Special Issue: Patient-reported outcomes
Scale assessment for irritable bowel syndrome (IBS) has been widely implemented in clinical practice, how to choose an appropriate assessment tool is very important. In view of this, we used literature research methodology to search patient-reported outcomes and disease-specific health-related quality of life instruments for IBS, summarized their main contents and psychometric properties, then put forward recommendations on the selection of the instruments. A total of 37 commonly used instruments for IBS were retrieved, which have proven to have good reliability, validity and responsiveness, and can be better applied in clinical practice. We suggest medical professionals and researchers choose an assessment instrument for IBS according to the purpose and content of their research.
Patient-reported outcome (PRO) refers to any information about the patient's own health condition, functional status, and feelings about treatment that directly comes from the patient without interpretation by physicians, nurses or anyone else. Measuring outcomes from the patient perspective is conducive to improving the quality of medical services and treatment satisfaction. We summarized recent applications of PRO in many fields, such as drug review and approval, drug and medical equipment supervision, quality evaluation of medical services, health technology assessment, and comprehensive clinical evaluation of drugs, and reviewed the latest advances in research on PRO, such as the development of PRO instruments, the construction of a health state utility value set, the health measurement in children and adolescents, and the quality evaluation of PRO instruments. Then we put forward recommendations to further promote the studies and use of PRO in China: strengthening the development of PRO instruments applicable to China, valuing the use of PRO in clinical practice, enhancing the interdisciplinary research on PRO, broadening the profile of research on PRO, and intensifying relevant research.
Patient-reported outcome (PRO) can provide evidence for medical decision making, health care policy development, and also serve as symptom parameters for condition monitoring to provide timely care targeting personal needs. With the widespread applications of PRO in clinical research, related ethical issues have received deeply attention. Published in 2022, the Ethical Considerations for the Inclusion of Patient-Reported Outcomes in Clinical Research (the PRO Ethics Guidelines) , is an important consensus-based reference developed for addressing ethical considerations of PRO in clinical research. Addressing ethical issues in PRO can improve the quality of PRO data while minimizing the risk, burden, and harm to participants and protecting the rights of participants and researchers. We interpreted the guideline, aiming to provide a guide for colleagues addressing ethical issues of PRO, and to increase their attention to these issues.
Patients with diabetic foot undergoing endovascular therapy for lower extremity arterial disease exhibit poor outcomes and a high mortality rate. It is unclear whether malnutrition assessed by geriatric nutritional risk index (GNRI) is associated with clinical outcomes in these patients.
To investigate the association of baseline malnutrition assessed by GNRI and clinical outcomes of endovascular therapy for lower extremity arterial disease in patients with diabetic foot.
Ninety-five patients who were admitted in Department of Endocrinology, Sun Yat-sen Memorial Hospital due to diabetic foot and lower extremity arterial disease were included from January 2011 to December 2016. All of them received endovascular therapy and followed up for two years. Baseline GNRI was assessed, and divided into three levels: normal nutrition (43 cases) , mild malnutrition (31 cases) and moderate to severe malnutrition (21 cases) . Clinical characteristics were collected, including sex, age, diabetes duration, smoking, body mass index, prevalence of cardiovascular and cerebrovascular diseases, diabetic foot classification, systolic and diastolic blood pressure, and blood test results containing white blood cell count, platelet count, lymphocyte count, neutrophil count, neutrophil-to-lymphocyte ratio, hemoglobin, fasting blood glucose, glycosylated hemoglobin, total cholesterol, triglyceride, high-density lipoprotein cholesterol and low-density lipoprotein cholesterol, albumin, creatinine, and estimated glomerular filtration rate. The primary endpoint was all-cause mortality, and the secondary endpoint was major lower extremity amputation. The Kaplan-Meier curve was used for survival analysis. Univariate and multivariate Cox proportional hazards regression analysis were analyzed to evaluate the risk factors of all-cause mortality.
Fifty-two cases (54.7%) were assessed with malnutrition (GNRI≤98) . Normal nutrition, mild malnutrition, and moderate to severe malnutrition patients had significant differences in average body mass index, leukocyte count, neutrophil count, neutrophil-to-lymphocyte ratio, hemoglobin, triglyceride and albumin (P<0.05) . During the follow-up, 16 patients died, 10 of whom were due to cardiovascular or cerebrovascular diseases; five patients underwent major lower extremity amputation. The two-year survival rate was 92.1%, 75.6% and 50.1% in normal nutrition, mild malnutrition, and moderate to severe malnutrition patients, respectively, showing statistically significant difference (Log-rank test: χ2=10.812, P=0.004) . GNRI≤98〔HR=3.937, 95%CI (1.070, 13.942) , P=0.037〕 was an independent risk factor for all-cause mortality.
The two-year survival rate of patients in normal nutrition group (GNRI>98) was higher than that in malnutrition group (GNRI≤98) . Baseline GNRI-assessed malnutrition may be an independent risk factor for all-cause mortality in diabetic foot patients with lower extremity arterial disease treated by endovascular therapy, so assessing and improving the nutritional status may better improve the clinical outcome of these patients.
Increased bronchiectasis (BE) prevalence has imposed heavy economic burden on patients and their families, greatly affecting the quality of life of patients. With increased use of patient reported outcome (PRO) in bronchiectasis treatment assessment, researchers not only focus on statistical pre-and post-treatment differences (P<0.05) , but also pay attention to minimal clinically important difference (MCID) . Currently, there is still a lack of relevant data about MCID in BE-PRO assessment tools. We reviewed the latest research on the use of MCID in BE-PRO assessment tools, aiming at providing a quantitative basis for outcome assessment in BE treatment.
The prevalence of inflammatory bowel disease (IBD) increases annually in China. The integrated traditional Chinese and western medicine is helpful to alleviate and control symptoms of IBD. Quality of life (QoL) is an important index to evaluate the clinical efficacy of IBD. However, there are few studies about IBD patient-reported outcomes (PROs) based on TCM theories.
To determine the framework and items, and then use them to develop the IBD PROs scale using TCM theories.
Under the guidance of the TCM theory of "body and mind harmony, and man's adaptation to his total environment" , literature review, core-group discussion and expert consultation were carried out. The conceptual framework and item pool of the draft of an IBD PROs scale were developed, then the corresponding items were determined, forming the draft of the IBD PROs scale. Then the draft scale was pre-tested in IBD patients treated in Department of Spleen and Stomach Diseases, the First Affiliated Hospital of Guangzhou University of Chinese Medicine from January to June 2021. The dispersion tendency, Cronbach's α and correlation analysis were used for further item selection.
The final IBD PROs scale is composed of two domains: body and mind harmony (12 body-related items, and 9 mind-related items) and man's adaptation to his total environment (6 items) .
The IBD PROs scale developed based on theories of TCM could be used to evaluate the QoL of IBD patients. But further clinical research is still needed to verify its reliability and validity.
Research Status and Thinking of Minimal Clinically Important Difference in Patient-reported Outcome Assessment Tool for Allergic Rhinitis
As the patient-reported outcome (PRO) assessment tool has been increasingly used in the clinical efficacy evaluation of patients with allergic rhinitis (AR) , the judgment and interpretation of changes in measurement results of assessment tools have become increasingly important. For the judgment of clinical research results, not only the statistical differences, but also the minimum clinically important difference (MCID) must be paid attention to. This paper systematically sorts out the MCID of the AR-PRO assessment tool, in order to provide a reference for AR-related clinical diagnosis and treatment decision-making and the objectification of the PRO assessment tool.