Measuring Patient-Reported Outcomes Adaptively: Multidimensionality Matters!
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ABSTRACT:
SUBMITTER: Paap MCS
PROVIDER: S-EPMC6009175 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:BackgroundA critical component that influences the measurement properties of a patient-reported outcome (PRO) instrument is the rating scale. Yet, there is a lack of general consensus regarding optimal rating scale format, including aspects of question structure, the number and the labels of response categories. This study aims to explore the characteristics of rating scales that function well and those that do not, and thereby develop guidelines for formulating rating scales.MethodsSeventeen existing PROs designed to measure vision-related quality of life dimensions were mailed for self-administration, in sets of 10, to patients who were on a waiting list for cataract extraction. These PROs included questions with ratings of difficulty, frequency, severity, and global ratings. Using Rasch analysis, performance of rating scales were assessed by examining hierarchical ordering (indicating categories are distinct from each other and follow a logical transition from lower to higher value), evenness (indicating relative utilization of categories), and range (indicating coverage of the attribute by the rating scale).ResultsThe rating scales with complicated question format, a large number of response categories, or unlabelled categories, tended to be dysfunctional. Rating scales with five or fewer response categories tended to be functional. Most of the rating scales measuring difficulty performed well. The rating scales measuring frequency and severity demonstrated hierarchical ordering but the categories lacked even utilization.ConclusionDevelopers of PRO instruments should use a simple question format, fewer (four to five) and labelled response categories.
| S-EPMC3503574 | biostudies-literature
Project description:Importance:Aesthetic eye treatments can dramatically change a person's appearance, but outcomes are rarely measured from the patient perspective. The patient perspective could be measured using an eye-specific patient-reported outcome measure. Objective:To describe the development and psychometric evaluation of FACE-Q scales and an adverse effect checklist designed to measure outcomes following cosmetic eye treatments. Design, Setting, and Participants:Pretreatment and posttreatment patients 18 years and older who had undergone facial aesthetic procedures were recruited from plastic surgery clinics in United States and Canada and completed FACE-Q scales between June 6, 2010, and July 14, 2014. We used Rasch Measurement Theory, a modern psychometric approach, to refine the scales and to examine psychometric properties. Main Outcomes and Measures:The FACE-Q Eye Module, which has 4 scales that measure appearance of the eyes, upper and lower eyelids, and eyelashes. Scale scores range from 0 (worst) to 100 (best). The module also includes a checklist measuring postblepharoplasty adverse effects. Results:Overall, 233 patients (81% response rate) 18 years and older participated. Adverse effects included being bothered by eyelid scars, dry eyes, and eye irritation. In Rasch Measurement Theory analysis, each scale's items had ordered thresholds and good item fit. Person Separation Index and Cronbach ? were greater than or equal to 0.83. Higher scores on the eye scales correlated with fewer adverse effects (range, -0.26 to -0.36). In the pretreatment group, older age correlated with lower scores (range, -0.42 to -0.51) on the scales measure appearance of the eyes and upper and lower eyelids. Compared with the pretreatment group, posttreatment participants reported significantly better scores on the scales measuring appearance of eyes overall, as well as upper and lower eyelids. Conclusions and Relevance:The FACE-Q Eye Module can be used in clinical practice, research and quality improvement to collect evidence-based outcomes data. Level of Evidence:NA.
