Showing papers in "Journal of Patient-Reported Outcomes in 2018"
TL;DR: The findings show thatPROMs completion is not a neutral act of information retrieval but can change how patients think about their condition and reveal that the ways in which clinicians use PROMs is shaped by their relationships with patients and professional roles and boundaries.
Abstract: In this paper, we report the findings of a realist synthesis that aimed to understand how and in what circumstances patient reported outcome measures (PROMs) support patient-clinician communication and subsequent care processes and outcomes in clinical care We tested two overarching programme theories: (1) PROMs completion prompts a process of self-reflection and supports patients to raise issues with clinicians and (2) PROMs scores raise clinicians’ awareness of patients’ problems and prompts discussion and action We examined how the structure of the PROM and care context shaped the ways in which PROMs support clinician-patient communication and subsequent care processes PROMs completion prompts patients to reflect on their health and gives them permission to raise issues with clinicians However, clinicians found standardised PROMs completion during patient assessments sometimes constrained rather than supported communication In response, clinicians adapted their use of PROMs to render them compatible with the ongoing management of patient relationships Individualised PROMs supported dialogue by enabling the patient to tell their story In oncology, PROMs completion outside of the consultation enabled clinicians to identify problematic symptoms when the PROM acted as a substitute rather than addition to the clinical encounter and when the PROM focused on symptoms and side effects, rather than health related quality of life (HRQoL) Patients did not always feel it was appropriate to discuss emotional, functional or HRQoL issues with doctors and doctors did not perceive this was within their remit This paper makes two important contributions to the literature First, our findings show that PROMs completion is not a neutral act of information retrieval but can change how patients think about their condition Second, our findings reveal that the ways in which clinicians use PROMs is shaped by their relationships with patients and professional roles and boundaries Future research should examine how PROMs completion and feedback shapes and is influenced by the process of building relationships with patients, rather than just their impact on information exchange and decision making
247 citations
TL;DR: There is underestimation in the assessment of adverse events in Japan, and that the Japanese version of the PRO-CTCAE had acceptable reliability and validity for common and clinically important symptoms.
Abstract: The Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) was developed by the National Cancer Institute as an adverse event assessment system to evaluate patients’ symptoms, which tend to be underestimated in cancer clinical trials. The aim of this study was to assess the psychometric properties of the Japanese version of the PRO-CTCAE and the degree of adverse event assessment discordance between clinicians and patients. A total of 187 cancer patients receiving systemic therapy were enrolled. Reproducibility, criterion validity, and responsiveness of the Japanese version of PROCTCAE were assessed. The EORTC QLQ-C30 was used as an external anchor. Discordance of assessment of adverse events between clinician and patients were also assessed using the CTCAE and PRO-CTCAE. A total of 187 participants (187 for criterion validity, 80 for reproducibility, and 100 for responsiveness), were analyzed (Mage = 62.4 years). All patients responded to at least one symptom item (M = 16). The mean (SD) intra-class correlation coefficients of overall reproducibility for the Japanese PRO-CTCAE was 0.63 (0.02). The correlation coefficient for the corresponding items in the EORTC QLQ-C30 and the Japanese PRO-CTCAE was high (Pearson r = 0.56–0.76). The analysis of responsiveness revealed significant dose-response trends (Jonckheere-Terpstra test, ps < 0.001). Depending on the adverse events, a discrepancy was observed in evaluation between the clinician and patient. These results revealed that there is underestimation in the assessment of adverse events in Japan, and that the Japanese version of the PRO-CTCAE had acceptable reliability and validity for common and clinically important symptoms.
241 citations
TL;DR: Having an implementation lead overseeing the process and developing the process based on feedback were identified as facilitating implementation, and organisations need to invest time and resources in ‘designing’ the PROMs strategy and ‘preparing” the organisation to usePROMs.
Abstract: There is increasing interest in using Patient Reported Outcome Measures (PROMs) within organisations delivering health related services. However, organisations have had mixed success in implementing PROMs and there is little understanding about why this may be. Thus, the purpose of this study was to identify the facilitators and barriers to implementing PROMs in organisations. A systematic review of reviews was undertaken. Searches were conducted of five electronic databases: MEDLINE, EMBASE, CINAHL, PsycINFO and the Cochrane Database of Systematic Reviews, during the week of the 20th February 2017. Additional search methods included website searching and reference checking. To be included, a publication had to be a review of the literature, describe its methods and include information related to implementing PROMs. The reviews were extracted using a standardised form and assessed for their risk of bias using the Risk of Bias in Systematic Reviews tool. The findings were synthesised using the Consolidated Framework for Implementation Research. The protocol was registered on the International Prospective Register of Systematic Reviews database (PROSPERO) (CRD42017057491). Initially 2047 records were identified. After assessing eligibility, six reviews were included. These reviews varied in their review type and focus. Different issues arose at distinct stages of the implementation process. Organisations needed to invest time and resources in two key stages early in the implementation process: ‘designing’ the processes for using PROMs within an organisation; and ‘preparing’ an organisation and its staff. The ‘designing’ stage involved organisations planning not just which PROMs to use and how to administer them, but also how the data would be used for clinical purposes. The ‘preparing’ stage involved getting an organisation and its staff ready to use PROMs, particularly persuading clinicians of the validity and value of PROMs, delivering training, and developing electronic systems. Having an implementation lead overseeing the process and developing the process based on feedback were also identified as facilitating implementation. Organisations implementing PROMs need to invest time and resources in ‘designing’ the PROMs strategy and ‘preparing’ the organisation to use PROMs. Focusing on these earlier stages may prevent problems arising when PROMs are used in practice.
154 citations
TL;DR: The framework outlined in this paper is designed to encourage health outcomes researchers to apply MMR to their research and to facilitate innovative, patient-centred methodological solutions to address the complex challenges of the field.
Abstract: Mixed methods research (MMR) has found an increased interest in the field of health outcomes research. Consideration for both qualitative and quantitative perspectives has become key to contextualising patient experiences in a clinically meaningful measurement framework. The purpose of this paper is to outline a process for incorporating MMR in health outcomes research to guide stakeholders in their understanding of the essence of mixed methods inquiry. In addition, this paper will outline the benefits and challenges of MMR and describe the types of support needed for designing and conducting robust MMR measurement studies. MMR involves the application of a well-defined and pre-specified research design that articulates purposely and prospectively, qualitative and quantitative components to generate an integrated set of evidence addressing a single research question. Various methodological design options are possible depending on the research question. MMR designs allow a research question to be studied thoroughly from different perspectives. When applied, it allows the strengths of one approach to complement the restrictions of another. Among other applications, MMR can be used to enhance the creation of conceptual models and development of new instruments, to interpret the meaningfulness of outcomes in a clinical study from the patient perspective, and inform health care policy. Robust MMR requires research teams with experience in both qualitative and quantitative research. Moreover, a thorough understanding of the underlying principles of MMR is recommended at the point of study conception all the way through to implementation and knowledge dissemination. The framework outlined in this paper is designed to encourage health outcomes researchers to apply MMR to their research and to facilitate innovative, patient-centred methodological solutions to address the complex challenges of the field.
