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Aims Patient-reported outcome measures (PROMs) to assess health-related quality of life (HRQoL) are increasingly used to guide decision-making in cardiovascular care. However, many of the existing PROMs are developed with limited patient involvement and overlook personal health preferences. We aim to develop a cardiovascular disease (CVD)-specific patient-centred preference-based PROM to assess and monitor HRQoL in CVD patients. Methods and results A mixed-methods study consisting of several phases was conducted to identify important health items: (i) a scoping literature review, (ii) first- and second-round expert group meetings, (iii) interviews with CVD patients, and (iv) an online survey asking CVD patients to indicate from a large set those health items that are considered the most important. The literature review, expert group meetings, and patient interviews resulted in a list of 55 items potentially important to CVD patients. In total, 666 CVD patients responded to the survey. The following nine items were considered the most important by CVD patients: mobility, activities, self-reliance, fatigue, shortness of breath, chest pain, palpitations, anxiety/worrying, and sexual limitations. An electronic preference-based PROM consisting of these nine items was developed within a cloud-based environment for clinical implementation. Conclusion Nine items considered the most important for health by CVD patients were identified and included in a new preference-based patient-centred PROM. This new CVD-specific PROM can be easily implemented using the electronic application and has the potential to improve quality of care for CVD patients.

OBJECTIVES: Patient-reported outcome measures (PROMs) are widely applied to assess perceived health status. To date, no transplant-specific PROM is available for generating a single, standardized score regarding the health status of transplant recipients. The objective of this study is to generate health items for a new patient-centered PROM for organ recipients: the Transplant PROM (TXP).STUDY DESIGN AND SETTING: A five-phase, mixed-method approach was applied to identify and select the health items: scoping literature review, expert meetings, focus-group meetings with organ recipients, a special judgmental task within an online survey, and expert meetings for final selection of health items.RESULTS: Based on a previously published scoping literature review, a first round of expert meetings, and a total of four focus-group meetings with kidney, lung, and liver transplant recipients (N = 18), a list of 83 relevant health items relating to post-transplant life was selected. In an online survey, 183 transplant recipients selected the 10 most important health items from this list. After evaluating the frequency of selected health items and combining items that assess closely related or similar concepts in the second round of expert meetings, nine health items were chosen to be included in TXP: fatigue, skin, worry/anxiety, self-reliance, activities, weight, sexuality, stooling, and memory/concentration.CONCLUSION: The nine TXP health items reflect the most prominent issues transplant recipients experience. The TXP can be administered by means of a mobile phone app.

Background More than two-thirds of patients diagnosed with type 2 diabetes mellitus (T2DM) in Indonesia encounter medical-related problems connected to routine self-management of medication and the social stigma related to T2DM. The current study aims to explore distress and coping strategies in Indonesian T2DM outpatients in a Primary Healthcare Centre (PHC) in Surabaya, East Java, Indonesia. Methods We conducted a qualitative study using two different data collection methods: focus group discussions and in-depth interviews. The guideline of interviews and discussions were developed based on seventeen questions derived from the DDS17 Bahasa Indonesia (a Bahasa Indonesia version of the Diabetes Distress Scale questionnaire), which covered physician distress domain, emotional burden domain, regimen distress domain and interpersonal distress domain. Results The majority of the 43 participants were females and aged 50 or older. Our study discovered two main themes: internal and external diabetes distress and coping strategies. Internal diabetes distress consists of disease burden, fatigue due to T2DM, fatigue not due to T2DM, emotional burden (fear, anxiety, etc.) and lack of knowledge. Internal coping strategies comprised spirituality, positive attitude, acceptance and getting more information about T2DM. External diabetes distress was evoked by distress concerning healthcare services, diet, routine medication, monthly blood sugar checks, interpersonal distress (family) and financial concern. External coping strategies included healthcare support, traditional medicine, vigilance, self-management, social and family support and obtaining information about health insurance. Conclusion Our study shows that for Indonesian T2DM-patients, spirituality and acceptance are the most common coping mechanisms for reducing DD. Furthermore, our study revealed an overall positive attitude towards dealing with T2DM as well as a need for more information about T2DM and potential coping strategies. Finally, an important finding of ours relates to differences in DD between males and females, potential DD associated with health services provision and the specific challenges faced by housewives with T2DM.

PURPOSE: To determine which items regarding prosthesis use were considered most important by adults with major unilateral upper limb absence (ULA) and to develop a patient-reported outcome measure to assess the preferred usage features of upper limb prostheses: PUF-ULP.MATERIALS AND METHODS: Based on a qualitative meta-synthesis combined with input from patients and clinicians a graphical diagram of 79 items related to prosthesis use was developed. Adults with ULA (N = 358; mean age = 55.4 ± 16.5 years; 52.0% male/40.8% female/7.3% unknown) selected their top-10 of most important items from this diagram. This study is registered in the Netherlands Trial Register: NL7682.RESULTS: Most selected items were "wearing comfort" (54.0% of cases), "grabbing, picking up, and holding" (34.3%), and "weight" (31.4%). All subpopulations (i.e. age, sex, origin of ULA, level of ULA, and prosthesis type), except multi-grip myoelectric hand prosthesis users (MHP), selected "wearing comfort" most. Nine items were included in the PUF-ULP: "wearing comfort," "functionality," "independence," "work, hobby, and household," "user-friendly," "life-like appearance," "phantom limb pain," "overuse complaints," and "reliability."CONCLUSIONS: All prosthesis users, except MHP-users, considered wearing comfort most important, which might be of interest for future research and industry. The PUF-ULP can be used to reflect the match between users and their prostheses.IMPLICATIONS FOR REHABILITATIONAll persons with upper limb absence, except multi-grip myoelectric hand prosthesis users, considered "wearing comfort" most important regarding prosthesis use, which highlights that prosthesis wearing comfort deserves more attention in future research to increase the value placed by the user on their upper limb prosthesis.Regarding prosthesis use, men considered "ease of control" more important compared to the overall population, while women considered "independence," "household," "life-like appearance," "overuse complaints," and "anonymity" more important.Persons with a mono- or multi-grip myoelectric upper limb prosthesis rated function-related items as more important compared to the overall population, while persons with a passive/cosmetic prosthesis rated comfort-related and appearance-related items as more important.The newly developed measurement tool, also called the PUF-ULP, provides a single score that represents the match between the user and their upper limb prosthesis.

