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Biomedical subjects

D Spiegelhalter

Publications and source records attributed to D Spiegelhalter.

At least 19 recordsLinked to original sources

What determines the cost-effectiveness of diabetes screening?

AIMS/HYPOTHESIS: The cost-effectiveness of screening for diabetes is unknown but has been modelled previously. None of these models has taken account of uncertainty. We aimed to describe these uncertainties in a model where the outcome was CHD risk. SUBJECTS AND METHODS: Our model used population data from the Danish Inter99 study, and simulations were run in a theoretical population of 1,000,000 individuals. CHD risk was estimated using the UK Prospective Diabetes Study (UKPDS) risk engine, and risk reduction from published randomised clinical trials. Probabilistic sensitivity analysis was used to provide confidence intervals for modelled outputs. Uncertain parameter values were independently simulated from distributions derived from existing literature and deterministic sensitivity analysis performed using multiple model runs under different strategy choices and using extreme parameter estimates. RESULTS: In the least conservative model (low costs and multiplicative risk reduction for combined treatments), the 95% confidence interval of the incremental cost-effectiveness ratio varied from pound23,300-82,000. The major contributors to this uncertainty were treatment risk reduction model parameters: the risk reduction for hypertension treatment and UKPDS risk model intercept. Overall cost-effectiveness ratio was not sensitive to decisions about which groups to screen, nor the costs of screening or treatment. It was strongly affected by assumptions about how treatments combine to reduce risk. CONCLUSIONS/INTERPRETATION: Our model suggests that there is considerable uncertainty about whether or not screening for diabetes would be cost-effective. The most important but uncertain parameter is the effect of treatment. In addition to directly influencing current policy decisions, health care modelling can identify important unknown or uncertain parameters that may be the target of future research.

Adult↗

Comparison of UK paediatric cardiac surgical performance by analysis of routinely collected data 1984-96: was Bristol an outlier?

BACKGROUND: Reports of high mortality after paediatric cardiac surgery at the Bristol Royal Infirmary, UK, led to the establishment of an independent public inquiry. A key question was whether or not the mortality statistics in Bristol were unusual compared with other specialist centres. To answer this question, we did a retrospective analysis of mortality in the UK using two datasets. METHODS: Data from the UK Cardiac Surgical Register (CSR; January, 1984, to March, 1996) and Hospital Episode Statistics (HES; April, 1991, to December, 1995) were obtained for all 12 major centres in which paediatric cardiac surgery is done in the UK. The main outcome measure was mortality within 30 days of a cardiac surgical procedure. We estimated excess deaths in Bristol using a random-effects model derived from the remaining 11 centres. Additionally, a sensitivity analysis was done and case-mix examined. FINDINGS: For children younger than 1 year, in open operations, the mortality rate in Bristol was around double that of the other centres during 1991-95: within the CSR, there were 19.0 excess deaths (95% interval 2-32) among 43 deaths; and in HES, there were 24.1 excess deaths (12-34) among 41 deaths recorded. There was no strong evidence for excess mortality in Bristol for closed operations or for open operations in children older than 1 year. INTERPRETATION: Our results suggest that Bristol was an outlier, and we do not believe that statistical variation, systematic bias in data collection, case-mix, or data quality can explain a divergence in performance of this size.

Adolescent↗

Factors associated with functional decline and improvement in a very elderly community-dwelling population.

The objective of this study was to analyze the factors associated with functional decline and improvement in a community-dwelling population of people aged 75 years and older. A representative sample of elderly people living at home in the city of Sherbrooke (Quebec, Canada) was assessed yearly on three occasions (1991-1993) by a nurse. A health questionnaire, together with standardized instruments measuring disabilities, cognitive status, and depressive mood, was administered on the three assessments. From the 655 subjects who agreed to participate, a total of 504 subjects completed the study. The most important factors associated with functional decline were the number of days off regular activities (odds ratio (OR) = 1.31), the number of hot meals per day (OR = 1.59), and cognitive status (OR = 0.96), whereas weight loss (OR = 0.37) and living alone (OR = 0.54) were significant protective factors. Previous declines in functional autonomy, cognitive state, or mood were not independent risk factors. The most important factors associated with functional improvement were the disability score (OR = 1.08), previous functional decline (OR = 6.06), and decline in the disability score (OR = 1.09). Perceiving health to be worse than it was the previous year was a significant factor against improvement (OR = 0.24). This study identifies risk factors that can be helpful for targeting high-risk subgroups within the very elderly population who may benefit from a preventive program.

