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A nonparametric comparison of the effectiveness of treatments: a multivariate toxicity-penalized approach
Pascale Tubert-Bitter1, Alexia Letierce, Daniel A Bloch
1INSERM U 472, Villejuif, France. tubert@vjf.inserm.fr
Abstract:
In cancer clinical trials, the amount of treatment dose actually received by a patient may be limited by severe toxicity or lack of efficacy. A nonparametric approach is proposed for comparing the effectiveness of treatments based on the bivariate relationship defined by the doses at which efficacy and toxicity are observed to occur. Simulation studies are used to contrast the performance of the new procedure with the usual method of comparing percentages of patients who have effective results. Data from a randomized clinical trial of patients with metastatic nonseminomatous germ cell tumors are used to illustrate the method.
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