Real-World Estimates of Adrenal Insufficiency-Related Adverse Events in Children With Congenital Adrenal Hyperplasia.

Background: Although congenital adrenal hyperplasia (CAH) is known to be associated with adrenal crises (AC), its association with patient- or clinician-reported sick day episodes (SDE) is less clear.

Methods: Data on children with classic 21-hydroxylase deficiency CAH from 34 centers in 18 countries, of which 7 were Low or Middle Income Countries (LMIC) and 11 were High Income (HIC), were collected from the International CAH Registry and analyzed to examine the clinical factors associated with SDE and AC.

Results: A total of 518 children-with a median of 11 children (range 1, 53) per center-had 5388 visits evaluated over a total of 2300 patient-years.

Evidence synthesis for constructing directed acyclic graphs (ESC-DAGs): a novel and systematic method for building directed acyclic graphs.

Background: Directed acyclic graphs (DAGs) are popular tools for identifying appropriate adjustment strategies for epidemiological analysis. However, a lack of direction on how to build them is problematic. As a solution, we propose using a combination of evidence synthesis strategies and causal inference principles to integrate the DAG-building exercise within the review stages of research projects.

Effects of Vildagliptin on Ventricular Function in Patients With Type 2 Diabetes Mellitus and Heart Failure: A Randomized Placebo-Controlled Trial.

Objectives: This study sought to examine the safety of the dipeptidyl peptidase-4 inhibitor, vildagliptin, in patients with heart failure and reduced ejection fraction.

Background: Many patients with type 2 diabetes mellitus have heart failure and it is important to know about the safety of new treatments for diabetes in these individuals.

Methods: Patients 18 to 85 years of age with type 2 diabetes and heart failure (New York Heart Association functional class I to III and left ventricular ejection fraction [LVEF] <0.

Estimation of Survival Probabilities for Use in Cost-effectiveness Analyses: A Comparison of a Multi-state Modeling Survival Analysis Approach with Partitioned Survival and Markov Decision-Analytic Modeling.

Modeling of clinical-effectiveness in a cost-effectiveness analysis typically involves some form of partitioned survival or Markov decision-analytic modeling. The health states progression-free, progression and death and the transitions between them are frequently of interest. With partitioned survival, progression is not modeled directly as a state; instead, time in that state is derived from the difference in area between the overall survival and the progression-free survival curves.

Cost-effectiveness Analysis in R Using a Multi-state Modeling Survival Analysis Framework: A Tutorial.

This tutorial provides a step-by-step guide to performing cost-effectiveness analysis using a multi-state modeling approach. Alongside the tutorial, we provide easy-to-use functions in the statistics package R. We argue that this multi-state modeling approach using a package such as R has advantages over approaches where models are built in a spreadsheet package.

Scottish Keep Well health check programme: an interrupted time series analysis.

Background: Effective interventions are available to reduce cardiovascular risk. Recently, health check programmes have been implemented to target those at high risk of cardiovascular disease (CVD), but there is much debate whether these are likely to be effective at population level. This paper evaluates the impact of wave 1 of Keep Well, a Scottish health check programme, on cardiovascular outcomes.

Characterization of subgroups of heart failure patients with preserved ejection fraction with possible implications for prognosis and treatment response.

Background: Patients with heart failure and preserved ejection fraction (HFpEF) have a poor prognosis, and no therapies have been proven to improve outcomes. It has been proposed that heart failure, including HFpEF, represents overlapping syndromes that may have different prognoses. We present an exploratory study of patients enrolled in the Irbesartan in Heart Failure with Preserved Ejection Fraction Study (I-PRESERVE) using latent class analysis (LCA) with validation using the Candesartan in Heart failure: Assessment of Reduction in Mortality and morbidity (CHARM)-Preserved study to identify HFpEF subgroups.

HMG-coenzyme A reductase inhibition, type 2 diabetes, and bodyweight: evidence from genetic analysis and randomised trials.

Background: Statins increase the risk of new-onset type 2 diabetes mellitus. We aimed to assess whether this increase in risk is a consequence of inhibition of 3-hydroxy-3-methylglutaryl-CoA reductase (HMGCR), the intended drug target.

