On Mar 23, 2010, at 11:05 AM, Prew, Paul wrote:

> Hello,  I am looking for a sample size function for samples sizes, to test 
> proportions that are not binomial proportions.  The proportions represent a 
> ratio of (final measure) / (baseline measure) on the same experimental unit.  
> Searches using RSeek and such bring multiple hits for binomial proportions, 
> but that doesn't seem to fit my situation.  Perhaps there's some standard 
> terminology from a different field that would provide better hits than 
> deeming this a 'rate' or a 'proportion'.
> 
> Of course, most sample size functions assume a normal distribution, while 
> this data will be bounded between 0 and 1.  The scientist I'm working with 
> feels it's important to make fair comparisons, any weight loss must account 
> for the baseline weight.  A logistic transformation seems appropriate, but 
> that term also didn't yield hits I recognized as useful.
> 
> Loss of weight --- compare treatments:
> Treatment A:  1 - Final weight / Initial weight
> Treatment B:  1 - Final weight / Initial weight
> 
> This appears to be a situation that would be common, but I'm not framing it 
> in a way that matches an R package.  Any guidance is appreciated.
> 
> Regards, Paul


If you and the scientist are in a position of being open to better options of 
analyzing "change from baseline" data, I would recommend that you both read the 
following two papers:

 
Statistics notes: analysing controlled trials with baseline and follow up 
measurements. 
Vickers AJ, Altman DG.
BMJ 2001;323:1123–4.
http://www.bmj.com/cgi/content/full/323/7321/1123
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1121605/pdf/1123.pdf

 
The use of percentage change from baseline as an outcome in a controlled trial 
is statistically inefficient: a simulation study. 
Vickers AJ.
BMC Med Res Methodol 2001;1:6.
http://www.biomedcentral.com/1471-2288/1/6
http://www.biomedcentral.com/content/pdf/1471-2288-1-6.pdf


and review an additional web site:

  http://biostat.mc.vanderbilt.edu/wiki/Main/MeasureChange


Once you are hopefully in a position of adopting a regression based approach 
(eg. FinalWeight ~ BaseWeight + Treatment), there are various options for 
calculating sample sizes.  The key advantage of this approach is that you get 
the baseline adjusted between-group comparison (the regression beta coefficient 
and confidence intervals for Treatment) which is the key outcome of interest in 
comparing treatments in a parallel design.

The easiest, albeit conservative approach for sample size, is to use 
power.t.test() on your assumptions of the inter-group delta for actual weight 
change (not percent change), the std dev for actual change, desired power and 
target alpha. 

I am not aware off-hand of any power/sample size functions in R for regular 
linear regression, though they may exist. There are third party programs that 
do provide that functionality. 

If you are willing to code and experiment a bit, you could construct a monte 
carlo simulation with a linear model, using data generated with rnorm() based 
upon reasonable assumptions about the distribution of your data in each group 
for the baseline and final values.

Once you get your actual data collected and ready for analysis, you will also 
need to test for a baseline*treatment interaction (FinalWeight ~ BaseWeight * 
Treatment), which can make the interpretation of treatment effects more 
complicated, since the treatment effect will be conditional upon the baseline 
weight, rather than being able to report a mean treatment effect.

HTH,

Marc Schwartz

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