Patient-reported outcome II: selecting appropriate measures for musculoskeletal care

The first article in this series outlined the important contribution that well developed patient reported outcome measures (PROMs) can make to healthcare, clinical decision making and evaluation. The increasing focus on patient reported health means that there are now hundreds of questionnaires available; selecting the appropriate measure is both challenging and essential. This article will introduce potential users of PROMs to key information to consider when selecting and using PROMs in research, routine practice or quality assurance. Important concepts, such as measurement reliability, validity and responsiveness to important change in health, will be explored and illustrated with examples from musculoskeletal healthcare. Practical properties, such as acceptability and feasibility, will also be introduced.     

PROMs in inflammatory arthritis: moving from static to dynamic

There are several advantages in using patient-reported outcome measures (PROMs) in standard clinical practice, particularly if a questionnaire is distributed to each patient at each visit as a standard in the infrastructure usual care. The patients, being the most knowledgeable persons concerning their pain and global estimate, do most of the work by completing a questionnaire. Completion of the questionnaire helps the patients prepare for their visit as well as improving doctor–patient communication. Recently, the role of PROMs has expanded from the static phase of capturing and measuring outcomes at a single point of time to a more dynamic role. This dynamic role is aiming at driving improvement not only in the quality of inflammatory arthritis care but also in the patients' reported experience. Therefore, in addition to its value in tailoring treatment targets adapted to the patient's needs, PROMs also have the potential of modifying the disease impact through improving the patients' adherence to therapy and allowing the patients to monitor the changes in their condition. Though more attention has been given to the use of PROMs in routine clinical care, little was published regarding what could be done with the plethora of data gained from PROMs and how dynamic it can be enhancing the “patient-centered care” approach and improving patients' experience. This article highlights the value of adopting PROMs for arthritic patients in standard clinical practice and its impact on long-term patients' management.     

Patient-reported outcome measures in inflammatory bowel disease

Patient-reported outcome measures (PROMs) are increasingly used in both research and clinical health settings. With the recent development of United States Food and Drug Administration guidance on PROMs, more attention is being devoted to their role and importance in health care. Several methodological challenges in the development, validation and implementation of PROMs must be resolved to ensure their appropriate utilization and interpretation. The present review discusses recent developments and updates in PROMs, with specific focus on the area of inflammatory bowel disease.     

It is time for an organised,scientific approach to the application of patient-reported outcome measures in clinical studies and trials

Clinical Rheumatology - Too often, clinicians and researchers assume that patient-reported outcome measures (PROMs) are simply designed, can be changed to suit specific circumstances and should be...     

A call for health systems to monitor the health-related quality of life of people living with HIV

Introduction

The World Health Organization's (WHO's) new global health strategy on HIV represents a major step toward a broader conceptualization of HIV care. It recognizes the importance of addressing chronic care more fully and–for the first time ever–the health-related quality of life (HRQoL) of people living with HIV (PLHIV).

Methods

A thorough literature review was conducted in order to analyse how the WHO strategy on HIV for 2022-2030 addresses the monitoring of the HRQoL of PLHIV for the next decade and compared it to that of other countries and health authorities.

Results

Unlike for other issues, the strategy does not include quantitative targets for 2030, thus falling short of committing to monitoring global progress in improving the long-term well-being of PLHIV.

Conclusions

We urge national health systems not to wait for WHO to lead on this issue. Seeking good HRQoL outcomes for PLHIV can confer far-reaching benefits on health systems. The feasibility of monitoring population-level HRQoL has been demonstrated through the use of simple tools like patient-reported outcome measures (PROMs) and patient-reported experience measures (PREMs). Many countries can already set HRQoL monitoring targets, similar to those presented in this viewpoint, while we work toward an agreed minimum metric for use by all countries.     

Saving time and effort: Best practice for adapting existing patient-reported outcome measures in hepatology

It is increasingly recognised that collecting patient reported outcome measures (PROMs) data is an important part of healthcare and should be considered alongside traditional clinical assessments. As part of a more holistic view of healthcare provision, there has been an increased drive to implement PROM collection as part of routine clinical care in hepatology. This drive has resulted in an increase in the number of PROMs currently developed to be used in various liver conditions. However, the development and validation of a new PROM is time-consuming and costly. Therefore, before deciding to develop a new PROM, researchers should consider identifying existing PROMs to assess their appropriateness and, if necessary, make adaptations to existing PROMs to ensure their rigour when used with the target population. Little is written in the literature on how to identify and adapt the existing PROMs in hepatology. This article aims to provide a summary of the current literature and guidance regarding identifying and adapting existing PROMs in clinical practice.     