Methods: We used single nucleotide polymorphisms in the HMGCR gene, rs17238484 (for the main analysis) and rs12916 (for a subsidiary analysis) as proxies for HMGCR inhibition by statins.

Fatigue as a predictor of outcome in patients with heart failure: analysis of CORONA (Controlled Rosuvastatin Multinational Trial in Heart Failure).

Objectives: The purpose of this study was to examine the relationship between fatigue and clinical outcomes, using dyspnea as a comparator, in patients with left ventricular ejection fraction (LVEF) ≤35% enrolled in the CORONA (Controlled Rosuvastatin Multinational Trial in Heart Failure) study.

Background: Although fatigue is a common symptom in heart failure (HF), little is known about its association with prognosis.

Methods: At baseline in CORONA, fatigue "during the past few days" was measured using a 5-point exertion scale (0 = none, 1 = heavy exertion, 2 = moderate exertion, 3 = slight exertion, 4 = rest); a 4-point scale was used for dyspnea (1 to 4 as for fatigue).

Time trends in 30 day case-fatality following hospitalisation for asthma in adults in Scotland: a retrospective cohort study from 1981 to 2009.

Background: The risk of case-fatality following hospitalisation for asthma has not been well characterised. We describe trends in 30 day case-fatality following hospitalisation for asthma in adults in Scotland from 1981 to 2009.

Methods: Using the Scottish Morbidity Record Scheme (SMR01) with all asthma hospitalisations for adults (≥18 years) with ICD9 493 and ICD10 J45-J46 in the principal diagnostic position at discharge (1981-2009).

Angina and intermittent claudication in 7403 participants of the 2003 Scottish Health Survey: impact on general and mental health, quality of life and five-year mortality.

Background: Angina and intermittent claudication impair function and mobility and reduce health-related quality of life. Both symptoms have similar etiology, yet the physical and psychological impacts of these symptoms are rarely studied in community-based cohorts or in individuals with isolated symptoms.

Methods: The 2003 Scottish Health Survey was a cross-sectional survey which enrolled a random sample of individuals aged 16-95 years living in Scotland.

Trends in incidence and in short term survival following a subarachnoid haemorrhage in Scotland, 1986-2005: a retrospective cohort study.

Background: To examine age and sex specific incidence and 30 day case fatality for subarachnoid haemorrhage (SAH) in Scotland over a 20 year period.

Methods: A retrospective cohort study using routine hospital discharge data linked to death records.

Results: Between 1986 and 2005, 12,056 individuals experienced an incident SAH.

Estimating the impact of stroke unit care in a whole population: an epidemiological study using routine data.

Background And Purpose: Randomised trials indicate that organised inpatient (stroke unit) care has an important impact on patient outcomes with an absolute risk difference (ARD) of 3% for survival and 5% for returning home. However, it is unclear what impact this complex intervention actually has in routine practice. A comprehensive national dataset was used to study the impact of stroke unit implementation.

Improved survival with bisoprolol in patients with heart failure and renal impairment: an analysis of the cardiac insufficiency bisoprolol study II (CIBIS-II) trial.

Aims: Information on the effectiveness of beta-blockade in patients with heart failure (HF) and concomitant renal impairment is scarce and beta-blockers are underutilized in these patients.

Methods And Results: The Cockcroft-Gault formula normalized for body surface-area was used to estimate renal function (eGFR(BSA)) in 2622 patients with HF, left ventricular ejection fraction < or =35%, New York Heart Association class III/IV and serum creatinine <300 micromol/L (3.4 mg/dL) in the second Cardiac Insufficiency Bisoprolol Study II.

Treatment of type 2 diabetes and outcomes in patients with heart failure: a nested case-control study from the U.K. General Practice Research Database.

Objective: Diabetes and heart failure commonly coexist, and prior studies have suggested better outcomes with metformin than other antidiabetic agents. We designed this study to determine whether this association reflects a beneficial effect of metformin or a harmful effect of other agents.

Research Design And Methods: We performed a case-control study nested within the U.

Mathematical coupling may account for the association between baseline severity and minimally important difference values.

