The COSMIN-RoB checklist includes three sections with a total of 10 boxes, which is used to evaluate risk of bias of studies on content validity, internal structure, and other measurement properties. COSMIN classifies reliability, measurement error, criteria validity, hypothesis testing for construct validity, and responsiveness as other measurement properties, which primarily focus on the quality of the (sub)scale as a whole, rather than on the item level. Among the five measurement properties, reliability, measurement error and criteria validity are the most widely used in the studies. Therefore, this paper aims to interpret COSMIN-RoB checklist with examples to guide researchers to evaluate the risk of bias of the studies on reliability, measurement error and criteria validity of PROMs.
Patient reported outcome measures (PROM) are widely used in clinical research and practice. To aid the interpretation of PROM, researchers have proposed the minimal important difference (MID), the smallest change or difference that patients perceive as important. However, the estimation methods of MID are numerous and inconsistent, which brings difficulties to selecting the optimal MID estimate to interpret PROM results. To address this issue, a research team from McMaster University in Canada has proposed an approach for selecting the optimal MID. This method includes three core steps: evaluating the credibility of MID estimates, assessing the consistency among credible MID estimates, and selecting the optimal value based on contextual factors. The credibility evaluation instrument for anchor-based MID examines five core criteria, including the data sources of PROM and anchor, the interpretability of anchor, the correlation between anchor and PROM, the precision of MID estimates, and the judgment of anchor thresholds. When there are multiple credible MID estimates, the optimal MID estimate is selected by evaluating the consistency among the estimates and considering contextual factors that affect the variability among the estimates, such as the type of intervention, follow-up time, and disease severity. In addition, the team provided recommendations to improve the reporting quality of MID studies. This article provides a detailed introduction and interpretation of these developments, aiming to enhance researchers' and clinicians' understanding and application of MID, thereby supporting clinical research and healthcare decision-making.
Esophageal carcinoma is a malignant tumor with high morbidity and mortality worldwide, and surgery is the main treatment currently. With the development of patient-centered care, the effect of surgery should not be limited to the improvement of the incidence of postoperative complications, mortality and other indicators. It is also important to provide experience related to disease and surgery from the perspective of patients. Therefore, more and more attention is paid to patient-reported outcomes by scholars. This paper will provide an overview of the international widely used, reliable and effective scales and researches about patient-reported outcomes in esophageal carcinoma.
With the transformation of modern medical models, patient-reported outcomes, clinician-reported outcomes, observer-reported outcomes, and performance outcomes have become internationally recognized clinical outcome assessment indicators, and scales have also become important evaluation tools, among which translation and cross-cultural adaptation are one of the important sources of scales. However, at present, there are fewer guidelines for scale translation in China. At present, domestic scale translation has not yet been unified and standardized in clinical reporting. Most translation reports provide readers with incomplete information, which affects the development of scale translation, and the methodology related to the translation of clinical outcome assessment scales still focuses on patient-reported outcome scales, which creates a gap in terms of the recommendations for the rest of the types of translations, a gap which leads to inconsistencies in the translation methodology and process. In this paper, we will develop specific translation methods and processes for each of the four current types of clinical outcome assessments by combining scale translation guidelines to support a standardized approach to translation, cross-cultural adaptation, and linguistic validation for use in standardizing the process of recommending translations of patient-reported outcome scales, clinical-reported outcome scales, observer-reported outcome scales, and behavioral outcome scales.
Objective To evaluate the reporting quality and influencing factors of patient-reported outcome (PRO) data in randomized controlled trials (RCTs) of lung cancer. Methods RCTs of lung cancer with PRO as either primary or secondary endpoints were searched from PubMed, EMbase, Medline, CNKI, Wanfang Data, and VIP databases between January 1, 2010 and April 20, 2024. Reporting quality of included RCTs were assessed based on the CONSORT-PRO extension. Descriptive statistics and bivariate regression analysis were used to describe the reporting quality and analyze the factors influencing the reporting quality. Results A total of 740 articles were retrieved. After screening, 53 eligible RCTs of lung cancer with 22 780 patients were included. The patients were mainly with non-small cell lung cancer (84.91%), with the median sample size of the included studies was 364.0 (160.5, 599.5) patients. The primary PRO tool used was the EORTC QLQ-C30 (60.38%). There were 52 (98.11%) studies whose PRO measured the domain of "symptom management of cough, dyspnea, fatigue, pain, etc.", and 45 (84.91%) studies measured "health-related quality of life". Multicenter studies accounted for 84.91%, and randomized non-blind trials accounted for 62.26%. PRO was used as the primary endpoint in 33.96% of the studies and as secondary endpoints in 66.04%. The reliability and validity of the PRO tools were explicitly mentioned in 11.32% and 7.55% of the studies, respectively. The average completeness of reporting according to the CONSORT-PRO guidelines was 60.00%, ranging from 25.00% to 93.00%. The main factors affecting the completeness of CONSORT-PRO reporting included sample size and publication year. For every increment in sample size, the completeness of reporting increased by 27.5% (SE=0.00, t=2.040, P=0.046). Additionally, studies published after 2018 had a 67.2% higher completeness of reporting compared to those published in or before 2018 (SE=17.8, t=–3.273, P=0.006). Conclusion The study reveals that the overall reporting quality of PRO in lung cancer RCTs is poor. Particularly, the reporting of PRO measures reliability and validity, PRO assumptions, applicability, and handling of missing data need further improvement. Future research should emphasize comprehensive adherence to the CONSORT-PRO guidelines.