Activities of Daily Living↗

Randomised controlled trial of laparoscopic versus open mesh repair for inguinal hernia: outcome and cost.

OBJECTIVE: To compare tension-free open mesh hernioplasty under local anaesthetic with transabdominal preperitoneal laparoscopic hernia repair under general anaesthetic. DESIGN: A randomised controlled trial of 403 patients with inguinal hernias. SETTING: Two acute general hospitals in London between May 1995 and December 1996. SUBJECTS: 400 patients with a diagnosis of groin hernia, 200 in each group. MAIN OUTCOME MEASURES: Time until discharge, postoperative pain, and complications; patients' perceived health (SF-36), duration of convalescence, and patients' satisfaction with surgery; and health service costs. RESULTS: More patients in the open group (96%) than in the laparoscopic group (89%) were discharged on the same day as the operation (chi2 = 6.7; 1 df; P=0.01). Although pain scores were lower in the open group while the effect of the local anaesthetic persisted (proportional odds ratio at 2 hours 3.5 (2.3 to 5.1)), scores after open repair were significantly higher for each day of the first week (0.5 (0.3 to 0.7) on day 7) and during the second week (0.7 (0.5 to 0.9)). At 1 month there was a greater improvement (or less deterioration) in mean SF-36 scores over baseline in the laparoscopic group compared with the open group on seven of eight dimensions, reaching significance on five. For every activity considered the median time until return to normal was significantly shorter for the laparoscopic group. Patients randomised to laparoscopic repair were more satisfied with surgery at 1 month and 3 months after surgery. The mean cost per patient of laparoscopic repair was 335 pounds (95% confidence interval 228 pounds to 441 pounds) more than the cost of open repair. CONCLUSION: This study confirms that laparoscopic hernia repair has considerable short term clinical advantages after discharge compared with open mesh hernioplasty, although it was more expensive.

Activities of Daily Living↗

Incidence of functional decline and improvement in a community-dwelling, very elderly population.

With the aging of the population, functional decline is one of the major challenges to health care systems. The objective of this study was to estimate the incidence of functional decline and improvement in a community-dwelling population of people aged 75 years and above. A representative sample of elderly people living at home in the city of Sherbrooke (Québec, Canada) was assessed yearly on three occasions (1991-1993) by a nurse. Disabilities were measured by the Functional Autonomy Measurement System, a 29-item rating scale developed according to the World Health Organization classification of disabilities. From the 655 subjects who agreed to participate, a total of 572 subjects completed the study, including 68 who subsequently died. The probability of declining was 20.1% for the first year and 12.4% for the second year. The incidence of functional decline among previously stable subjects was 11.9% (95% confidence interval (CI) 8.9-15.9). Age was strongly related to decline (relative risk (RR) = 1.15/year, 95% CI 1.09-1.21), but there was no significant sex effect (RRmale = 0.88, 95% CI 0.55-1.39). The incidence of improvement among previously disabled subjects was estimated at 7.5% (95% CI 5.1-10.9) for the first year and 17.9% (95% CI 14.0-23.0) for the second year. Neither age (RR = 1.07, 95% CI 0.99-1.15) nor sex (RRmale = 1.70, 95% CI 0.90-3.18) was significantly associated with the probability of improving. This study stresses the importance of precise measurement of disabilities and the complex and dynamic process of functional transitions.

Activities of Daily Living↗

Presentation and attrition in complex pulmonary atresia.

