Designing randomised controlled trials in rheumatology – What,why, and how

Randomised controlled trials (RCTs) have become the workhorse for evaluating efficacy of various healthcare interventions. The basic plan is to compare the outcome experience of two or more prognostically similar groups of patients with study intervention being the only difference between them. It involves key elements such as random allocation, concealment of allocation, and blinding to guard against design-related biases. This article explains these key features and the steps involved in designing an RCT in the context of rheumatological diseases.     

Performance of the Steno type 1 risk engine for cardiovascular disease prediction in Italian patients with type 1 diabetes

Background and aimsPremature cardiovascular disease cause excess mortality in type 1 diabetes (T1D). The Steno T1D Risk Engine was developed and validated in northern European countries but its validity in other populations is unknown. We evaluated the performance of the Steno T1D Risk Engine in Italian patients with T1D.Materials and methodsWe included patients with T1D with a baseline visit between July 2013 and April 2014, who were free of cardiovascular disease and had complete information to estimate risk. The estimated cardiovascular risk score was compared with the 5-year rate of cardiovascular events by means of logistic regression.ResultsAmong 223 patients (mean age 43 ± 13 years, 34.5% male, mean duration of diabetes 22 ± 12 years) the mean estimated cardiovascular risk at 5 years was 5.9% (95% C.I. 5.2–6.5%). At baseline, high estimated risk discriminated the presence of asymptomatic atherosclerosis better than microangiopathy, and was not associated with markers of inflammation or endothelial activation. After a mean follow-up of 4.7 ± 0.5 years, only 3 cardiovascular events were observed and nonetheless the risk score was significantly associated with their incidence (OR 1.22; 95% C.I. 1.08–1.39, p = 0.001). However, the observed event rate was significantly lower than the estimated one (3 vs 13; 95% C.I. 12–14; p < 0.001).ConclusionThe Steno T1D Risk Score identified subjects with subclinical atherosclerosis and high cardiovascular risk in an Italian T1D population. However, the absolute risk was significantly overestimated. Further studies in larger population are needed to confirm these results.     

Widening the participation gap: The effect of educational track on reported voting in England

This article explores the effect of tracked education in upper secondary on voting behaviour. It discusses two causal mechanisms that link tracked education to greater disparities of political participation: the curriculum and peer socialization. Data of Waves 1, 2, 5 and 7 of the Longitudinal Study of Young People in England (LSYPE) is used to assess the hypothesis that educational track has an independent effect on voting. Controlling for several pre- and post-track influences, the paper shows that students who have taken vocational courses in less prestigious schools indeed have lower reported voting levels at age 20 than those who have pursued an academic qualification (A levels) in prestigious schools. It is proposed that the effect of tracked education on political participation is likely to vary across Europe and that this variation may well be explained by differences across Europe in the extent to which the academic and vocational tracks are integrated, both in terms of the curriculum and in their social intake.     

Family History for Cardio-Metabolic Diseases: A Predictor of Major Adverse Cardiovascular Events in Men With Erectile Dysfunction

