Barriers and facilitators to national guideline implementation for palliative cancer care in a Danish cross-sectoral healthcare setting: A qualitative study of healthcare professionals' experiences.

OBJECTIVE: Patients with incurable cancer should receive general palliative care according to their needs, as provided through collaboration between hospital departments, municipalities, and general practices and as outlined in national guidelines. However, the implementation of general palliative care in Denmark has been inadequate. This study aimed to investigate the healthcare professionals' (HCPs') perceptions on barriers to and facilitators of the implementation of the Danish National Guideline (NG) for general palliative care. METHODS: This descriptive, qualitative study was guided by the Consolidated Framework for Implementation Research (CFIR). Qualitative focus group and individual interviews were conducted with 23 HCPs. The interview guide, coding, analysis, and reporting of findings were developed within the CFIR framework. RESULTS: The main barriers to implementing NG were as follows: lack of knowledge about the NG, lack of an implementation plan, and insufficient communication and collaboration across sectors. Important facilitators were as follows: HCP motivation to meet palliative care needs, HCPs with special functions taking responsibility for incorporating NG into local guidelines, and the role of district nurses specialised in palliative care as opinion leaders providing security and continuity for the HCPs working in palliative care. CONCLUSIONS: To address the needs of patients with incurable cancer, greater efforts are required on implementing general palliative care. Although HCPs in our setting were motivated to improve NG implementation, financial resources and strategies are necessary to ensure sufficient knowledge uptake and accommodate identified barriers in order to translate the NG into practice.

Validation of the psychosocial consequences of screening in lung cancer questionnaire in the international lung screen trial Australian cohort.

BACKGROUND: Evaluation of psychosocial consequences of lung cancer screening with LDCT in high-risk populations has generally been performed using generic psychometric instruments. Such generic instruments have low coverage and low power to detect screening impacts. This study aims to validate an established lung cancer screening-specific questionnaire, Consequences Of Screening Lung Cancer (COS-LC), in Australian-English and describe early results from the baseline LDCT round of the International Lung Screen Trial (ILST). METHODS: The Danish-version COS-LC was translated to Australian-English using the double panel method and field tested in Australian-ILST participants to examine content validity. A random sample of 200 participants were used to assess construct validity using Rasch item response theory models. Reliability was assessed using classical test theory. The COS-LC was administered to ILST participants at prespecified timepoints including at enrolment, dependent of screening results. RESULTS: Minor linguistic alterations were made after initial translation of COS-LC to English. The COS-LC demonstrated good content validity and adequate construct validity using psychometric analysis. The four core scales fit the Rasch model, with only minor issues in five non-core scales which resolved with modification. 1129 Australian-ILST participants were included in the analysis, with minimal psychosocial impact observed shortly after baseline LDCT results. CONCLUSION: COS-LC is the first lung cancer screening-specific questionnaire to be validated in Australia and has demonstrated excellent psychometric properties. Early results did not demonstrate significant psychosocial impacts of screening. Longer-term follow-up is awaited and will be particularly pertinent given the announcement of an Australian National Lung Cancer Screening Program. TRIAL REGISTRATION: NCT02871856.

Data driven or data informed? How general practitioners use data to evaluate their own and colleagues' clinical work in clusters.

In contemporary policy discourses, data are presented as key assets for improving health-care quality: policymakers want health care to become 'data driven'. In this article, we focus on a particular example of this ambition, namely a new Danish national quality development program for general practitioners (GPs) where doctors are placed in so-called 'clusters'. In these clusters, GPs are obliged to assess their own and colleagues' clinical quality with data derived from their own clinics-using comparisons, averages and benchmarks. Based on semi-structured interviews with Danish GPs and drawing on Science and Technology Studies, we explore how GPs understand these data, and what makes them trust-or question-a data analysis. The GPs describe how they change clinical practices based on these discussions of data. So, when and how do data for quality assurance come to influence their perceptions of quality? By exploring these issues, we carve out a role for a sociological engagement with evidence in everyday medical practices. In conclusion, we suggest a need to move from the aim of being data driven to one of being data informed.

Psychometric validation of the Danish version of the Major Depression Inventory using data from the Lolland-Falster health study (LOFUS).