OBJECTIVES: This study summarized patterns of presentation and attrition in complex pulmonary atresia. BACKGROUND: Assessment of the potential impact of surgical strategies for managing complex pulmonary atresia requires information about variability in age and physiology at presentation of the condition. METHODS: We performed a retrospective review of age at presentation, referral source, pulmonary artery and collateral anatomy and surgical history of 218 patients from two institutions dealing with congenital heart disease throughout life. RESULTS: Approximately 65% of pulmonary atresia appears in infancy, with 50% of patients severely symptomatic from cyanosis and 25% from heart failure. Compared with those presenting undiagnosed, patients referred secondarily for specialist management tend to be older when first seen, and care must be taken when generalizing about the natural history of the condition from their survival experience. Overall actuarial survival, including the effects of operation, suggests that 60% (95% confidence limits [CL] 43 to 73) of patients presenting in infancy survive to their first birthday, 65% (95% CL 51 to 74) of those alive at 1 year old survive to the age of 10, and 16% (95% CL 5 to 31) of those alive at 10 years old survive to age 35. CONCLUSIONS: Novel surgical approaches have generally been applied beyond infancy in patients selected by their survival through the period of greatest attrition for this disease. Unless successful application in symptomatic infants is demonstrated, we cannot assume that these serial and complicated operations will have a major impact on the outlook of most patients with complex pulmonary atresia.

Actuarial Analysis↗

Pregnancy in cyanotic congenital heart disease. Outcome of mother and fetus.

In a series of 416 women with congenital heart disease seen in the Royal Brompton National Heart and Lung Hospital, London, and the Hospital Giovanni Bosco, Torino, Italy, there were 822 pregnancies. The outcomes of 96 pregnancies in 44 patients with cyanotic congenital heart disease were studied. Patients with the Eisenmenger reaction were excluded. Patients were divided arbitrarily into groups according to the type of maternal congenital cardiac anomaly, and factors influencing maternal and fetal outcome were evaluated. The incidence of maternal cardiovascular complications was high (32%), with one death from endocarditis 2 months after delivery. Forty-one (43%) of 96 pregnancies resulted in a live birth; 15 (37%) were premature. Mean weight of full-term infants was 2575 g. Univariate analysis suggested that maternal disease, Ability Index, hemoglobin, and arterial oxygen saturation before the pregnancy were factors that discriminated between successful and unsuccessful fetal outcome, with hemoglobin and arterial oxygen saturation being the most important predictors. Women with cyanotic congenital heart disease can go through pregnancy with a low risk to themselves, with frequent treatable complications, but there is a high incidence of miscarriage, premature births, and low birth weights. An incidence of congenital heart disease in the fetus of 4.9% (2 of 41 live births) is higher than that found in the normal population.

Adolescent↗

Analysis of a cluster of surgical failures. Application to a series of neonatal arterial switch operations.

A pediatric cardiac surgeon performed 104 neonatal arterial switch operations for transposition of the great arteries with or without ventricular septal defect between June 1987 and February 1993. Initial euphoria on having only one death in the first 52 patients gave way to increasing concern when patients 53, 55, 59, 63, 64, 67, and 68 died. Sensing a problem, the surgeon visited a low-risk institution after patients 55 and 64 had died and then decided to re-train after patient 68 died. One death has occurred since. To find out whether the cluster of failures could have been related to chance alone, to variability of risk factors across time, or to suboptimal performance, we conducted the following analyses: First, identification of trends with the cumulative sum procedure was undertaken and actual mortality compared with the mortality predicted from an equation derived from a multi-institutional study. Second, logistic regression analysis of risk factors was done. If a mechanism of continuous monitoring had been in place, unfavorable trends and a need for change in protocol would have been detected earlier. Retrospective risk factor analysis suggested an excessive risk for patients with origin of the circumflex or left anterior descending coronary arteries from sinus 2 and a protective effect of phenoxybenzamine. However, about half of the risk associated with the cluster of failures was not accounted for by the variables analyzed. There was therefore an indication of suboptimal performance that appears to have been neutralized by retraining.

