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Abstract Background Autosomal dominant polycystic kidney disease (ADPKD) is considered the most common inherited renal disease. Patient-Reported Outcomes (PROs) and patient experience in ADPKD are difficult to quantify and have not been well studied, particularly in the early stages of the disease. There is evidence to suggest that early-stage ADPKD patients have a lower Health-Related Quality of Life (HRQoL) than the general population due to the signs and symptoms of early-stage ADPKD. However, no research has been carried out on the HRQoL of early-stage ADPKD patients using validated ADPKD-specific PRO measures. Additionally, a new disease progression delaying treatment option has recently emerged for ADPKD. Patient preference for this treatment and unmet treatment needs have not yet been investigated. Methods The ACQUIRE study is a prospective, observational study investigating the influence of early-stage ADPKD-related symptoms and treatments on PROs. It aims to collect real-world data on patient demographics, treatment patterns, clinical outcomes, and PROs such as HRQoL, treatment satisfaction and treatment preference in early-stage ADPKD. Adult ADPKD patients in stages 1–3 of chronic kidney disease (CKD) with evidence of rapidly progressing disease are being recruited from seven European countries. At baseline and every 3 months, for a follow-up period of 18 months, general and disease-specific questionnaires are completed remotely to capture patients’ own assessment of their overall and ADPKD-related HRQoL. A Discrete Choice Experiment (DCE) is also used to investigate the value patients place on different attributes of hypothetical treatment options (e.g. treatment outcomes, side effects) and the role each attribute plays in determining overall patient treatment preference. Discussion The results of this study will highlight the real-world effects of ADPKD-related challenges on PROs including HRQoL, treatment experience and satisfaction; and help physicians gain greater insight into likely disease outcomes based on early-stage patient symptoms and patients’ experience with treatment. Data captured by the DCE may inform ADPKD treatment decision-making from a patient perspective. The DCE will also provide insights into which patients are more likely to perceive benefit from treatments based on the value and trade-offs they place on specific treatment attributes. Trial registration NCT02848521 . Protocol Number/Version: 156–303-00096/Final

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Background and objectives: Systemic lupus erythematosus (SLE) is an autoimmune condition that can highly impact patients' quality of life (QoL). However, there is a lack of knowledge about SLE, affecting the general population and health care professionals (HCPs) alike. This lack of knowledge has negative implications for patients and the healthcare system, worsening prognosis, negatively impacting QoL, and increasing healthcare utilization. The aim of this paper is to draw attention, according to the perspective of the participants of this study, to the lack of awareness of SLE and its consequences in Spain, and to suggest improvements. Patients and methods: This qualitative, descriptive, observational, multicenter, and cross-sectional study included 40 patients with moderate or severe SLE, recruited during their routine visits in six university hospitals in Spain. The study also included 11 caregivers and 9 HCPs. All participants were individually interviewed. Data from the interviews were coded and analyzed thematically by two anthropologists following a phenomenological perspective. Results: Our study identified a lack of disease awareness among primary care physicians, emergency medicine doctors, and other specialists treating SLE symptomatology. This led to diagnostic delays, which had a clinical and emotional impact on patients. Furthermore, symptom awareness was found to be context dependent. Differences in symptom awareness between HCPs and patients led to a mismatch between the severity evaluation made by doctors and patients. Some HCPs did not consider the limitations of the current severity evaluation of SLE, and therefore attributed symptoms potentially caused by SLE to the unfavorable socioeconomic conditions patients lived in. Finally, a lack of social awareness among friends, family members, and romantic partners led to lower social support, increased isolation, and negative physical and emotional impact for patients. Gender differences in the provision of support were identified. Conclusion: This study highlights the need to increase SLE awareness among patients, HCPs, and the broader public in order to improve patient QoL. Being aware of the clinical and emotional impact of such lack of awareness, as well as the role played by context on the patient experience of SLE, is a crucial step towards achieving this goal. [ABSTRACT FROM AUTHOR]

