The relationship between baseline value and its change: problems in categorization and the proposal of a new method.

Oral health researchers have shown great interest in the relationship between the initial status of diseases and subsequent changes following treatment. Two main approaches have been adopted to provide evidence of a positive association between baseline values and their changes following treatment. One approach is to use correlation or regression to test the relationship between baseline measurements and subsequent change (correlation/regression approach). The second approach is to categorize the lesions into subgroups, according to threshold values, and subsequently compare the treatment effects across the two (or more) subgroups (categorization approach). However, the correlation/regression approach suffers a methodological weakness known as mathematical coupling. Consequently, the statistical procedure of testing the null hypothesis becomes inappropriate. Categorization seems to avoid the problem of mathematical coupling, although it still suffers regression to the mean. We show, first, how the appropriate null hypothesis may be established to analyze the relationship between baseline values and change in the correlation approach and, second, we use computer simulations to investigate the impact of regression to the mean on the significance testing of the differences in the average treatment effects (or average baseline values) in the categorization approach. Data available from previous literature are reanalyzed by testing the appropriate null hypotheses and the results are compared to those from testing the usual (incorrect) null hypothesis. The results indicate that both the correlation and categorization approaches can give rise to misleading conclusions and that more appropriate methods, such as Oldham's method and our new approach of deriving the correct null hypothesis, should be adopted.