| S-EPMC5247311 | biostudies-literature
Project description:BACKGROUND:The patient's perspective of their facial scar after skin cancer surgery influences perception of care and quality of life (QoL). Appearance satisfaction after surgery is also an important but often overlooked treatment outcome. OBJECTIVES:To report the psychometric validation of the FACE-Q Skin Cancer Module consisting of five scales, measuring appearance satisfaction (Satisfaction with Facial Appearance, Appraisal of Scars), QoL (Cancer Worry, Appearance-related Psychosocial Distress) and the patient experience (Satisfaction with Information: Appearance). METHODS:Participants underwent Mohs surgery for facial basal or squamous cell carcinoma or excision of early facial melanoma. Cohort 1 received a set of scales before and after surgery. Cohort 2 received the scales on two occasions in the postoperative period for test-retest reliability. Rasch measurement theory was used to select (item-reduce) the most clinically meaningful items for the scales. Reliability, validity, floor and ceiling effects and responsiveness were also analysed. RESULTS:Of 334 patients, 209 (response rate 62·6%) were included. Rasch analysis reduced the total scale items from 77 to 41. All items had ordered thresholds and good psychometric fit. Reliability was high (Person separation index and Cronbach's ? ? 0·90) and scales measuring similar constructs were correlated. High floor and ceiling effects were seen for the scales. The Cancer Worry scale demonstrated responsiveness (P = 0·004). CONCLUSIONS:The FACE-Q Skin Cancer Module meet the requirements of the Rasch model providing linearized measurement. Discriminating between patients with minimal appearance or worry impairment may be a limitation. The scales can be used for larger validation studies, clinical practice and research.
| S-EPMC6115303 | biostudies-literature
Project description:Background:Multiple Sclerosis (MS) clinical trials increasingly focus on progressive and advanced MS, with upper limb function (ULF) as a key outcome. Within clinical trials, Patient Reported Outcomes (PROs) quantify clinical variables and establish meaningfulness of changes. Scientific standards and regulatory criteria (from Food and Drug Administration [FDA]) require PROs be "fit-for-purpose": well-defined and reliable measures of specific concepts in defined contexts. Objective:To identify, from literature, existing PROs measuring ULF and determine which satisfy scientific and regulatory clinical trials requirements. Method:We screened PubMed/Web of Science using multiple relevant terms. Abstracts and full texts were screened using suitability criteria. PRO development papers were evaluated using recently expanded Consensus Standards for Measurement Instruments (COSMIN) criteria for content development. Results:We identified 3619 articles; 485 used 24 different ULF PROs. No PRO satisfied scientific and regulatory requirements as a well-defined measure of a clearly defined construct in a specific clinical context. Conclusions:Existing ULF PROs don't meet fit-for-purpose criteria. MS clinical trials require new measures with greater emphasis on patient engagement to derive theoretical frameworks, concepts of interest, and contexts of use followed by systematic literature searches, expert input, and qualitative research to support item generation. Until then, trials will miss aspects of meaningful within-patient change and thereby misrepresent (likely underestimating) treatment effects.
| S-EPMC7125352 | biostudies-literature
Project description:ObjectivesMeasuring patient-reported outcomes (PROs) has become increasingly important for assessing quality of care and guiding patient management. However, PROs have yet to be integrated with traditional clinical outcomes (such as length of hospital stay), to evaluate perioperative care. This study aimed to use longitudinal PRO assessments to define the postoperative symptom recovery trajectory in patients undergoing thoracic surgery for lung cancer.MethodsNewly diagnosed patients (N = 60) with stage I or II non-small cell lung cancer who underwent either standard open thoracotomy or video-assisted thoracoscopic surgery lobectomy reported multiple symptoms from before surgery to 3 months after surgery, using the MD Anderson Symptom Inventory. We conducted Kaplan-Meier analyses to determine when symptoms returned to presurgical levels and to mild-severity levels during recovery.ResultsThe most-severe postoperative symptoms were fatigue, pain, shortness of breath, disturbed sleep, and drowsiness. The median time to return to mild symptom severity for these 5 symptoms was shorter than the time to return to baseline severity, with fatigue taking longer. Recovery from pain occurred more quickly for patients who underwent lobectomy versus thoracotomy (8 vs 18 days, respectively; P = .022). Patients who had poor preoperative performance status or comorbidities reported higher postoperative pain (all P < .05).ConclusionsAssessing symptoms from the patient's perspective throughout the postoperative recovery period is an effective strategy for evaluating perioperative care. This study demonstrates that the MD Anderson Symptom Inventory is a sensitive tool for detecting symptomatic recovery, with an expected relationship among surgery type, preoperative performance status, and comorbid conditions.
