Wilms’ Tumour: Trials and Tribulation

Abstract

As the treatment of any medical condition becomes more effective, it is difficult to make further progress in management. Where there is general agreement over the role of treatment modalities, the subject of trials and their design, which might give further insight into therapy, becomes more important; yet for rare lesions individual units are unable to gain sufficient accrual of patients to perform what would be regarded as an acceptable clinical trial. Where the subject under investigation has a very low incidence in the population, it may be impossible to organise a comparative clinical trial as such on a national basis, as is the case in the United Kingdom for patients with Wilms’ tumour. Areas with larger populations, such as the United States or mainland Europe, have been able to arrange trials comparing two treatments concurrently, but, with large numbers of patients in different centres, the control of the study and assessment of individuals does become a serious problem. Central collation of data, as in the United Kingdom Children’s Cancer Study Group (UKCCSG), often reveals minor irregularities in treatment protocols when close scrutiny is made of each case, which are insufficient to withdraw a patient from the study but may explain some of the minor variations in results. The dangers of assessing current therapy against historical data are well illustrated by the different results obtained from the same chemotherapy protocol for stage II, stage III, and stage IV patients with Wilms’ tumour in the first national study in the United States, and the poorer results from the second national study [1].

… For though the father sets the price, the children pay the cost.

Don McLean