Diagnostic and prognostic research最新文献

Background: Current clinical guidelines recommend the use of clinical prognostic models (CPMs) for therapeutic decision-making in sarcoma patients. However, the number and quality of developed and externally validated CPMs is unknown. Therefore, we aimed to describe and critically assess CPMs for sarcomas.

Methods: We performed a systematic review including all studies describing the development and/or external validation of a CPM for sarcomas. We searched the databases MEDLINE, EMBASE, Cochrane Central, and Scopus from inception until June 7th, 2022. The risk of bias was assessed using the prediction model risk of bias assessment tool (PROBAST).

Results: Seven thousand six hundred fifty-six records were screened, of which 145 studies were eventually included, developing 182 and externally validating 59 CPMs. The most frequently modeled type of sarcoma was osteosarcoma (43/182; 23.6%), and the most frequently predicted outcome was overall survival (81/182; 44.5%). The most used predictors were the patient's age (133/182; 73.1%) and tumor size (116/182; 63.7%). Univariable screening was used in 137 (75.3%) CPMs, and only 7 (3.9%) CPMs were developed using pre-specified predictors based on clinical knowledge or literature. The median c-statistic on the development dataset was 0.74 (interquartile range [IQR] 0.71, 0.78). Calibration was reported for 142 CPMs (142/182; 78.0%). The median c-statistic of external validations was 0.72 (IQR 0.68-0.75). Calibration was reported for 46 out of 59 (78.0%) externally validated CPMs. We found 169 out of 241 (70.1%) CPMs to be at high risk of bias, mostly due to the high risk of bias in the analysis domain.

Discussion: While various CPMs for sarcomas have been developed, the clinical utility of most of them is hindered by a high risk of bias and limited external validation. Future research should prioritise validating and updating existing well-developed CPMs over developing new ones to ensure reliable prognostic tools.

Trial registration: PROSPERO CRD42022335222.

Background: Many chronic conditions, such as epilepsy and asthma, are typified by recurrent events-repeated acute deterioration events of a similar type. Statistical models for these conditions often focus on evaluating the time to the first event. They therefore do not make use of data available on all events. Statistical models for recurrent events exist, but it is not clear how best to evaluate their performance. We compare the relative performance of statistical models for analysing recurrent events for epilepsy and asthma.

Methods: We studied two clinical exemplars of common and infrequent events: asthma exacerbations using the Optimum Patient Clinical Research Database, and epileptic seizures using data from the Standard versus New Antiepileptic Drug Study. In both cases, count-based models (negative binomial and zero-inflated negative binomial) and variants on the Cox model (Andersen-Gill and Prentice, Williams and Peterson) were used to assess the risk of recurrence (of exacerbations or seizures respectively). Performance of models was evaluated via numerical (root mean square prediction error, mean absolute prediction error, and prediction bias) and graphical (calibration plots and Bland-Altman plots) approaches.

Results: The performance of the prediction models for asthma and epilepsy recurrent events could be evaluated via the selected numerical and graphical measures. For both the asthma and epilepsy exemplars, the Prentice, Williams and Peterson model showed the closest agreement between predicted and observed outcomes.

Conclusion: Inappropriate models can lead to incorrect conclusions which disadvantage patients. Therefore, prediction models for outcomes associated with chronic conditions should include all repeated events. Such models can be evaluated via the promoted numerical and graphical approaches alongside modified calibration measures.

Background: As populations are aging, the number of older patients with multiple chronic diseases demanding complex care increases. Although clinical guidelines recommend care to be personalized accounting for life expectancy, there are no tools to estimate life expectancy among multimorbid patients. Our objective was therefore to develop and internally validate a life expectancy estimator specifically for older multimorbid adults.