Objectives: Efforts to evaluate the health of solid organ transplant recipients are hampered by the lack of adequate patient-reported outcome measures (PROMs) targeting this group. We developed the Transplant ePROM (TXP), which is based on a novel measurement model and administered through a mobile application to fill this gap. The main objective of this article is to elucidate how we derived the weights for different items, and to report initial empirical results. Study design and setting: The nine health items in the TXP were fatigue, skin, worry, self-reliance, activities, weight, sexuality, stooling, and memory. Via an online survey solid organ recipient participating in the TransplantLines Biobank and Cohort study (NCT03272841) were asked to describe and then compare their own health state with six other health states. Coefficients for item levels were obtained using a conditional logit model. Results: A total of 232 solid organ transplant recipients (mean age: 54 years) participated. The majority (106) were kidney recipients, followed by lung, liver, and heart recipients. Fatigue was the most frequent complaint (54%). The strongest negative coefficients were found for activities and worry, followed by self-reliance and memory. Conclusion: A set of coefficients and values were developed for TXP. The TXP score approximated an optimal health state for the majority of respondents and recipients of different organs reported comparable health states. (c) 2021 The Authors. Published by Elsevier Inc. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/)

OBJECTIVES: The patients' perspective on health has become increasingly important when assessing treatment outcomes. Recently, the Endoscopic Endonasal Sinus and Skull Base Surgery Questionnaire (EES-Q) was developed to determine the impact of endoscopic endonasal surgery on health-related quality of life (HRQoL). The aim of this study was to evaluate the test-retest reliability, construct validity, and responsiveness of the EES-Q.DESIGN: Prospective cohort study.SETTING: University Medical Center Groningen, tertiary referral hospital, the Netherlands.PARTICIPANTS: One hundred patients who underwent endoscopic endonasal surgery because of sinus or anterior skull base pathology.MAIN OUTCOME MEASURES: Test-retest reliability, construct validity and responsiveness.RESULTS: The EES-Q domains exhibited good test-retest reliability (ICC > 0.90). Construct validity was corroborated by significant positive and negative correlations between the EES-Q and the SNOT-22 and postoperative health status (p < 0.01) respectively. The correlation between the social EES-Q domain and the ability to move and perform usual activities (EQ-5D-3L) was significant positive (p < 0.01). In patients with paranasal sinus pathology, the EES-Q was responsive to clinical change (Cohen's d = 0.6).CONCLUSION: The EES-Q is a reliable and acceptable responsive disease-specific HRQoL instrument. The expected construct validity of the EES-Q is supported by the results in this study. Inconveniences in social functioning had the greatest negative impact on postoperative health status rating. This reflects the importance of a multidimensional HRQoL assessment after EES. The results indicate that the EES-Q is a promising disease-specific tool for the HRQoL assessment after endoscopic endonasal sinus or anterior skull base surgery. This article is protected by copyright. All rights reserved.

The overall aim of this scoping review of the literature is twofold: (1) to provide an overview of all instruments that have been used to assess health-related quality of life (HRQoL) after solid organ transplantation and (2) to provide a list of health items they include to support future studies on the development of a new-generation HRQoL instrument. All studies that administered any form of HRQoL instrument to post-transplant solid organ recipients were identified in a comprehensive search of PubMed (MEDLINE), Embase, and Web of Science, with a cut-off date of May 2018. The search used various combinations of the following keywords: lung, heart, liver, kidney, or pancreas transplantation; quality of life; well-being; patient-reported outcome; instrument; questionnaire; and health survey. In total, 8013 distinct publications were identified and 1218 of these were selected for review. Among the instruments applied, 53 measured generic, 51 organ-specific, 271 domain-specific, and 43 transplant-specific HRQoL. A total of 78 distinct health items grouped into 16 sub-domains were identified and depicted graphically. The majority of publications did not report a logical rationale for the choice of specific HRQoL instrument. The most commonly used types of instruments were generic health instruments, followed by domain-specific instruments. Despite the availability of transplant-specific instruments, few studies applied these types of instruments. Based on the 78 items, further research is planned to develop a patient-centered, transplant-specific HRQoL instrument that is concise, easy to apply (mobile application), and specifically related to the health issues of solid organ recipients. Electronic supplementary material The online version of this article (10.1007/s40271-018-0335-3) contains supplementary material, which is available to authorized users.

OBJECTIVES: To present EuroQol-5D (EQ-5D) index scores in Indonesian type 2 diabetes mellitus (T2DM) outpatients and to investigate the associations between EQ-5D and socio-demographic characteristics and clinical condition.METHODS: Socio-demographic data were collected by interviewing participants, clinical data were obtained from treating physicians and self-reporting. Participants originated from primary and secondary care facilities in the Java and Sulawesi regions. Ordinal regression analysis was conducted with the quintiles of the EQ-5D index scores as the dependent variable to investigate the multivariate association with the participants' socio-demographic characteristics and clinical condition.RESULTS: 907 participants completed the five-level Indonesian version of the EQ-5D. The mean age of the participants was 59.3 (SD 9.7), and 57% were female. The overall EQ-5D index score was 0.77 (0.75-0.79). Male participants had a higher EQ-5D index score compared to females, and the highest percentage of self-reported health problems was in the pain/discomfort dimension (61%). Factors identified as being significantly associated with lower EQ-5D index scores were: (i) treatment in secondary care, (ii) lower educational level, (iii) dependency on caregivers, (iv) not undergoing T2DM therapy, and (v) being a housewife.CONCLUSION: This study provides estimates of EQ-5D index scores that can be used in health economic evaluations. As housewives were found to experience more T2DM-related pain/discomfort and anxiety/depression, targeted approaches to reduce these problems should be aimed specifically at this group of patients. Potential approaches could involve disease-specific-counselors (health literacy partners) who provide routine monitoring of T2DM therapy as well as improved health promotion among T2DM communities.

