustainedly. In fully adjusted models of the entire study population, LLDAS attainment and lower disease activity were associated with favourable PROs, irrespective of SDI. Conclusion: In one of the longest observational studies to date, we demonstrated that low disease activity and being sustainedly in LLDAS were coupled with favourable HRQoL, pain, fatigue and overall health experience, irrespective of organ damage.

Objective To estimate the prevalence and incidence of SLE in Sweden using a recent patient cohort, and to estimate the proportion and survival of patients with moderate-to-severe disease defined from register data. Methods This observational cohort study utilized data from national registries. Adult patients were included if they had at least two secondary care visits with a primary diagnosis of SLE from 1 July 2005 to 31 December 2020. Incident patients were defined as those with no prior visits for SLE in at least the previous 4 years. Disease severity was defined using an algorithm based on previous studies.1 2 Overall survival was defined for incident patients from date of first SLE visit (presumed diagnosis date) until death, stratified by severity in the year after diagnosis. Results In total, 10,186 patients were identified, of which 5,076 were diagnosed after 2006. Prevalence increased from 2006 to 2019. The estimated point prevalence of adult SLE was 93.8 per 100,000 on 31 December 2019, and the estimated average incidence rate between 2015 and 2019 was 4.1 per 100,000 (figure 1). Of incident patients (mean age 49.9, 85% females), 61% had a clinical presentation of moderate-to-severe SLE at some point during the year following diagnosis, however patients can transition between severity states over time. The proportion of patients with moderate-to-severe disease stabilised at around 45% by 4 years after diagnosis until the end of follow-up. Compared to mild SLE patients, moderate-to-severe patients had poorer survival. After 10 years, patients with severe SLE in the year following diagnosis had an 80% survival probability compared to 86% for those with mild SLE (HR 1.49, P<0.001). Patients with moderate SLE had an 81% survival probability (HR vs. mild 1.42, P<0.001) (figure 2). Conclusions The estimated incidence and prevalence of SLE in Sweden is consistent with previous studies. This is the first study to evaluate moderate-to-severe SLE in Sweden from registers. Previous research has shown that survival in SLE is poorer than for controls, and we show that survival is poorer in patients presenting with moderate-to-severe disease than in mild disease, highlighting the importance of improving care for this patient group. Acknowledgements This study was sponsored by AstraZeneca. References Garris C, Jhingran P, Bass D, et al. Healthcare utilization and cost of systemic lupus erythematosus in a US managed care health plan. J Med Econ 2013;16:667–77. Samnaliev M, Barut V, Weir S, et al. Health care utilization and costs in adults with systemic lupus erythematosus in the United Kingdom: A real-world observational retrospective cohort study. EULAR European E-Congress of Rheumatology. Online2020.

Purpose:Qualitative studies that highlight the patient perspective of heart failure (HF) and its impact on the lives of patients are limited. Our study objective was to describe the patient's perspective on HF, including the diagnosis, treatment journey and healthcare interactions, and how HF impacts patients' lives and specifically their health-related quality of life (HRQoL) and work capacity. Patients and Methods:This cross-sectional, non-interventional, mixed-methods patient experience study comprised: (i) a quantitative online survey with study-specific questions and assessments of HRQoL and work impairment among 101 patients with HF in Sweden and (ii) 35 qualitative interviews to gain in-depth understanding of the patients' experiences. Results:Patients were found to experience a highly symptomatic and detrimental impact of HF on their HRQoL and work capacity. Fatigue was the most frequently reported symptom, and it was detrimental to all areas of patients' lives limiting them mentally, socially, and physically. Two-thirds of patients were not aware of the type of HF they had, one-third did not check their body weight regularly, and around half did not increase their physical exercise as recommended by both guidelines and healthcare practitioners. Patients preferred specialist to primary care, desired greater access to healthcare, and continuity in whom they interact with in primary care. Conclusion:Patients with HF experience a highly symptomatic burden that affects them physically, mentally, and socially. Our study highlights a major gap in patients' knowledge about HF and HF-related healthcare. These results demonstrate a challenge for the Swedish healthcare system particularly as regards providing patients with continuity, accessibility, and proximity to primary care.