PURPOSE: The Major Depression Inventory (MDI) is a widely used self-rating depression scale commonly in primary care in Denmark. It has not been subject to robust psychometric validation in a general population setting. The aim of this study was to evaluate the psychometric measurement properties of the MDI when applied in the general population. METHODS: We evaluated statistical psychometric validity using modern test theory (confirmatory factor analysis, item response theory models and Rasch measurement theory) testing local independence and differential item function across groups defined by gender, age, education, and chronic disease status. Separate analyses across different strata and across different statistical models were employed. RESULTS: Regarding structural validity we consistently identified local dependence for the item two pairs (MDI2,MDI3) and (MDI4,MDI5) across strata. This result was confirmed by bifactor CFA models and item screening. We further identified substantial differential item functioning with respect to age group and with respect to chronic disease. We identified quantified the magnitude of this lack of measurement invariance. CONCLUSION: The MDI is psychometrically valid in homogenous sub populations, but the disclosed evidence of local dependence means that published estimates of its reliability cannot be trusted. The lack of measurement invariance means that the instrument cannot be used to compare individuals or groups unless they are similar in terms of age group and chronic disease status.

Colon cancer diagnosed in patients with non-specific symptoms - comparisons between diagnostic paradigms.

Background In Denmark, the Cancer Patient Pathway for Non-Specific Signs and Symptoms (NSSC-CPP) has been implemented with variations: in some areas, general practitioners (GPs) do the initial diagnostic work-up (GP paradigm); in other areas, patients are referred directly to the hospital (hospital paradigm). There is no evidence to suggest the most beneficial organisation. Therefore, this study aims to compare the occurrence of colon cancer and the risk of non-localised cancer stage between the GP and hospital paradigms.Material and Methods In this registry-based case-control study, we applied multivariable binary logistic regression models to estimate the odds ratios (OR) of colon cancer and non-localised stage associated with the GP paradigm and hospital paradigm. All cases and controls were assigned to a paradigm based on their diagnostic activity (CT scan or CPP) six months before the index date. As not all CT scans in the control group were part of the cancer work-up as a sensitivity analysis, we investigated the impact of varying the fraction of these, which were randomly removed using a bootstrap approach for inference.Results The GP paradigm was more likely to result in a cancer diagnosis than the hospital paradigm; ORs ranged from 1.91-3.15 considering different fractions of CT scans as part of cancer work-up. No difference was found in the cancer stage between the two paradigms; ORs ranged from 1.08-1.10 and were not statistically significant.Conclusion Patients in the GP paradigm were diagnosed with colon cancer more often, but we cannot conclude that the distribution of respectively localised or non-localised extent of disease is different from that of patients in the hospital paradigm.

Women's experiences of age-related discontinuation from mammography screening: A qualitative interview study.

INTRODUCTION: In Denmark, women are discontinued from mammography screening at age 69 due to decreased likelihood of benefits and increased likelihood of harm. The risk of harm increases with age and includes false positives, overdiagnosis and overtreatment. In a questionnaire survey, 24 women expressed unsolicited concerns about being discontinued from mammography screening due to age. This calls for further investigation of experiences related to discontinuation from screening. METHODS: We invited the women, who had left comments on the questionnaire, to participate in in-depth interviews with the purpose to explore their reactions, preferences, and conceptions about mammography screening and discontinuation. The interviews lasted 1-4 h and were followed up with a telephone interview 2 weeks after the initial interview. RESULTS: The women had high expectations of the benefits of mammography screening and felt that participation was a moral obligation. Following that, they perceived the screening discontinuation as a result of societal age discrimination and consequently felt devalued. Further, the women perceived the discontinuation as a health threat, felt more susceptible to late diagnosis and death, and therefore sought out new ways to control their risk of breast cancer. CONCLUSION: Our findings indicate that the age-related discontinuation from mammography screening might be of more importance than previously assumed. This study raises important questions about screening ethics, and we encourage research to explore this in other settings. PATIENT AND PUBLIC CONTRIBUTION: This study was conducted as a result of the women's unsolicited concerns about being discontinued from screening. This particular group contributed to the study with their own statements, interpretations and perspectives on the discontinuation of screening, and the initial analysis of data was discussed with the women during follow-up interviews.