| S-EPMC4554973 | biostudies-literature
Project description:Incorporation of the patient perspective into research and clinical practice will enrich our understanding of the status and management of patients with glomerular disease and may result in therapies that better address patient needs. In recent years, the importance of the patient experience of glomerular disease has become clear, and significant efforts have been undertaken to systematically capture and describe the patient's disease experience. Patient-reported outcome instruments provide a means to assess the patient's experience in a quantitative manner, thus enabling for comparisons within and between patients. Patient-reported outcome assessments are solely on the basis of a patient report about the status of their health without amendment or interpretation by a clinician or others. Patient-reported outcome assessments provide an opportunity to incorporate the patient perspective into clinical care, research, and clinical trials. Our paper provides an overview of terminology and development methods for patient-reported outcomes and reviews (1) currently available patient-reported outcome instruments appropriate for use in glomerular disease, (2) existing patient-reported outcome data in glomerular disease, and (3) opportunities for incorporating patient-reported outcome instruments into clinical care and research.
| S-EPMC5220669 | biostudies-other
Project description:Ensuring that the patient's voice is routinely incorporated in all aspects of health care in oncology is essential to provide quality care. Patient reported outcomes (PROs) are standardized measures that are used to obtain the patient's perspective and are increasingly used in all aspects of health care to ensure optimal delivery of patient-centered care. The US Food and Drug Administration encourages that PROs be used in studies for label indications. There are no uniform standardized methods to use PROs nor is there consensus on which PROs are best for regulatory approval, comparative effectiveness research, toxicity assessment, health-related quality of life, or symptom monitoring. For this review, we conducted a literature search using PubMed and Google Scholar, and herein summarize the evidence related to the use of PROs in clinic care and research. Using valid, reliable, and easily interpretable PROs developed in comparable populations will provide the most useful results. Various ways that PROs can be used successfully in oncology have been exemplified in this overview to provide clinicians and researchers practical guidance.
| S-EPMC6832764 | biostudies-literature
Project description:Patient-reported outcomes (PROs) are reports of a person's health status that provide a global perspective of patient well-being. PROs can be classified into 4 primary domains: global, mental, physical, and social health. In this descriptive review, we focus on how PROs can be used in cardiac clinical trials, with an emphasis on cardiac surgical trials for patients with coronary heart disease and heart failure. We also highlight ongoing challenges and provide specific suggestions and novel opportunities to advance cardiac clinical trials. Current challenges include the long-term measurement of PROs in clinical trials beyond 1 year, inconsistency in the choice of the outcome measures among studies, and the lack of measurement of PROs across multiple domains. Opportunities for advancement include measuring PROs using consumer health informatics tools, including returning information back to participants in formats that they can understand using visualization. Future opportunities include quantifying cohort-specific minimal clinically important differences for PROs.
| S-EPMC8487900 | biostudies-literature
Project description: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.
| S-EPMC2647603 | biostudies-literature
Project description:Patient-reported outcomes (PROs) include questionnaires or surveys that ask patients for their perceptions about things like symptoms they are experiencing or quality of life. For incurable, morbid, life-shortening conditions like idiopathic pulmonary fibrosis (IPF), PROs are particularly germane: They elucidate for clinicians and researchers what it is like for patients to live with such a disease, and they may detect important treatment effects not captured by other metrics (eg, pulmonary physiology). However, a relative paucity of research on PROs in IPF has left significant knowledge gaps in this area and contributed to the timidity investigators have about using PROs as prominent outcomes in IPF drug trials. Additional research on existing instruments is needed to establish or bolster their basic psychometric properties in IPF. When PROs are used as end points in therapeutic trials, analyzing PRO response data can be challenging, but these challenges can be overcome with a transparent, thoughtful, and sophisticated statistical approach. In this article, we discuss some of the basics of PRO assessment, existing knowledge gaps in IPF-related PRO research, and the potential usefulness of using PROs in IPF trials and conclude by offering specific recommendations for an approach to analyzing repeated-measures PRO data from IPF trials.
| S-EPMC3425334 | biostudies-other