BackgroundAdvanced chronic kidney disease (CKD) severely impacts patients' lives. Nevertheless, little is known about patients' own experiences of living with CKD in Sweden. The objective of this study was to describe the patient's perspective on CKD-including diagnosis, treatment journey, and healthcare interactions-and how CKD impacts patients' health-related quality of life (HRQoL) and work capacity.Subject and methodsA mixed-methods approach was used that incorporated (i) a quantitative online survey with study-specific questions and assessments of HRQoL and work impairment among 178 patients with CKD in Sweden, and (ii) 40 qualitative interviews to gain an in-depth understanding of the patient experience.ResultsHRQoL and work capacity were notably impacted by CKD and deteriorated with advancing disease stage. Patients experienced numerous symptoms long before diagnosis, but still found diagnosis unexpected. Fatigue was the most frequently reported symptom impacting all areas of patients' lives. In the interviews, patients reported that they worried what their future life would be like, a dread of dialysis and organ transplantation, and how they implemented lifestyle changes to mitigate progression. In primary healthcare, patients struggled to have their symptoms taken seriously. Once referred to kidney specialists, patients were satisfied with healthcare. Patients wished they had been diagnosed sooner in their disease journey.ConclusionThese findings highlight an unmet need from the patient's perspective in primary healthcare, driven primarily by lack of early CKD diagnosis. In Sweden, there is a need for increased education on CKD in primary care, and a need for increased availability of specialized kidney care.

Purpose Fatigue is a prominent symptom in individuals with chronic lymphocytic leukemia (CLL). This work evaluates the content validity and psychometric properties of the Functional Assessment of Chronic Illness Therapy-Fatigue scale (FACIT-Fatigue) in patients with CLL to determine if it is fit for purpose in CLL research. Methods The FACIT-Fatigue yields a 13-item total score from a five-item symptom subscale and an eight-item impact subscale. To evaluate content validity, cognitive debriefing interviews were conducted with 40 patients with CLL in the first-line or relapsed or refractory setting. Psychometric properties, including structural validity, internal consistency, construct and known-groups validity, were investigated using data from a phase 3 trial in relapsed or refractory CLL (NCT02970318). Results Interviewed patients considered the FACIT-Fatigue items relevant to their CLL experience, understood the terminology and agreed with response options. Confirmatory factor analysis confirmed the presence of symptom and impact subscales, but also supported unidimensionality of the FACIT-Fatigue. The FACIT-Fatigue total, symptom and impact subscales demonstrated good internal consistency (Cronbach’s coefficient α > 0.85 and McDonald’s omega ω > 0.90), and strong correlations with relevant EORTC QLQ-C30 scales (all Spearman’s r ≥ 0.5). Known-groups validity was shown by significant differences between groups defined by baseline performance status, hemoglobin level and constitutional symptoms (all p < .0001). Cluster analysis supported FACIT-Fatigue score thresholds of 30 and 34 to define a severe fatigue population. Conclusions Content validity and psychometric evaluation in patients with CLL demonstrated that the FACIT-Fatigue has good psychometric properties and is fit for purpose in CLL.