Objective: To generate anchor-based values for the "minimally important difference" (MID) for a number of commonly used patient-reported outcome (PRO) measures and to examine whether these values could be applied across the continuum of preoperative patient severity.

Study Design And Setting: Six prospective cohort studies of patients undergoing elective surgery at hospitals in England and Wales. Patients completed questionnaires about their health and health-related quality of life before and after surgery.

Age- and sex-specific trends in fatal incidence and hospitalized incidence of stroke in Scotland, 1986 to 2005.

Background: Temporal trends in stroke incidence are unclear. We aimed to examine age- and sex-specific temporal trends in incidence of fatal and nonfatal hospitalized stroke in Scotland from 1986 to 2005.

Methods And Results: Mean age at the time of first stroke was 70.

Discordant short- and long-term outcomes associated with diabetes in patients with heart failure: importance of age and sex: a population study of 5.1 million people in Scotland.

Background: Diabetes and heart failure frequently coexist. Our aim was to assess the association between diabetes and short- and long-term outcomes in all patients admitted to the hospital for the first time with heart failure in Scotland between 1986 and 2003.

Methods And Results: A total of 116 556 patients were studied, of whom 13% (n=15 161) had a diagnosis of diabetes.

The impact of serum potassium concentration on mortality after liver transplantation: a cohort multicenter study.

Background: Potassium plays a key role in human metabolism in both health and disease. The impact of recipient serum potassium concentration [K] on mortality after liver transplantation has not been described previously.

Methods: We assessed the effect of recipient [K] on the survival of adult first single-organ liver transplant recipients in the United Kingdom and Ireland between March 1, 1994, and February 28, 2007 (n=5942), adjusting for recipient, donor, and graft characteristics.

Sex differences in incidence, mortality, and survival in individuals with stroke in Scotland, 1986 to 2005.

Background And Purpose: The aim of this study was to examine the effect of sex across different age groups and over time for stroke incidence, 30-day case-fatality, and mortality.

Methods: All first hospitalizations for stroke in Scotland (1986 to 2005) were identified using linked morbidity and mortality data. Age-specific rate ratios (RRs) for comparing women with men for both incidence and mortality were modeled with adjustment for study year and socioeconomic deprivation.

Long-term trends in first hospitalization for heart failure and subsequent survival between 1986 and 2003: a population study of 5.1 million people.

Background: We examined whether population-level hospitalization rates for heart failure (HF) and subsequent survival have continued to improve since the turn of the century. We also examined trends in the prescribing of evidence-based pharmacological treatment for HF.

Methods And Results: All patients in Scotland hospitalized with a first episode of HF between 1986 and 2003 were followed up until death or the end of 2004.

Diathermy power settings as a risk factor for hemorrhage after tonsillectomy.

Objective: To investigate bipolar diathermy power settings as a risk factor for postoperative hemorrhage following tonsillectomy.

Study Design And Setting: A prospective cohort study was undertaken between July 2003 and September 2004 in National Health Service (NHS) and independent hospitals in England and Northern Ireland. Data were collected on patient characteristics, tonsillectomy technique, and postoperative hemorrhage within 28 days of surgery.

Continuous monitoring tools for pediatric surgical outcomes: an example using biliary atresia.

Purpose: Continuous monitoring tools can be used to monitor surgical outcomes over time. We illustrate the use of CUmulative SUM (CUSUM) charts in monitoring outcomes of Kasai portoenterostomy for treatment of biliary atresia at a supraregional unit.

Methods: Data on 57 consecutive infants who underwent a Kasai portoenterostomy performed by a single surgeon between June 1994 and June 2006 were collected.

The impact of serum sodium concentration on mortality after liver transplantation: a cohort multicenter study.

Modification of the current allocation system for donor livers in the United States to incorporate recipient serum sodium concentration ([Na]) has recently been proposed. However, the impact of this parameter on posttransplantation mortality has not been previously examined in a large risk-adjusted analysis. We assessed the effect of recipient [Na] on the survival of all adults with chronic liver disease who received a first single organ liver transplant in the UK and Ireland during the period March 1, 1994 to March 31, 2005 (n=5,152) at 3 years, during the first 90 days, and beyond the first 90 days, adjusting for a wide range of recipient, donor, and graft characteristics.