Clinical decision making: from theory to practice. Applying cost-effectiveness analysis. The inside story.
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Biomedical subjects
Publications and source records attributed to D M Eddy.
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The use of meta-analysis is becoming more common in the medical literature, but it is not common in the environmental literature. Although meta-analysis cannot combine a group of poorly executed, conflicting studies to get an unequivocal answer, there are certain situations where it can be helpful. The inability of studies to produce similar results may be a function of the power of the studies rather than a reflection of their quality. The literature on the effects of nitrogen dioxide on the odds of respiratory illness in children is such an example. Three quantitative methods for the synthesis of this evidence are presented. Although the methods produce slightly different results, the conclusion from all three methods is that the increase in the odds of respiratory illness in children exposed to a long-term increase of 30 micrograms/m3 (comparable to the increase resulting from exposure to a gas stove) is about 20 percent. This estimated increase is not sensitive to the method of analysis.
PURPOSE: This review was undertaken to examine the evidence of effectiveness of high-dose chemotherapy and autologous bone marrow transplantation (HDC/ABMT) for the treatment of metastatic breast cancer and to compare the magnitudes of the benefits and harms of HDC/ABMT with those of conventional doses of chemotherapy. DESIGN: Published studies were reviewed and analyzed. RESULTS: No randomized controlled trials have been published that evaluate HDC/ABMT. Only one internally controlled study has been conducted; it demonstrated that HDC/ABMT and conventional treatment have virtually identical outcomes. Comparisons of uncontrolled clinical series are confounded by patient selection and other biases. Gross comparisons indicate that, compared with conventional-dose chemotherapy, HDC/ABMT achieves (1) higher complete response rates (36% v 8%), (2) higher overall response rates (70% v 39%), (3) similar median response durations (8 months v 9.6 months), (4) similar median survival durations (16 months v 16.6 months), and (5) similar overall survival rates (eg, 43% 2-year survival v 39%). Observations of cases with longer-term disease-free survival are promising but not conclusive. High-dose chemotherapy with ABMT has a higher treatment-related mortality rate (5% to 15% v 1%), a high rate of nonmortal toxicity (approximately 30%), and a high rate of side effects (approaching 100%). CONCLUSIONS: Firm conclusions are not possible because of the lack of controlled studies and the presence of numerous biases. However, the existing evidence does not demonstrate that HDC/ABMT is superior to conventional-dose chemotherapy for the treatment of metastatic breast cancer. Randomized controlled trials are needed.
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Considerable indirect evidence, based on the natural history of colorectal cancer and the ability of tests to detect adenomas and invasive cancers, suggests that screening for colorectal cancer reduces mortality. Without screening, a 50-year-old person at average risk has approximately a 530-in-10,000 chance of developing invasive colorectal cancer in the rest of his or her life and approximately a 250-in-10,000 chance of dying from it. Analysis of indirect evidence with a mathematic model indicates that screening persons for 25 years, from the age of 50 to the age of 75 years should reduce the chance of developing or dying from colorectal cancer by 10% to 75%, depending on which screening tests are used and how often screening is done. Screening for colorectal cancer is optional. A possible recommendation is that annual fecal occult blood tests and 65-cm flexible sigmoidoscopy every 3 to 5 years be done for average-risk men and women who are between 50 and 75 years of age. In addition to having annual fecal occult blood tests, persons with first-degree relatives with colorectal cancer can be offered barium enemas instead of sigmoidoscopies every 3 to 5 years.
Indirect evidence indicates that cervical cancer screening should reduce the incidence and mortality of invasive cervical cancer by about 90%. In the absence of screening, a 20-year-old average-risk woman has about a 250 in 10,000 chance of developing invasive cervical cancer during the rest of her life, and about a 118 in 10,000 chance of dying from it. Screening at least every 3 years from 20 to 75 years of age will decrease these probabilities by about 215 in 10,000 and 107 in 10,000, respectively, and will increase a 20-year-old woman's life expectancy by about 96 days. The particular age at which screening is begun (for example, 17 or 20 years), the requirement of several initial annual examinations before reducing the frequency, and screening every 1 or 2 years compared with every 3 years improves the effectiveness by less than 5%. Screening is recommended at least every 3 years from about age 20 to about age 65 years.
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