There is a need to understand how patients assess perceived benefits and risks of treatments. The study aimed to (i) elucidate how patients evaluate treatment experiences and (ii) develop a brief patient-reported outcome (PRO) instrument for use across disease areas for perceived benefit–risk evaluation of a new medicine in a clinical trial setting. Concepts relating to patient-perceived benefit–risk were identified from literature reviews and qualitative concept elicitation interviews with patients across a variety of primary medical conditions. Draft instrument items were developed from identified concepts and evaluated for clarity, relevance and appropriateness of response options in cognitive interviews. Items were iteratively revised to address patient feedback. Qualitative interviews were conducted with 47 patients (primary condition: 20 oncological, 12 respiratory, 10 metabolic, 5 cardiovascular), of whom 32 contributed to concept elicitation and 42 to cognitive debriefing. Elicited concepts could be grouped into four medication-related categories: effectiveness of treatment, burden of side effects, convenience of use and overall acceptance/satisfaction. Cost, trial experience and altruism were additional concept categories unrelated to medication. The final instrument contained one item each on the medication’s effectiveness, side effects and convenience, and an overall item capturing patient benefit–risk assessment. An upfront question was included to separate out non-medication aspects of patients’ experiences. We developed a brief PRO instrument, the Patient Global Impression of Benefit–Risk (PGI-BR), which can be applied across disease areas to assess patient views of benefit–risk of a new medicine in the clinical trial setting.

Purpose To evaluate the influence of recall periods on the assessment of physical function, we compared, in cancer and general population samples, the standard administration of PROMIS Physical Function items without a recall period to administrations with 24-hour and 7-day recall periods. Methods We administered 31 items from the PROMIS Physical Function v2.0 item bank to 2400 respondents (n = 1001 with cancer; n = 1399 from the general population). Respondents were randomly assigned to one of three recall conditions (no recall, 24-hours, or 7-days) and one of two "reminder" conditions (with recall periods presented only at the start of the survey or with every item). We assessed items for potential differential item functioning (DIF) by recall time period. We then tested recall and reminder effects with analysis of variance controlling for demographics, English fluency, and co-morbidities. Results Based on conservative pre-set criteria, no items were flagged for recall time period-related DIF. Using analysis of variance, each condition was compared to the standard PROMIS administration for Physical Function (no recall period). There was no evidence of significant differences among groups in the cancer sample. In the general population sample, only the 24-hour recall condition with reminders was significantly different from the "no recall" PROMIS standard. At the item level, for both samples, the number of items with non-trivial effect size differences across conditions was minimal. Conclusions Compared to no recall, the use of a recall period has little to no effect upon PROMIS physical function responses or scores. We recommend that PROMIS Physical Function be administered with the standard PROMIS "no recall" period.

Understanding the patient’s perception of their disease is vital for guiding care decisions. The current study aimed to identify the most predominant experiences in women diagnosed with, and treated for, ovarian cancer in terms of disease-related symptoms, treatment-attributed side effects and their impacts. Semi-structured qualitative interviews about disease-related symptoms, treatment-attributed side effects and their impacts were conducted with women who were being treated for ovarian cancer in Europe (n = 55) or in the USA (n = 9). The women were also asked to rate the bothersomeness of the symptoms, side effects and impacts that they mentioned during the interview. Symptoms, side effects and impacts were identified from coded interview transcripts using an iterative coding framework. Bloating, abdominal pain, tiredness and frequent urination were the most frequently expressed symptoms, and were reported by 72%, 67%, 64% and 55% of women, respectively, which together constituted approximately 30% of all symptom expressions. The most bothersome symptoms were reported as bloating, abdominal pain, pain in the side, tiredness and fatigue. The most frequently expressed side effects were hair loss, neuropathy, tiredness and nausea, which were reported by 84%, 63%, 61% and 61% of women, respectively. The most bothersome reported side effects were constipation, nausea, diarrhoea, pain in general, fatigue, weakness, reduced sleep quality and hair loss. Feelings of anxiety, concerns about the future, physical functioning, work limitations and the adoption of coping strategies were the most frequently expressed impacts and were reported by 72–80% of women. Impacts reported as the most difficult to deal with were concerns about the future, emotional difficulties in general, physical functioning, sexual functioning, negative self-image, fatigue, sleep difficulties, financial burden and work limitations. In our qualitative study, the most common and most bothersome experiences reported by women treated for ovarian cancer were symptoms of bloating, abdominal pain and tiredness; side effects of hair loss, nausea and tiredness/fatigue; and impacts relating to concerns about the future, physical functioning and work limitations. We suggest that clinicians measure these experiences consistently and take them into consideration when making treatment decisions.

