Background: PARADIGM-HF demonstrated superiority of sacubitril/valsartan (sac/val) over enalapril in patients with heart failure with reduced ejection fraction (HFrEF). However, patients in clinical practice may differ in their characteristics and overall risk compared with patients in clinical trials, and additional outcomes can be observed in real world (RW). Hence, a systematic review was conducted to identify and describe RW data on sac/val. Methods: RW studies evaluating the effects of sac/val in adult patients with HFrEF with a sample size ≥100 were identified via MEDLINE® and Embase® from 2015 to January 2020. Citations were screened, critically appraised and relevant data were extracted. Results: A total of 68 unique studies were identified. Nearly half of the studies were conducted in Europe (n = 34), followed by the US (n = 15) and Asia (n = 11). Median follow-up period varied from 1 to 19 months. Mean age ranged between 48.7 and 79.0 years; patients were mostly male and in New York Heart Association (NYHA) functional class II/III, and mean left ventricular ejection fraction varied between 23%and 38%. Of studies performing comparisons, most reported superior efficacy of sac/val in reducing the risk of HF hospitalisations, allcause hospitalisations, and all-cause mortality as compared to standard-of-care. Many studies reported significant improvements in NYHA functional class and reduction in biomarker levels post sac/val. Hypotension and hyperkalaemia were the most frequently reported adverse events. Conclusions: This comprehensive overview of currently available RW evidence on sac/val complements the evidence from randomised controlled trials, substantiating its effectiveness in heterogeneous real-world HF populations.

This study was funded by Novartis Pharmaceuticals. Park and Ko are employed by Novartis Pharmaceuticals in East Hanover, New Jersey, and Ko holds stock in Novartis. Raza, George, and Agrawal are employed by Novartis Healthcare in Hyderabad, India. Study concept and design were contributed primarily by Park and Ko, along with the other authors. Raza, George, and Agrawal collected the data, along with Park and Ko. Data interpretation was performed by Agrawal, Raza, George, Park, and Ko. The manuscript was written and revised by Raza, George, and Park, along with Ko and Agrawal. Results from this systematic literature review were presented at the AMCP Annual Meeting 2016; San Francisco, California; April 19-22, 2016.

While the epidemiology of adult heart failure has been extensively researched, this systematic review addresses the less well characterized incidence and prevalence of pediatric HF. The search strategy used Cochrane methodology and identified 83 unique studies for inclusion. Studies were categorized according to whether the HF diagnosis was reported as primary (n = 10); associated with other cardiovascular diseases (CVDs) (n = 49); or associated with non-CVDs (n = 24). A narrative synthesis of the evidence is presented. For primary HF, the incidence ranged from 0.87/100,000 (UK and Ireland) to 7.4/100,000 (Taiwan). A prevalence of 83.3/100,000 was reported in one large population-based study from Spain. HF etiology varied across regions with lower respiratory tract infections and severe anemia predominating in lower income countries, and cardiomyopathies and congenital heart disease major causes in higher income countries. Key findings for the other categories included a prevalence of HF associated with cardiomyopathies ranging from 36.1% (Japan) to 79% (US); associated with congenital heart disease from 8% (Norway) to 82.2% (Nigeria); associated with rheumatic heart diseases from 1.5% (Turkey) to 74% (Zimbabwe); associated with renal disorders from 3.8% (India) to 24.1% (Nigeria); and associated with HIV from 1% (US) to 29.3% (Brazil). To our knowledge, this is the first systematic review of the topic and strengthens current knowledge of pediatric HF epidemiology. Although a large body of research was identified, heterogeneity in study design and diagnostic criteria limited the ability to compare regional data. Standardized definitions of pediatric HF are required to facilitate cross-regional comparisons of epidemiological data.Electronic supplementary materialThe online version of this article (10.1007/s00246-017-1787-2) contains supplementary material, which is available to authorized users.

This study was funded by Novartis Pharma AG, Basel, Switzerland. Deschaseaux, McSharry, Hudson, Agrawal, and Turner are permanent employees of Novartis. Concept and study design were contributed by Deschaseaux, Hudson, and Turner, along with McSharry. McSharry took the lead in data collection, along with Deschaseaux, Hudson, and Turner. Data interpretation was performed by Hudson, along with the other authors. The manuscript was written by Agrawal, along with Deschaseaux and Turner, and revised by Deschaseaux and Turner, along with the other authors.