BackgroundIn many countries, medical interventions are reimbursed on the basis of recommendations made by advisory boards and committees that apply multiple criteria in their assessment procedures. Given the diversity of these criteria, it is difficult to find common ground to determine what information is required for setting priorities.ObjectiveTo investigate whether society and patients share the same interests and views concerning healthcare priorities.MethodsWe applied a framework of discrete choice models in which respondents were presented with judgmental tasks to elicit their preferences. They were asked to choose between two hypothetical scenarios of patients receiving a new treatment. The scenarios graphically presented treatment outcomes and patient characteristics. Responses were collected through an online survey administered among respondents from the general population (N = 1,253) and patients (N = 1,389) and were analyzed using conditional logit and mixed logit models.ResultsThe respondents' preferences regarding new medical treatments revealed that they attached the most relative importance to additional survival years, age at treatment, initial health condition, and the cause of disease. Minor differences in the relative importance assigned to three criteria: age at treatment, initial health, and cause of disease were found between the general population and patient samples. Health scenarios in which patients had higher initial health-related quality of life (i.e., a lower burden of disease) were favored over those in which patients' initial health-related quality of life was lower.ConclusionsOverall, respondents within the general population expressed preferences that were similar to those of the patients. Therefore, priority-setting studies that are based on the perspectives of the general population may be useful for informing decisions on reimbursement and other types of priority-setting processes in health care. Incorporating the preferences of the general population may simultaneously increase public acceptance of these decisions.

Background There is no consensus on the optimal treatment for displaced midshaft clavicle fractures. Several studies indicate superior patient satisfaction in favour of operative reconstruction. It is unknown what drives superior satisfaction in this treatment group. The aim of this study was to explore patient satisfaction and identify contributors to patient satisfaction after operative and nonoperative treatment for displaced midshaft clavicle fractures in adults using a focus group approach. Methods Four face-to-face and two web-based focus groups were hosted. A total of 24 participants who were treated nonoperatively (n = 14) or operatively (n = 10) agreed to participate. Participants were selected using purposive sampling, ensuring variation in gender, age, treatment complications and outcomes. A question script was developed to systematically explore patient expectations, attitudes and satisfaction with different dimensions of care. All focus groups were voice-recorded and transcribed at verbatim. Thematic analysis was conducted on all face-to-face and web-based transcripts. Results The main emerging themes across treatment groups were; need for more information, functional recovery, speed of recovery and patient-doctor interaction. There was no difference in themes observed between operative and nonoperative focus groups. The lack of information was the most important complaint in dissatisfied patients. Conclusion Our study shows that informing patients about their injury, treatment options and expectations for recovery is paramount for overall patient satisfaction after treatment for a displaced midshaft clavicle fracture. Level of evidence Level III, focus group study.

Background The EuroQoL five-dimensional instrument (EQ-5D) is the favoured preference-based instrument to measure health-related quality of life (HRQoL) in several countries. Two versions of the EQ-5D are available: the 3-level version (EQ-5D-3 L) and the 5-level version (EQ-5D-5 L). This study aims to compare specific measurement properties and scoring of the EQ-5D-3 L (3 L) and EQ-5D-5 L (5 L) in Indonesian type 2 diabetes mellitus (T2DM) outpatients. Methods A survey was conducted in a hospital and two primary healthcare centres on Sulawesi Island. Participants were asked to complete the two versions of the EQ-5D instruments. The 3 L and 5 L were compared in terms of distribution and ceiling, discriminative power and test-retest reliability. To determine the consistency of the participants’ answers, we checked the redistribution pattern, i.e., the consistency of a participant’s scores in both versions. Results A total of 198 T2DM outpatients (mean age 59.90 ± 11.06) completed the 3 L and 5 L surveys. A total of 46 health states for 3 L and 90 health states for 5 L were reported. The ‘11121’ health state was reported most often: 17% in the 3 L and 13% in the 5 L. The results suggested a lower ceiling effect for 5 L (11%) than for 3 L (15%). Regarding redistribution, only 6.1% of responses were found to be inconsistent in this study. The 5 L had higher discriminative power than the 3 L version. Reliability as reflected by the index score was 0.64 for 3 L and 0.74 for 5 L. Pain/discomfort was the dimension mostly affected, whereas the self-care dimension was the least affected. Conclusions This study suggests that the 5 L-version of the EQ-5D instrument performs better than the 3 L-version in T2DM outpatients in Indonesia, regarding measurement and scoring properties. As such, our study supports the use of the 5 L as the preferred health-related quality of life measurement tool. We did not do a trial but this study was approved by the Medical Ethics Committee of Universitas Gadjah Mada Yogyakarta, Indonesia (document number KE/FK/1188/EC, 12 November 2014, amended 16 March 2015).

Background The EQ-5D is a widely used preference-based instrument to measure health-related quality of life. Some methodological drawbacks of its three-level version (EQ-5D-3L) prompted development of a new format (EQ-5D-5L). There is no clear evidence that the new format outperforms the standard version. Objective The objective of this study was to make a head-to-head comparison of the EQ-5D-3L and EQ-5D-5L in a discrete choice model setting giving special attention to the consistency and logical ordering of coefficients for the attribute levels and to the differences in health-state values. Methods Using efficient designs, 240 pairs of EQ-5D-3L health states and 240 pairs of EQ-5D-5L health states were generated in a pairwise choice format. The study included 3698 Dutch general population respondents, analyzed their responses using a conditional logit model, and compared the values elicited by EQ-5D-3L and EQ-5D-5L for different health states. Results No inconsistencies or illogical ordering of level coefficients were observed in either version. The proportion of severe health states with low values was higher in the EQ-5D-5L than in the EQ-5D-3L, and the proportion of mild/moderate states was lower in the EQ-5D-5L than in the EQ-5D-3L. Moreover, differences were observed in the relative weights of the attributes. Conclusion Overall distribution of health-state values derived from a large representative sample using the same measurement framework for both versions showed differences between the EQ-5D-3L and EQ-5D-5L. However, even small differences in the phrasing (language) of the descriptive system or in the valuation protocol can produce differences in values between these two versions.