Background and Objective Understanding the patient experience is important for identifying the unmet need in chronic lymphocytic leukemia. The current study aimed to develop a comprehensive chronic lymphocytic leukemia conceptual model. Methods The conceptual model was based on literature searches, review of chronic lymphocytic leukemia patient blogs/forums, and interviews with five expert clinicians, with 20 patients who received at most one treatment (first line) for their chronic lymphocytic leukemia, and with 20 patients with relapsed or refractory chronic lymphocytic leukemia. De-identified interviews were transcribed, coded, and evaluated using qualitative data analysis software. Results Thirty-five prevalent chronic lymphocytic leukemia-related symptom and impact concepts were identified from literature searches, patient blogs/forums, and clinician interviews. Patient interviews confirmed the identified concepts and revealed five additional concepts. Fatigue-related sub-components were identified from how patients described their fatigue, covering symptoms (tiredness/need for sleep, lack of energy, weakness, cognitive fatigue), and impacts (decreased ability to maintain their social, familial, or professional role, decreased physical functioning, frustration). Three versions of the conceptual model were created: an overall model with all concepts; a model highlighting the most prominent concepts in first line; and a model highlighting the most prominent concepts in relapsed or refractory disease. Prominent concepts in both first line and relapsed or refractory disease were fatigue-related symptoms and impacts, muscle/joint aches, night sweats, bruising, fever, recurrent infections/illness, insomnia, decreased cognitive/emotional functioning, anxiety/worry, stress, depression, financial difficulty, and fear of death. Dyspnea and cough were prominent in first line only, and enlarged lymph nodes, headaches, pain/discomfort, weight loss, nausea/vomiting, and infusion reactions were prominent in relapsed or refractory disease only. Conclusions The results show that fatigue is a dominant issue affecting patients with chronic lymphocytic leukemia. The three versions of the conceptual model can help researchers to understand patients’ unmet needs and guide the patient-reported outcome strategy for clinical trials.

e19001 Background: The study aimed to create a conceptual model (CM) of disease- and treatment-related signs, symptoms and impacts in patients with first-line (1L) or relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL). Methods: The CM was based on literature searches, review of CLL patient blogs/forums, interviews with expert hematologists, and semi-structured concept elicitation interviews with 40 patients with CLL (20 1L, 20 R/R; with fatigue, weight loss, fever and/or night sweats in previous week). To identify salient concepts, the number of patients mentioning each concept and the concept’s mean disturbance rating (from 0 [not at all] to 10 [very]) were mapped. Results: Literature searches identified 32 prevalent concepts, with the most common being fatigue/tiredness. Patient blogs/forums revealed further symptoms (muscle/joint aches, headaches, weakness) and impacts (stress, fear of death, uncertainty). Clinicians reported recurrent infections and swollen lymph nodes as key signs of CLL and noted that fatigue is related to both symptoms (e.g. tiredness) and impacts (e.g. needing to rest and limit daily activities). In patient interviews, 50 concepts were revealed, with 42 present in both 1L and R/R; fatigue was the most prominent and impactful. Fatigue-related sub-concepts were identified, covering symptoms (tiredness/need to sleep, lack of energy, weakness, cognitive fatigue) and impacts (decreased ability to maintain social/familial/professional role, decreased physical functioning, frustration). Three versions of the CM were created: an overall model with all identified concepts, and two models highlighting the most salient concepts in 1L and R/R. Muscle/joint aches, night sweats, bruising, fatigue-related symptoms, fever and recurrent infections/illness were salient symptoms in both 1L and R/R, as were impacts such as anxiety/worry, stress, depression, financial difficulty, and fear of death. Shortness of breath and coughing were salient in 1L only, and swollen lymph nodes, headaches, pain/discomfort, weight loss, nausea/vomiting, and infusion reactions were salient in R/R only. Conclusions: The three versions of the CM can help to inform patient-centered end points to evaluate new treatments for 1L and/or R/R CLL.