101 citations
TL;DR: Evidence is provided supporting the reliability and responsiveness of the newly translated Swedish RAND-36 and the user-friendliness of the electronic version of the questionnaire.
Abstract: The Short Form 36-Item Survey is one of the most commonly used instruments for assessing health-related quality of life. Two identical versions of the original instrument are currently available: the public domain, license free RAND-36 and the commercial SF-36. RAND-36 is not available in Swedish. The purpose of this study was threefold: to translate and culturally adapt the RAND-36 into Swedish; to evaluate its reliability and responsiveness using Svensson’s method for paired ordered categorical data; and to assess the usability of an electronic version of the questionnaire. The translation process included forward and backward translations and reconciliation. Test-retest reliability was examined during a period of two-weeks in 84 patients undergoing dialysis for chronic kidney disease. Responsiveness was examined in 97 patients before and 2 months after a cardiac rehabilitation program. Usability tests and cognitive debriefing of the electronic questionnaire were carried out with 18 patients. The Swedish translation of the RAND-36 was conceptually equivalent to the English version. Test-retest reliability was supported by non-significant relative position (RP) values among dialysis patients for all RAND-36 subscales (range − 0.02 to 0.10; all confidence intervals (CI) included zero). Responsiveness was demonstrated by significant improvements in RP values among cardiac rehabilitation patients for all subscales (range 0.22–0.36; lower limits of all CI > 0.1) except two subscales (General health, RP -0.02; CI -0.13 to 0.10; and Role functioning/emotional, RP 0.03; CI -0.09 to 0.16). In cardiac rehabilitation patients, sizable individual variation (RV > 0.2) was also shown for the Pain, Energy/fatigue and Social functioning subscales. The electronic version of RAND-36 was found easy and intuitive to use. Our results provide evidence supporting the reliability and responsiveness of the newly translated Swedish RAND-36 and the user-friendliness of the electronic version. Svensson’s method for paired ordinal data was able to characterize not only the direction and size of differences among the patients’ responses at different time points but also variations in response patterns within groups. The method is therefore, besides being suitable for ordinal data, also an important and novel tool for gaining insights into patients’ response patterns to treatment or interventions, thus informing individualized care.
91 citations
TL;DR: Both empirical studies and review articles provide evidence that the 11-point NRS is likely the optimal response scale to evaluate pain among adult patients without cognitive impairment.
Abstract: The purpose of this literature review was to examine the existing patient-reported outcome measurement literature to understand the empirical evidence supporting response scale selection in pain measurement for the adult population The search strategy involved a comprehensive, structured, literature review with multiple search objectives and search terms The searched yielded 6918 abstracts which were reviewed against study criteria for eligibility across the adult pain objective The review included 42 review articles, consensus guidelines, expert opinion pieces, and primary research articles providing insights into optimal response scale selection for pain assessment in the adult population Based on the extensive and varied literature on pain assessments, the adult pain studies typically use simple response scales with single-item measures of pain—a numeric rating scale, visual analog scale, or verbal rating scale Across 42 review articles, consensus guidelines, expert opinion pieces, and primary research articles, the NRS response scale was most often recommended in these guidance documents When reviewing the empirical basis for these recommendations, we found that the NRS had slightly superior measurement properties (eg, reliability, validity, responsiveness) across a wide variety of contexts of use as compared to other response scales Both empirical studies and review articles provide evidence that the 11-point NRS is likely the optimal response scale to evaluate pain among adult patients without cognitive impairment
66 citations
TL;DR: The PRO Consortium translation process will play an important role in maintaining the validity of the data generated through these measures by ensuring that they are translated by qualified linguists following a standardized and rigorous process that reflects best practice.
Abstract: This paper describes the rationale and goals of the Patient-Reported Outcome (PRO) Consortium's instrument translation process. The PRO Consortium has developed a number of novel PRO measures which are in the process of qualification by the U.S. Food and Drug Administration (FDA) for use in clinical trials where endpoints based on these measures would support product labeling claims. Given the importance of FDA qualification of these measures, the PRO Consortium's Process Subcommittee determined that a detailed linguistic validation (LV) process was necessary to ensure that all translations of Consortium-developed PRO measures are performed using a standardized approach with the rigor required to meet regulatory and pharmaceutical industry expectations, as well as having a clearly defined instrument translation process that the translation industry can support. The consensus process involved gathering information about current best practices from 13 translation companies with expertise in LV, consolidating the findings to generate a proposed process, and obtaining iterative feedback from the translation companies and PRO Consortium member firms on the proposed process in two rounds of review in order to update existing principles of good practice in LV and to provide sufficient detail for the translation process to ensure consistency across PRO Consortium measures, sponsors, and translation companies. The consensus development resulted in a 12-step process that outlines universal and country-specific new translation approaches, as well as country-specific adaptations of existing translations. The PRO Consortium translation process will play an important role in maintaining the validity of the data generated through these measures by ensuring that they are translated by qualified linguists following a standardized and rigorous process that reflects best practice.
55 citations
TL;DR: Before the promise of practice-based PRO assessment in oncology can be truly realized, investigators need to advance the state-of-the-art of real-time PRO score interpretation as well as developing guidance on how to use PRO insights to drive clinically-meaningful patient-management strategies.
Abstract: To explore the existing evidence of the real-world implementation of patient-reported outcomes (PROs) in oncology clinical practice and address two aims: (1) summarize available evidence of PRO use in clinical practice using a framework based on the International Society for Quality of Life Research (ISOQOL) PRO Implementation Guide; and (2) describe reports of real-world, standardized PRO administration in oncology conducted outside of scope of a research study. A Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) protocol was developed to guide the systematic literature review (SLR) that was conducted in MEDLINE and Embase databases. A two step search strategy was implemented including two searches based on previously completed reviews. Studies published from 2006 to 2017 were synthesized using a framework based on the ISOQOL PRO Implementation Guide. After screening 4427 abstracts, 36 studies met the eligibility criteria. Most elements of the ISOQOL PRO Implementation Guide were followed. Two notable exceptions were found: 1) providing PRO score interpretation guidelines (39% of studies); and 2) providing patient-management guidance for addressing issues identified by PROs (25% of studies). Of the 22 studies with an intervention component, 19 (86%) reported intervention effects on study outcomes. The European Organisation for Research and Treatment of Cancer Quality-of-Life Questionnaire-Core 30 (EORTC QLQ-C30) was the most commonly used PRO (n = 10, 28%); use of 38 other PRO measures was also reported. Only three studies (8%) reported real-world PRO implementation. Reports of real-world PRO implementation are limited. Reports from studies conducted in clinical settings suggest gaps in information on PRO score interpretation and the use of PRO results to inform patient management. Before the promise of practice-based PRO assessment in oncology can be truly realized, investigators need to advance the state-of-the-art of real-time PRO score interpretation as well as developing guidance on how to use PRO insights to drive clinically-meaningful patient-management strategies.
