Clinical Research Papers:
Predictors of early treatment discontinuation in patients enrolled on Phase I oncology trials
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Abstract
David M. Hyman1,4,*, Anne A. Eaton2,*, Mrinal M. Gounder1,4, Erika G. Pamer1, Jasmine Pettiford1, Richard D. Carvajal1,4, S. Percy Ivy3, Alexia Iasonos2,4, David R. Spriggs1,4
1Developmental Therapeutics, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY 10065, USA
2Department of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, NY 10065, USA
3The National Cancer Institute, Bethesda, MD 20892, USA
4Weill Cornell Medical College, New York, NY 10065, USA
*These authors have contributed equally to this work
Correspondence to:
David M. Hyman, e-mail: [email protected]
Keywords: Phase I trials, Early Discontinuation, Drug Development
Received: October 21, 2014 Accepted: December 11, 2014 Published: February 17, 2015
ABSTRACT
Purpose
Patients who do not complete one cycle of therapy on Phase I trials for reasons other than dose limiting toxicity (DLT) are considered inevaluable for toxicity and must be replaced.
Methods
Individual records from patients enrolled to NCI-sponsored Phase I trials activated between 2000 and 2010 were used. Early discontinuation was defined as the failure to begin cycle 2 for reasons other than a DLT during cycle 1. A multinomial logistic regression with a 3-level nominal outcome (early discontinuation, DLT during cycle 1, and continuation to cycle 2) was used with continuation to cycle 2 serving as the reference category. The final model was used to create two risk scores. An independent external cohort was used to validate these models.
Results
Data from 3079 patients on 127 Phase I trials were analyzed. ECOG performance status (1, ≥ 2, two-sided P = .0315 and P = .0007), creatinine clearance (<60 ml/min, P = .0455), alkaline phosphatase (>2.5xULN, P = .0026), AST (>ULN, P = .0076), hemoglobin (<10 g/dL, P < .0001), albumin (<3.5 g/dL, P < .0001), and platelets (<400x109/L, P = .0732) were predictors of early discontinuation. The c-index of the final model was 0.63.
Conclusion
Knowledge of risk factors for early treatment discontinuation in conjunction with clinical judgment can help guide Phase I patient selection.
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