Introduction: The FACIT-Fatigue is a 13-item patient-reported outcome instrument (PRO) that was designed to assess fatigue-related symptoms and impacts on daily functioning. This study assessed the content validity of the FACIT-Fatigue in patients with CLL to help determine if it is fit-for-purpose in this population. Methods: Forty adults with first-line (1L) or relapsed or refractory (R/R) CLL took part in an interview study comprising concept elicitation and cognitive debriefing. All participants had experienced CLL-related symptoms (fatigue, weight loss, fever or night sweats) in the past week. For concept elicitation, interviewers used a standardized semi-structured interview guide with open-ended and prompted questions to explore patients' experiences of the signs, symptoms and impacts of CLL and its treatments. Patients were asked to provide a disturbance rating of these concepts on a 0-10 scale of severity. All interviewed patients completed the FACIT-Fatigue. Item scores can range from 0 ("not at all") to 4 ("very much"), and the total score from 0 to 52; lower scores indicate greater fatigue. The recall period for each item is the past 7 days. The general population mean score is 43 (Cella et al. 2002; Montan et al. 2018). During cognitive debriefing, the clarity of the FACIT-Fatigue items, their relevance to the patients, and the comprehensiveness of the response options were assessed. Interviews were carried out by telephone and lasted about 60-75 minutes. De-identified audio-recorded interviews were transcribed, coded and analyzed using qualitative data analysis software. Results: Median age of participants was 58 years (range: 28-73 years), and gender distribution was equal. During concept elicitation, fatigue was identified as the most prominent and impactful concept. It was the only concept mentioned by 100% of patients, and almost all (1L: 95%; R/R: 85%) mentioned it spontaneously without prompting. Fatigue had a high mean disturbance rating (1L: 7.1; R/R: 6.9). Seven fatigue-related sub-components were identified from the way that patients described their fatigue, covering symptoms (tiredness/need for sleep, lack of energy, weakness, cognitive fatigue) and impacts (decreased ability to maintain social/familial/professional role, decreased physical functioning, frustration). Mean (standard deviation) FACIT-Fatigue score was 28.9 (13.6) for patients with 1L CLL and 29.3 (11.5) for those with R/R CLL, indicating greater fatigue than the general population and providing further evidence that fatigue is a core component of CLL. The items that scored highest were: "I feel fatigued" (item 1; mean score: 2.4) and "I feel tired" (item 4; mean score: 2.4). The items that scored lowest were: "I am too tired to eat" (item 10; mean score: 0.4) and "I need help doing my usual activities" (item 11; mean score: 1.0). During cognitive debriefing, 100% of patients confirmed that the FACIT-Fatigue was reflective of their experiences with fatigue resulting from CLL. All patients found the response options provided by the FACIT-Fatigue to be sufficient. Patients confirmed that most of the FACIT-Fatigue terminology was clear. An exception was the item "I feel listless/washed out" (item 3), which was not consistently understood: most patients linked it to an absence of both physical and mental energy, but some interpreted it just as a lack of physical energy. Overall, patients considered the FACIT-Fatigue items to be relevant and distinct from each other, and found that the impact items captured both the mental and physical impacts of fatigue. One item, "I am too tired to eat", was not considered to be highly pertinent because, although respondents could imagine that some patients with CLL might be too tired to eat, they themselves could only recall being too tired to prepare a meal, not being too tired to eat. However, respondents felt that the item was still relevant and that it did not detract from the applicability or clarity of the FACIT-Fatigue. Conceptual relevance of the FACIT-Fatigue was supported by mapping of its items to the 7 fatigue-related sub-components identified during concept elicitation (Table). Conclusions: Results from concept elicitation and cognitive debriefing interviews demonstrated content validity of the FACIT-Fatigue in patients with CLL with 1L or R/R disease, thus providing evidence that it is fit-for-purpose in this population. Disclosures Chung: AstraZeneca: Employment, Equity Ownership. Eek:AstraZeneca: Employment, Equity Ownership. Eyre:Janssen: Honoraria; Gilead: Consultancy, Honoraria, Other: commercial research support; Abbvie: Honoraria; Roche: Honoraria.