53 citations
TL;DR: University of Utah Health is deploying system-wide standard and customized PRO collection with the goals of providing better patient care, improving physician-patient communication, and ultimately improving the value of the care given.
Abstract: Patient-reported outcomes (PROs) have traditionally been implemented through a manual process of paper and pencil with little standardization throughout a Healthcare System. Each practice has asked patients specific questions to understand the patient’s health as it pertains to their specialty. These data were rarely shared and there has not been a comparison of patient’s health across different specialty domains. We sought to leverage interoperable electronic systems to provide a standardization of PRO assessments across sites of care. University of Utah Health is comprised of four hospitals, 12 community clinics, over 400,000 unique annual patients, and more than 5000 providers. The enterprise wide implementation of PROs started in November of 2015. Patients can complete an assessment at home via email, or within the clinic on a tablet. Each specialty has the opportunity to add additional specialty-specific instruments. We customized the interval with which the patient answers the assessments based on specialty preference in order to minimize patient burden, while maximizing relevant data for clinicians. Barriers and facilitators were identified in three phases: Pre-implementation, Implementation, and Post-implementation. Each phase was further broken down into technical challenges, content inclusion and exclusion, and organizational strategy. These phases are unique and require collaboration between several groups throughout the organization with support from executive leadership. We are deploying system-wide standard and customized PRO collection with the goals of providing better patient care, improving physician-patient communication, and ultimately improving the value of the care given. Standardized assessment provides any clinician with information to quickly evaluate the overall, physical and mental health of a patient. This information is available real time to aid in patient communication for the clinician.
53 citations
TL;DR: Overall, the studies showed that NASH affects HRQoL, especially physical functioning, with many patients reporting being fatigued, and a range of symptoms, especially fatigue, and the impact on their lives is broad.
Abstract: Nonalcoholic steatohepatitis (NASH) is a form of chronic liver disease (CLD): patients have an increased risk of developing cirrhosis, liver failure, and complications (e.g. hepatocellular carcinoma). NASH has a high clinical burden, and likely impairs patients’ health-related quality of life (HRQoL), but there are currently no licensed therapies. The objective of this robust pragmatic literature review was to identify and describe recent studies on the HRQoL burden of NASH from the patient perspective. English-language primary research studies were identified that measured HRQoL in adults with NASH (population-based studies or clinical trials of pharmacological therapy). Searches were conducted in the following bibliographical databases: MEDLINE, EMBASE, Cochrane Database of Systematic Reviews (CDSR), Cochrane Central Register of Controlled Trials (CENTRAL), Database of Abstracts of Reviews of Effects (DARE), and Health Technology Assessment Database (HTA). Abstracts from selected congresses (2015/2016) were hand searched. Articles were assessed for relevance by two independent reviewers, and HRQoL data were extracted. A total of 567 de-duplicated abstracts were identified, and 20 full-text articles were reviewed. Eight studies were included: five quantitative, two interventional, and one qualitative. The quantitative and interventional studies measured HRQoL using the Short-Form 36 (SF-36) and the Chronic Liver Disease Questionnaire (CLDQ), and the qualitative study involved focus groups and individual interviews. Overall, the studies showed that NASH affects HRQoL, especially physical functioning, with many patients reporting being fatigued. In quantitative studies, overall, patients with NASH had a reduced HRQoL versus normative populations and nonalcoholic fatty liver disease (NAFLD) patients, but not versus chronic liver diseases. A longitudinal study showed that when weight loss was achieved, HRQoL improvement over 6 months was greater in patients with NASH versus NAFLD. Qualitative research suggested that, in addition to fatigue, other symptoms are also burdensome, having a broad negative impact on patients’ lives. The impact of pharmacological treatment on HRQoL was explored in only two included studies. HRQoL is impaired in patients with NASH. Patients experience a range of symptoms, especially fatigue, and the impact on their lives is broad. Further research is needed to understand the HRQoL burden of NASH (e.g. assessing NASH-specific impacts not captured by SF-36 and CLDQ) and the impact of future NASH therapies on HRQoL.
39 citations
TL;DR: The VRS, NRS, and VAS are acceptable response scale types in the development of PRO instruments and more empirical evidence needs to be generated.
Abstract: In the development of patient-reported outcome (PRO) instruments, little documentation is provided on the justification of response scale selection The selection of response scales is often based on the developers’ preferences or therapeutic area conventions The purpose of this literature review was to assemble evidence on the selection of response scale types, in PRO instruments The literature search was conducted in EMBASE, MEDLINE, and PsycINFO databases Secondary search was conducted on supplementary sources including reference lists of key articles, websites for major PRO-related working groups and consortia, and conference abstracts Evidence on the selection of verbal rating scale (VRS), numeric rating scale (NRS), and visual analogue scale (VAS) was collated based on pre-determined categories pertinent to the development of PRO instruments: reliability, validity, and responsiveness of PRO instruments, select therapeutic areas, and optimal number of response scale options A total of 6713 abstracts were reviewed; 186 full-text references included There was a lack of consensus in the literature on the justification for response scale type based on the reliability, validity, and responsiveness of a PRO instrument The type of response scale varied within the following therapeutic areas: asthma, cognition, depression, fatigue in rheumatoid arthritis, and oncology The optimal number of response options depends on the construct, but quantitative evidence suggests that a 5-point or 6-point VRS was more informative and discriminative than fewer response options The VRS, NRS, and VAS are acceptable response scale types in the development of PRO instruments The empirical evidence on selection of response scales was inconsistent and, therefore, more empirical evidence needs to be generated In the development of PRO instruments, it is important to consider the measurement properties and therapeutic area and provide justification for the selection of response scale type
TL;DR: Results suggest the Crohn’s Disease Patient-reported Outcomes Signs and Symptoms (CD-PRO/SS) is a reliable and valid measure of gastrointestinal symptom severity in CD patients.