During the last three decades lung transplantation (LTx) has become a proven modality for increasing both survival and health‐related quality of life (HRQoL) in patients with various end‐stage lung diseases. Most previous studies have reported improved HRQoL shortly after LTx. With regard to long‐term effects on HRQoL, however, the evidence is less solid. This prospective cohort study was started with 828 patients who were on the waiting list for LTx. Then, in a longitudinal follow‐up, 370 post‐LTx patients were evaluated annually for up to 15 years. For all wait‐listed and follow‐up patients, the following four HRQoL instruments were administered: State‐Trait Anxiety Inventory, Zung Self‐rating Depression Scale, Nottingham Health Profile, and a visual analogue scale. Cross‐sectional and generalized estimating equation (GEE) analysis for repeated measures were performed to assess changes in HRQoL during follow‐up. After LTx, patients showed improvement in all HRQoL domains except pain, which remained steady throughout the long‐term follow‐up. The level of anxiety and depressive symptoms decreased significantly and remained constant. In conclusion, this study showed that HRQoL improves after LTx and tends to remain relatively constant for the entire life span., This prospective cohort study follows lung transplant patients up to 15 years after transplantation and shows that health‐related quality of life improves after transplantation and tends to remain relatively constant for the patient's entire life span.

Background The patients’ perspective of health outcomes has become important input for assessing treatment effects. However, existing endoscopic endonasal surgery (EES) instruments are not fully aligned with the concept of health-related quality of life (HRQoL). A prospective cohort study was therefore conducted to develop a suitable quality-of-life tool to assess nasal morbidity after EES: the Endoscopic Endonasal Sinus and Skull Base Surgery Questionnaire (EES-Q). Methods The study included 300 patients: 207 with sinus pathology and 93 with anterior skull base pathology. The EES-Q was administered prior to surgery and postoperatively (2 weeks, 3 months, and 1 year). Psychometric instrument properties were tested and relevant health domains were formulated. Seventy-two items were generated by the conventional psychometric approach. An exploratory factor analysis was used to test construct validity. The optimal number of factors to retain was determined by using the eigenvalues-greater-than-1 rule and scree plot. Orthogonal varimax rotation was used to enhance interpretability. Internal consistency was assessed using the Cronbach α. Results The factor analysis yielded a 3-factor solution, representing physical, psychological, and social functioning. The final version of the instrument consisted of 30 items with a high internal consistency (>0.80) for all 3 HRQoL domains. Conclusions The EES-Q is a comprehensive, multidimensional, disease-specific instrument. A distinguishing characteristic is that, apart from the physical and psychological domains, the EES-Q also encompasses a social domain. Understanding different HRQoL aspects in patients undergoing EES may help caregivers restore, improve, or preserve the patient's health through individualized care, which depends on identifying their specific needs.

OBJECTIVES: To translate, revise, and validate the Diabetes Distress Scale (DDS) instrument for Indonesian type 2 diabetes mellitus (T2DM) outpatients with various complications.METHODS: Participants were recruited from four hospitals and two primary health care centers. The study was performed with forward and backward translations, an adaptation testing with a small subset of participants, and validation analysis. Factor analysis with maximum likelihood estimation and promax rotation was then used to investigate the instrument structure. Internal consistency among the items was estimated using Cronbach α for each domain of the DDS.RESULTS: In total, 324 participants (246 from the hospitals and 78 from the primary health care centers) were involved in this study. To improve participant comprehension of the exact meaning of questions, examples of daily activities for patients with T2DM (e.g., diet, exercise, and adherence to therapy) were added to some questions after the translation and revision procedures. The factor analysis revealed a correlation among the four factors ranging from 0.40 to 0.67. The factor loadings of selected items from the four factors ranged from 0.41 to 0.98. The order of the four factors in the factor analysis was as follows: interpersonal distress, emotional burden, physician distress, and regimen distress. The internal consistency for the four domains ranged from 0.78 to 0.83. The instrument resulting from this study was labeled "DDS17 Bahasa Indonesia."CONCLUSIONS: The DDS17 Bahasa Indonesia provides an initial psychometric validation study, factor structure, and internal consistency for assessing the distress of Indonesian T2DM outpatients. Use of this instrument in future research and clinical trials is recommended for the Indonesian context.

Background: Children with intestinal failure (IF) depend on parenteral nutrition (PN). The goal in the treatment of IF is to wean children off PN through intestinal rehabilitation (IR). Although the healthcare burden of IF is enormous, to our knowledge there has been no previous cost-effectiveness analysis in pediatric IF including IR.Objective: We sought to determine the cost-effectiveness of IR in terms of costs and life-years.Design: We simulated the treatment of IF in children in a discrete-event model. Data for this model were derived from patient records, the Dutch Registry of Intestinal Failure and Transplantation, the Intestinal Transplant Registry, and the literature. The time horizon of the model was 40 y. Simulated patients were enrolled at a rate of 40 patients/mo for 10 y. Actual costs were calculated for hospital admissions, surgical interventions, endoscopies, PN, and immunosuppressive medication. We evaluated the cost-effectiveness of IR by comparing 1 scenario with IR with 1 scenario without IR. In the scenario with IR, a proportion of patients who represented those with the ability to wean off PN were assigned to IR. In the scenario without IR, all patients progressed to. home PN (HPN). In both scenarios, a proportion of patients receiving HPN were eventually eligible for an intestinal transplantation.Results: IR prolonged survival; the mean number of life-years per patient was 19.4 in the scenario with IR compared with 18.2 in the scenario without IR. Average total costs per patient were 819,292 in the scenario with IR compared with 1,176,830 in the scenario without IR (equivalent to 1,129,230 US$ and 1,622,025 US$, respectively, in January 2014); costs mainly included hospital admissions and PN.Conclusions: On the basis of our simulations, we concluded that IR improved the survival of children with IF and was associated with cost savings. Therefore, we consider IR to be a cost-effective treatment for children with IF.

OBJECTIVES: To develop patient-centered health content for a novel generic instrument (Château Santé Base [CS-Base]) that is suitable to generate values for health status.METHODS: Candidate items were drawn from existing health frameworks of generic health status instruments and placed in a diagram (HealthFANTM, Zeist, the Netherlands). Through an online survey, patients with a wide range of diseases were asked to select the 9 items that were most important to them. The importance of the items for the whole study group was determined by means of frequency distributions.RESULTS: After handling duplicates and overlap, the remaining set of 47 items was placed in the HealthFAN. Among the 2256 Dutch patients who started the survey, the most common diagnoses were neck and back pain, diabetes, and asthma/chronic obstructive pulmonary disease. The 5 health items mentioned most frequently as most important were pain, personal relationships, fatigue, memory, and vision. Hearing and vision, anxiety and depression, and independence and self-esteem seemed highly intertwined, so we chose to pair these items.CONCLUSIONS: A total of 12 health items were included in CS-Base. Its content is largely based on patient input and enables classification of patients' health status. CS-Base can be administered by means of an app on a mobile phone, which makes it a convenient and attractive tool for patients and researchers.

