Advanced Thyroid Cancer Patients in Phase I Clinical Trials: Outcome Assessment and Literature Review

BACKGROUND: To describe the basic demographics, analyze the response and survival experience of advanced thyroid cancer subjects treated in Phase I clinical trials. We also reviewed early Phase studies using new targeted agents in thyroid cancer. METHODS: We conducted a retrospective observational study in 32 advanced thyroid cancer patients who participated in 21 Phase I trials at our center between 2002 and 2012. Descriptive statistics and Kaplan Meier analyses were used to examine clinical outcomes and survival. RESULTS: The median age of subjects was 57.5 years (range 21-81) at the time of study enrollment; more men (53.1%) than women were enrolled. A large number of study participants were Hispanic Americans. Nearly half (46.8%) of patients had ECOG performance status of zero, 53.1% were ECOG one and above. The most common histological subtypes were papillary (37.5%), medullary (28.1%), follicular (18.7) and anaplastic (15.6%). More than half of patients had ≥ 2 metastatic sites (62.6%). Of the 30 evaluable patients for tumor response, 2 confirmed partial responses (6.6%) were observed, whereas 17 patients had stable disease (SD) as best response. Among patients with stable disease, 10 patients (33.3%) achieved SD lasting ≥ 6 months. The median overall survival from the time of enrollment in a Phase I trial was 16.1 months. No treatment related death was observed among these patients treated with an investigational agent. CONCLUSION: Advanced thyroid cancer patients with no standard treatment options could participate in early Phase clinical trials of novel agents.