Cost-of-illness studies based on massive data: a prevalence-based,top-down regression approach

Despite the increasing availability of routine data, no analysis method has yet been presented for cost-of-illness (COI) studies based on massive data. We aim, first, to present such a method and, second, to assess the relevance of the associated gain in numerical efficiency. We propose a prevalence-based, top-down regression approach consisting of five steps: aggregating the data; fitting a generalized additive model (GAM); predicting costs via the fitted GAM; comparing predicted costs between prevalent and non-prevalent subjects; and quantifying the stochastic uncertainty via error propagation. To demonstrate the method, it was applied to aggregated data in the context of chronic lung disease to German sickness funds data (from 1999), covering over 7.3 million insured. To assess the gain in numerical efficiency, the computational time of the innovative approach has been compared with corresponding GAMs applied to simulated individual-level data. Furthermore, the probability of model failure was modeled via logistic regression. Applying the innovative method was reasonably fast (19 min). In contrast, regarding patient-level data, computational time increased disproportionately by sample size. Furthermore, using patient-level data was accompanied by a substantial risk of model failure (about 80 % for 6 million subjects). The gain in computational efficiency of the innovative COI method seems to be of practical relevance. Furthermore, it may yield more precise cost estimates.     

Smoking-attributable mortality in Morocco: results of a prevalence-based study in Casablanca

Background

Tobacco control measurements’ had little impact on smoking prevalence in Morocco. The aim of this study is to provide first data on smoking attributable mortality in Morocco.

Method

The Smoking-Attributable Mortality, Morbidity and Economic Costs (SAMMEC) software was used to estimate the smoking attributable mortality (SAM) in Casablanca region in 2012. Smoking prevalence and mortality data of people aged 35 years or older were obtained from the national survey on tobacco “Marta” and from Health Ministry Mortality System, respectively.

Results

Of the 5261deaths of persons aged 35 years and older, 508 (9.7%) were attributable to cigarette smoking. This total represents 16.2% of all male deaths (n =448) and 2.0% (n =80) of all female deaths in this region. The leading four causes of smoking attributable deaths were lung cancer (177), chronic airways obstruction (76), ischemic heart disease (39), and cerebrovascular disease (31).

Conclusion

Tobacco use caused one out of six deaths in Casablanca in 2012. Four leading causes (lung cancer, ischemic heart disease, cerebrovascular disease and chronic airways obstruction,) accounted for 51.6% of SAM. Effective and comprehensive actions must be taken in order to slow this epidemic in Morocco.     

Understanding genetic disease in a socio-historical context: a case study of cystic fibrosis

In this article I present a socio-historical analysis of the definition and diagnosis of one particular genetic disease--cystic fibrosis (CF)--in an effort the better to understand its social context both before and after the advent of molecular genetics. I begin my analysis with some background on the history of CF, before moving on to consider the emergence of the notion of classic CF, the development of the sweat test, early approaches to mild or variant forms of CF, the concept of CF as a genetic disease, the concept of CF as a collection of related disorders, and developments in the understanding and diagnosis of CF which came about in the wake of molecular genetics. I highlight a range of technological, professional and patient developments and how these stimulated new research, typologies and clinical tools. I also consider how different notions of CF were mobilised, either to support or undermine a particular approach to diagnosis or treatment, and consider how the dynamic and contextual characteristics of CF were accounted for by scientists and clinicians with an interest in CF. I end by discussing the implications of my analysis for the contemporary sociology of genetics, and related studies in the sociology of medicine more generally.     

Telehealth in cystic fibrosis: a systematic review

We conducted a systematic review of the use of telehealth in people with Cystic Fibrosis (CF). The studies reviewed were of adults and children with CF, and incorporated telehealth for monitoring symptoms, assessing adherence to prescribed therapies or providing a therapeutic intervention. Searches of four electronic databases returned 293 references. Eight studies met the inclusion criteria. Variability in study design and outcome measures precluded meta-analysis. Seven studies assessed telemonitoring feasibility for patient usability and acceptance, or for physiological monitoring. Two studies were randomised controlled trials, although only one showed differences in outcome between the intervention and usual care with improved spirometry stability and significantly increased antibiotic use in the intervention group. In four studies participants were asked to transmit data on spirometry (FEV(1)) or symptoms. Participant non-compliance with data reporting ranged from 43-63%. Generally, participants reported being able to use the required technology. There is insufficient evidence to reach a firm conclusion about the benefits of telehealth in people with CF, but it remains a promising area for future investigation.     