Background: Research on new treatments for dementia is gaining pace worldwide in an effort to alleviate this growing health care problem. The optimal evaluation of such interventions, however, calls for a practical and credible patient-reported outcome measure. Objectives: To describe the refinement of the Dementia Quality-of life Instrument (DQI) and present its revised version. Methods: A prototype of the DQI was adapted to cover a broader range of health related quality of life (HRQOL) and to improve consistency in the descriptions of its domains. A valuation study was then conducted to assign meaningful numbers to all DQI health states. Pairs of DQI states were presented to a sample of professionals working with people with dementia and a representative sample of the Dutch population. They had to repeatedly select the best DQI state, and their responses were statistically modeled to obtain values for each health state. Results: In total, 207 professionals working with people with dementia and 631 members of the general population completed the paired comparison tasks. Statistically significant differences between the two samples were found for the domains of social functioning, mood, and memory. Severe problems with physical health and severe memory problems were deemed most important by the general population. In contrast, severe mood problems were considered most important by professionals working with people with dementia. Discussion: The DQI is a simple and feasible measurement instrument that expresses the overall HRQOL of people suffering from dementia in a single meaningful number. Current results suggest that revisiting the discussion of using values from the general population might be warranted in the dementia context. (C) Copyright 2016, International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc.

Background The healthcare sector is evolving while life expectancy is increasing. These trends put greater pressure on healthcare resources, prompt healthcare reforms, and demand transparent arguments and criteria to assess the overall value of health interventions. There is no consensus on the core criteria by which to value and prioritize interventions, and individual stakeholders might value specific elements differently. The present study is based on a literature review that retrieved the most widely recognized arguments and criteria used in decision-making. The aim was to compile a smaller set of arguments and criteria that would seem most relevant to different stakeholders. Methods A literature review was performed in Medline and EMBASE. The initial search retrieved over 2000 articles and documents of relevant committees. A selection was made based on their reference to healthcare, policy issues, or social justice. Finally, 84 papers were included. Data extraction took place after appraisal of the articles. A full table was made, including all arguments and criteria found; next, identical or largely overlapping arguments were excluded. The remaining arguments and criteria were assessed for relevance and a reduced set was compiled. Results The final set included 25 arguments and criteria, categorized by type (clinical, social justice, ethical, and policy). For each argument and criterion, relevance to stakeholders was scored on three levels (not, partly, and completely relevant). Conclusions Many arguments and criteria play a role in making value judgments on health interventions, but not all are relevant to all interventions. Moreover, they may interact with each other. A viable way to deal with interacting and possibly conflicting arguments and criteria might be to arrange public discussions that would evoke different stakeholders’ perspectives.

Multi-attribute utility-based instruments (MAUIs) assess health status and provide an index score on the full health-dead scale, and are widely used to support reimbursement decisions for new healthcare interventions worldwide. A valuation study is a key part of the development of MAUIs, with the primary goal of developing a scoring algorithm through eliciting societal preferences. We developed the 21-item Checklist for REporting VAluaTion StudiEs (CREATE) by following a modified two-round Delphi panel approach plus an email survey. CREATE is intended to promote good reporting practice as well as guiding developers to thoroughly and carefully think through key methodological elements in designing valuation studies. Electronic supplementary material The online version of this article (doi:10.1007/s40273-015-0292-9) contains supplementary material, which is available to authorized users.

Purpose. Health state valuations, used to evaluate the effectiveness of healthcare interventions, can be obtained either by the patients or by the general population. The general population seems to value somatic conditions more negatively than patients, but little is known about valuations of psychological conditions. This study examined whether individuals with and without depression differ in their valuations of depression and whether perceptions regarding depression (empathy, perceived susceptibility, stigma, illness perceptions) and individual characteristics (mastery, self-compassion, dysfunctional attitudes) bias valuations of either individuals with or without depression. Methods. In an online study, a general population sample used a time-trade-off task to value 30 vignettes describing depression states (four per participant) and completed questionnaires on perceptions regarding depression and individual characteristics. Participants were assigned to depression groups (with or without depression), based on the PHQ-9. A generalized linear mixed model was used to assess discrepancies in valuations and identify their determinants. Results. The sample (N = 1268) was representative of the Dutch population on age, gender, education and residence. We found that for mild depression states, individuals with depression (N = 200) valued depression more negatively than individuals without depression (N = 1068) (p = .007). Variables related to perceptions of depression and individual characteristics were not found to affect valuations of either individuals with or individuals without depression. Conclusion. Since the general population values depression less negatively, using their perspective might result in less effectiveness for interventions for mild depression. Perceptions of depression or to individual characteristics did not seem to differentially affect valuations made by either individuals with or without depression. Keywords: Depression; Discrepancies; Valuations; Health-related quality of life; Time-trade-off

Background: Expanding vaccination programs for the older population might be important as older adults are becoming a larger proportion of the general population. The aim of this study is to determine the relative importance of vaccine and disease specific characteristics and acceptance for Dutch older adults, including pneumococcal disease, herpes zoster, pertussis vaccination, and influenza vaccination.Methods: A discrete choice experiment was conducted to generate choice data that was analyzed using a mixed multinomial logit statistical model.Results: Important factors that were associated with vaccination acceptance in older adults are high mortality risk of the infectious disease, high susceptibility of getting the infectious disease, and high vaccine effectiveness. Age, influenza vaccination in 2013 and self-perceived health score were identified as personal factors that affect vaccine preference. Potential vaccination rates of older adults were estimated at 68.1% for pneumococcal vaccination, 58.1% for herpes zoster vaccination, 53.9% for pertussis vaccination and 54.3% for influenza vaccination. For persons aged 50-65, potential vaccination rates were estimated at 58.1% for pneumococcal vaccination, 49.5% for herpes zoster vaccination, 43.9% for pertussis vaccination and 42.2% for influenza vaccination. For persons aged 65 and older, these were respectively 76.2%, 67.5%, 57.5% and 65.5%.Discussion: Our results suggest that older adults are most likely to accept pneumococcal vaccination of the four vaccines. Information provision accompanied with the implementation of a new vaccine has to be tailored for the individual and the vaccine it concerns. Special attention is needed to ensure high uptake among persons aged 50-65 years. (C) 2017 Elsevier Ltd. All rights reserved.