Methods: We analyzed data from the OPERAM (OPtimising thERapy to prevent avoidable hospital admissions in multimorbid older people) study in Bern, Switzerland. Participants aged 70 years old or more with multimorbidity (3 or more chronic medical conditions) and polypharmacy (use of 5 drugs or more for > 30 days) were included. All-cause mortality was assessed during 3 years of follow-up. We built a 3-year mortality prognostic index and transformed this index into a life expectancy estimator. Mortality risk candidate predictors included demographic variables (age, sex), clinical characteristics (metastatic cancer, number of drugs, body mass index, weight loss), smoking, functional status variables (Barthel-Index, falls, nursing home residence), and hospitalization. We internally validated and optimism corrected the model using bootstrapping techniques. We transformed the mortality prognostic index into a life expectancy estimator using the Gompertz survival function.

Results: Eight hundred five participants were included in the analysis. During 3 years of follow-up, 292 participants (36%) died. Age, metastatic cancer, number of drugs, lower body mass index, weight loss, number of hospitalizations, and lower Barthel-Index (functional impairment) were selected as predictors in the final multivariable model. Our model showed moderate discrimination with an optimism-corrected C statistic of 0.70. The optimism-corrected calibration slope was 0.96. The Gompertz-predicted mean life expectancy in our sample was 5.4 years (standard deviation 3.5 years). Categorization into three life expectancy groups led to visually good separation in Kaplan-Meier curves. We also developed a web application that calculates an individual's life expectancy estimation.

Conclusion: A life expectancy estimator for multimorbid older adults based on an internally validated 3-year mortality risk index was developed. Further validation of the score among various populations of multimorbid patients is needed before its implementation into practice.

Trial registration: ClinicalTrials.gov NCT02986425. First submitted 21/10/2016. First posted 08/12/2016.

Background: Endometrial cancer (EC) is the most prevalent gynaecological cancer in the UK with a rising incidence. Various models exist to predict the risk of developing EC, for different settings and prediction timeframes. This systematic review aims to provide a summary of models and assess their characteristics and performance.

Methods: A systematic search of the MEDLINE and Embase (OVID) databases was used to identify risk prediction models related to EC and studies validating these models. Papers relating to predicting the risk of a future diagnosis of EC were selected for inclusion. Study characteristics, variables included in the model, methods used, and model performance, were extracted. The Prediction model Risk-of-Bias Assessment Tool was used to assess model quality.

Results: Twenty studies describing 19 models were included. Ten were designed for the general population and nine for high-risk populations. Three models were developed for premenopausal women and two for postmenopausal women. Logistic regression was the most used development method. Three models, all in the general population, had a low risk of bias and all models had high applicability. Most models had moderate (area under the receiver operating characteristic curve (AUC) 0.60-0.80) or high predictive ability (AUC > 0.80) with AUCs ranging from 0.56 to 0.92. Calibration was assessed for five models. Two of these, the Hippisley-Cox and Coupland QCancer models, had high predictive ability and were well calibrated; these models also received a low risk of bias rating.

Conclusions: Several models of moderate-high predictive ability exist for predicting the risk of EC, but study quality varies, with most models at high risk of bias. External validation of well-performing models in large, diverse cohorts is needed to assess their utility.

Registration: The protocol for this review is available on PROSPERO (CRD42022303085).

Background: Pressure injuries (PIs) place a substantial burden on healthcare systems worldwide. Risk stratification of those who are at risk of developing PIs allows preventive interventions to be focused on patients who are at the highest risk. The considerable number of risk assessment scales and prediction models available underscores the need for a thorough evaluation of their development, validation, and clinical utility. Our objectives were to identify and describe available risk prediction tools for PI occurrence, their content and the development and validation methods used.

Methods: The umbrella review was conducted according to Cochrane guidance. MEDLINE, Embase, CINAHL, EPISTEMONIKOS, Google Scholar, and reference lists were searched to identify relevant systematic reviews. The risk of bias was assessed using adapted AMSTAR-2 criteria. Results were described narratively. All included reviews contributed to building a comprehensive list of risk prediction tools.

