Sample size in obesity trials: patient perspective versus current practice.
OBJECTIVE
To evaluate patient opinions on acceptable risks in exchange for a given degree of weight loss and their implications for sample size determination in obesity randomized clinical trials (RCTs).


METHODS
. Survey of patients entering RCTs for weight loss in a university-based clinical research setting and power calculations based on their responses. Participants. Men (n = 8) and women (n = 66) between 24 and 73 years of age with body mass indices ranging from 26.8 to 40.5 kg/m(2). Measurements. Survey responses to questions assessing the added risk of serious adverse events (SAEs) or death one is willing to assume for a given degree of weight loss.


RESULTS
For 5% and 10% weight loss against risk for death per se, the mean acceptable risk tended to be about 3.5%, but the median (0.00) and mode (0.00) suggested that for most individuals, only a risk of < or = 1% would be acceptable. Figures, estimated dropout rates, and base rates of SAEs (including deaths) from recent obesity trials indicate that 1-year 2-group obesity RCTs would need tens of thousands of participants per group to have 80% power to detect risks that are meaningful to patients at the 2-tailed 0.05 alpha level.


CONCLUSIONS
Patient education is needed to explain which risks are realistically detectable in RCTs so that patients may provide truly informed consent, or RCT standards should be modified to meet patients' implicit expectations.
Allison
David B
DB
Department of Nutrition Sciences and the Clinical Nutrition Research Center, University of Alabama at Birmingham, Birmingham, AL 35294, USA. dallison@uab.edu
Elobeid
Mai A
MA
Cope
Mark B
MB
Brock
David W
DW
Faith
Myles S
MS
Vander Veur
Stephanie
S
Berkowitz
Robert
R
Cutter
Gary
G
McVie
Theresa
T
Gadde
Kishore M
KM
Foster
Gary D
GD
D000328:Adult; D000368:Aged; D003625:Data Collection; D005260:Female; D006801:Humans; D007258:Informed Consent; D008297:Male; D008875:Middle Aged; D009765:Obesity; D010361:Patients / Q000523:psychology*; D016032:Randomized Controlled Trials as Topic; D018570:Risk Assessment; D018401:Sample Size; D015431:Weight Loss / Q000502:physiology; D055815:Young Adult
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