Cost-effectiveness of newborn screening for cystic fibrosis determined with real-life data.

BACKGROUND: Previous cost-effectiveness studies using data from the literature showed that newborn screening for cystic fibrosis (NBSCF) is a good economic option with positive health effects and longer survival. METHODS: We used primary data to compare cost-effectiveness of four screening strategies for NBSCF, i.e. immunoreactive trypsinogen-testing followed by pancreatitis-associated protein-testing (IRT-PAP), IRT-DNA, IRT-DNA-sequencing, and IRT-PAP-DNA-sequencing, each compared to no-screening. A previously developed decision analysis model for NBSCF was fed with model parameters mainly based on a study evaluating two novel screening strategies among 145,499 newborns in The Netherlands. RESULTS: The four screening strategies had cost-effectiveness ratios varying from €23,600 to €29,200 per life-year gained. IRT-PAP had the most favourable cost-effectiveness ratio. Additional life-years can be gained by IRT-DNA but against higher costs. When treatment costs reduce with 5% due to early diagnosis, screening will lead to financial savings. CONCLUSION: NBSCF is as an economically justifiable public health initiative. Of the four strategies tested IRT-PAP is the most economic and this finding should be included in any decision making model, when considering implementation of newborn screening for CF.

Newborn screening for cystic fibrosis.

Newborn screening for cystic fibrosis (CF) was considered over 3 decades ago in 1970; however, the technology did not exist then for an accurate neonatal screening test. With the development of immunoreactive trypsinogen analysis, alone or coupled with DNA mutation analysis, the means were developed for CF newborn screening. Studies have demonstrated benefits of newborn screening in the areas of nutrition, cognitive function, pulmonary function, and survival.

Analysis of the costs of diagnosing cystic fibrosis with a newborn screening program.

OBJECTIVES: To compare the cost of diagnosing cystic fibrosis (CF) through a newborn screening program with the traditional method and to estimate the cost of CF diagnosis if a national newborn screening program is implemented. STUDY DESIGN: Surveys were conducted to determine the annual number of sweat tests in 1991 and in 2000 after implementation of statewide screening. A national survey of sweat test costs was used to estimate the annual expense for diagnosing CF in the United States through newborn screening. RESULTS: Since the introduction of newborn screening for CF, the numbers of sweat tests ordered annually have decreased from 1670 to 804 (including 134 follow-up tests from screening). The current estimated annual cost of Wisconsin CF newborn screening and diagnosis is $4.58 per newborn infant. The estimated annual cost per newly diagnosed CF infant using the traditional method is $4.97 per newborn infant. If no additional sweat tests were ordered outside of the newborn screening program, the estimated annual cost of a Wisconsin CF newborn screening and diagnosis is $2.66 per newborn and $2.47 per newborn for a national CF newborn screening program. CONCLUSIONS: A CF newborn screening program provides a potentially cost-saving alternative to the traditional method of diagnosis of CF.

The usefulness of laboratory tests in the early assessment of severity of acute pancreatitis.

Acute pancreatitis is a disorder that affects approximately 200,000 individuals in the U.S. annually. While most cases are mild, up to 30% of patients will have a complicated course with prolonged hospitalization and significant morbidity and mortality. Early institution of several therapeutic interventions, such as enteral nutrition, prophylactic antibiotics, endoscopic retrograde cholangiopancreatography (ERCP) and intensive care monitoring, have been shown to decrease the morbidity associated with severe acute pancreatitis. However, the ability of clinicians to predict, upon presentation, which patient will have mild or severe pancreatitis has remained poor over the years, thus leading to a delay in the institution of such treatments. Researchers have focused on markers that might improve upon clinical prediction alone. While data have shown the predictive value of tools such as Ranson's and Glasgow's criteria, C-reactive protein (CRP) and the APACHE score, their application in clinical practice has been limited by a time delay of at least 48 h in the former two and by being cumbersome in the latter. Thus, our focus is to critically appraise the evidence available for various biochemical markers in their ability to distinguish mild and severe acute pancreatitis early and more accurately than currently available tools.

Assessment of the role of pancreatic proteases in human abdominal aortic aneurysms and occlusive disease.

Previous studies on the pathogenesis of abdominal aortic aneurysms have shown both elastase-like activity in the aortic wall and a decreased elastin content. The present study, using specific radioimmunoassays for pancreatic elastase 2 (IRE2) and cationic trypsin(ogen) (IRCT), investigates the concentrations of these proteases which are known to circulate in blood, in abdominal aortic aneurysms. Aortic specimens were obtained from 32 patients with aneurysms and 21 patients with atherosclerotic occlusive disease. Aortic tissue, obtained at autopsy from young adults, served as controls. Elastase-like activity was 300% and 800% higher, respectively, in aortic homogenates from aneurysms in comparison to occlusive disease and control aortic tissue. This was associated with 1.4-fold higher level of IRE2 and 2.7-fold higher levels of IRCT as compared to occlusive disease. Although there was no significant difference in the aortic collagen concentration among all 3 groups, the elastin content of aneurysmal aorta was 85% and 74% lower, respectively, in comparison to control and occlusive aorta. The results of this investigation demonstrate the presence of pancreatic elastase 2 and cationic trypsin(ogen) in abdominal aortic aneurysmal tissue and suggest that circulating pancreatic proteases contribute to the pathophysiology of aneurysms of the infrarenal aorta.