Abstract: The clinical course of Crohn’s disease (CD) and the effect of its treatment are monitored through patient-reported signs and symptoms (SS n = 29), then refined through cognitive interviews of CD patients (n = 20). Measurement properties, including item-level statistics, scaling structure, reliability, and validity, were examined using secondary analyses of baseline and two-week clinical trial data of adults with moderate-to-severe CD (n = 238). Findings from qualitative interviews identified nine SS reproducibility (intraclass correlation coefficient > 0.80), and validity, with the last including moderate correlations with the Inflammatory Bowel Disease Questionnaire bowel symptom score and select items (ranging from r = 0.43–0.54). Scores distinguished patients categorized by patient global ratings of disease severity (p < 0.0001). Results suggest the CD-PRO/SS is a reliable and valid measure of gastrointestinal symptom severity in CD patients. Additional longitudinal data are needed to evaluate the ability of the CD-PRO/SS scores to detect responsiveness and inform the selection of responder definitions.
TL;DR: In this paper, the authors explored how patients with epilepsy experience the use of patient-reported outcome (PRO) measures to improve the quality and effectiveness of health care in three outpatient clinics in the Central Denmark Region.
Abstract: There is an increasing focus on the use of patient-reported outcome (PRO) measures to improve the quality and effectiveness of health care PRO-based follow-up is a new model of service delivery, where the patient’s PRO measures are used as the very basis for outpatient follow-up This study aimed to explore how patients with epilepsy experience the use of PRO-based follow-up in three outpatient clinics in the Central Denmark Region We also sought to explain how these experiences relate to self-management Interpretive description was the methodological approach We conducted in-depth individual interviews with 29 patients referred to PRO-based follow-up, each of whom had completed at least two PRO questionnaires Participants were sampled based on purposive and theoretical sampling PRO-based follow-up may support patients’ self-management by a) increasing awareness of psychosocial problems, b) improving communication, c) increasing understanding of symptoms, d) facilitating change in health behavior and e) strengthening autonomy Inhibitors for PRO measures as a means of self-management support were identified as a) feelings of rejection and disconnection, b) incomprehension of purpose of PRO-based follow-up, c) PRO measures being too standardized and negative and d) lack of confidence in own ability to assess PRO questionnaires The findings demonstrate broad variation in the influences of PRO measures on patient’s self-management in life with epilepsy Sense of ownership may explain this variation We suggest supplementary clinical initiatives in order to enhance the benefits from PRO-based follow-up, particularly on how patients are allocated to this health care service
TL;DR: Empirical cumulative distribution functions of change in FACT-Cog demonstrating possible MCTs showed that anchor change of none, minimally better and much better were well separated and can help in the design, analysis and interpretation of self-reported cognitive function in cancer patients and survivors.
Abstract: We estimated clinically important, group-level differences in self-reported cognitive function for the Functional Assessment of Cancer Therapy-Cognitive Function (FACT-Cog) instrument. We also investigated individual level change that could be considered meaningful for cancer survivors affected by cognitive impairment following chemotherapy, and that could be used for responder analyses. We used data from a multi-site randomized controlled trial in 242 participants that evaluated a web-based intervention for improving self-reported cognitive functioning in adult cancer survivors who reported cognitive impairment and who had adjuvant chemotherapy in the previous 6–60 months. We used anchor and distribution methods to estimate a range of clinically important differences (CIDs) and investigated meaningful change thresholds (MCTs) for the FACT-Cog and the Perceived Cognitive Impairments (PCI) subscale, post-intervention and at six-month follow-up with empirical cumulative distribution functions. Our primary anchor was the patient reported cognitive function subscale of the European Organization for Research and Treatment of Cancer Quality of Life-Cognitive Functioning Scale (EORTC-CF). Most participants were female (95%) breast cancer survivors (89%). Correlation of changes in the FACT-Cog and the EORTC-CF were 0.55 post-intervention and 0.61 at follow-up. Anchor-based CID estimates for the FACT-Cog using our primary anchor were 11.3 points (post) and 8.8 (follow-up), which corresponds to a standardized effect size of 0.49 and 0.38; 8.6% and 6.6% of the total scale’s range. Anchor-based CID estimates for the FACT-Cog PCI subscale were 7.4 (post) and 4.6 points (follow-up), which corresponds to a standardized effect size of 0.50 and 0.31; 10.3% and 6.4% of the PCI range). Empirical cumulative distribution functions of change in FACT-Cog demonstrating possible MCTs showed that anchor change of none, minimally better and much better were well separated. The CID and MCT estimates from this manuscript can help in the design, analysis and interpretation of self-reported cognitive function in cancer patients and survivors.
TL;DR: This paper presents emerging Good Practices for Translatability Assessment of Patient-Reported Outcome (PRO) Measures and how these can guide the future use of TA in the development of PROs.
Abstract: This paper presents emerging Good Practices for Translatability Assessment (TA) of Patient-Reported Outcome (PRO) Measures. The ISOQOL Translation and Cultural Adaptation Special Interest Group (TCA-SIG) undertook the review of several TA approaches, with the collaboration of organizations who are involved in conducting TA, and members of the TCA-SIG. The effort led to agreement by the writing group on Good Practices for 1) the terminology to be used in referring to translatability process, 2) the best definition of TA, 3) the methodology that is recommended at each step of the process, 4) the persons involved in TA, 5) the timing of assessment, 6) the review criteria for TA, and 7) the recommendations to be made at the end of the TA process. With input from the TCA-SIG membership and in consultation with experts in the field, these emerging good practices can guide the future use of TA in the development of PROs.
TL;DR: Results suggest that the UC-PRO/SS is a reliable and valid measure of gastrointestinal symptom severity in UC patients, and additional longitudinal data are needed to evaluate the ability of theUC- PRO/SS scores to detect responsiveness and inform the selection of responder definitions.
Abstract: The clinical course of ulcerative colitis (UC) and the effects of treatment are assessed through patient-reported signs and symptoms (SS reproducibility (intraclass correlation coefficient = 0.81, 0.71) and validity, including moderate-to-high correlations with the Partial Mayo Score (0.79; 0.45) and Inflammatory Bowel Disease Questionnaire (IBDQ) total score (− 0.70; − 0.61). Scores discriminated by level of disease severity, as defined by the Partial Mayo Score, Patient Global Rating, and Clinician Global Rating (p < 0.0001). Results suggest that the UC-PRO/SS is a reliable and valid measure of gastrointestinal symptom severity in UC patients. Additional longitudinal data are needed to evaluate the ability of the UC-PRO/SS scores to detect responsiveness and inform the selection of responder definitions.
TL;DR: The process of designing and integrating a QPSS in palliative care for patients with life-limiting conditions and their family caregivers is complex and requires extensive consultation with clinicians, administrators, patients, and family caregivers to inform successful implementation.
