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A novel test to compare two treatments based on endpoints involving both nonfatal and fatal?events.

ABSTRACT: In a clinical trial comparing two treatment groups, one commonly-used endpoint is time to death. Another is time until the first nonfatal event (if there is one) or until death (if not). Both endpoints have drawbacks. The wrong choice may adversely affect the value of the study by impairing power if deaths are too few (with the first endpoint) or by lessening the role of mortality if not (with the second endpoint). We propose a compromise that provides a simple test based on the time to death if the patient has died or time since randomization augmented by an increment otherwise. The test applies the ordinary two-sample Wilcoxon statistic to these values. The formula for the increment (the same for experimental and control patients) must be specified before the trial starts. In the simplest (and perhaps most useful) case, the increment assumes only two values, according to whether or not the (surviving) patient had a nonfatal event. More generally, the increment depends on the time of the first nonfatal event, if any, and the time since randomization. The test has correct Type I error even though it does not handle censoring in a customary way. For conditions where investigators would face no easy (advance) choice between the two older tests, simulation results favor the new test. An example using a renal-cancer trial is presented.

SUBMITTER: Potthoff RF

PROVIDER: S-EPMC4503508 | biostudies-literature | 2015 Jul-Aug

REPOSITORIES: biostudies-literature

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A novel test to compare two treatments based on endpoints involving both nonfatal and fatal events.

Potthoff Richard F RF Halabi Susan S

Pharmaceutical statistics 20150420 4

In a clinical trial comparing two treatment groups, one commonly-used endpoint is time to death. Another is time until the first nonfatal event (if there is one) or until death (if not). Both endpoints have drawbacks. The wrong choice may adversely affect the value of the study by impairing power if deaths are too few (with the first endpoint) or by lessening the role of mortality if not (with the second endpoint). We propose a compromise that provides a simple test based on the time to death if ...[more]

PMID: 25894200

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Project description:Blacks have higher coronary heart disease (CHD) mortality compared with whites. However, a previous study suggests that nonfatal CHD risk may be lower for black versus white men.We compared fatal and nonfatal CHD incidence and CHD case-fatality among blacks and whites in the Atherosclerosis Risk in Communities study (ARIC), the Cardiovascular Health Study (CHS), and the Reasons for Geographic and Racial Differences in Stroke study (REGARDS) by sex. Participants 45 to 64 years of age in ARIC (men=6479, women=8488) and REGARDS (men=5296, women=7822), and ?65 years of age in CHS (men=1836, women=2790) and REGARDS (men=3381, women=4112), all without a history of CHD, were analyzed. Fatal and nonfatal CHD incidence was assessed from baseline (ARIC=1987-1989, CHS=1989-1990, REGARDS=2003-2007) through up to 11 years of follow-up.Age-adjusted hazard ratios comparing black versus white men 45 to 64 years of age in ARIC and REGARDS were 2.09 (95% confidence interval, 1.42-3.06) and 2.11 (1.32-3.38), respectively, for fatal CHD, and 0.82 (0.64-1.05) and 0.94 (0.69-1.28), respectively, for nonfatal CHD. After adjustment for social determinants of health and cardiovascular risk factors, hazard ratios in ARIC and REGARDS were 1.19 (95% confidence interval, 0.74-1.92) and 1.09 (0.62-1.93), respectively, for fatal CHD, and 0.64 (0.47-0.86) and 0.67 (0.48-0.95), respectively, for nonfatal CHD. Similar patterns were present among men ?65 years of age in CHS and REGARDS. Among women 45 to 64 years of age in ARIC and REGARDS, age-adjusted hazard ratios comparing blacks versus whites were 2.61 (95% confidence interval, 1.57-4.34) and 1.79 (1.06-3.03), respectively, for fatal CHD, and 1.47 (1.13-1.91) and 1.29 (0.91-1.83), respectively, for nonfatal CHD. After multivariable adjustment, hazard ratios in ARIC and REGARDS were 0.67 (95% confidence interval, 0.36-1.24) and 1.00 (0.54-1.85), respectively, for fatal CHD, and 0.70 (0.51-0.97) and 0.70 (0.46-1.06), respectively, for nonfatal CHD. Racial differences in CHD incidence were attenuated among older women. CHD case fatality was higher among black versus white men and women, and the difference remained similar after multivariable adjustment.After accounting for social determinants of health and risk factors, black men and women have similar risk for fatal CHD compared with white men and women, respectively. However, the risk for nonfatal CHD is consistently lower for black versus white men and women.

Project description:ImportanceFirearm injury research in the US has focused on fatal injuries. The incidence and epidemiologic factors associated with nonfatal firearm injuries are less understood.ObjectiveTo evaluate estimates of incidence and trends over time of fatal and nonfatal firearm injuries.Design, setting, and participantsA cross-sectional, ecologic study was conducted using data throughout the US from 2009 to 2017. Data on fatal injuries from the Centers for Disease Control and Prevention were combined with national data on emergency department visits for nonfatal firearm injury from the Nationwide Emergency Department (ED) sample. Data analysis was conducted from August 2019 to September 2020.ExposuresFirearm injuries identified with International Classification of Diseases external cause of injury codes and categorized by intent of injury, age group, and urban-rural location.Main outcomes and measuresIncidence, case fatality rate, and trends over time of firearm injury according to intent, age group, and urban-rural location.ResultsFrom 2009 to 2017, there was a mean of 85 694 ED visits for nonfatal firearm injury and 34 538 deaths each year. An annual mean of 26 445 deaths (76.6%) occurred outside of the hospital. Assault was the most common overall mechanism (38.9%), followed by unintentional injuries (36.9%) and intentional self-harm (19.6%). Self-harm, which accounted for 21 128 deaths (61.2%), had the highest case fatality rate (89.4%; 95% CI, 88.5%-90.4%), followed by assault (25.9%; 95% CI, 23.7%-28.6%) and legal intervention (23.4%; 95% CI, 21.6%-25.5%). Unintentional injuries were the most common nonfatal injuries (43 729 [51.0%]) and had the lowest case fatality rate (1.2%; 95% CI, 1.1%-1.3%). Self-harm deaths, 87.8% of which occurred outside the hospital, increased in all age groups in both rural and urban areas during the study period and were most common among people aged 55 years and older. The rate of fatal assault injuries was higher in urban than in rural areas (16.6 vs 9.0 per 100 000 per year) and highest among people aged 15 to 34 years (38.6 per 100 000 per year). Rates of unintentional injury were higher in rural than in urban areas (18.5 per 100 000 vs 12.4 per 100 000).Conclusions and relevanceIn this cross-sectional study, suicide appears to be the most common cause of firearm injury death in the US, and most people who die from suicide never reach the hospital. These findings suggest that assaults and unintentional injuries account for most nonfatal and overall firearm injuries and for most of the injuries that are treated in hospitals.

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A novel test to compare two treatments based on endpoints involving both nonfatal and fatal?events.

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A novel test to compare two treatments based on endpoints involving both nonfatal and fatal events.

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