The impact of influences in a medical screening programme invitation: a randomized controlled trial.

BACKGROUND: Invitations to screening programmes may include influences that are intending to increase the participation rates. This study had two objectives: (i) to assess if different categories of influences had a significant effect on the intention to participate in a screening programme for a fictitious disease and (ii) whether participants were aware of the influences, and if the intention to participate was associated to this awareness. METHODS: A seven-armed randomized controlled trial. Six hundred passers-by were randomly allocated to receive one of seven pamphlets inviting to a fictitious screening programme (neutral, relative risk reductions, misrepresentation of harms, pre-booked appointment, recommendation of participation, fear appeals, all combined). Participants were surveyed to assess (i) intention to participate (ITP) in the screening programme and (ii) awareness of an exerted influence. Chi-squared test was used to calculate the effect of the influences on ITP and the association of ITP with indicating awareness of an exerted influence and correctly locating an influence. RESULTS: Five hundred and eighty-nine participants were included for analysis. ITP was significantly increased (P < 0.05) in three pamphlets (misrepresentation of harms, fear appeals, all combined) [adjusted odds ratio (OR) 4.84, 95% confidence interval (CI): 2.54-9.23; OR 2.45, 95% CI: 1.31-4.59; OR 9.02, 95% CI: 4.44-18.34]. A percentage of 60.0-78.3 participants did not indicate awareness. Awareness was associated with a decreased ITP for those who could locate the influence (OR 0.39, 95% CI: 0.21-0.72) and those who failed to locate the influence (OR 0.47, 95% CI: 0.30-0.74). CONCLUSION: The application of influences should be carefully considered for interventions where an informed choice is desired.

General Practitioners' Perspectives on Appropriate Use of Ultrasonography in Primary Care in Denmark: A Multistage Mixed Methods Study.

PURPOSE: Researchers aimed to describe general practitioners' understanding of appropriate ultrasound use, to record actual scanning practices of early adopters in general practice, and to identify differences between attitudes and actual practice via a mixed methods analysis. METHODS: This study was part of a larger multistage mixed methods research framework exploring the use of ultrasound in general practice in Denmark. We used an exploratory sequential approach in the data collection with initial qualitative findings from an interview study applied to building a quantitative questionnaire utilized in a cohort study. In addition, we merged the qualitative and quantitative data using joint display analysis to compare and contrast the results from the 2 stages of the study. RESULTS: In the interviews, general practitioners described appropriate ultrasound use as point-of-care examinations with a clear purpose and limited to predefined specific conditions within delimited anatomic areas. They stated that general practitioners should receive formalized ultrasound training and be skilled in the examinations they perform. In the cohort study, general practitioners performed ultrasound examinations of anatomic areas with or without a defined clinical suspicion. Some performed ultrasound examinations for which they had no previous training or skills. CONCLUSIONS: We found a difference between the ideas about the appropriate uses for ultrasound in general practice and the actual use by early adopters in clinical practice. Our findings suggest a need for evidence-based guidelines to support general practitioners in choosing which examinations to perform and strategies for developing and maintaining scanning competency.

Is it okay to choose to receive bad news by telephone? An observational study on psychosocial consequences of diagnostic workup for lung cancer suspicion.

BACKGROUND: In-person meeting is considered the gold standard in current communication protocols regarding sensitive information, yet one size may not fit all, and patients increasingly demand or are offered disclosure of bad news by, e.g., telephone. It is unknown how patients' active preference for communication modality affect psychosocial consequences of receiving potentially bad news. AIM: To explore psychosocial consequences in patients, who themselves chose to have results of lung cancer workup delivered either in-person or by telephone compared with patients randomly assigned to either delivery in a recently published randomised controlled trial (RCT). METHODS: An observational study prospectively including patients referred for invasive workup for suspected lung cancer stratified in those declining (Patient's Own Choice, POC group) and those participating in the RCT. On the day of invasive workup and five weeks later, patients completed a validated, nine-dimension, condition-specific questionnaire, Consequences of Screening in Lung Cancer (COS-LC). Primary outcome: difference in change in COS-LC dimensions between POC and RCT groups. RESULTS: In total, 151 patients were included in the POC group versus 255 in the RCT. Most (70%) in the POC group chose to have results by telephone. Baseline characteristics and diagnostic outcomes were comparable between POC and RCT groups, and in telephone and in-person subgroups too. We observed no statistically significant between-groups differences in any COS-LC score between POC and RCT groups, or between telephone and in-person subgroups in the POC group. CONCLUSION: Continually informed patients' choice between in-person or telephone disclosure of results of lung cancer workup is not associated with differences in psychosocial outcomes. The present article supports further use of a simple model for how to prepare the patient for potential bad news.