Patient-reported outcomes in cystic fibrosis

Over the past 20 years, there has been tremendous progress in the area of patient-reported outcomes (PROs). A PRO instrument is defined as any measure of a patient's health status that is elicited directly from the patient and assesses how the patient "feels or functions with respect to his or her health condition." The advances seen in clinical research regarding PROs has been mirrored in research in cystic fibrosis (CF). A large number of instruments have been used for both therapeutic and nontherapeutic clinical research for many chronic conditions. This review will summarize a history of the development of PROs and how PROs are viewed by the U.S. Food and Drug Administration. We will then review the current state of the art of patient-reported outcomes in CF, specifically addressing the evaluation of different PRO instruments in terms of their reliability and validity. Finally, we will delineate further areas for development of PROs in CF. We believe that the future of CF research will incorporate a more diverse selection of PRO outcome measures; these outcome measures ultimately may be incorporated into clinical care to standardize symptom assessment and provide information regarding the need for specific clinical interventions to improve the quality of care delivered to these patients.     

Are all outcomes in chronic heart failure rated equally? An argument for a patient-centred approach to outcome assessment

Chronic heart failure (CHF) is a multi-dimensional and complex syndrome. Outcome measures are important for determining both the efficacy and quality of care and capturing the patient’s perspective in evaluating the outcomes of health care delivery. Capturing the patient’s perspective via patient-reported outcomes is increasingly important; however, including objective measures such as mortality would provide more complete account of outcomes important to patients. Currently, no single measure for CHF outcomes captures all dimensions of the quality of care from the patient’s perspective. To describe the role of outcome measures in CHF from the perspective of patients, a structured literature review was undertaken. This review discusses the concepts and methodological issues related to measurement of CHF outcomes. Outcome assessment at the level of the patient, provider and health care system were identified as being important. The perspectives of all stakeholders should be considered when developing an outcomes measurement suite to inform CHF health care. This paper recommends that choice of outcome measures should depend on their ability to provide a comprehensive, comparable, meaningful and accurate assessment that are important to patient.     

Patient‐reported outcome measures in advanced musculoskeletal physiotherapy practice: a systematic review

Objective

Advanced practice physiotherapists (APPs), also known as extended scope physiotherapists, provide a new model of service delivery for musculoskeletal (MSK) disorders. Research to date has largely focused on health service efficiencies, with less emphasis on patient outcomes. The present systematic review aimed to identify the patient‐reported outcome measures (PROMs) being utilized by APPs.

Method

A wide search strategy was employed, including the PubMed, Embase, CINAHL, CENTRAL and PEDro databases, to identify studies relating to PROMs utilized by APPs in MSK healthcare settings. PROMs identified were classified into predetermined outcome domains, with additional contextual data extracted.

Results

Of the initial 12,302 studies, 38 met the inclusion criteria. These involved APPs across different settings, utilizing 72 different PROMs and most commonly capturing: Patient Satisfaction, Quality of Life (QoL), Functional Status, and Pain; and, less frequently: Global Status (i.e. overall improvement), Psychological Well‐Being, Work ability, and Healthcare Consumption and Costs. The quality of the PROMs varied greatly, with Satisfaction most commonly measured utilizing non‐standardized locally‐devised tools; the EuroQol five‐dimensions questionnaire (EuroQoL‐5D) and 36‐Item Short‐Form (SF‐36) cited most frequently to capture QoL; and the Visual Analogue Scale (VAS) to capture Pain. No key measure was identified to capture Functional Status, with 15 different tools utilized.

Conclusion

APPs utilized a multiplicity of PROMs across a range of MSK disorders. The present review will act as an important resource, informing the selection of outcomes for MSK disorders, with a view to greater standardization of outcome measurement in MSK clinical practice, service evaluation and research.     

Patient-reported outcome measures (PROMs) in pancreatic cancer: a systematic review