Prenatal screening for cystic fibrosis: an economic analysis

Cystic fibrosis (CF) is the most common life-shortening genetically transmitted disease in Denmark with a birth prevalence of 1 in 4700, resulting in 12-15 new cases of cystic fibroses annually.The aim of this study is to disclose the societal resource implications of introducing a population wide prenatal screening programme for cystic fibrosis in Denmark. The present analysis is limited to the monetary consequences of introducing a screening programme, where costs of screening are compared to the potential benefits measured in cost savings involved if births of CF patients are avoided.Screening costs in a Danish setting were estimated at DKK 2 771 262 ( pound sterlings 231 438) per aborted affected fetus in the first screening round, stabilising at DKK 1 864 594 ( pound sterlings 155 383) per aborted affected fetus at subsequent screening rounds. Comparing this figure with the estimated benefits of avoiding a CF case (DKK 2.1-4.4 million; pound sterlings 175 000-366 667) suggests that introducing a screening programme for cystic fibrosis will be net cost saving irrespective of the perspective of the analysis, assumptions on replacement children and method of estimating long-term production gains/losses.     

Growth hormone as a nutritional adjunct in cystic fibrosis: results of a pilot study

Growth hormone (GH) stimulates linear growth and improves nitrogen balance in many catabolic states, including sepsis, and in malnutrition associated with chronic obstructive pulmonary disease. In children with cystic fibrosis (CF), these anabolic effects could aid survival and enhance suitability for transplantation. We assessed response to 0.49 IU/kg/week of recombinant human growth hormone (hGH) for 6–12 months in seven children with cystic fibrosis. All showed an initial increase in height velocity (0.33-4.14 cm/year) and height standard deviation score (P<0.01). Knemometry also demonstrated a significant improvement in lower leg growth [P < 0.05). Weight velocity increased in five patients but there was no change in body mass index or skinfold thickness. The number of respiratory exacerbations decreased during therapy, although there was no measurable improvement in respiratory function. Growth hormone may be a useful adjunct therapy in cystic fibrosis.     

Implementation of preconceptional carrier screening for cystic fibrosis and haemoglobinopathies: a sociotechnical analysis

OBJECTIVE: To obtain more insight into the process of potential implementation of a screening program, which aims to identify carriers of cystic fibrosis and haemoglobinopathies before pregnancy, in order to enable couples at high risk of having a child with these disorders, to make informed reproductive decisions. METHODS: Use of sociotechnical analysis, based on a model of co-evolution between technology and society, and, for comparison, the study of the implementation processes of two already existing health care programs with similar aspects to the screening program at issue. RESULTS: Factors important for success appeared to be the existence of sociotechnical niches, in which technological options can be developed and studied in an experimental setting; a structural approach of providing information to future parents; a party that can articulate demand; governmental involvement in the attunement between various stakeholders; and a screening infrastructure in which large-scale DNA diagnostic services are available. CONCLUSIONS: Successful implementation of preconceptional carrier screening for cystic fibrosis and haemoglobinopathies will depend on changes at both regime and landscape level, including the establishment of a new preconceptional health care setting and a clearly visible public health authority which can coordinate, monitor and evaluate such an initiative in public health care.     

Appetite stimulants in cystic fibrosis: a systematic review

BACKGROUND: The use of appetite stimulants in cystic fibrosis (CF) is controversial because of doubts concerning efficacy and side-effects. The aim of the review was to establish whether appetite stimulants should be recommended in CF. MATERIALS AND METHODS: Medline, AMED, British Nursing Index, Cinahl, Embase, The Cochrane Library, National Research Register and Google were searched. Unpublished studies, case reports and conference abstracts were sought from experts, including authors of identified published articles, and suppliers of appetite stimulants. RESULTS: Fifty-four articles were located. Fifteen studies were suitable for inclusion in the review: 10 studied, megesterol acetate (MA); 1, dronabinol; 2, mirtazapine; and 2, cyproheptadine. MA showed a beneficial effect on lung function, weight, appetite, fat free and fat mass. Adverse effects for MA included adrenal suppression, abnormalities of glycaemic control, mood changes and testicular failure. There was no consistency in the dose, frequency and duration of therapy for MA. CONCLUSIONS: The review shows MA may have a role in the management of anorexia associated with CF. It is not possible to be conclusive for the other agents given the low numbers in the studies. Larger randomized, controlled trials of MA are necessary to confirm its safety and validate efficacy in CF.     