Use and diagnostic outcomes of cancer patient pathways in Denmark - is the place of initial diagnostic work-up an important factor?

INTRODUCTION: The Cancer Patient Pathway for Non-specific Symptoms and Signs of Cancer (NSSC-CPP) has been implemented in Denmark with regional and intra-regional differences. In some places, the initial diagnostic work-up (often including a CT scan) is performed by general practitioners (GPs) and in others by hospitals. Variations may influence the use of Organ Specific Cancer Patient Pathways (OS-CPPs) and prognostic outcomes for the patients. Therefore, the aims were: 1) To analyse how a CT scan referred from GP or hospital is followed by OS-CPPs and NSSC-CPPs at the national and regional level, and 2) To analyse, nationally and regionally, the diagnostic outcomes of persons referred to CT scan by either GP or hospital six months after and mortality one year after CT scan. METHODS: A nationwide population-based study including individuals with a first CT scan in 2013-2016, either referred from GP or hospital. RESULTS: Overall, individuals with a CT scan referred from GPs were more likely to start a NSSC-CPP or an OS-CPP than individuals with a CT scan referred by hospitals. Across the five Regions in Denmark, CT scans referred by GPs were associated with reduced odds of total mortality in all regions; (North, OR=0.78 [0.73 0.83], Central, OR=0.92 [0.87 0.96], South, OR=0.85 [0.81 0.89], Capital, OR=0.96 [0.91 1.00] and Zealand, OR=0.85 [0.79 0.90]) and increased odds of cancer-specific mortality in four regions, ORs ranging from 1.15-1.51 with no difference in Region North (1.00 [0.91 1.10]). CONCLUSION: No obvious association between more CT scans and CPPs and reduced diagnoses and mortality was observed. The different diagnostic models might not explain the prognostic outcomes, but the different use of CT scans in, and between Regions play a large role in the differences in incidence and mortality.

Learning strategies of general practitioners striving to achieve point-of-care ultrasound competence: a qualitative study.

BACKGROUND: Point-of-care ultrasonography (POCUS) is increasingly used in general practice despite the lack of official educational programmes or guidelines for general practitioners (GPs). AIM: To explore how GPs have learnt to use POCUS and which barriers they have encountered in their learning process. DESIGN AND SETTING: Qualitative study conducted in office-based general practice in Denmark. METHODS: Semi-structured interviews were conducted with 13 GPs who had implemented POCUS without supporting guidelines or regulations. Analysis was carried out using systematic text condensation. The interview data for this study were collected along with data used in a previous study. RESULTS: The participating GPs described having composed their own ultrasound education following a continuous learning process. Basic POCUS competences were achieved through formalized training sessions at hospital departments or courses. The GPs further developed and expanded their scanning skills through additional courses and continuous self-study practice on patients often while consulting internet sources, textbooks or colleagues. Lack of available ultrasound courses, supervision, and clinical guidelines together with time constraints and financial aspects were mentioned as barriers to their ultrasound training. CONCLUSION: This study showed how GPs had composed their own ultrasound education individually and differently, guided by their own experiences and beliefs about good clinical practice. Formalized ultrasound training was considered a prerequisite for achieving basic ultrasound competences while continuous practice was considered paramount to develop and maintain scanning skills. There were several obstacles to overcome in the learning process including lack of supervision, guidance, and opportunity for practicing skills.Key pointsLittle is known about the educational needs of general practitioners striving to achieve ultrasound competences.General practitioners described using formalized training to achieve basic scanning competences and continuous self-study and practice to further develop their skills.Lack of time, supervision, clinical guidelines and ultrasound courses were considered barriers in the learning process together with financial aspects.