Objective To analyze the changes of perioperative symptoms of lung cancer patients by using patient-reported outcomes at different time points. MethodsA total of 109 patients who underwent thoracoscopic lung cancer resection in the department of thoracic surgery of our hospital from March to April 2021 were selected, including 55 (50.46%) males and 54 (49.54%) females. The mean age was 55.19±12.12 years. The postoperative symptom scale for lung cancer patients was used to investigate the changes of symptoms before surgery, 1 day after surgery, the day of discharge, and 30 days after surgery. Results The mean hospital stay was 6.89±2.25 days. None of the patients reported any clinical symptoms related to lung cancer before surgery. The most prominent symptoms 1 day after surgery were pain (3.33±0.96 points), nausea (2.81±1.18 points), dizziness (2.00±0.85 points), fatigue (1.89±0.79 points) and shortness of breath (1.79±1.37 points). The patients with dizziness, nausea, fatigue and other symptoms gradually decreased, and the symptoms were relieved significantly (P<0.05). However, the symptoms of conscious pain, cough and shortness of breath lasted for a long time. At 30 days after surgery, 70.64%, 64.22% and 33.03% of patients felt pain, cough and shortness of breath, respectively, and the degree of cough was aggravated (P<0.001). Conclusion Pain, cough, dizziness, shortness of breath and fatigue are the core postoperative symptoms of lung cancer patients. Most postoperative adverse symptoms can be effectively controlled in a short period of time, but pain, cough and shortness of breath still present persistent characteristics, which deserve further study.
ObjectiveTo develop a symptom and function assessment scale for patients after Nuss procedure for pectus excavatum and to test its reliability and validity. MethodsFollowing the principles and procedures of patient-reported outcome (PRO) scale development stipulated by the U.S. Food and Drug Administration, an initial draft was formed through literature analysis, qualitative interviews, and Delphi expert consultation. The preliminary draft was used to conduct a pre-survey on patients who underwent Nuss procedure for pectus excavatum at Guangdong Provincial People's Hospital, and the reliability and validity of the scale were tested. ResultsA preliminary PRO-based symptom and function scale was constructed, covering two domains: symptoms and impact on daily functions. The symptom dimension includes six items: chest tightness, palpitations, pain, shortness of breath, foreign body sensation of the steel plate, and distress; while the impact on daily functions includes four items: difficulty in getting out of bed or lying down, raising arms, bending over, and standing or sitting for a long time. A total of 73 patients who underwent Nuss procedure for pectus excavatum were included in the questionnaire survey, with 70 valid questionnaires collected, including 64 males and 6 females, with 56 patients aged 12-17 years and 14 patients≥18 years. Through exploratory factor analysis, two common factors were extracted, with a cumulative variance contribution rate of 70%. The Cronbach's α coefficient of the scale is 0.917. ConclusionThe scale developed in this study has good reliability and validity, high reliability and stability, and can be used as an evaluation tool for the recovery status of patients after Nuss procedure for pectus excavatum.
The estimation of the minimal important difference (MID) in patient-reported outcomes (PRO) relies on various selection principles and statistical methods, resulting in varying degrees of credibility among studies. When applying these findings, it is crucial to consider their evaluation outcomes. In the context of widely accepted MID studies based on the anchoring method, the credibility of the MID of PRO is influenced by the selection of anchors and the statistical methods employed for estimation. Variations in the anchors utilized, differences in clinical trial designs, disparities in the characteristics of measurement subjects and environment, as well as the control of biases in studies, can all contribute to inconsistencies in the MID of PRO. In response to this, McMaster University in Canada has developed a credibility evaluation tool specifically for MID studies in PRO. The tool comprises five core items and four additional items. The five core items encompass an evaluation framework that assesses: (1) Is the patient or necessary proxy responding directly to both the PRO and the anchor? (2) Is the anchor easily understandable and relevant for patients or necessary proxy? (3)Has the anchor shown good correlation with the PRO? (4) Is the MID precise? (5) Does the threshold or difference between groups on the anchor used to estimate the MID reflect a small but important difference? The four additional items concerning transition-rated anchors assess: (1) Is the amount of elapsed time between baseline and follow-up measurement for MID estimation optimal? (2) Does the transition item have a satisfactory correlation with the PRO score at follow-up? (3) Does the transition item correlate with the PRO score at baseline? (4) Is the correlation of the transition item with the PRO change score appreciably greater than the correlation of the transition item with the PRO score at follow-up? Given the relative weights of each item in the tool are uncertain and environment-dependent, items are not scored; instead, an overall judgment is made using a qualitative rating approach. This article introduces the specific items of this tool and illustrates the evaluation process through a case study to improve its use in optimizing PRO results presentation and interpretation in clinical trials, reviews, assessments, and guidelines.
Telephone follow-up is one of the important ways to follow up patients. High-quality follow-up can benefit both doctors and patients. However, clinical research-related follow-up is often faced with problems such as time-consuming, laborious and poor patient compliance. The authors belong to a team that has been committed to the study of patient-reported outcomes for a long time. The team has carried out long-term follow-up of symptoms, daily function and postoperative complications of more than 1 000 patients after lung cancer surgery, and accumulated certain experience. In this paper, the experience of telephone follow-up was summarized and discussed with relevant literatures from the aspects of clarifying the purpose of clinical research follow-up, understanding the needs of patients in follow-up, and using follow-up skills.
Patient-reported outcome measures (PROM) measure attribute studies refer to studies conducted by investigators to validate the measurement attributes of PROM. The consensus-based standards for the selection of health measurement instruments (COSMIN), an international consensus standard for the selection of health measurement instruments, divides this attribute into three aspects: reliability, validity and responsiveness, and adds interpretability as an additional important feature for evaluating PROM. The purpose of this paper is to introduce the verification methods and principles of the three major measurement attributes in the COSMIN consensus, as well as the significance and direction of interpretability evaluation, and to provide international methodological experience and reference for the development of high-quality PROM psychometric attribute verification in China.