BackgroundThe aim of this systematic review is to examine patient-reported outcome measures (PROMs), their attributes and application in patients with pancreatic cancer (PC).MethodA systematic literature search was undertaken of articles published to June 2018 to identify PROMs applied in primary studies in PC. Characteristics of the included studies and PROMs were described with identified scales grouped into five domains. The psychometric properties of the identified PROMs were further assessed for reliability and validity among patients with PC.ResultsFrom 1688 studies screened, 170 were included. Almost half (48%) were conducted in patients with unresectable PC; the majority of these (68%) were evaluated in randomized controlled trials. Median questionnaire completion rates fell below 10% of the original cohort within 12 months in patients with unresectable PC compared to 75% in patients with resectable PC. Seventy PROMs were identified, 32 measuring unidimensional parameters (e.g. pain) and 35 measuring multidimensional (e.g. quality of life) constructs. Only five (7%) PROMs were disease-specific and 13 (19%) were validated in patients with PC. Fifty scales were grouped into 19 physical, 9 psychological, 6 psychiatric, 9 social and 7 other domains.ConclusionThree multidimensional PROMs, the: (i) FACT-HEP in unresectable PC; (ii) QLQ-PAN26 (in conjunction with its core QLQ-C30 PROM) in resectable PC; and (iii) MDASI-GI are recommended as instruments to capture quality of life in patients with PC. Summarised scales and psychometric evaluation provide a framework to choose PROMs for scales not captured by the recommended PROMs.     

Protecting seriously ill populations during pragmatic clinical trials

Pragmatic clinical trials (PCTs) emphasize real-world effectiveness methodology to address the limitations of results from explanatory randomized clinical trials (RCTs), which often fail to translate to real-world medical practice. An inherent tension in the conduct of PCTs is that the research must impose a minimal burden on patients and health care institutions. PCTs prioritize outcome measures from existing data sources to minimize data collection burden; however, a lack of patient-reported outcomes may result in gaps in safety for vulnerable populations, such as those with serious illnesses. One proposed standard for judging the readiness of a study for a pragmatic trial is a ranking system that assigns PCTs a lower rank if they impose additional data collection burdens. However, this results in the wide use of measures of health care utilization and costs while patient experience measures, which could capture adverse unintended consequences, are omitted. In this article, we make the case for a risk-based approach to imposing additional data collection in PCTs to capture potential safety and patient experience outcomes, using examples from “real life” implemented interventions to improve end-of-life care through the Liverpool Pathway and through the implementation of Physician Orders for Life Sustaining Treatment (POLST) in Oregon.     

Update on the epidemiology,risk factors and disease outcomes of osteoarthritis

Osteoarthritis (OA) is the most frequent form of arthritis and a leading cause of pain and disability worldwide. OA can affect any synovial joint, although the hip, knee, hand, foot and spine are the most commonly affected sites. Knowledge about the occurrence and risk factors for OA is important to define the clinical and public health burden of the disease to understand mechanisms of disease occurrence and may also help to inform the development of population-wide prevention strategies. In this article, we review the occurrence and risk factors for OA and also consider patient-reported outcome measures that have been used for the assessment of the disease.     

Patient-Reported Outcomes and Risk of Hospitalization and Readmission in Patients with Inflammatory Bowel Diseases

Digestive Diseases and Sciences - Patient-reported outcome measures (PROMs) provide a wholesome view of patient well-being. We conducted a retrospective cohort study to evaluate whether PROMs...     

OMERACT 10 Patient Perspective Virtual Campus: valuing health; measuring outcomes in rheumatoid arthritis fatigue, RA sleep, arthroplasty, and systemic sclerosis; and clinical significance of changes in health

This workshop reviewed progress in a number of areas related to patient perspective outcomes that were not specifically included within other areas of the program. A substantial review of the work of the valuing health outcomes group (the "QALY" working group) with participation and feedback from the plenary audience resulted in guidance to refocus on the use of patient preferences in the elaboration of more robust outcome measures for patient-reported outcomes and life impact measures. Progress and developments in the areas of fatigue and sleep in rheumatoid arthritis, outcome measures in hip and knee arthroplasty clinical trials, and scleroderma were outlined, and the challenge of truly understanding the nature of clinically important improvement was reviewed.     

Choosing the right survey—patient reported outcomes in esophageal surgery

Patient reported outcomes (PROs) fulfill a crucial and unique niche in patient management, providing health-care providers a glimpse into their patients’ health experience. This is of utmost importance in patients with benign and malignant disorders of esophagus requiring surgery, which carries significant morbidity, in part due to a high burden of symptoms affecting health-related quality of life (HRQOL). There are a variety of generic and disease-specific patient reported outcome measures (PROMs) available for use in esophageal surgery. This article provides a broad overview of commonly used HRQOL instruments in esophageal surgery, including their utility in comparative effectiveness research, prognostication and shared decision-making for patients undergoing surgery for benign and malignant disorders of the esophagus.     