Four of five frequently used orthopedic PROMs possess inadequate content validity: a COSMIN evaluation of the mHHS, HAGOS, IKDC-SKF, KOOS and KNEES-ACL.

PURPOSE: Content validity is the most important property of PROMs. The COSMIN initiative has published guidelines for evaluating the content validity of PROMs, but they have only sparsely been applied to relevant PROMs for musculoskeletal conditions. The aim of this study was to use the COSMIN Risk of Bias checklist to evaluate the content validity of five PROMs, that are highly relevant in musculoskeletal research and used by the arthroscopic surgery community: the modified Harris' Hip Score (mHHS), the Copenhagen Hip and Groin Outcome Score (HAGOS), the International Knee Documentation Committee Subjective Knee evaluation Form (IKDC-SKF), the Knee injury and Osteoarthritis Outcome Score (KOOS) and the Knee Numeric-Entity Evaluation Score ACL (KNEES-ACL). METHODS: The development articles for the five PROMs were identified through searches in PubMed and SCOPUS. A literature search was performed to identify additional studies assessing content validity of the PROMs. Additional information, necessary for the assessments, was obtained from the PROM developers after direct request. To evaluate the quality of the development studies and rate the content validity, the COSMIN Risk of Bias checklist was applied to all studies. RESULTS: All five development studies were identified. Three subsequent content validity studies were identified, all evaluating KOOS and one also IKDC. One content validity study was of inadequate quality and excluded from further analysis. The development of mHHS, IKDC-SKF, and KOOS was rated inadequate and possess insufficient content validity for their target populations. Due to the irrelevance of multiple items, KOOS was in particular inappropriate to evaluate patients with an ACL injury. The development of HAGOS was also rated inadequate, although the insufficiency aspects can be regarded as minor. KNEES-ACL possessed sufficient content validity. CONCLUSION: Out of five PROMs, only KNEES-ACL possessed sufficient content validity. Particularly, KOOS should not be used as an outcome for patients with an ACL injury. There is an urgent need for condition-specific PROMs for musculoskeletal conditions, developed with adequate methods. LEVEL OF EVIDENCE: III.

Re: Hofmann et al. Overdiagnosis, one concept, three perspectives, and a model.

It is with great interest we have read the article "Overdiagnosis: one concept, three perspectives, and a model" by Hofmann and colleagues. We share the authors' ambition of understanding what overdiagnosis is and what it isn't. In our research, we define overdiagnosis on the basis of two interrelated phenomena: overdetection and overdefinition. Overdetection is the labelling of a person with a disease or abnormal condition, that would not have caused the person harm, e.g., symptoms or death, if left undiscovered. Overdefinition is the creation of new diagnoses by overmedicalising ordinary life experiences or expanding existing diagnoses by lowering thresholds or widening diagnostic criteria, without evidence of improved outcomes. These phenomena have different causes and thereby often different drivers. However, they have one important consequence in common: people are turned into patients unnecessarily, i.e., overdiagnosed. On a personal level, overdiagnosis cause various types of harms, including physical, psychological, social and financial harm. On a societal level, overdiagnosis may also cause harm to public health, cause resource waste, and cultural changes with overmedicalisation of normal life events. By definition, none of the aforementioned phenomena lead to any clinical benefit. Therefore, we disagree with Hofmann and colleagues' definition of overdiagnosis as diagnoses that "…on balance, do more harm than good.". We argue that introducing balance and benefits to the definition of overdiagnosis complicates the concept unnecessarily and cause problems operationalising overdiagnosis.

Choosing the most appropriate PROM for clinical studies in sports medicine.

