IMPORTANCE Strategies for reliable selection of high-risk patients with hypertrophic cardiomyopathy (HCM) for prevention of sudden cardiac death (SCD) with implantable cardioverter/defibrillators (ICDs) are incompletely resolved. OBJECTIVE To assess the reliability of SCD prediction methods leading to prophylactic ICD recommendations to reduce the number of SCDs occurring in patients with HCM. DESIGN, SETTING, AND PARTICIPANTS In this observational longitudinal study, 2094 predominantly adult patients with HCM consecutively evaluated over 17 years in a large HCM clinical center were studied. All patients underwent prospective ICD decision making relying on individual major risk markers derived from the HCM literature and an enhanced American College of Cardiology/American Heart Association (ACC/AHA) guidelines-based risk factor algorithm with complete clinical outcome follow-up. Data were collected from June 2017 to February 2018, and data were analyzed from February to July 2018. MAIN OUTCOMES AND MEASURES Arrhythmic SCD or appropriate ICD intervention for ventricular tachycardia or ventricular fibrillation. RESULTS Of the 2094 study patients, 1313 (62.7%) were male, and the mean (SD) age was 51 (17) years. Of 527 patients with primary prevention ICDs implanted based on 1 or more major risk markers, 82 (15.6%) experienced device therapy-terminated ventricular tachycardia or ventricular fibrillation episodes, which exceeded the 5 HCM-related SCDs occurring among 1567 patients without ICDs (0.3%), including 2 who declined device therapy, by 49-fold (95% CI, 20-119; P = .001). Cumulative 5-year probability of an appropriate ICD intervention was 10.5% (95% CI, 8.0-13.5). The enhanced ACC/AHA clinical risk factor strategy was highly sensitive for predicting SCD events (range, 87%-95%) but less specific for identifying patients without SCD events (78%). The C statistic calculated for enhanced ACC/AHA guidelines was 0.81 (95% CI, 0.77-0.85), demonstrating good discrimination between patients who did or did not experience an SCD event. Compared with enhanced ACC/AHA risk factors, the European Society of Cardiology risk score retrospectively applied to the study patients was much less sensitive than the ACC/AHA criteria (34% [95% CI, 22-44] vs 95% [95% CI, 89-99]), consistent with recognizing fewer high-risk patients. CONCLUSIONS AND RELEVANCE A systematic enhanced ACC/AHA guideline and practice-based risk factor strategy prospectively predicted SCD events in nearly all at-risk patients with HCM, resulting in prophylactically implanted ICDs that prevented many catastrophic arrhythmic events in this at-risk population.

BackgroundThe relation of sex to clinical presentation and course in hypertrophic cardiomyopathy (HCM) remains incompletely resolved. We assessed differences in clinical outcomes between men and women within our large HCM cohort.Methods and ResultsOf 2123 consecutive patients, a minority (38%) were women who were diagnosed with HCM at older ages or referred for subspecialty evaluation later than men (50±19 versus 44±16 and 55±18 versus 49±16; P<0.001). Women more commonly developed advanced New York Heart Association class III/IV symptoms (53% versus 35% in men; P<0.001), predominantly secondary to outflow obstruction. While end‐stage heart failure with systolic dysfunction (ejection fraction <50%) was similar in men (5% versus 4% in women; P=0.33), women were 3‐fold more likely to develop heart failure with preserved systolic function (7.5% versus 2.6%; P=0.002). Sudden death events terminated by defibrillator therapy were similar in women (0.9%/year) versus men (1.0%/year; hazard ratio, 0.92; 95% CI, 0.6–1.5; P=0.73). HCM mortality was uncommon, with identical rates in both sexes (0.3%/year; hazard ratio, 1.5; 95% CI, 0.7–3.4;, P=0.25). Age‐adjusted all‐cause mortality also did not differ between women and men (1.7% versus 1.3%/year; hazard ratio, 1.32; 95% CI, 0.92–1.91; P=0.13).ConclusionsSurvival was not less favorable in women with HCM. Contemporary treatments including surgical myectomy to reverse heart failure and defibrillators to prevent sudden death, were effective in both sexes contributing to low mortality. However, despite more frequent outflow obstruction, women with HCM are underrecognized and referred to centers later than men, often with more advanced heart failure. Greater awareness of HCM in women should lead to earlier diagnosis and treatment, with implications for improved quality of life.