Abstract: Quality of life (QOL) assessment instruments, including patient-reported outcome measures (PROMs) and patient-reported experience measures (PREMs), are increasingly promoted as a means of enabling clinicians to enhance person-centered care. However, integration of these instruments into palliative care clinical practice has been inconsistent. This study focused on the design of an electronic Quality of Life and Practice Support System (QPSS) prototype and its initial use in palliative inpatient and home care settings. Our objectives were to ascertain desired features of a QPSS prototype and the experiences of clinicians, patients, and family caregivers in regard to the initial introduction of a QPSS in palliative care, interpreting them in context. We applied an integrated knowledge translation approach in two stages by engaging a total of 71 clinicians, 18 patients, and 17 family caregivers in palliative inpatient and home care settings. Data for Stage I were collected via 12 focus groups with clinicians to ascertain desirable features of a QPSS. Stage II involved 5 focus groups and 24 interviews with clinicians and 35 interviews with patients or family caregivers during initial implementation of a QPSS. The focus groups and interviews were recorded, transcribed, and analyzed using the qualitative methodology of interpretive description. Desirable features focused on hardware (lightweight, durable, and easy to disinfect), software (simple, user-friendly interface, multi-linguistic, integration with e-health systems), and choice of assessment instruments that would facilitate a holistic assessment. Although patient and family caregiver participants were predominantly enthusiastic, clinicians expressed a mixture of enthusiasm, receptivity, and concern regarding the use of a QPSS. The analyses revealed important contextual considerations, including: (a) logistical, technical, and aesthetic considerations regarding the QPSS as a technology, (b) diversity in knowledge, skills, and attitudes of clinicians, patients, and family caregivers regarding the integration of electronic QOL assessments in care, and (c) the need to understand organizational context and priorities in using QOL assessment data. The process of designing and integrating a QPSS in palliative care for patients with life-limiting conditions and their family caregivers is complex and requires extensive consultation with clinicians, administrators, patients, and family caregivers to inform successful implementation.
TL;DR: The International Consortium for Health Outcomes Measurement (ICHOM) Standard Set was developed to capture the outcomes of care for costly conditions including osteoarthritis in public and private hospital settings as mentioned in this paper.
Abstract: There is growing international momentum for standardising patient outcome assessment and using patient-reported outcome measures (PROMs) to capture outcomes that matter to patients. The International Consortium for Health Outcomes Measurement (ICHOM) Standard Sets were developed to capture the outcomes of care for costly conditions including osteoarthritis. This study evaluated the feasibility of implementing the ICHOM Standard Set for Hip and Knee Osteoarthritis in ‘real world’ public and private hospital settings. A mixed-methods design was used to capture comprehensive data on patient outcomes, implementation costs, and the implementation experiences of patients, clinicians and administrative staff. The ICHOM Standard Set was implemented at two hospital sites (1 public, 1 private) in May 2016. Patients undergoing primary hip or knee replacement for osteoarthritis were recruited from pre-admission clinics and a private orthopaedic clinic. Baseline Standard Set data were collected before surgery and at pre-determined post-operative timepoints. Data on the costs of Standard Set implementation were also collected. Semi-structured interviews were conducted with key stakeholders (n = 15) to evaluate the ease of implementation, and explore barriers and enablers to implementation and sustainability. The cost of Standard Set implementation and ongoing data collection for 17 months totalled $AUD94,955. Preference data (collected prior to completing the Standard Set) revealed that most participants preferred paper-based (83%) or web-based questionnaire completion (14%), with only a small proportion preferring iPad-based completion (3%). Several PROMs within the Standard Set were responsive to change (effect size range 0.19–0.85), with significant improvements in important health outcomes identified 6 weeks after surgery. Patient interviews showed a variable understanding of why patient-reported data collection is undertaken; however, patients perceived that PROMs provided relevant information to treating clinicians, and that the burden of questionnaire completion was minimal. Staff interviews revealed that PROMs are considered valuable, dedicated personnel are required to support data collection, gaps in information technology resources must be addressed, and that the Standard Set offers benefits beyond what currently-used measures provide. The Standard Set can be feasibly implemented in hospital settings, but with important caveats around staffing and technical support, consideration of patient preferences, and promotion of active clinician engagement.
TL;DR: The evidence supports the WPAI as having test-retest reliability (reproducibility) over time, convergent validity, and ability (sensitivity) to detect change, as indicated by substantial improvement in scores for patients who achieve remission, accompanied by substantial worsening of scores for Patients who relapse.
Abstract: Patients with ulcerative colitis, a type of inflammatory bowel disease, report negative impacts of disease symptoms on work-related outcomes, including absenteeism and presenteeism. As a way to better understand the impact of this disease and its treatment on work-related outcomes, the current review examines the use of the Work Productivity and Activity Impairment Questionnaire (WPAI), a patient-reported outcomes measure of absenteeism, presenteeism, and impairment in other activities, in studies of patients with ulcerative colitis. This review assesses the measurement properties of the WPAI in this patient population: its reliability, construct validity, ability to detect change, and responsiveness to effective treatments. Relevant data were extracted from 13 sources (journal articles and conference posters) identified following a systematic review of the published and gray literature. The evidence supports the WPAI as having test-retest reliability (reproducibility) over time; convergent validity, as indicated by moderate correlations with measures of quality of life and moderate-to-strong correlations with measures of disease activity; known-groups validity, as indicated by differences in WPAI scores between patients with active and inactive disease; ability (sensitivity) to detect change, as indicated by substantial improvement in scores for patients who achieve remission, accompanied by substantial worsening of scores for patients who relapse; and, responsiveness to treatment, with improvements in scores following treatments that reduce disease activity. Limitations included a lack of available evidence from randomized-controlled trials that could speak more directly to the WPAI’s responsiveness to treatment. In conclusion, we recommend the use of the WPAI for measuring work outcomes in both observational studies and interventional trials that include patients with ulcerative colitis.
TL;DR: The positive association between low comorbidity medication adherence and the number of unhealthy days suggests that addressing barriers toComorbidities medication adherence during cancer treatment may be an avenue for improving or maintaining HRQoL for older patients with cancer and comor bid conditions.