John FP Bridges,1 Kathleen Beusterien,2 Stephan Heres,3 Pedro Such,4 Joaquín Sánchez-Covisa,5 Anna-Greta Nylander,4 Elcie Chan,6 Anne de Jong-Laird5 1Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA; 2Outcomes Research Strategies in Health, Washington DC, USA; 3Department of Psychiatry and Psychotherapy, Technische Universität München, Munich, Germany; 4Global Medical Affairs, H. Lundbeck A/S, Valby, Denmark; 5Medical Department, Otsuka Pharmaceutical Europe Ltd., Wexham, UK; 6HEOR/Market Access, Otsuka Pharmaceutical Europe Ltd., Wexham, UK Objective: This study seeks to quantify the treatment goals of people recently diagnosed with schizophrenia and explore their impact on treatment plan.Methods: People aged 18–35 years with a confirmed diagnosis of schizophrenia within the past 5 years were surveyed in the UK, Germany, and Italy. Treatment goals were assessed via a validated best–worst scaling instrument, where participants evaluated subsets of 13 possible treatment goals identified using a balanced incomplete block design. Participants identified the most and least important goals within each task. Data were also collected on current treatment and preference for daily oral versus long-acting injectable (LAI) treatment. Hierarchical Bayes was used to identify preference weights for the goals, and latent class analysis was used to identify segments of people with similar goals. The segments were compared with the current treatment and preference for oral versus LAI treatment.Results: Across 100 participants, the average age was 26 years, 75% were male and 50% were diagnosed within 2 years ago. Overall, preferences were most favorable for reduced disease symptoms, think clearly, reduced hospitalizations, reduced anxiety, and take care of self. A total of 61% preferred oral medication and 39% LAI. Two groups were identified with different treatment goals; 50% of participants emphasized clinical goals, including reduced disease symptoms (preference weight =19.7%), reduced hospitalizations (15.5%), and reduced anxiety (10.5%). The other 50% emphasized functional goals, including improved relationships with family/friends (11.4%), increased interest in work (10.6%), experiencing a fuller range of emotions (8.4%), and ability to socialize (7.5%). Those emphasizing functional goals were more likely to be on LAI (44% versus 26%; p=0.059) and preferred LAI (46% versus 32%; p=0.151).Conclusions: People with recent-onset schizophrenia may focus more on clinical goals or functional goals, a discussion of which may help facilitate patient engagement. Keywords: recent-onset schizophrenia, preferences, treatment goals

The utility measure is a method to quantify health-related quality of life according to the preference values that patients attach to their health status. This study aimed to estimate the utility measure of patients with controlled and uncontrolled severe asthma (SA) in Spain, separately. Additionally, other characteristics (sociodemographic, clinical, and healthcare resource use [HCRU]) were also assessed for both SA populations. This cross-sectional study included 159 patients with SA in Spain. Data were collected from medical records and directly from the patients during the study visit. Asthma Control Questionnaire (ACQ)-5 was used to classify patients with controlled and uncontrolled SA. Most of the patients were female (72.0% uncontrolled SA and 63.6% controlled SA). The mean (SD) EuroQol-5D (EQ-5D-5L) score was 0.88 (0.14) and 0.70 (0.25) in controlled and uncontrolled SA, respectively. The mean (SD) Asthma Quality-of-Life-5D (AQL-5D) score was 0.93 (0.09) and 0.85 (0.09) in controlled and uncontrolled SA, respectively. Emergency visits (19.2 vs. 2.7%) and hospitalizations (7.7% vs. no hospitalization) were more common among uncontrolled SA than controlled SA. Mean (SD) number of visits to primary care and pneumologists in uncontrolled SA vs. controlled SA was 4.1 (2.8) vs. 2.5 (3.0) and 3.7 (3.5) vs. 2.8 (2.2), respectively. The study provides data on utility measures among patients with SA in Spain for the first time. Patients with uncontrolled SA had lower HRQoL and higher HCRU than patients with controlled SA. Therefore, the implementation of measures that improve HRQoL among patients with uncontrolled SA is highly recommended. Despite the existence in Spain of validated asthma questionnaires, the impact of severe asthma on quality of life, depending on whether it is controlled or not, had never been assessed. This study, which included 159 patients, was conducted to fill the gap above by obtaining two utility measures for quality of life, a generic one using the EQ-5D questionnaire (which can be used for comparison with other chronic conditions) and an asthma-specific one using the AQL-5D questionnaire. Patients with uncontrolled SA had a lower utility measure than patients with controlled disease and, therefore, a lower quality of life. In addition, patients with uncontrolled SA also had higher use of healthcare resources. These results highlight that the implementation of measures that improve the quality of life among patients with uncontrolled SA is highly recommended. [ABSTRACT FROM AUTHOR]

A 28-day randomized open-label multicenter study was conducted to assess the efficacy of bromhexine plus standard of care (SOC) (n = 98) vs. SOC alone (n = 93) in 191 outpatients with mild-to-moderate COVID-19 in the primary health care setting. Bromhexine three daily doses of 10 mL (48 mg/day) were administered for seven days. The primary efficacy endpoint was the reduction of viral load estimated as the cycle thresholds (Ct) to detect ORF1ab, N Protein, and S Protein genes by RT-qPCR in saliva samples on day 4 as compared with baseline. Ct values of the three genes increased from baseline throughout days 4 to 14 (p < 0.001) but significant differences between the study groups were not found. Differences in the percentages of patients with low, medium, and high viral loads at 4, 7, and 14 days were not found either. In summary, treatment with bromhexine plus SCO was associated with a viral load reduction of ORF1ab, N Protein, and S Protein genes at day 4, which was not significantly different than similar viral load reductions observed with SOC alone. The present findings do not seem to favor the use of bromhexine as an antiviral in patients with COVID-19. [ABSTRACT FROM AUTHOR]