Treatment evaluation of combined modality therapy: 'what can we obtain today from phase II trials?'

Demonstration of activity is the major goal of Phase II studies. Other end points-duration of response, survival (relapse free in complete responders), progression free interval, changes in performance status and quality of life also provide useful information. From these data a decision to continue or discard a particular therapy can be considered. Various handicaps including variability in response reporting, heterogeneity of patient populations, inadequate reporting of failure patterns, causes of death, performance status changes and quality of life hinder interpretation of Phase II data. Nevertheless, Phase II studies have defined prognostic groups, beneficial changes in performance status and have helped formulate novel management approaches. Targeting particular prognostic groups, investigating dose-intensive regimens with haemopoietic growth-factor support and novel combined-modality approaches have all been generated through Phase II investigations.