Abstract: Studies have demonstrated that comorbidities compound the adverse influence of cancer on health-related quality of life (HRQoL). Comorbidities adversely impact adherence to cancer treatment. Additionally, adherence to medications for comorbidities is positively associated with HRQoL for various diseases. This study used the Center for Disease Control and Prevention’s Healthy Days measure of HRQoL to explore the association between HRQoL and adherence to comorbidity medication for elderly patients with cancer and at least one comorbid condition. We conducted a cross-sectional survey combined with retrospective claims data. Patients with metastatic breast, lung or colorectal cancer were surveyed regarding their HRQoL, comorbidity medication adherence and cancer-related symptoms. Patients reported the number of physical, mental and total unhealthy days in the prior month. The Morisky Medication Adherence 8-point scale was differentiated into moderate/high (> 6) and low (≤ 6) comorbidity medication adherence. Of the 1847 respondents, the mean age was 69.2 years, most were female (66.8%) and the majority of the sample had Medicare coverage (88.2%). Low comorbidity medication adherence was associated with significantly more total, mental and physical unhealthy days. Low comorbidity medication adherence was associated with the presence of patient-reported cancer-related symptoms. Patients reporting low, as compared to moderate/high, comorbidity medication adherence had 23.4% more unhealthy days in adjusted analysis, P = 0.007. The positive association between low comorbidity medication adherence and the number of unhealthy days suggests that addressing barriers to comorbidity medication adherence during cancer treatment may be an avenue for improving or maintaining HRQoL for older patients with cancer and comorbid conditions.
TL;DR: For youth with JIA, HRQOL is multidimensional, reflecting disease as well as treatment factors, and should be assessed routinely to improve wellbeing.
Abstract: Children with Juvenile Idiopathic Arthritis (JIA) often have poor health-related quality of life (HRQOL) despite advances in treatment. Patient-centered research may shed light on how patient experiences of treatment and disease contribute to HRQOL, pinpointing directions for improving care and enhancing outcomes. Parent proxies of youth enrolled in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry shared patient-reported outcomes about their child’s HRQOL and experiences of disease and treatment burden (pain interference, morning stiffness, history of medication side effects and methotrexate intolerance). Contributions of these measures to HRQOL were estimated using generalized estimating equations accounting for site and patient demographics. Patients (N = 180) were 81.1% white non-Hispanic and 76.7% female. Mean age was 11.8 (SD = 3.6) years, mean disease duration was 7.7 years (SD = 3.5). Mean Total Pediatric Quality of Life was 76.7 (SD = 18.2). Mean pain interference score was 50.1 (SD = 11.1). Nearly one-in-five (17.8%) youth experienced >15 min of morning stiffness on a typical day, more than one quarter (26.7%) reported ≥1 serious medication side effect and among 90 methotrexate users, 42.2% met criteria for methotrexate intolerance. Measures of disease and treatment burden were independently negatively associated with HRQOL (all p-values <0.01). Negative associations among measures of treatment burden and HRQOL were attenuated after controlling for disease burden and clinical characteristics but remained significant. For youth with JIA, HRQOL is multidimensional, reflecting disease as well as treatment factors. Adverse treatment experiences undermine HRQOL even after accounting for disease symptoms and disease activity and should be assessed routinely to improve wellbeing.
TL;DR: The French version of the FCCHL provides a brief reliable and valid measure to explore the dimensions of health literacy and could be used by health professionals to screen for health literacy level in order to develop this skill and to tailor health communication.
Abstract: Health literacy is a key asset, defined as the capacity to acquire, understand and use information in ways which promote and maintain good health To assess the reliability and validity of the French translation of the Functional, Communicative and Critical Health Literacy (FCCHL) scale A cross-sectional survey using an online questionnaire was proposed to all members of Seintinelles association Exploratory and confirmatory factorial analyses were conducted Data from 2342 respondents (458% had cancer history) were analysed The FCCHL scale was well-accepted (missing value by item ≤07%) Factor analysis revealed an acceptable fit of three-factor model (comparative fit index = 0922, root mean square error of approximation = 0065 and standardized root mean square residual = 0052) The FCCHL showed satisfactory reliability (α = 077) and scalar invariance was reached for education and deprivation, but not for age Known group validity was verified as mean scale scores differed according to education, deprivation and age, as expected The French version of the FCCHL provides a brief reliable and valid measure to explore the dimensions of health literacy It could be used by health professionals to screen for health literacy level in order to develop this skill and to tailor health communication
TL;DR: The German IKDC-SKF is a reliable outcome measure with good hypotheses testing and responsiveness, but its MIC and structural/content validity need further analysis.
Abstract: To examine the measurement properties of the German International Knee Documentation Committee Subjective Knee Form (IKDC-SKF) in knee disorder patients. Three hundred twelve consecutive patients undergoing surgery for anterior cruciate ligament, meniscus and/or cartilage injuries completed the IKDC-SKF, Lysholm Score, Tegner Activity Scale, and Short Form-12 Health Survey before and 6 months post-surgery. IKDC-SKF measurement properties were calculated and patients were also asked to rate the relevance/comprehensibility of the questionnaire items. Reliability was good with high Cronbach’s alpha and intraclass correlation coefficients, and standard error of measurement values of 4.4 to 6.0. The smallest detectable change (SDC) ranged from 12.3 to 16.7 points. Validity was good with 90% of all hypotheses confirmed. Confirmatory factor analysis did not show adequate fitting indices within the model. Over half of the items were rated as essential, and all were well comprehended. The majority of hypotheses for responsiveness were confirmed. No floor and ceiling effects were observed. The area under the curve ranged from 0.82 to 0.89 and the minimal important difference was smaller than the SDC. The German IKDC-SKF is a reliable outcome measure with good hypotheses testing and responsiveness, but its MIC and structural/content validity need further analysis.
TL;DR: Informal caregivers, especially partners, are faced with providing care of greater intensity to elderly patients during the first six months after a hip fracture, and this study shows that a considerable group of informal caregivers for elderly patients with a hip fractures experienced relational, physical and mental health problems that stemmed from providing intensive informal care.
Abstract: Reforms in the Dutch healthcare system in combination with the aging of the population will lead to a strong increase in the demand for informal care in the Netherlands. A hip fracture is one of the most important causes of hospital admissions among frail elderly and informal caregivers experience stress that may have significantly negative impact on the caregivers’ Quality of Life. The purpose of the study was to determine the nature, intensity and the care-related Quality of Life (CarerQoL) of informal caregivers of elderly patients in the first six months after a hip fracture. In this cross-sectional study, were interviewed the primary informal caregivers of patients with a hip fracture about the informal care provided after one, three or six months following the injury. The CarerQoL of the informal caregivers was measured with the CarerQoL-7D instrument. In total, 123 primary informal caregivers were included. The CarerQoL-7D score was on average 83.7 (SD 15.0) after one, three and six months, and there were no major differences between the measurement time points. The average amount of informal care provided per patient per week was 39.5 during the first six months. Partners of patients with a hip fracture provided significantly more hours of informal care (β 34.0; 95% CI: 20.9 – 47.1). Female informal caregivers stated a significantly lower level of CarerQoL (β -7.8; 95% CI: -13.3 – -2.3). Female caregivers were 3.0 times more likely to experience relational problems (aOR 3.02; 95% CI 1.08-8.43). Caregivers provided care at 6 months were associated with physical health problems (aOR 2.54; 95% CI 1.05-6.14). Informal caregivers, especially partners, are faced with providing care of greater intensity to elderly patients during the first six months after a hip fracture. The CarerQoL was not associated with the intensity of the provided informal care. However, this study shows that a considerable group of informal caregivers for elderly patients with a hip fracture experienced relational, physical and mental health problems that stemmed from providing intensive informal care during the first six months.
