BACKGROUND: Osteogenesis imperfecta (OI) is a group of inherited connective tissue disorders of varying severity characterized by bone fragility. The primary objective of this international multidisciplinary collaboration initiative was to reach a consensus for a standardized set of clinician and patient-reported outcome measures, as well as associated measuring instruments for dental care of individuals with OI, based on the aspects considered important by both experts and patients. This project is a subsequent to the Key4OI project initiated by the Care4BrittleBones foundation which aims to develop a standard set of outcome measures covering a large domain of factors affecting quality of life for people with OI. An international team of experts comprising orthodontists, pediatric dentists, oral and maxillofacial surgeons, and prosthetic dentists used a modified Delphi consensus process to select clinician-reported outcome measures (CROMs) and patient-reported outcome measures (PROMs) to evaluate oral health in individuals with OI. Important domains were identified through a literature review and by professional expertise (both CROMs and PROMs). In three focus groups of individuals with OI, important and relevant issues regarding dental health were identified. The input from the focus groups was used as the basis for the final set of outcome measures: the selected issues were attributed to relevant CROMs and, when appropriate, matched with validated questionnaires to establish the final PROMs which represented best the specific oral health-related concerns of individuals with OI.
RESULTS: Consensus was reached on selected CROMs and PROMs for a standard set of outcome measures and measuring instruments of oral health in individuals with OI.
CONCLUSIONS: Our project resulted in consensus statements for standardization oral health PROMs and CROMs in individuals with OI. This outcome set can improve the standard of care by incorporating recommendations of professionals involved in dental care of individuals with OI. Further, it can facilitate research and international research co-operation. In addition, the significant contribution of the focus groups highlights the relevance of dental and oral health-related problems of individuals with OI.

The aim of this network meta-analysis was to compare the efficacy of various commonly used drugs in treating patients with hypertrophic cardiomyopathy (HCM). Randomized controlled trials on drugs for HCM treatment were retrieved from PubMed, Embase, Cochrane Library, and Web of Science (search cutoff: January 10, 2024). Quality assessment was performed using the risk of bias tool, and data analysis used R software. Seventeen studies (1,133 patients with HCM) were included. The network meta-analysis indicated that mavacamten and perhexiline improved peak oxygen consumption compared with placebo. Mavacamten reduced N-terminal pro-B-type natriuretic peptide, left ventricular mass index, left atrial volume index, and septal E/e\' ratio. Losartan decreased systolic blood pressure, whereas candesartan, mavacamten, and valsartan reduced maximum wall thickness. Perhexiline had better efficacy in increasing peak oxygen consumption, and candesartan in reducing maximum wall thickness. No drug significantly improved left ventricular ejection fraction compared with placebo. In conclusion, on the basis of current studies, commonly used drugs may effectively improve some of the outcome measures in patients with HCM, whereas the novel drug mavacamten showed significant therapeutic effects in most of the remaining outcome measures except for left ventricular ejection fraction.

OBJECTIVE: To determine whether physical performance measures commonly used in clinical settings can discriminate fallers from nonfallers and predict falls in older adults with dementia.
METHODS: Systematic review and meta-analysis.
METHODS: Older adults with dementia residing in the community, hospitals, and residential care facilities.
METHODS: MEDLINE, Embase, PsycINFO, CINAHL, SPORTDiscus, the Cochrane Library, and the PEDro databases were searched from inception until December 27, 2023 (PROSPERO registration number: CRD42022303670). Retrospective or prospective studies that evaluated the associations between physical performance measures and falls in older adults with dementia were included. A random effects model was used to calculate the standardized mean difference (SMD) and 95% CI for each physical performance measure between fallers and nonfallers. Sensitivity analyses were conducted on the longitudinal studies to determine the ability of physical performance measures to predict future falls.
RESULTS: Twenty-eight studies were included in this review (n = 3542). The 5-time chair stand test [SMD = 0.23 (0.01, 0.45)], the Berg Balance Scale [SMD = -0.52 (-0.87, -0.17)], postural sway when standing on the floor [SMD = 0.25 (0.07, 0.43)] and on a foam surface [SMD = 0.45 (0.25, 0.66)], and the Short Physical Performance Battery total score [SMD = -0.46 (-0.66, -0.27)] could discriminate fallers from nonfallers. Sensitivity analyses showed that gait speed could predict future falls in longitudinal cohort studies [SMD = -0.29 (-0.49, -0.08)]. Subgroup analyses showed that gait speed [SMD = -0.21 (-0.38, -0.05)] and the Timed Up and Go test [SMD = 0.54 (0.16, 0.92)] could identify fallers staying in residential care facilities or hospitals.
CONCLUSIONS: The 5-time chair stand test, the Berg Balance Scale, postural sway when standing on the floor and a foam surface, and the Short Physical Performance Battery can be used to predict falls in older adults with dementia. Gait speed and the Timed Up and Go test can be used to predict falls in institutionalized older adults with dementia. Clinicians are recommended to use these physical performance measures to assess fall risk in older adults with dementia.