IMPORTANCEPatent foramen ovale (PFO)-associated strokes comprise approximately 10% of ischemic strokes in adults aged 18 to 60 years. While device closure decreases stroke recurrence risk overall, the best treatment for any individual is often unclear.OBJECTIVE To evaluate heterogeneity of treatment effect of PFO closure on stroke recurrence based on previously developed scoring systems.DESIGN, SETTING, AND PARTICIPANTS Investigators for the Systematic, Collaborative, PFO Closure Evaluation (SCOPE) Consortium pooled individual patient data from all 6 randomized clinical trials that compared PFO closure plus medical therapy vs medical therapy alone in patients with PFO-associated stroke, and included a total of 3740 participants. The trials were conducted worldwide from 2000 to 2017.EXPOSURES PFO closure plus medical therapy vs medical therapy alone. Subgroup analyses used the Risk of Paradoxical Embolism (RoPE) Score (a 10-point scoring system in which higher scores reflect younger age and the absence of vascular risk factors) and the PFO-Associated Stroke Causal Likelihood (PASCAL) Classification System, which combines the RoPE Score with high-risk PFO features (either an atrial septal aneurysm or a large-sized shunt) to classify patients into 3 categories of causal relatedness: unlikely, possible, and probable. MAIN OUTCOMES AND MEASURES Ischemic stroke.RESULTS Over a median follow-up of 57 months (IQR, 24-64), 121 outcomes occurred in 3740 patients. The annualized incidence of stroke with medical therapy was 1.09% (95% CI, 0.88%-1.36%) and with device closure was 0.47% (95% CI, 0.35%-0.65%) (adjusted hazard ratio [HR], 0.41 [95% CI, 0.28-0.60]). The subgroup analyses showed statistically significant interaction effects. Patients with low vs high RoPE Score had HRs of 0.61 (95% CI, 0.37-1.00) and 0.21 (95% CI, 0.11-0.42), respectively (P for interaction = .02). Patients classified as unlikely, possible, and probable using the PASCAL Classification System had HRs of 1.14 (95% CI, 0.53-2.46), 0.38 (95% CI, 0.22-0.65), and 0.10 (95% CI, 0.03-0.35), respectively (P for interaction = .003). The 2-year absolute risk reduction was −0.7% (95% CI, −4.0% to 2.6%), 2.1% (95% CI, 0.6%-3.6%), and 2.1% (95% CI, 0.9%-3.4%) in the unlikely, possible, and probable PASCAL categories, respectively. Device-associated adverse events were generally higher among patients classified as unlikely; the absolute risk increases in atrial fibrillation beyond day 45 after randomization with a device were 4.41% (95% CI, 1.02% to 7.80%), 1.53% (95% CI, 0.33% to 2.72%), and 0.65% (95% CI, −0.41% to 1.71%) in the unlikely, possible, and probable PASCAL categories, respectively.CONCLUSIONS AND RELEVANCE Among patients aged 18 to 60 years with PFO-associated stroke, risk reduction for recurrent stroke with device closure varied across groups classified by their probabilities that the stroke was causally related to the PFO. Application of this classification system has the potential to guide individualized decision-making.

The SEHC appears to measure a single general job satisfaction construct. The scale has adequate reliability and validity to recommend its use to assess satisfaction among multidisciplinary, U.S. healthcare staff. Our findings suggest that this survey is a good candidate for reduction to a short-form, and future research should validate this survey in other healthcare populations.

Objective: To assess whether the Prediction model Risk Of Bias ASsessment Tool (PROBAST) and a shorter version of this tool can identify clinical prediction models (CPMs) that perform poorly at external validation.Study Design and Setting: We evaluated risk of bias (ROB) on 102 CPMs from the Tufts CPM Registry, comparing PROBAST to a short form consisting of six PROBAST items anticipated to best identify high ROB. We then applied the short form to all CPMs in the Registry with at least 1 validation (n = 556) and assessed the change in discrimination (dAUC) in external validation cohorts (n = 1,147).Results: PROBAST classified 98/102 CPMS as high ROB. The short form identified 96 of these 98 as high ROB (98% sensitivity), with perfect specificity. In the full CPM registry, 527 of 556 CPMs (95%) were classified as high ROB, 20 (3.6%) low ROB, and 9 (1.6%) unclear ROB. Only one model with unclear ROB was reclassified to high ROB after full PROBAST assessment of all low and unclear ROB models. Median change in discrimination was significantly smaller in low ROB models (dAUC -0.9%, IQR -6.2-4.2%) compared to high ROB models (dAUC -11.7%, IQR -33.3-2.6%; P < 0.001). Conclusion:High ROB is pervasive among published CPMs. It is associated with poor discriminative performance at validation, supporting the application of PROBAST or a shorter version in CPM reviews.

No abstract

BackgroundWhile many clinical prediction models (CPMs) exist to guide valvular heart disease treatment decisions, the relative performance of these CPMs is largely unknown. We systematically describe the CPMs available for patients with valvular heart disease with specific attention to performance in external validations.Methods and ResultsA systematic review identified 49 CPMs for patients with valvular heart disease treated with surgery (n=34), percutaneous interventions (n=12), or no intervention (n=3). There were 204 external validations of these CPMs. Only 35 (71%) CPMs have been externally validated. Sixty‐five percent (n=133) of the external validations were performed on distantly related populations. There was substantial heterogeneity in model performance and a median percentage change in discrimination of −27.1% (interquartile range, −49.4%–−5.7%). Nearly two‐thirds of validations (n=129) demonstrate at least a 10% relative decline in discrimination. Discriminatory performance of EuroSCORE II and Society of Thoracic Surgeons (2009) models (accounting for 73% of external validations) varied widely: EuroSCORE II validation c‐statistic range 0.50 to 0.95; Society of Thoracic Surgeons (2009) Models validation c‐statistic range 0.50 to 0.86. These models performed well when tested on related populations (median related validation c‐statistics: EuroSCORE II, 0.82 [0.76, 0.85]; Society of Thoracic Surgeons [2009], 0.72 [0.67, 0.79]). There remain few (n=9) external validations of transcatheter aortic valve replacement CPMs.ConclusionsMany CPMs for patients with valvular heart disease have never been externally validated and isolated external validations appear insufficient to assess the trustworthiness of predictions. For surgical valve interventions, there are existing predictive models that perform reasonably well on related populations. For transcatheter aortic valve replacement (CPMs additional external validations are needed to broadly understand the trustworthiness of predictions.

No abstract