BACKGROUND: Intestinal bile salts (BSs) may be implicated in NEC development. We hypothesized that fecal BS levels are higher in preterm infants at risk for NEC.METHODS: We compared the composition and concentration of fecal BSs in ten preterm infants who developed NEC (Bell's Stage ≥ II) with twenty matched control infants without NEC. Conjugated and unconjugated fecal BSs were measured after birth (T1) and twice prior to NEC (T2, T3). Data are presented as medians and interquartile ranges.RESULTS: GA and BW were similar in all preterms: ~27+4 weeks and ~1010 g. Age of NEC onset was day 10 (8-24). T1 was collected 2 (1-3) days after birth. T2 and T3 were collected 5 (5-6) days and 1 (0-2) day before NEC or at corresponding postnatal ages in controls. The composition of conjugated BSs did not differ between the two groups. Total unconjugated BSs were 3-fold higher before NEC compared to controls at corresponding ages (0.41 μmol/g feces (0.21-0.74) versus 0.14 μmol/g feces (0.06-0.46), p < 0.05).CONCLUSION: Fecal BS concentrations are higher in preterm infants who develop NEC compared to infants without NEC. Further study is needed to determine the predictive value of fecal BSs in the development of NEC.

The quality-adjusted life year combines the two main components of health, namely the quantity and quality of life, into a single measure. Health summary measures can also be generated by the disability-adjusted life year approach, which combines years of life lost to premature death and years lived with a disability or disease, weighted for the severity of the condition. The Q-TWiST, another summary measure, is known for its clinical orientation; many of its applications are based on randomized controlled trials. This chapter examines the way these summary measures are constructed.

Health promotion programmes for patients with severe mental illness (HPP) are not uniformly evaluated. We discuss the evaluation of HPP in theory and practice, as a prerequisite for future uniform evaluation. We explored the expected outcome and mechanism of HPP in the current literature. Based on this theoretical exploration we selected measures assessing the expected outcome and mechanism in current practice. The individual properties of these measures were described. Based on our theoretical exploration the outcome of HPP can be expressed in several aspects of health. Health can be improved through several mechanisms. In the current evaluation of HPP only some of the expected outcomes were evaluated. The measures used for evaluation were not all representative for the constructs they should assess. Important aspects of HPP are currently not evaluated, based on a comparison between our theoretical exploration of expected outcome and mechanism and current practice. Additionally, not all measures in use are suitable for evaluation of HPP. Therefore, development and identification of suitable measures is necessary. Our framework offers valuable directions for the development of such measures and the future evaluation of HPP. Copyright © 2014 John Wiley & Sons, Ltd.

Objective: To present the results of a literature review on factors related to vaccine uptake by elderly persons. Methods: A systematic literature search was performed using Medline, Embase, and SciSearch to collect all publications available on factors related to vaccine uptake from 1966 until October 2012 for West European and North American societies. In total, 1001 articles were identified and 60 were included in the review. Results: We identified six main themes that influence the willingness to be vaccinated: 1) attitudes and beliefs regarding vaccination in general including positive and negative attitudes and beliefs; 2) perceived risk and severity including knowledge, perceived susceptibility and severity and personal experience; 3) vaccine characteristics including side-effects, effectiveness, content of the vaccine and knowledge; 4) advice and information including influence of the healthcare worker and relatives and the information source and format; 5) general health-related behavior including previous vaccinations, visiting GP or senior center and other preventive behaviors; and 6) accessibility and affordability including logistics, combinations of vaccines and costs. Conclusion: The most important factors related to vaccine uptake are people's attitudes and beliefs regarding vaccination (especially their negative attitudes), recommendations of healthcare workers, side effects and effectiveness of the vaccine, and perceived susceptibility. (C) 2014 Elsevier Inc. All rights reserved.

Item does not contain fulltext BACKGROUND: Recent research suggests that the value of health states may depend on their duration, implying a more complex relationship between quality and quantity of life than the standard linear relationship assumed in the quality-adjusted life years (QALY) model. OBJECTIVE: This study models how duration affects the value of health states, using discrete choice data. METHODS: A study was conducted comprising health profiles including various durations. Health states were described with the EQ-5D. Duration was introduced as a sixth domain with six levels (1, 5, 10, 15, 30, 50 years). Sixty pairs of health profiles were selected, applying a Bayesian approach. Two hundred eight university students self-completed the computerized response tasks. Data were analyzed with a conditional logistic regression model. RESULTS: There is clear evidence for non-linear values for duration, that is, preferences for duration are more accurately described by a logarithmic function. CONCLUSIONS: In discrete choice experiments using health states with different durations, these data present the first evidence for decreasing marginal utility for duration. Our results suggest that refinement of the standard QALY framework may be needed for states with a long duration.

OBJECTIVES: To describe the research that has been undertaken by the EuroQol Group to improve current methods for health state valuation, to summarize the results of an extensive international pilot program, and to outline the key elements of the five-level EuroQol five-dimensional (EQ-5D-5L) questionnaire valuation protocol, which is the culmination of that work.METHODS: To improve on methods of health state valuation for the EQ-5D-5L questionnaire, we investigated the performance of different variants of time trade-off and discrete choice tasks in a multinational setting. We also investigated the effect of three modes of administration on health state valuation: group interviews, online self-completion, and face-to-face interviews.RESULTS: The research program provided the basis for the EQ-5D-5L questionnaire valuation protocol. Two different types of tasks are included to derive preferences: a newly developed composite time trade-off task and a forced-choice paired comparisons discrete choice task. Furthermore, standardized blocked designs for the selection of the states to be valued by participants were created and implemented together with all other elements of the valuation protocol in a digital aid, the EuroQol Valuation Technology, which was developed in conjunction with the protocol.CONCLUSIONS: The EuroQol Group has developed a standard protocol, with accompanying digital aid and interviewer training materials, that can be used to create value sets for the EQ-5D-5L questionnaire. The use of a well-described, consistent protocol across all countries enhances the comparability of value sets between countries, and allows the exploration of the influence of cultural and other factors on health state values.