Background In the phase 3 SOLO2 trial (ENGOT Ov-21), maintenance therapy with olaparib tablets significantly prolonged progression-free survival (primary endpoint) compared with placebo in patients with a germline BRCA1 or BRCA2 (BRCA1/2) mutation and platinum-sensitive, relapsed ovarian cancer who had received two or more lines of previous chemotherapy. The most common subjective adverse effects included fatigue, nausea, and vomiting, which were typically low grade and self-limiting. Our a-priori hypothesis was that maintenance olaparib would not negatively affect health-related quality of life (HRQOL) and additionally that the prolongation of progression-free survival with olaparib would be underpinned by additional patient-centred benefits. Methods In SOLO2, 196 patients were randomly assigned to olaparib tablets (300 mg twice daily) and 99 to placebo. Randomisation was stratified by response to previous chemotherapy (complete vs partial) and length of platinum-free interval (>6-12 vs >12 months). The prespecified primary HRQOL analysis evaluated the change from baseline in the Trial Outcome Index (TOI) score during the first 12 months of the study. To be assessable, patients had to have an evaluable score at baseline and at least one evaluable follow-up form. Secondary planned quality-of-life (QOL) analyses included the duration of good quality of life (defined as time without significant symptoms of toxicity [TWiST] and quality-adjusted progression-free survival [QAPFS]). Efficacy and QOL outcomes were analysed in all randomly assigned patients (the full analysis set), and safety outcomes were analysed in all randomly assigned patients who received at least one dose of study drug. Findings The adjusted average mean change from baseline over the first 12 months in TOI was -2.90 (95% CI -4.13 to -1.67) with olaparib and -2.87 (-4.64 to -1.10) with placebo (estimated difference -0.03; 95% CI -2.19 to 2.13; p=0.98). Mean QAPFS (13.96 [SD 10.96] vs 7.28 [5.22] months; difference 6.68, 95% CI 4.98-8.54) and mean duration of TWiST (15.03 [SD 12.79] vs 7.70 [6.42] months; difference 7.33, 95% CI 4.70-8.96) were significantly longer with olaparib than with placebo. Interpretation Olaparib maintenance therapy did not have a significant detrimental effect on HRQOL compared with placebo. There were clinically meaningful patient-centred benefits in both TWiST and QAPFS despite the adverse effects associated with olaparib. These patient-centred endpoints support the improvement in progression-free survival, the primary endpoint in SOLO2, and should be included in future trials of maintenance therapies. Copyright (C) 2018 Elsevier Ltd. All rights reserved.

In an experiment we investigate preferences for allocation of a public good among group members who contributed unequally in providing the public good. Inducing the group goal of productivity resulted in preferences for equitable allocations, whereas inducing the group goals of harmony and social concern resulted in preferences for equal final outcomes. The study makes a contribution by simultaneously treating provision and allocation of a public good, thus viewing these as related processes. Another contribution is that a new paradigm is introduced that bears closer resemblance to real life public good dilemmas than previous research paradigms do.

