title: A nonparametric comparison of the effectiveness of treatments: a multivariate toxicity-penalized approach.
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.
authors.affiliation: INSERM U 472, Villejuif, France. tubert@vjf.inserm.fr
authors.forename: Pascale
authors.initials: P
authors.lastname: Tubert-Bitter
authors.forename: Alexia
authors.initials: A
authors.lastname: Letierce
authors.forename: Daniel A
authors.initials: DA
authors.lastname: Bloch
authors.forename: Andrew
authors.initials: A
authors.lastname: Kramar
mesh_terms: D000970:Antineoplastic Agents / Q000633:toxicity; D064420:Drug-Related Side Effects and Adverse Reactions / Q000453:epidemiology*; D006801:Humans; D015999:Multivariate Analysis; D016032:Randomized Controlled Trials as Topic / Q000706:statistics & numerical data*; D018709:Statistics, Nonparametric
chemical_list: D000970:Antineoplastic Agents