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Patient Selection for Oncology Phase I Trials: A Multi-Institutional Study of Prognostic Factors

Lookup NU author(s): Professor Ruth Plummer


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PurposeThe appropriate selection of patients for early clinical trials presents a major challenge. Previous analyses focusing on this problem were limited by small size and by interpractice heterogeneity. This study aims to define prognostic factors to guide risk-benefit assessments by using a large patient database from multiple phase I trials.Patients and MethodsData were collected from 2,182 eligible patients treated in phase I trials between 2005 and 2007 in 14 European institutions. We derived and validated independent prognostic factors for 90-day mortality by using multivariate logistic regression analysis.ResultsThe 90-day mortality was 16.5% with a drug-related death rate of 0.4%. Trial discontinuation within 3 weeks occurred in 14% of patients primarily because of disease progression. Eight different prognostic variables for 90-day mortality were validated: performance status (PS), albumin, lactate dehydrogenase, alkaline phosphatase, number of metastatic sites, clinical tumor growth rate, lymphocytes, and WBC. Two different models of prognostic scores for 90-day mortality were generated by using these factors, including or excluding PS; both achieved specificities of more than 85% and sensitivities of approximately 50% when using a score cutoff of 5 or higher. These models were not superior to the previously published Royal Marsden Hospital score in their ability to predict 90-day mortality.ConclusionPatient selection using any of these prognostic scores will reduce non-drug-related 90-day mortality among patients enrolled in phase I trials by 50%. However, this can be achieved only by an overall reduction in recruitment to phase I studies of 20%, more than half of whom would in fact have survived beyond 90 days. J Clin Oncol 30: 996-1004. (C) 2012 by American Society of Clinical Oncology

Publication metadata

Author(s): Olmos D, A'Hern RP, Marsoni S, Morales R, Gomez-Roca C, Verweij J, Voest EE, Schoffski P, Ang JE, Penel N, Schellens JH, del Conte G, Brunetto AT, Evans TRJ, Wilson R, Gallerani E, Plummer R, Tabernero J, Soria JC, Kaye SB

Publication type: Article

Publication status: Published

Journal: Journal of Clinical Oncology

Year: 2012

Volume: 30

Issue: 9

Pages: 996-1004

Print publication date: 21/02/2012

ISSN (print): 0732-183X

ISSN (electronic): 1527-7755

Publisher: American Society of Clinical Oncology


DOI: 10.1200/JCO.2010.34.5074


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Funder referenceFunder name
ASCO Cancer Foundation
Cancer Research UK
Departments of Health
Drug Development Unit of the Royal Marsden National Health Service (NHS) Foundation
ESMO Foundation
European Drug Development Network (EDDN) centres
Institute of Cancer Research
110722Experimental Cancer Medicine Centre
CA51/A7401National Institute of Health Research Biomedical Research Centre