Survival of patients in a Phase 1 Clinic: the M. D. Anderson Cancer Center experience. Cancer 2009 Mar 01;115(5):1091-9
Date
01/24/2009Pubmed ID
19165805DOI
10.1002/cncr.24018Scopus ID
2-s2.0-61449180271 (requires institutional sign-in at Scopus site) 68 CitationsAbstract
BACKGROUND: 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.
METHODS: 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.
RESULTS: 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 >or=440 x 10(9)/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).
CONCLUSIONS: 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.
Author List
Wheler J, Tsimberidou AM, Hong D, Naing A, Jackson T, Liu S, Feng L, Kurzrock RAuthor
Razelle Kurzrock MD Center Associate Director, Professor in the Medicine department at Medical College of WisconsinMESH terms used to index this publication - Major topics in bold
AdolescentAdult
Aged
Aged, 80 and over
Antineoplastic Agents
Child
Clinical Trials, Phase I as Topic
Drug Delivery Systems
Female
Humans
Liver Neoplasms
Male
Middle Aged
Neoplasms
Prognosis
Retrospective Studies
Risk Assessment
Survival Analysis
Thrombocytosis
Thromboembolism
Treatment Outcome