This study assesses the status of outcome measures in the randomized controlled trial(RCT) involving the kidney-tonif-ying and blood-activating method for treating knee osteoarthritis(KOA), aiming to establish a theoretical foundation for the development of a core set of outcome measures in traditional Chinese medicine(TCM) treatment of KOA. The relevant articles were retrieved from CNKI, Wanfang, VIP, SinoMed, PubMed, EMbase, Cochrane Library, and Web of Science, in addition to ClinicalTrials.gov and the China Clinical Trial Registration Center, with the time interval from inception to August 2023. The RCT of treating KOA with the kidney-tonifying and blood-activating method was included. Two assessors independently conducted literature screening, data collection, and qualitative analysis to compile the outcome measure results. A total of 350 RCTs were included, involving 165 outcome measures with the total frequency of 1 462. These outcome measures were categorized into six domains: symptom and sign measures(23) with the frequency of 718(49.1%), TCM symptom and syndrome measures(3) with the frequency of 53(3.6%), physical examination measures(130) with the frequency of 506(34.6%), quality of life measures(4) with the frequency of 20(1.3%), long-term efficacy measures(2) with the frequency of 6(0.4%), and safety measures(3) with the frequency of 159(10.9%). Additionally, 53 studies used TCM syndrome and symptom scores as indicators of efficacy, employing eight distinct measurement tools. The RCTs involving the kidney-tonifying and blood-activating method for treating KOA had a variety of problems, such as unclear prio-ritization of outcome measures, diversity in measurement tools, absence of standardized assessment criteria for specific measures, and non-standardized usage. These problems affected the research quality and reliability. Hence, it is advisable to draw upon international expertise, improve research design, and merge TCM efficacy characteristics with clinical research to establish a core set of KOA outcome measures aligned with TCM principles.

暂无摘要。

OBJECTIVE: A core outcome set (COS) is an agreed standardized set of outcomes that should be measured and reported, as a minimum, in specific areas of health or health care. A COS is developed through a consensus process to ensure health care outcomes to be measured are relevant to decision-makers, including patients and health-care professionals. Use of COS in guideline development is likely to increase the relevance of the guideline to those decision-makers. Previous work has looked at the uptake of COS in trials, systematic reviews, health technology assessments and regulatory guidance but to date there has been no evaluation of the use of COS in practice guideline development. The objective of this study was to investigate the representation of core outcomes in a set of international practice guidelines.
METHODS: We searched for clinical guidelines relevant to ten high-quality COS (with focus on the United Kingdom, Germany, China, India, Canada, Denmark, United States and World Health Organisation). We matched scope between COS and guideline in terms of condition, population and outcome. We calculated the proportion of guidelines mentioning or referencing COS and the proportion of COS domains specifically, or generally, matching to outcomes specified in each guideline populations, interventions, comparators and outcome (PICO) statement.
RESULTS: We found 38 guidelines that contained 170 PICO statements matching the scope of the ten COS and of sufficient quality to allow data extraction. None of the guidelines reviewed explicitly mentioned or referenced the relevant COS. The median (range) of the proportion of core outcomes covered either specifically or generally by the guideline PICO was 30% (0%-100%).
CONCLUSIONS: There is no evidence that COS are being used routinely to inform the guideline development process, and concordance between outcomes in published guidelines and those in COS is limited. Further work is warranted to explore barriers and facilitators in the use of COS when developing clinical guidelines.

Purpose: To evaluate the patient-reported outcomes of patients treated with commercially approved antibody-drug conjugates (ADC) reported in randomized controlled trials (RCT) published up to September 2023. Methods: A meta-analysis of 6430 patients from 12 randomized controlled trials was conducted. Results: No significant change was observed between the groups from baseline to end of treatment and end of follow-up, with a standardized mean difference of -0.08 (95% CI: -0.27-0.12) and 0.01 (95% CI: -0.11-0.12), respectively. Treatment with ADCs delayed the deterioration of patients\' clinical condition compared with treatment with non-ADCs, with a hazard ratio of 0.78 (95% CI: 0.67-0.92). Conclusion: ADCs have a good correlation with delay of clinical deterioration in patients with cancer.