Choosing the most appropriate patient-reported outcome measure (PROM) for a clinical study is essential in order to achieve trustworthy results. This choice will depend on (a) the objective of the study and hence the research question; (b) the choice of a theoretical framework, such as the World Health Organization's International Classification of Functioning, Disability, and Health (ICF); (c) whether there currently is a PROM that possesses high content validity and high construct validity for the specific patient group and objective, and if not; (d) the decision on whether to use a suboptimal PROM or develop and validate a new PROM. This paper presents the steps that should be followed in order to assess the relevance of PROMs and suggests ways to enhance the choice depending on the goal of the study.

A catalogue of PROMs in sports science: Quality assessment of PROM development and validation.

Choosing the most adequate PROM for a study is a non-trivial process. The aim of this study was to provide a catalogue with analyses of content and construct validity of PROMs relevant to research in sports science, including all published local translations. The most commonly used PROMs in sports research were selected from a PubMed search "patient reported outcome measures sports", identifying 439 articles and 194 different PROMs. Articles describing development of the 61 selected PROMs were assessed for content validity, and all articles regarding construct validity of each PROM and all published translations (in total 622 articles) were analyzed. A catalogue with assessments of the 61 PROMs was produced. The majority were of inferior validity, with few exceptions. The most common reason for this was that the PROM had not been developed by methods that ensure high content validity. Another major reason for inferior validity was that construct validity had not been secured by adequate statistical methods. In conclusion, this catalogue provides a tool for researchers to facilitate choosing the most valid PROM for studies in sports research. Furthermore, it shows for popular PROMs where further validation is needed, and for fields in musculoskeletal medicine where valid PROMs are lacking. It is suggested that a targeted effort is made to develop valid PROMs for major conditions in musculoskeletal research. The current method is easier to practice compared with assessment after COSMIN guidelines.

Are adequate PROMs used as outcomes in randomized controlled trials? an analysis of 54 trials.

Results by patient-reported outcome measures (PROMs) from randomized controlled trials (RCTs) in musculoskeletal research often influence healthcare strategies. We aimed to evaluate to which extent these RCTs use adequate PROMs, and how this influences the results and conclusions. We identified RCTs of sports research relevance with PROMs as primary outcomes published in 13 preselected journals between January 1, 2008, and November 1, 2019; all journals regularly publish results from musculoskeletal research. Five journals have a high impact factor (>15), and eight with lower impact factors are widely read journals. It was assessed whether the RCTs had used PROMs with high content validity and whether the most adequate PROMs were used (ie, the most well developed and well validated for the patients enrolled in the study). We registered journal impact factor, year of publication, existence of a registered protocol, and whether the study showed significant difference between interventions. A total of 54 RCTs with 56 primary outcomes comprising 26 different PROMs were identified. For 13 RCTs (24%), a protocol was not published. In only 24 of RCTs (44%), the most appropriate PROM had been used as primary outcome, independent of a registered protocol, ranking of the journal, and year of publication. In seven cases, PROMs were used to evaluate a condition that they had not been developed for. RCTs that used the most adequate PROM showed significantly more often (46%) difference in outcomes in contrast to RCTs that used inadequate PROMs (22%) (P = 0.0483). In conclusion, in the majority of RCTs, the most adequate PROM had not been used. Studies, in which the most adequate PROM had been used as outcome, were significantly more likely to show significant difference between interventions. The extent to which protocols were not available was surprisingly high. Journals should request that adequate PROMs are used in RCTs, and if this is not the case that it is discussed how it might influence the results and conclusions. Likewise, it should be requested that a protocol is published or registered.

Potential problems in the use of patient reported outcome measures (PROMs) and reporting of PROM data in sports science.

To use an inadequate patient reported outcome measure (PROM) or use a PROM in an inappropriate way potentially influences the quality of measurement. The objectives of this study were to define potential inadequate uses of PROMs in sports research studies and estimate how often they occur. A consensus group consisting of medical researchers, statisticians, and psychometricians identified and defined potentially irregular applications of PROMs. Occurrence of these in 349 consecutive articles in sports medicine in which PROMs were used as primary outcomes was reviewed. In all, 14 different potential problems were defined, and one or several occurred in 172 of the articles (49%). These were as follows: using a PROM that was developed for a different patient group (100 cases), using two or more PROMs with identical questions (94), aggregation of domain sum scores (82), combinations of subjective and objective measures (27), using a PROM to diagnose or evaluate the individual patient (7), using a PROM for a single limb (3), recall bias (3), exclusion of domains or items (3), construction of a PROM for a specific occasion (2), categorization of the scale (2), and mixing different versions of a PROM (1). Adaption of scale scores (e. g., to percentage) when results are reported (144) carries a risk of miscalculation and distorted impression of results. Data related to uncertainty about completing the PROM and the handling of missing data were not provided in the manuscripts. In conclusion, potential problems in the use and reporting of PROMs are common in sports research, and this can influence the validity of reported results.