Objective: To present a systematic review of the evaluation of randomized interventions directed toward improving somatic health for patients with severe mental illness (SMI). Method: A systematic search in PubMed, Embase, Cinahl, and PsycInfo was performed. The scope of the search was prospective studies for patients aged 18-70, published from January 2000 till June 2011. Randomized interventions directed toward improving somatic health for patients with SMI were selected. We excluded studies on elderly, children, and studies performed before 2000. Information on population, type of intervention, follow-up, outcome measures, and on authors' conclusions were drawn from the original articles. Results: Twenty-two original studies were included, presenting four types of interventions: health education (n = 9), exercise (n = 6), smoking cessation (n = 5), and changes in health care organization (n = 2). To evaluate the effect of these studies 93 different outcome measures were used in 16 categories. Conclusion: Many interventions directed toward improving somatic health for patients with SMI have been started. These studies did not apply similar evaluations, and did not use uniform outcome measures of the effect of their interventions. Valuable comparisons on effectiveness are therefore almost impossible. © 2013 John Wiley & Sons A/S.

The willingness of older adults to receive vaccination against influenza, pneumococcal disease, herpes zoster and pertussis and the role of the general practitioners

Background and purpose: Proton therapy is an emerging technique in radiotherapy which results in less dose to the normal tissues with similar target dose than photon therapy, the current standard. Patient-level simulation models support better decision making on which patients would benefit most.Materials and methods: A simulation model was developed tracking individual patients' status regarding the primary tumour and multiple complications. As a proof of principle, the model was populated based on information from a cohort of 1013 head and neck cancer patients. Dose-volume parameters for photon and proton radiation treatment plans were then fed into the model to compare outcomes in terms of length and quality of life and select patients that would benefit most.Results: The illustrative model could adequately replicate the outcomes of photon therapy in the cohort. Improvements from proton therapy varied considerably between patients. The model projects medium-term outcomes for specific individuals and determines the benefits of applying proton rather than photon therapy.Conclusions: While the model needs to be fed with more and especially recent data before being fully ready for use in clinical practice, it could already distinguish between patients with high and low potential benefits from proton therapy. Benefits are highest for patients with both good prognosis and high expected damage to adjacent organs. The model allows for selecting such patients a priori based on patient relevant outcomes. (C) 2016 Elsevier Ltd. All rights reserved.

BackgroundThere are numerous health-related quality of life (HRQol) measurements used in coronary heart disease (CHD) in the literature. However, only values assessed with preference-based instruments can be directly applied in a cost-utility analysis (CUA).ObjectiveTo summarize and synthesize instrument-specific preference-based values in CHD and the underlying disease-subgroups, stable angina and post-acute coronary syndrome (post-ACS), for developed countries, while accounting for study-level characteristics, and within- and between-study correlation.MethodsA systematic review was conducted to identify studies reporting preference-based values in CHD. A multivariate meta-analysis was applied to synthesize the HRQoL values. Meta-regression analyses examined the effect of study level covariates age, publication year, prevalence of diabetes and gender.ResultsA total of 40 studies providing preference-based values were detected. Synthesized estimates of HRQoL in post-ACS ranged from 0.64 (Quality of Well-Being) to 0.92 (EuroQol European"tariff"), while in stable angina they ranged from 0.64 (Short form 6D) to 0.89 (Standard Gamble). Similar findings were observed in estimates applying to general CHD. No significant improvement in model fit was found after adjusting for study-level covariates. Large between-study heterogeneity was observed in all the models investigated.ConclusionsThe main finding of our study is the presence of large heterogeneity both within and between instrument-specific HRQoL values. Current economic models in CHD ignore this between-study heterogeneity. Multivariate meta-analysis can quantify this heterogeneity and offers the means for uncertainty around HRQoL values to be translated to uncertainty in CUAs.

Contains fulltext : 81763.pdf (Publisher’s version ) (Closed access) INTRODUCTION: The clinical presentation of Complex Regional Pain Syndrome type I (CRPS I) in children differs compared to the presentation in adults. Reported results of treatment of CRPS I in children are usually more favourable and seem better than the reported treatment of adults with CRPS I. We investigated the quality of life (QoL) in adults who have been treated for childhood-onset CRPS I. METHODS: We performed a retrospective chart review on signs, symptoms and treatment of all patients, seen and treated for CRPS I in childhood (age

Contains fulltext : 80310.pdf (Publisher’s version ) (Closed access) With the increasing possibilities for surgical treatment of colorectal liver metastases, careful selection of patients who may benefit from surgical treatment becomes critical. The addition of PET to (18)F-FDG may significantly improve conventional staging by CT. Up to now, definitive evidence that the addition of (18)F-FDG PET to conventional staging leads to superior clinical results and improved clinical management in these patients has been lacking. In this randomized controlled trial in patients with colorectal liver metastases, we investigated whether the addition of (18)F-FDG PET is beneficial and reduces the number of futile laparotomies. METHODS: A total of 150 patients with colorectal liver metastases selected for surgical treatment by imaging with CT were randomly assigned to CT only (n = 75) or CT plus (18)F-FDG PET (n = 75). Patients were followed up for at least 3 y. The primary outcome measure was futile laparotomy, defined as any laparotomy that did not result in complete tumor treatment, that revealed benign disease, or that did not result in a disease-free survival period longer than 6 mo. RESULTS: Patient and tumor characteristics were similar for both groups. The number of futile laparotomies was 34 (45%) in the control arm without (18)F-FDG PET and 21 (28%) in the experimental arm with (18)F-FDG PET; the relative risk reduction was 38% (95% confidence interval, 4%-60%, P = 0.042). CONCLUSION: The number of futile laparotomies was reduced from 45% to 28%; thus, the addition of (18)F-FDG PET to the work-up for surgical resection of colorectal liver metastases prevents unnecessary surgery in 1 of 6 patients.