Little evidence exists regarding an integrated multidimensional evaluation methodology to analyze the within-patient effects of medical treatment for chronic rhinosinusitis with nasal polyps (CRSwNP). We aimed to use an integrated evaluation model to analyze the effects of short-course oral corticosteroid (OCS) followed by intranasal corticosteroid spray (INCS) therapy in patients with severe CRSwNP.
In all, 32 patients with severe CRSwNP received oral methylprednisolone for three weeks followed by intranasal budesonide spray for nine weeks in this prospective single-arm study. An evaluation model integrating the concepts of the core outcome set (COS), clinical control and minimum clinically important difference (MCID) was longitudinally evaluated.
All uncontrolled patients at baseline showed similar progressive improvements from baseline and more than 1 MCID response across core outcomes during the OCS period, with severe CRSwNP being partly controlled in 31 (96.9%) patients and uncontrolled in 1 (3.1%) patient at 3 weeks. During the subsequent INCS period, 14 (43.8%) patients gradually deteriorated to an uncontrolled status at 12 weeks, whereas 18 (56.2%) exhibited partly controlled CRSwNP until 12 weeks.
In more than half of the patients, severe CRSwNP was partly controlled with the initial OCS followed by INCS therapy. An integrated evaluation model was used to facilitate the comprehensive evaluation of within-patient response, especially in patients with different responses to the same treatment.
ChiCTR1900024287.

UNASSIGNED: The Spinal Cord Independence Measure is a comprehensive functional rating scale for individuals with spinal cord lesion (SCL).
UNASSIGNED: To validate the scores of the three subscales of SCIM IV, the fourth version of SCIM, using advanced statistical methods.
UNASSIGNED: Multi-center cohort study.
UNASSIGNED: Nineteen SCL units in 11 countries.
UNASSIGNED: SCIM developers created SCIM IV following comments by experts, included more accurate definitions of scoring criteria in the SCIM IV form, and adjusted it to assess specific conditions or situations that the third version, SCIM III, does not address. Professional staff members assessed 648 SCL inpatients, using SCIM IV and SCIM III, at admission to rehabilitation, and at discharge. The authors examined the validity and reliability of SCIM IV subscale scores using Rasch analysis.
UNASSIGNED: The study included inpatients aged 16-87 years old. SCIM IV subscale scores fit the Rasch model. All item infit and most item outfit mean-square indices were below 1.4; statistically distinct strata of abilities were 2.6-6; most categories were properly ordered; item hierarchy was stable across most clinical subgroups and countries. In a few items, however, we found misfit or category threshold disordering. We found SCIM III and SCIM IV Rasch properties to be comparable.
UNASSIGNED: Rasch analysis suggests that the scores of each SCIM IV subscale are reliable and valid. This reinforces the justification for using SCIM IV in clinical practice and research.

BACKGROUND: Performance-based physical tests have been widely used as objective assessments for individuals with knee osteoarthritis (KOA), and the core set of tests recommended by the Osteoarthritis Research Society International (OARSI) aims to provide reliable, valid, feasible and standardized measures for clinical application. However, few studies have documented their validity in roentgenographically mild KOA. Our goal was to test the validity of five performance-based tests in symptomatic KOA patients with X-ray findings of Kellgren and Lawrence (K-L) grade 0-2.
METHODS: We recruited a convenience sample of thirty KOA patients from outpatient clinics and 30 age- and sex-matched asymptomatic controls from the community. They performed five OARSI-recommended physical tests and the KOA group answered the Western Ontario and McMaster Universities (WOMAC) Osteoarthritis Index. The tests included the 9-step stair-climbing test (9 s-SCT), timed up and go (TUG) test, 30-second chair-stand test (30sCST), 40-m fast walking-test (40MFPW) and 6-minute walking test (6MWT). The discriminant validity of these physical tests were assessed by comparisons between the KOA and control groups, receiver operating curve and multivariate logistic regression analysis. The convergent/divergent validity was assessed by correlation between the physical tests results and the three subscale scores of the WOMAC in the KOA group.
RESULTS: The KOA group had significantly worse performance than the control group. The percentage of difference was the largest in the 9 s-SCT (57.2%) and TUG tests (38.4%). Meanwhile, Cohen\'s d was above 1.2 for the TUG test and 6MWT (1.2 ~ 2.0), and between 0.8 and 1.2 for the other tests. The areas under the curve to discriminate the two groups were mostly excellent to outstanding, except for the 30sCST. Convergent validity was documented with a moderate correlation between the 9 s-SCT and the physical function (WOMAC-PF) subscale scores (Spearman\'s ρ = 0.60).
CONCLUSIONS: The OARSI recommended core set was generally highly discriminative between people with K-L grade 0-2 KOA and their controls, but convergent/divergent validity was observed only in the 9 s-SCT. Further studies are required to evaluate the responsiveness of these tests and understand the discordance of physical performance and self-reported measures.