TL;DR: The core-questionnaire Consequences of Screening was found to be relevant for men offered regular follow-up of an asymptomatic screening-detected abdominal aortic aneurysm and a new questionnaire called COS-AAA covers in two parts the psychosocial experience in abdominal aortal aneurYSm screening.
Abstract: In interview studies, men under surveillance for screening-detected abdominal aortic aneurysms have reported ambivalence towards this diagnosis: the knowledge was welcomed together with worries, feelings of anxiety and existential thoughts about life’s fragility and mortality due to the diagnosis. Previous surveys about health-related quality of life aspects among men under surveillance for screening-detected aneurysm have all used generic patient-reported outcomes. Therefore, the aim of this study was to extend the core-questionnaire Consequences of Screening for use in abdominal aortic aneurysm screening by testing for comprehension, content coverage, dimensionality, and reliability. In interviews, the suitability, content coverage, and relevance of the core-questionnaire Consequences of Screening were tested on men under surveillance for a screeningdetected abdominal aortic aneurysm. The results were thematically analysed to identify the key consequences of abnormal screening results. Item Response Theory and Classical Test Theory were used to analyse data. Dimensionality, differential item functioning, local response dependency and reliability were established by item analysis, examining the fit between item responses and Rasch models. The core-questionnaire Consequences of Screening was found to be relevant for men offered regular follow-up of an asymptomatic screening-detected abdominal aortic aneurysm. Fourteen themes especially relevant for men diagnosed with a screening-detected abdominal aortic aneurysm were extracted from the interviews: ‘Uncertainty about the result of the ultra sound examination’, ‘Change in body perception’, ‘Guilt’, ‘Fear and powerlessness’, ‘Negative experiences from the examination’, ‘Emotional reactions’, ‘Change in lifestyle’, ‘Better not knowing’, ‘Fear of rupture’, ‘Sexuality’, ‘Information’, ‘Stigmatised’, ‘Self-blame for smoking’, ‘Still regretful smoking’. Altogether, 55 new items were generated: 3 were single items and 13 were only relevant for former or current smokers. 51 of the 52 items belonging to a theme were confirmed to fit Rasch models measuring fourteen different constructs. No differential item functioning and only minor local dependency was revealed between some of the 51 items. The reliability and the dimensionality of a condition-specific measure with high content validity for men under surveillance for a screening-detected abdominal aortic aneurysm have been demonstrated. This new questionnaire called COS-AAA covers in two parts the psychosocial experience in abdominal aortic aneurysm screening.
TL;DR: Qualitative research yielded two draft questionnaires grounded in patient perceptions and ready for psychometric validation studies with larger samples of patients with type 2 diabetes, which are the first developed specifically to assess perceptions of non-insulin injection delivery systems.
Abstract: Previous research has examined patient perceptions of insulin injection devices. However, injectable medications other than insulin are increasingly used to treat type 2 diabetes, including GLP-1 receptor agonists. No patient-reported outcome (PRO) instruments have been developed taking into account the experiences of patients using newer injection devices, which are often different from devices used for insulin. Therefore, the purpose of this qualitative study was to develop two draft PRO instruments focusing on patients’ experiences with these newer injection devices (one instrument assessing perceptions of a single injection device, and another assessing preferences between two devices). Questionnaire development proceeded in six steps: literature review, interviews with six device experts, concept elicitation interviews with patients (N = 32), preliminary translatability assessment, cognitive interviews with patients (N = 20), and final translatability assessment. Literature review and expert interviews were conducted to inform a concept elicitation interview guide. In concept elicitation in the US, UK, and Germany, patients with type 2 diabetes reported a range of injection features that influenced their perceptions of non-insulin injection devices (e.g., requirements for preparation of the medication/device, issues related to the needle, ease-of-use, portability). Two draft “item pools” were developed based on the literature review, expert interviews, and concept elicitation results. In cognitive interviews, patients recommended minor revisions and indicated that the draft instruments were generally clear, comprehensible, and relevant to their experience with non-insulin injectable medication. The instruments were refined based on the cognitive interviews and translatability assessment, resulting in two questionnaires. The various steps of qualitative research support the content validity of these new PRO instruments, which are the first developed specifically to assess perceptions of non-insulin injection delivery systems. Despite some overlap with insulin-focused questionnaires, the new instruments are distinct from previous instruments (omitting content that would not be relevant to patients receiving non-insulin injectable treatment, while including content that is not included in the insulin focused instruments). This qualitative research yielded two draft questionnaires that are grounded in patient perceptions and ready for psychometric validation studies with larger samples of patients with type 2 diabetes.
TL;DR: This psychometric evaluation supports the reliability and validity of the Diabetes Injection Device - Experience Questionnaire, while providing initial information on the performance of the DID-PQ.
Abstract: Previous research has examined patient perceptions of insulin injection devices However, a range of injectable medications other than insulin are now used to treat type 2 diabetes No patient-reported outcome (PRO) instruments have been developed taking into account the perceptions of patients using newer injection devices, which are often different from those used in the past Therefore, the primary purpose of this study was to evaluate a new PRO instrument focusing on patients’ experiences with injection devices, including those used for newer treatments such as GLP-1 receptor agonists Patients with T2D treated with non-insulin injectable medications were recruited via advertisements and six clinical sites in the US All participants completed the draft Diabetes Injection Device - Experience Questionnaire (DID-EQ) and additional measures administered for validity assessment Participants who had experience with two non-insulin injection devices also completed the draft Diabetes Injection Device - Preference Questionnaire (DID-PQ) Analyses focused on item reduction (item performance, exploratory factor analysis), reliability, and validity One hundred fourty two patients (mean age = 630y; 563% female) participated Item reduction yielded a 10-item version of the DID-EQ, including a 7-item Device Characteristics subscale and three global items assessing satisfaction, ease of use, and convenience of the injection device The DID-EQ demonstrated good internal consistency reliability (Cronbach’s alpha of Device Characteristics subscale = 080) and 7-day test-retest reliability (ICCs: 092 for Device Characteristics subscale; 065 to 091 for the three global items) Construct validity was demonstrated via correlations with previously validated instruments (eg, correlations with the DTSQ treatment satisfaction subscale ranged from 056 to 060, all p < 00001; correlations with the TRIM-D Device ranged from 063 to 077, all p < 00001) Descriptive analyses of the DID-PQ were conducted with a subset of 27 participants who were able to use it to compare two devices This psychometric evaluation supports the reliability and validity of the DID-EQ, while providing initial information on the performance of the DID-PQ These brief questionnaires complement measures of treatment efficacy and provide a more thorough picture of patients’ experiences with non-insulin injectable treatments for type 2 diabetes
TL;DR: The ANMS GCSI-DD was easily understood, found to contain the most important symptoms for patients with idiopathic and diabetic gastroparesis, and no changes were recommended.
