Fax: (713) 563-0566
Survival of patients in a Phase 1 clinic
The M. D. Anderson Cancer Center Experience
Version of Record online: 22 JAN 2009
Copyright © 2009 American Cancer Society
Volume 115, Issue 5, pages 1091–1099, 1 March 2009
How to Cite
Wheler, J., Tsimberidou, A. M., Hong, D., Naing, A., Jackson, T., Liu, S., Feng, L. and Kurzrock, R. (2009), Survival of patients in a Phase 1 clinic. Cancer, 115: 1091–1099. doi: 10.1002/cncr.24018
- Issue online: 18 FEB 2009
- Version of Record online: 22 JAN 2009
- Manuscript Accepted: 7 AUG 2008
- Manuscript Revised: 27 JUL 2008
- Manuscript Received: 15 MAY 2008
- phase 1;
- predictive factors;
- targeted therapies;
- biologic agents;
- liver metastases
Patients with advanced malignancies for whom standard therapy is ineffective may participate in phase 1 trials. To gain a better understanding of the clinical features that could influence benefit versus risk, the authors of this report assessed prognostic factors and survival for patients who were referred to a phase 1 clinic focused primarily on targeted agents.
The medical records of 200 sequential patients who presented to the Phase 1 Clinic at The University of Texas M. D. Anderson Cancer Center were reviewed, and their characteristics and survival were analyzed.
The median patient age was 58 years (range, 12-85 years), and 57% of patients were men. The median number of prior therapies was 4. Of 200 patients, 182 were treated on at least 1 phase 1 clinical trial. The median follow-up of surviving patients was 21 months, and the median overall survival was 9 months (95% confidence interval [CI], 7.4-10.8). In univariate analysis, the factors that predicted shorter survival were primary tumor in the gastrointestinal tract; a history of thrombosis, liver metastases, and elevated levels of serum lactate dehydrogenase; platelet count; carbohydrate antigen 9 (Ca19-9) and Ca-125 levels; aspartate aminotransferase levels, and alkaline phosphatase levels (P < .05 for each). In multivariate analysis, independent factors that predicted shorter survival were a history of thromboembolism (hazard ratio [HR], 2.38; 95% CI, 1.29-4.39; P = .005), platelets ≥440 × 109/L (HR, 1.72; 95% CI, 1.12-2.65; P = .014), and the presence of liver metastases (HR, 1.51; 95% CI, 1.09-2.09; P = .013).
Patients who were referred to phase 1 studies had a short median survival (9 months). Patients with thrombocytosis, liver metastases, and a history of thromboembolism had worse outcomes. A prognostic score is proposed. Cancer 2009. © 2009 American Cancer Society.