BackgroundFamily history (FH) of cardiovascular (CV) disease is a known CV risk factor. However, it is rarely considered for CV risk stratification. Furthermore, FH for metabolic diseases is generally overlooked.AimTo evaluate, in a population of men with erectile dysfunction (ED), whether FH for cardio-metabolic diseases could provide insights into metabolic and sexual features and predict the occurrence of forthcoming major adverse CV events (MACE).MethodsA consecutive series of 4,693 individuals (aged 51.3 ± 13.3 years) attending an Andrology outpatient clinic for ED was studied. A subset of these (n = 1,595) was evaluated retrospectively for MACE occurrence.OutcomesSeveral metabolic and sexual function–related parameters were studied. For the retrospective study, information on an incident MACE was collected over a mean follow-up of 4.2 ± 2.5 years.ResultsA greater number of cardio-metabolic FH factors were associated with a worse metabolic profile, including higher waist circumference, triglycerides, glucose, glycosylated hemoglobin, and diastolic blood pressure, as well as lower high-density lipoprotein cholesterol. An increased number of FH factors were associated with worse erectile function (odds ratio = 1.14[1.07;1.23], P < .0001), impaired penile dynamic peak systolic velocity, and lower testosterone levels. In the retrospective study, a positive cardiometabolic FH was associated with a significantly higher incidence of MACEs, even after adjusting for age and comorbidities (hazard ratio = 1.51[1.06-2.16], P = .023). Interestingly, when dividing the sample into high- and low-risk categories according to several CV risk factors (age, previous MACEs, high-density lipoprotein cholesterol, and comorbidities), FH was confirmed as a predictor of incident MACE only among the low-risk individuals.Clinical ImplicationsInvestigating FH for cardio-metabolic diseases is a quick and easy task that could help clinicians in identifying, among individuals with ED, those who deserve careful evaluation of CV and metabolic risk factors. Moreover, considering FH for CV risk stratification could predict MACEs in individuals who, according to conventional CV risk factors, would be erroneously considered at low risk.Strengths & LimitationsThe large sample size and the systematic collection of MACEs through an administrative database, with no risk of loss at follow-up, represent strengths. The use of administrative database for MACE collection may lead to some misclassifications. The specific population of the study limits the generalizability of the results.ConclusionFH is simple and inexpensive information that should be part of the CV risk assessment in all men with ED because it helps in the identification of those who need lifestyle and risk factor modifications and whose risk would otherwise be overlooked.Rastrelli G, Yannas D, Mucci B, et al. Family History for Cardio-Metabolic Diseases: A Predictor of Major Adverse Cardiovascular Events in Men With Erectile Dysfunction. J Sex Med 2020;17:2370–2381.     

Activation of Stratification Strategies and Results of the interventions on frail patients of Healthcare Services (ASSEHS) DG Sanco Project No. 2013 12 04

To face the challenge of active and healthy ageing (AHA), European Health Systems and services should move towards proactive, anticipatory and integrated care. Health care systems thus need to personalize services, put patients at the centre of care and provide services using the adequate resources. Population health risk management is emphasized through the use of tools to stratify people with chronic diseases according to their risk. Effective screening of frailty is vital for optimizing the care of frail populations at risk. The Activation of Stratification Strategies and Results of the interventions on frail patients of Healthcare Services (ASSEHS) EU project (N° 2013 12 04) is an international effort whose aim is to bring together stratification-related professionals from Health Services, Academia and Research in the EU in order to (i) study current existing health risk stratification strategies and tools, (ii) spread their use and application on frail elderly patients, (iii) minimize deterioration of conditions and/or (iv) prevent emergency or hospital admissions. The analysis of Risk Stratification in different Health Systems will generate conclusions and risk stratification solutions, which will be transferable to a variety of regions in the future. ASSEHS is in line with Area 4 of the B3 Action Plan of the European Innovation Partnership on Active and Healthy Ageing (EIP on AHA).     

Coronary artery calcifications and 6-month mortality in patients with COVID-19 without known atheromatous disease

Central illustration. Six-month mortality according to category of coronary artery calcium (CAC). The mortality rate increased with the magnitude of calcifications according to a visual scoring of CAC on chest computed tomography. CAC was associated with 6-month mortality, independent of conventional cardiovascular risk-factors, in patients hospitalized for coronavirus disease 2019 without known atheromatous disease. CI: confidence interval; HR: hazard ratio.

     

Genomic Markers in Prostate Cancer Decision Making

Context

Although the widespread use of prostate-specific antigen (PSA) has led to an early detection of prostate cancer (PCa) and a reduction of metastatic disease at diagnosis, PSA remains one of the most controversial biomarkers due to its limited specificity. As part of emerging efforts to improve both detection and management decision making, a number of new genomic tools have recently been developed.