HCV感染患者肝脏纤维化255例队例研究

目的了解有偿献血者中丙型肝炎病毒(HCV)感染者肝脏纤维化状况和丙肝纤维化危险因素。方法选取河南省王营村有有偿献血史的单纯HCV感染者149例和丙型肝炎病毒/人类免疫缺陷病毒(HCV/HIV)混合感染者106例,进行回顾性队列研究和现场调查,采集血样进行HCV、HIV抗体、CD4、CD8T细胞检测,B超检查肝脏纤维化;采用COX回归分析比较HCV感染者肝脏纤维化的危险因素。结果 255例患者中,肝脏纤维化发生率为25.88%(66/255),不同性别、是否接受高效抗逆转录病毒治疗、CD4T和CD8T细胞计数、病毒感染类型的肝脏纤维化发生率差异均有统计学意义(P<0.05);以肝脏纤维化为结局,HCV混合感染组的中位生存时间比单纯HCV感染者早5.74年进入肝脏纤维化阶段,差异有统计学意义(χ2=47.41,P<0.01)。HCV/HIV混合感染是影响肝脏纤维化的风险因子(χ2=10.453,P<0.01)。结论与单纯HCV感染者比较,HCV/HIV混合感染能够增加肝脏纤维化发生危险性并加快其病程进程。     

Cost-of-illness study of pneumococcal disease in Italian children

Streptococcus pneumoniae (S pneumoniae, or pneumococcus) is a leading cause of illness in children, and causes illness and death among the elderly and persons with certain underlying conditions. A Cost-of-Illness (COI) estimate for each pneumococcal disease (meningitis, bacteremia, pneumonia, and otitis media) was determined using decision tree analysis that considered both direct and indirect costs. Information on the burden of pneumococcal disease in Italy, in terms of data on the incidence and seroprevalence of disease was collected from published and unpublished records, supplemented, and verified by Italian pediatric and infectious disease experts. The annual cost to society of caring for children with pneumococcal disease is estimated to be around 59,604,477 euro including both direct costs and indirect costs (productivity changes). Direct costs accounted for 39.9% of the total costs. The value of resources used to treat otitis media was 60.6% of the total direct costs; 31.9% was the value of resources for treating pneumonia; 6.5% for treating bacteremia; 1.0% for treating meningitis. A sensitivity analysis confirmed the robustness of the results.     

Fetuses with cystic hygroma. A retrospective study

BACKGROUND: The aim of this study is to analyse several theories of pathogenesis of cystic hygroma, its correlation with chromosomal abnormalities and the indicators of poor or good prognosis. METHODS: Experimental design: This is a retrospective study that evaluates all cases of cystic hygroma seen during the four-year period from January 1994 to December 1997. Setting: This study was performed in the center of prenatal diagnosis of institutional hospital in Caserta. Patients: All cases of fetuses with cystic hygroma were examined in 2100 pregnant female who visited the ambulatory. Interventions: The modality of diagnosis of this pathology and the presence of abnormal maternal serum levels of alpha-fetoprotein, human chorionic gonadotropin and unconjugated estriol were evaluated. Moreover, the presence of karyotype abnormalities or other non chromosomal abnormalities were also evaluated. Prognostic indicators such as the presence of septae seen by sonography were examined. RESULTS: Nine fetuses with cystic hygroma were diagnosed sonographically. Septae were identified in six cases. Chromosomal abnormalities were found in five cases. Two cases presented Turner's syndrome and one case Downs' syndrome. There were two cases with associated anomalies. The amniotic fluid alpha-fetoprotein (AFP) levels were high in all cases. CONCLUSIONS: Cystic hygroma is a malformation of the lymphatic system that is diagnosed by ultrasound very well from the first quarter of pregnancy. It is frequently associated with chromosomal and non chromosomal abnormalities. The presence of septae in it and amniotic fluid AFP levels are prognostic indicators.     