Results: We identified 32 eligible systematic reviews only seven of which described the development and validation of risk prediction tools for PI. Nineteen reviews assessed the prognostic accuracy of the tools and 11 assessed clinical effectiveness. Of the seven reviews reporting model development and validation, six included only machine learning models. Two reviews included external validations of models, although only one review reported any details on external validation methods or results. This was also the only review to report measures of both discrimination and calibration. Five reviews presented measures of discrimination, such as the area under the curve (AUC), sensitivities, specificities, F1 scores, and G-means. For the four reviews that assessed the risk of bias assessment using the PROBAST tool, all models but one were found to be at high or unclear risk of bias.

Conclusions: Available tools do not meet current standards for the development or reporting of risk prediction models. The majority of tools have not been externally validated. Standardised and rigorous approaches to risk prediction model development and validation are needed.

Trial registration: The protocol was registered on the Open Science Framework ( https://osf.io/tepyk ).

Background: The initial theme of the PROGRESS framework for prognosis research is termed overall prognosis research. Its aim is to describe the most likely course of health conditions in the context of current care. These average group-level prognoses may be used to inform patients, health policies, trial designs, or further prognosis research. Acquired brain injury, such as stroke, traumatic brain injury or encephalopathy, is a major cause of disability and functional limitations, worldwide. Rehabilitation aims to maximize independent functioning and meaningful participation in society post-injury. While some observational studies can allow for an inference of the overall prognosis of the level of independent functioning, the context for the provision of rehabilitation is rarely described. The aim of this protocol is to provide a detailed account of the clinical context to aid the interpretation of our upcoming overall prognosis study.

Methods: The study will occur at a Danish post-acute inpatient rehabilitation facility providing specialised inpatient rehabilitation for individuals with moderate to severe acquired brain injury. Routinely collected electronic health data will be extracted from the healthcare provider's database and deterministically linked on an individual level to construct the study cohort. The study period spans from March 2011 to December 2022. Four outcomes will measure the level of functioning. Rehabilitation needs will also be described. Outcomes and rehabilitation needs will be described for the entire cohort, across rehabilitation complexity levels and stratified for relevant demographic and clinical parameters. Descriptive statistics will be used to estimate average prognoses for the level of functioning at discharge from post-acute rehabilitation. The patterns of missing data will be investigated.

Discussion: This protocol is intended to provide transparency in our upcoming study based on routinely collected clinical data. It will aid in the interpretation of the overall prognosis estimates within the context of our current clinical practice and the assessment of potential sources of bias independently.

Background: We evaluated the performance of prognostic models for predicting mortality or ICU admission in hospitalized patients with COVID-19 in the World Health Organization (WHO) Global Clinical Platform, a repository of individual-level clinical data of patients hospitalized with COVID-19, including in low- and middle-income countries (LMICs).

Methods: We identified eligible multivariable prognostic models for predicting overall mortality and ICU admission during hospital stay in patients with confirmed or suspected COVID-19 from a living review of COVID-19 prediction models. These models were evaluated using data contributed to the WHO Global Clinical Platform for COVID-19 from nine LMICs (Burkina Faso, Cameroon, Democratic Republic of Congo, Guinea, India, Niger, Nigeria, Zambia, and Zimbabwe). Model performance was assessed in terms of discrimination and calibration.

Results: Out of 144 eligible models, 140 were excluded due to a high risk of bias, predictors unavailable in LIMCs, or insufficient model description. Among 11,338 participants, the remaining models showed good discrimination for predicting in-hospital mortality (3 models), with areas under the curve (AUCs) ranging between 0.76 (95% CI 0.71-0.81) and 0.84 (95% CI 0.77-0.89). An AUC of 0.74 (95% CI 0.70-0.78) was found for predicting ICU admission risk (one model). All models showed signs of miscalibration and overfitting, with extensive heterogeneity between countries.

Conclusions: Among the available COVID-19 prognostic models, only a few could be validated on data collected from LMICs, mainly due to limited predictor availability. Despite their discriminative ability, selected models for mortality prediction or ICU admission showed varying and suboptimal calibration.