Patient-reported outcome measures after endoscopic retrograde cholangiopancreatography: a prospective,multicentre study

Abstract

Objective. While patient-reported outcome measures (PROMs) in ERCP are scarce, these reports are important for making improvements in quality of care. This study sought to document patient satisfaction and specifically pain related to endoscopic retrograde cholangiopancreatography (ERCP) procedures and to identify predictors for these experiences. Methods. From 2007 through 2009, prospective data from consecutive ERCP procedures at 11 hospitals during normal daily practice were recorded. Information regarding undesirable events that occurred during a 30-day follow-up period was also reported. The patient-reported pain, discomfort and general satisfaction with the ERCP were recorded. Results. Data from 2808 ERCP procedures were included in this study. Patient questionnaires were returned for 52.6% of the procedures. Moderate or severe pain was experienced in 15.5% and 14.0% of the procedures during the ERCP and in 10.8% and 7.7% of the procedures after the ERCP, respectively. In addition, female gender, endoscopic sphincterotomy (EST), and longer procedure times served as independent predictors of increased pain during the ERCP. The performing hospitals and sedation regimens were independent predictors of the procedural pain experience. In 90.9% of the procedures, the patients were satisfied with the information overall, and in 98.3% of the procedures, the patients were satisfied with the treatment provided. Independent predictors of dissatisfaction with the treatment included the occurrence of specific complications after ERCP and pain during or after the procedure. Conclusions. Female gender, the performance of EST and longer procedure times were independent predictors for increased procedure-related pain. The individual hospital and sedation regimen predicts the patient's pain experience.     

Insight into patient dissatisfaction with asthma treatment

BACKGROUND: Measures of patient satisfaction or dissatisfaction with treatment are increasingly being used as indicators of quality of care. As these measures become more widely used, it is important to know if patient dissatisfaction is associated with important processes or outcomes of medical care. METHODS: Survey of patient-reported asthma management issues using the Asthma Therapy Assessment Questionnaire in a large health maintenance organization in the Pacific Northwest. Associations between patient dissatisfaction with asthma treatment and patient-reported measures of asthma control, patient-provider communication, and belief in asthma medications (self-efficacy) were examined. RESULTS: Of the 5181 adult members with asthma enrolled in the health maintenance organization, 30% indicated dissatisfaction with current treatment. Dissatisfaction was higher among patients with a higher number of asthma control problems, patient-provider communication problems, or belief in medication problems (eg, failure to believe their medications are useful and inability to take asthma medications as directed). The odds of dissatisfaction with treatment were 2.8 (95% confidence interval [CI], 2.4-3.3; P<.001) for asthma control problems, 2.0 (95% CI, 1.6-2.6; P<.001) for communication problems, and 8.0 (95% CI, 6.7-9.5; P<.001) for belief in medication problems compared with patients without these perceived problems. CONCLUSION: Patient dissatisfaction with treatment may be related to important asthma disease management issues.     

Health outcomes of pediatric rheumatic diseases

Measurement of health outcomes in pediatric rheumatic diseases is a critical component of clinical practice and research studies. Measures should include the biological, physical, and psychosocial dimensions of health. Health outcome measures are developed systematically, often using consensus methods. Prior to implementation into practice, health outcome measures must undergo evaluation of measurement properties such as reliability, validity, and responsiveness. There are several health outcome measures available for juvenile idiopathic arthritis, juvenile systemic lupus erythematosus, and juvenile dermatomyositis, many of which are composite measures of disease activity. In addition, tools exist for measuring physical functioning and health-related quality of life. There is increasing focus on the incorporation of patient-reported or parent-reported outcomes when measuring the health state of patients with pediatric rheumatic diseases. Further work is required to determine the optimal health outcome measures and approach for eliciting the patient's perception of their health state in pediatric rheumatology.     

Improving cirrhosis care: The potential for telemedicine and mobile health technologies

Decompensated cirrhosis is a condition associated with significant morbidity and mortality. While there have been significant efforts to develop quality metrics that ensure high-value care of these patients, wide variations in clinical practice exist. In this opinion review, we discuss the quality gap in the care of patients with cirrhosis, including low levels of compliance with recommended cancer screening and other clinical outcome and patient-reported outcome measures. We posit that innovations in telemedicine and mobile health (mHealth) should play a key role in closing the quality gaps in liver disease management. We highlight interventions that have been performed to date in liver disease and heart failurefrom successful teleconsultation interventions in the care of veterans with cirrhosis to the use of telemonitoring to reduce hospital readmissions and decrease mortality rates in heart failure. Telemedicine and mHealth can effectively address unmet needs in the care of patients with cirrhosis by increasing preventative care, expanding outreach to rural communities, and increasing high-value care. We aim to highlight the benefits of investing in innovative solutions in telemedicine and mHealth to improve care for patients with cirrhosis and create downstream cost savings.