Background: Transparent and robust real-world evidence sources are increasingly important for global health, including cardiovascular (CV) diseases. We aimed to identify global real-world data (RWD) sources for heart failure (HF), acute coronary syndrome (ACS), and atrial fibrillation (AF). Methods: We conducted a systematic review of publications with RWD pertaining to HF, ACS, and AF (2010–2018), generating a list of unique data sources. Metadata were extracted based on the source type (e.g., electronic health records, genomics, and clinical data), study design, population size, clinical characteristics, follow-up duration, outcomes, and assessment of data availability for future studies and linkage. Results: Overall, 11,889 publications were retrieved for HF, 10,729 for ACS, and 6,262 for AF. From these, 322 (HF), 287 (ACS), and 220 (AF) data sources were selected for detailed review. The majority of data sources had near complete data on demographic variables (HF: 94%, ACS: 99%, and AF: 100%) and considerable data on comorbidities (HF: 77%, ACS: 93%, and AF: 97%). The least reported data categories were drug codes (HF, ACS, and AF: 10%) and caregiver involvement (HF: 6%, ACS: 1%, and AF: 1%). Only a minority of data sources provided information on access to data for other researchers (11%) or whether data could be linked to other data sources to maximize clinical impact (20%). The list and metadata for the RWD sources are publicly available at www.escardio.org/bigdata. Conclusions: This review has created a comprehensive resource of CV data sources, providing new avenues to improve future real-world research and to achieve better patient outcomes.

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Objectives To summarize cost-effectiveness (CE) evidence of sacubitril/valsartan for the treatment of heart failure (HF) patients with reduced ejection fraction (HFrEF). The impact of different modeling approaches and parameters on the CE results is also described. Methods We conducted a systematic literature review using multiple databases: Embase®; MEDLINE®; MEDLINE®-In Process; NIHR CRD database including DARE, NHS EED, and HTA databases; and the Cost Effectiveness Analysis registry. We also reviewed HTA countries’ websites to identify CE reports of sacubitril/valsartan, published up to 25-July-2021. Articles published in English as full-texts, conference-abstracts, or HTA reports were included. Results We included 44 CE models [39 from 37 publications (22 full-texts; 15 conference-abstracts) and 5 HTAs; Europe, n = 20; North and South Americas, n = 14; Asia and Australia, n = 10]. Most models adopted a Markov structure with constant transition probabilities of events (n = 27) or a mix of Markov and regression-based models (n = 16), with variations in structural assumptions and chosen parameters. Study authors concluded sacubitril/valsartan to be a cost-effective therapy in 37/41 models in chronic HFrEF patients and 2/3 models in hospitalized patients stabilized after an acute decompensation for HF. CE models showing sacubitril/valsartan not to be a cost-effective treatment generally modeled a shorter time horizon. Effect of sacubitril/valsartan on cardiovascular and all-cause mortality, cost, duration of effect and time horizon was the main model drivers. Conclusions Most evidence indicated sacubitril/valsartan is cost-effective in HFrEF. The use of a lifetime horizon is recommended in future models as HF is a chronic disease. Data on the CE of sacubitril/valsartan in the inpatient setting were limited and further research is warranted.

A623 (QoL) and work ability (WA) in Slovak Republic. Methods: The sample consisted of 99 patients, 41 women and 58 men, with average age being 63,7 years. 15 patients were classified in NYHA I, 48 patients-NYHA II and 36 patients-NYHA III. The average duration of disease was 3,6 years. Primary method used for the analysis of QoL was a combined questionnaire consisting of 6 parts: A. Demography, B1. Clinical part filled out by physician, B2. Clinical part filled out by patient, C. Quality of life, D. Socioeconomic part, E. EQ5DL. QoL and WA were evaluated on numeric scales from 0-the worst to 10-the best. T-test, Mann-Whitney, Wilcoxon, Kruskal-Wallis and Friedman tests and Spearman correlation were used in results evaluation. Results: Significant statistical differences in QoL were found: in the time of best health-9,18, without CHF-8,15 in the time of diagnosis-5,76, in the time of acceptance in hospital-3,98 and in the time of full treatment-5,01. The results gained in WA were: 9,36 vs 8,43 vs 5,71 vs 3,69 vs 4,55. The results from QoL and WA were in strong correlation. Foreknowledge of disease (1-the worst, 5-the best) was 3,55, satisfaction with medical care-3,7and nursing care-3,8. Willingness to pay was for full health withou CHF was 67,9 € monthly by average monthly income 427,1 €. ConClusions: CHF has a significant impact on patients' QoL and WA. There are significant differences in both areas in duration of CHF. The treatment had positive impact on QoL and WA. Higher grade of NYHA had worse QoL and WA.