Contains fulltext : 80003.pdf (Publisher’s version ) (Closed access) Contains fulltext : 80003-1.pdf (Author’s version postprint ) (Open Access) In this study, we examined the in vivo relationship between functional tumor vasculature, determined by dynamic contrast-enhanced (DCE-) MRI, and tumor metabolism, determined by dynamic (18)F-FDG PET, during cytotoxic treatment of patients with colorectal liver metastases. METHODS: Twenty-three patients underwent DCE-MRI (using gadolinium dimeglumine) and dynamic (18)F-FDG PET at baseline and after 3 treatment cycles, unless treatment was terminated because of toxicity. Parameters for vasculature (rate constant between extravascular extracellular space and blood plasma [k(ep)] and volume transfer constant [K(trans)]), extracellular space (v(e)), tumor size (the maximal axial diameter of each included lesion [MAD]), and metabolism (glucose metabolic rates [MR(glc)]) were derived, and changes during treatment were correlated. Overall survival (OS) and progression-free survival (PFS) served as outcome measures for the predictive abilities of pretreatment parameters and of treatment-related parameter changes. RESULTS: Pretreatment MR(glc) and MAD were individually predictive for OS and PFS. During treatment, K(trans) increased significantly, but this increase could not be confirmed in a lesion-by-lesion analysis. MR(glc) decreased significantly (P < 0.001). No correlations were found for changes in DCE-MRI parameters and DeltaMR(glc). No relationship was found between changes in DCE-MRI parameters and OS or PFS. DeltaMR(glc) was able to predict OS (P = 0.008) after correction for confounders. CONCLUSION: The efficacy of cytotoxic chemotherapy assessed by reduction in tumor metabolism does not depend on pretreatment properties of the tumor vasculature determined by DCE-MRI. Cytotoxic chemotherapy does not alter DCE-MRI-derived properties of tumor vasculature but decreases glucose consumption of tumor cells.

Contains fulltext : 53587.pdf (Publisher’s version ) (Closed access) Complicating infectious foci resulting from haematogenous or local spread of microorganisms are observed frequently in patients with Staphylococcus aureus bacteraemia (SAB) or Streptococcus species bacteraemia (SSB). The aim of this study was to compare the epidemiology of complicating infectious foci during SAB and SSB in a university hospital in The Netherlands. The charts of all adult patients diagnosed with SAB or SSB (except for Streptococcus pneumoniae bacteraemia) from July 2002 until December 2004 were reviewed retrospectively. Overall, 180 immunocompetent patients were identified, 127 with SAB and 53 with SSB. The percentage of patients with complicating infectious foci (39% of SAB patients, 25% of SSB patients) did not differ significantly between the groups. Endocarditis and cerebral involvement, however, were significantly more common in the SSB group. Of all complicating infectious foci, 32% lacked guiding signs or symptoms and 10% were detected only at autopsy. Factors associated with the development of complicating infectious foci were a delay in treatment for more than 48 h after the onset of symptoms, community acquisition, persistently positive blood cultures, congenital heart disease, and the presence of foreign bodies or prosthetic valves. Infection-related mortality was 18% in SAB patients and 11% in SSB patients and was significantly higher in patients with complicating infectious foci (29 vs. 9%). In conclusion, complicating infectious foci develop in approximately one-third of all patients with SAB and SSB. An active approach that entails searching for the complicating infectious foci is warranted in these patients, because only two-thirds of complicated infectious foci have guiding symptoms or signs, and infection-related mortality is significantly increased in patients with complicating infectious foci compared to patients without these infections. 9 p.

Contains fulltext : 52858.pdf (Publisher’s version ) (Closed access) PURPOSE: Each pathological stage of non-small cell lung cancer (NSCLC) consists of a heterogeneous population containing patients at much higher risk than others. Noninvasive functional imaging modalities, such as 18F-fluorodeoxyglucose positron emission tomography (FDG-PET), could play a role in further characterization of NSCLCs. As many factors can influence the extent of FDG uptake, the underlying mechanisms for FDG accumulation in tumors, are still a matter of debate. The aim of the present study was to investigate these possible mechanisms in the primary site of early stage preoperatively untreated NSCLC. METHODS: 19 patients with early stage NSCLC, who had undergone both preoperative FDG-PET imaging and curative surgery, were enrolled in this study. Standardized uptake values (SUVs) were used for evaluation of primary tumor FDG uptake. Final diagnosis, tumor type, tumor cell differentiation and size of the primary tumors were confirmed histopathologically in resected specimens. Histologic sections were analyzed for amount of inflammation and necrosis. Expression of the glucose membrane transporters (GLUT-1 and GLUT-3); the isoforms of the glycolytic enzyme hexokinase (HK-I, HK-II and HK-III); and the cysteine protease caspase-3, was evaluated immunohistochemically. RESULTS: FDG uptake was significantly higher in squamous cell carcinomas (mean SUV 13.4+/-4.9, n=8) compared to adenocarcinomas (7.1+/-3.3, n=8, p=0.007), or large cell carcinomas (5.9+/-1.9, n=3, p=0.02). The degree of FDG accumulation seemed to depend especially on GLUT-1, GLUT-3 and tumor cell differentiation. The summed standardized values of these three parameters correlated significantly with the SUV (r=0.47, p=0.05). CONCLUSION: The present study supports the hypothesis that tumor cell differentiation in combination with overexpression of GLUT-1 and GLUT-3 determine the extent of FDG accumulation and that squamous cell carcinomas accumulate more FDG than adenocarcinomas or large cell carcinomas.

Aim: To evaluate the psychometric properties of the Dutch version of the 16-item Subjective Opiate Withdrawal Scale (SOWS). The SOWS measures withdrawal symptoms at the time of assessment. Methods: The Dutch SOWS was repeatedly administered to a sample of 272 opioid-dependent inpatients of four addiction treatment centers during rapid detoxification with or without general anesthesia. Examination of the psychometric properties of the SOWS included exploratory factor analysis, internal consistency, test-retest reliability, and criterion validity. Results: Exploratory factor analysis of the SOWS revealed a general pattern of four factors with three items not always clustered in the same factors at different points of measurement. After excluding these items from factor analysis four factors were identified during detoxification (‘temperature dysregulation’, ‘tractus locomotorius’, ‘tractus gastro-intestinalis’ and ‘facial disinhibition’). The 13-item SOWS shows high internal consistency and test-retest reliability and good validity at different stages of withdrawal. Conclusion: The 13-item SOWS is a reliable and valid instrument to assess opioid withdrawal during rapid detoxification. Three items were deleted because their content does not correspond directly with opioid withdrawal symptoms.