Abstract: The American Neurogastroenterology and Motility Society Gastroparesis Cardinal Symptom Index-Daily Diary (ANMS GCSI-DD) was developed to meet Food and Drug Administration (FDA) recommendations for patient-reported outcome (PRO) endpoints in gastroparesis studies, including therapeutic trials. The current version of the ANMS GCSI-DD contains five items pertaining to nausea, early satiety, post-prandial fullness, upper abdominal pain, and vomiting. The specific aims of this study were to determine if the appropriate symptoms are included in the ANMS GCSI-DD and to assess the content validity in patients with idiopathic (IG) and diabetic gastroparesis (DG). Patients diagnosed with IG or DG were recruited by five clinical sites in the United States for a cross-sectional, qualitative study involving one-on-one in-person concept elicitation and cognitive debriefing interviews. Concept elicitation included open-ended questions to elicit patients’ symptoms and impacts of gastroparesis, while cognitive debriefing was designed to assess the comprehensiveness of the ANMS GCSI-DD and clarity of the instructions, items, and response scales. The interviews were audio-recorded and transcribed. Transcripts were analyzed using a content analysis approach with ATLAS.ti. Of 25 patients interviewed, 15 (60%) had IG and 10 (40%) DG. Mean age of the sample was 42.3 years (range: 20–70 years), and most patients were female (n = 19, 76%) and white (n = 19, 76%). During concept elicitation, patients endorsed the following signs and symptoms as relevant and important to their condition: early satiety (n = 25, 100%), post-prandial fullness (n = 25, 100%), nausea (n = 22, 88%), upper abdominal pain (n = 18, 72%), vomiting (n = 15, 60%), and bloating (n = 11, 44%). Many patients (n = 20, 80%) experienced day-to-day symptom change. During cognitive debriefing, patients confirmed the ANMS GCSI-DD content was comprehensive and reflective of their gastroparesis experience. Patients could easily select a response option and describe how they arrived at their answers. Overall, patients found the instrument’s instructions, recall period, items, and response options clear and understandable. The ANMS GCSI-DD was easily understood, found to contain the most important symptoms for patients with IG and DG, and no changes were recommended. Results support the content validity of the ANMS GCSI-DD for clinical trials and clinical care among IG or DG patients.
TL;DR: It appears to be feasible to collect retrospective PROMs from patients admitted unexpectedly as emergencies for the two conditions studied, and this findings justify the case for a large, multi-site study that could explore unresolved concerns about selection bias, particularly those arising from the clinical characteristics of patients.
Abstract: Outcome of emergency admissions is usually limited to mortality with little attempt to capture the views of health status of survivors. This is because of the challenge of determining patient reported outcome measures (PROMs) for the period before their emergency admission. The aim was to assess the feasibility of collecting retrospective PROMs to capture the pre-admission health status of patients admitted as emergencies. Prospective study of two cohorts: patients undergoing primary coronary angioplasty for acute ST elevation myocardial infarction (STEMI) in five hospitals and emergency laparotomy (EL) for gastro-intestinal conditions in 11 hospitals. Three rates were calculated: proportion of patients eligible for inclusion; proportion of eligible patients invited to participate; proportion of invitees who participated. Staff views were thematically analysed to understand factors that affected recruitment. About 85% of patients were eligible of whom most were invited to participate (84% EL; 79% STEMI). The proportions of invitees agreeing to participate differed between STEMI (92%) and EL (72%), probably reflecting greater post-intervention morbidity in the latter. Variation between hospitals was observed in the proportion deemed eligible (EL 72–97%; STEMI 63–100%), proportion invited (EL 60–93%; STEMI 71–96%) and the proportion of invitees agreeing to participate (EL 55–92%; STEMI 67–100%). While this might reflect case-mix differences between hospitals, it suggests there is scope for less well performing hospitals to improve their recruitment processes. The extent to which this initial feasibility study was able to assess selection bias was limited to the age and sex of patients. There was no bias evident for EL patients but for STEMI, younger men were more likely to participate. It appears to be feasible to collect retrospective PROMs from patients admitted unexpectedly as emergencies for the two conditions studied. The relevance of these findings to other causes of emergency admissions needs to be established. In addition, these findings justify the case for a large, multi-site study that could explore unresolved concerns about selection bias, particularly those arising from the clinical characteristics of patients. It would also enable estimates of the extent of variation in PROMs between hospitals to determine the usefulness of using PROMs in emergency admissions.
Fred Hutchinson Cancer Research Center1, University of Utah2, Wayne State University3, National Institutes of Health4, Northwestern University5, Ohio State University6, Johns Hopkins University School of Medicine7, University of Washington8, University of California, Davis9, University of Southern California10
TL;DR: The SWOG S0421 PRO data showed little evidence of clinically meaningful differences by arm in either pain palliation or functional status, an important secondary endpoint of this large randomized trial.
Abstract: SWOG S0421 was a large randomized trial comparing docetaxel/prednisone plus placebo (DPP) to docetaxel/prednisone plus atrasentan over 12 cycles for patients with metastatic castration-resistant prostate cancer (mCRPC). The current report presents the PRO results for this trial, an important secondary endpoint. The trial specified two primary PRO endpoints. Palliation of worst pain was based on the Brief Pain Inventory (BPI), where a 2 point difference is defined as clinically meaningful. Improvement of functional status was based on the Functional Assessment of Cancer Therapy – Prostate Cancer Trial Outcome Index (FACT-P TOI); a 5-point difference has been defined as clinically meaningful. We compared rates by arm using chi-square tests. Longitudinal analyses using linear mixed models addressed changes by arm over time. Four-hundred eighty-nine patients on each arm were evaluable for PRO endpoint data. There were no differences by arm in clinically meaningful pain palliation (41.7% for DPP vs. 44.0% for DPA, p = .70) or functional status (24.2% for DPP vs. 28.7% for DPA, p = .13). Longitudinal comparisons indicated no differences over time by arm for BPI Worst Pain scores (0.13 points, p = .23). Patients on the DPA arm had improved functional status of 1.78 points on average, a statistically significant (p = .02) but not clinically meaningful difference. The SWOG S0421 PRO data showed little evidence of clinically meaningful differences by arm in either pain palliation or functional status.