The perils of adding up: a cautionary tale about modeling and regression analysis.

To turn incomplete or imperfectly reliable information into estimates useful for policy, some modeling is often necessary or helpful. It does not follow that every statistical relation constitutes a model. There has to be an underlying theory, and the numerical estimates must respect any definitional or accounting identities that constrain the results. Ad hoc relations that do not meet these criteria can lead to uninterpretable or meaningless values, especially when different partial values, separately estimated, are added together. The imputed values of "responsiveness" in the WHO World Health Report 2000 illustrate this danger.     

Smoking-attributable mortality and years of potential life lost in 16 Brazilian capitals, 2003: a prevalence-based study

Background  

To establish the impact of tobacco smoking on mortality is essential to define and monitor public health interventions in developing countries.     

A cost-effectiveness analysis of rhDNase in children with cystic fibrosis

OBJECTIVES: This study compared the relative cost-effectiveness of daily recombinant human deoxyribonuclease (rhDNase), with alternate day rhDNase and hypertonic saline (HS) for treating children with cystic fibrosis (CF). METHODS: A randomized controlled trial with a crossover design allocated 40 CF children consecutively to 12 weeks of daily rhDNase, alternate day rhDNase, or HS. The primary outcome measure was forced expiratory volume in 1 second (FEV1), a measure of lung function. All health resource use was prospectively documented for each patient and multiplied by unit costs to give a total health service cost for each 12-week treatment period. The nonparametric bootstrap method was used to present cost-effectiveness acceptability curves and net benefit statistics for each treatment comparison, for various hypothetical levels of the decision maker's ceiling ratio. RESULTS: Compared with HS, there was a 14% improvement in FEV1 for daily rhDNase (95% Cl, 5% to 23%), and a 12% improvement (95% Cl, 2% to 22%) for alternate day rhDNase. For a ceiling ratio of 200 pounds sterling per 1% gain in FEV1, the mean net benefits of daily and alternate day rhDNase compared with HS were 1,158 pounds sterling (95% Cl, -621pounds sterling to 2,842) and 1,188 pounds sterling (95% Cl, -847 to 3,343), respectively; the mean net benefit of daily compared with alternate day rhDNase was -30 pounds sterling (95% Cl, -2,091 pounds sterling to 1,576). CONCLUSIONS: If decision makers are prepared to pay 200 pounds sterling for a 1% gain in FEV1 over a 12-week period, then on average either rhDNase strategy is cost-effective.     

Nitrogen dioxide concentrations in neighborhoods adjacent to a commercial airport: a land use regression modeling study

Background  

There is growing concern in communities surrounding airports regarding the contribution of various emission sources (such as aircraft and ground support equipment) to nearby ambient concentrations. We used extensive monitoring of nitrogen dioxide (NO₂) in neighborhoods surrounding T.F. Green Airport in Warwick, RI, and land-use regression (LUR) modeling techniques to determine the impact of proximity to the airport and local traffic on these concentrations.     

Enteral-tube feeding as adjunct therapy in malnourished patients with cystic fibrosis: a clinical study and literature review

Eight children (aged 8 mo to 13 yr) with cystic fibrosis (CF) and growth failure were given home nocturnal nasogastric feeding of an elemental diet for 3 mo and re-evaluated 3 mo after cessation of tube feeding. An increase in energy intake (p less than 0.05) resulted in increased serum transferrin (p less than 0.005), retinol-binding protein (p less than 0.05), and clinical scores (p less than 0.05) during the study. Height and growth velocity increased by 60% (p less than 0.05), and weight growth velocity increased by 63%. Arm-muscle circumference, triceps skinfolds, serum albumin, vitamin A, vitamin E, zinc, and copper did not change significantly during tube feeding. Nocturnal feeding were safe and effective in promoting growth; however, most children with severe lung disease did not sustain the gains after cessation of tube feeding. Our findings indicate that continued nutritional support is needed to maintain the growth in malnourished children with severe lung disease.