How to develop a condition-specific PROM.

Developing new patient-reported outcome measures (PROMs) for application in clinical studies can be necessary if an adequate PROM does not exist. For adequate measurement, it is essential that the PROM has face validity (ie, is perceived to be relevant by clinicians and researchers) and has high content validity (ie, content relevance and content coverage for the targeted patient group). The steps needed to create PROMs that possess face and content validity for a specific condition are described in this paper. Face validity is achieved by item identification and generation through literature review. Content validity is confirmed through repetitive cognitive interviews of patients from the targeted patient group in order to generate a consensus-based pilot-version of the new PROM. This qualitative process ensures that items are appropriately worded, understandable, and minimizes doubts about how items should be answered. A practical example of this process is presented, which shows the development of the Knee Numeric-Entity Evaluation Score (KNEES-ACL), a condition-specific PROM for patients with deficiency of the anterior cruciate ligament (ACL).

Responsiveness, minimal important difference, minimal relevant difference, and optimal number of patients for a study.

Several terms are used to describe changes in PROM scores in relation to treatments. Whether the change is small, large, or relevant is defined in different ways, yet these change scores are used to recommend or oppose treatments. They are also used to calculate the necessary number of patients for a study. This article offers a theoretical explanation behind the terms responsiveness, minimal important difference (MID), minimal important change (MIC), minimal relevant difference (MIREDIF), and threshold of clinical importance. It also gives instructions on how these and the optimal number of patients for a study are calculated. Responses to two domains of the Knee Injury and Osteoarthritis Outcome Score (KOOS), before and 1 year after reconstruction of the anterior cruciate ligament of 164 patients, are used to illustrate the calculations. This paper presents the most common methods used to calculate and interpret MID. Results vary substantially across domains, patient location on the scale, and health conditions. The optimal number of patients depends on the minimal relevant difference (MIREDIF), the standard error of the measure (SEM), the desired statistical power for the measurement, and the responsiveness of the measurement instrument (the PROM). There is often uncertainty surrounding the calculation and interpretation of responsiveness, MID, and MIREDIF, as these concepts are complex. When MID is used to evaluate research results, authors should specify how the MID was calculated, and its relevance for the study population. These measures should only be used after thorough consideration to justify healthcare decisions.

Are PROMs used adequately in sports research? An analysis of 54 randomized controlled trials with PROMs as endpoint.

Deviations from adequate use and reporting of PROMs may be problematic and misleading. The aim of this study was to investigate the extent of such problems in randomized clinical trials (RCTs). RCTs involving sports medicine research that used PROMs as primary outcomes were identified in 13 preselected journals. The articles were reviewed for nine potential problems related to how the PROM was used and how the data had been reported. The potential problems were as follows: aggregating subscale scores; combining patient-reported scores with physical, clinical, or para-clinical measures; using a PROM to diagnose or evaluate the individual patient; using a PROM for one leg or arm; selectively excluding domains or items; constructing a PROM for the specific occasion; mixing PROM formats (ie, digital, paper, telephone, e-mail, in person); ambiguous instructions for how the PROM should be completed; and recall bias. As covariates, we registered journal impact factor, year of publication, and existence of a registered protocol. In 29 (53.7%) of 54 identified RCTs, at least one potential problem was identified, the most common being aggregation of domain scores. This was not different with a published protocol or dependent on journal rankings, except for exclusion of domains, which was most common in high-ranking journals. Aggregation of domain scores was significantly less common in recently published articles compared with older articles (P = .03). Potential problematic use of PROMs and reporting of PROM data are common in RCTs, also in high-ranking journals, but less so in more recent articles.