Background: Few studies have evaluated patient perspectives on neuromyelitis optica (NMO) and NMO spectrum disorder (NMOSD). Objective: Describe patient-reported clinical and treatment experience in NMOSD and compare disease characteristics of NMOSD with those of multiple sclerosis (MS). Methods: This retrospective, observational study included 522 members with NMO or NMOSD (hereafter collectively referred to as NMOSD) from PatientsLikeMe (PLM), an online patient community. Data describing member demographics, symptoms, and treatments were collected, analysed descriptively, and compared with data from PLM members with MS. Results: Fatigue, pain, and stiffness/spasticity were each rated as moderate to severe by more than half of NMOSD members, and 59% reported that their health limited the type of work or other activities they could perform all or most of the time. Overall, symptom severity and disability levels were comparable between NMOSD and MS members; however, NMOSD members were more likely than MS members to attribute disability to vision-related symptoms and were less likely to report moderate to severe cognitive and emotional symptoms, including brain fog, depressed or anxious mood, and emotional lability. Conclusion: This analysis underscores the challenges of living with fatigue, pain, stiffness/spasticity, and visual difficulties, prevalent NMOSD symptoms among members of the PLM community.

BACKGROUND:Advances in ovarian cancer treatment have improved outcomes. However, the gap between patients' hopes and expectations and their actual outcomes remains an understudied aspect of treatment decision making. This gap has been noted to be a predictor of poorer health-related quality of life. Incorporating patient hopes and expectations for treatment into clinical care may improve patient experiences.OBJECTIVES:The aim of this study is to describe the hopes and expectations of ovarian cancer patients at diagnosis and throughout treatment transitions.METHODS:This study sampled from an online community of patients with ovarian cancer. Thirty members completed an online qualitative survey about their diagnostic and treatment journey, including hopes and expectations around treatment.RESULTS:Initially, ovarian cancer patients hoped for a complete cure or removal of cancer. As they progressed through treatment, hopes and expectations centered on issues related to living with ovarian cancer. A subset of patients emphasized a lack of information about treatment side effects.CONCLUSIONS:The shift in expectations from survival to living with ovarian cancer may demonstrate an enhanced understanding of ovarian cancer prognosis as patients learn more about their condition. Patients underscored that a more involved discussion of side effect profiles with clinicians would enhance their treatment decision making and expectation setting process.IMPLICATIONS FOR PRACTICE:Expectation setting may be improved by contextualizing treatment in terms of the transitions ovarian cancer patients experience. Providing relevant information that aligns with patient needs, desires, and concerns at critical transitions may improve clinical care and decision making.

BACKGROUND:The aim of this study was to explore the need for a new disease-specific patient reported outcome (PRO) measure for use in clinical trials of drugs designed to target the underlying causes of polycystic ovary syndrome (PCOS), and in the process contribute to our understanding of the symptoms and impacts that define the patient experience with PCOS.METHODS:Semi-structured interviews were conducted in 20 women diagnosed with PCOS according to the Rotterdam criteria who had not menstruated in the previous month. The relative importance of PCOS symptoms and impact concepts to patients was determined by analyzing the frequency of their expression in the interview transcripts. These insights were compared to clinicians' perceptions of PCOS.RESULTS:Pain- and discomfort-related symptoms accounted for the highest proportion (27.6%) of the 735 patient expressions, although clinicians did not consider pain to be important to patients with PCOS. The most frequently expressed individual symptoms were cramping (70% of patients; 14.7% of concepts), irregular menstruation (95% of patients; 12.2% of concepts), facial hair growth (75% of patients; 10.6% of concepts), heavy bleeding (70% of patients; 8.8% of concepts), infertility (70% of patients; 5.4% of concepts), and bloating (60% of patients; 5.2% of concepts). Cramping, heavy bleeding, and bloating were not identified by clinicians as being important to patients with PCOS. The impacts most frequently reported by patients with PCOS related to emotional well-being (e.g. anxiety/stress) and coping behaviors (e.g. acne medication, hair removal).CONCLUSIONS:The only validated PCOS-specific PRO, the PCOSQ, does not capture some key PCOS symptoms and impacts expressed by patients with PCOS, most notably those related to pain and discomfort, bleeding intensity and coping behaviours. Furthermore, some key PCOS symptoms may be under-recognized in the clinic.