Cardiac Surgical Procedures↗

Implications for trials in progress of publication of positive results.

It is not easy to decide, when results from similar trials appear, whether a trial still underway should be stopped or not. The weight of the other evidence has to be taken into account--as indeed it has to be for decisions by clinicians and by health service managers outside the settings of a trial. Taking the Systolic Hypertension in the Elderly Trial (SHEP) as an example, we show how its results are not unequivocal (ie, there is no proof beyond reasonable doubt). This verdict justifies the continuation of similar trials in progress. More generally--but again for individual clinicians and for trial organisers and again with SHEP as the example--we illustrate a bayesian approach to trial-termination decisions.

Aged↗

Prostacyclin (epoprostenol) and heart-lung transplantation as treatments for severe pulmonary hypertension.

OBJECTIVE: To determine whether epoprostenol (prostacyclin, PGI2) or heart-lung transplantation (HLT), or both improves survival of patients with severe pulmonary hypertension. DESIGN: This was a prospective study where the effects of epoprostenol were compared with conventional treatment. Also, the benefits of epoprostenol and HLT were assessed by comparing survival in this group with that of 120 patients at the Mayo Clinic before HLT and epoprostenol treatment became available. PATIENTS AND INTERVENTIONS: Forty four patients were studied; 25 received continuous epoprostenol over a four year period (mean (SD) cardiac index 1.8 (0.4) 1 min-1 m-2 and mean (SD) pulmonary artery pressure (PAP) 70 (16) mm Hg) and 19 did not (cardiac index 2.1 (0.6) 1 min-1 m-2 and PAP 64 (13) mm Hg). Ten patients underwent HLT: seven had received epoprostenol, and three had not. RESULTS: The therapeutic intervention with epoprostenol, or HLT, or both improved survival compared with the Mayo clinic patients (p = 0.05). Most of the benefit was conferred by epoprostenol, which prolonged survival twofold from a median time of eight to 17 months and doubled the changes of successful HLT. The improved survival with epoprostenol was not related to its immediate capacity to cause pulmonary vasodilation. Those patients who had limited acute pulmonary vasodilation when treated with epoprostenol showed the greatest improvement in survival. CONCLUSIONS: These preliminary results indicate that those pulmonary hypertensive patients with the poorest chance of survival can be helped by epoprostenol and by HLT.

Adult↗

Quality of life measures in health care. I: Applications and issues in assessment.

Many clinicians remain unsure of the relevance of measuring quality of life to their clinical practice. In health economics quality of life measures have become the standard means of assessing the results of health care interventions and, more controversially, the means of prioritising funding; but they have many other applications. This article--the first of three on measuring quality of life--reviews the instruments available and their application in screening programmes, audit, health care research, and clinical trials. Using the appropriate instrument is essential if outcome measures are to be valid and clinically meaningful.

Clinical Protocols↗

Field trials of medical decision-aids: potential problems and solutions.

Only clinical trials can assess the impact of prototype medical decision-aids, but they are seldom performed before dissemination. Many problems are encountered when designing such studies, including ensuring generality, deciding what to measure, feasible study designs, correcting for biases caused by the trial itself and by the decision-aid, resolving the "Evaluation Paradox", and potential legal and ethical doubts. These are discussed in this paper.

Bias↗

Predicting difficult intubation.

The amount of larynx seen at intubation was assessed in 633 adult patients undergoing routine surgery. Various measurements of the head and neck were made in an attempt to discover which features were associated with difficulty with laryngoscopy (defined as the inability to see even the arytenoids). In addition 38 patients, reported by colleagues because they had been "difficult to intubate", were measured. Five useful risk factors, measured at three levels of severity, were identified. A simple predictive rule was developed and tested on a prospective set of 778 patients, in 1.5% of whom laryngoscopy was found to be difficult. Depending on the threshold chosen, the rule allowed the detection of, for example, 75% of the "difficult" laryngoscopies at a cost of falsely identifying 12% of the "not difficult" patients.

Adult↗