Phase I clinical trials in 56 patients with thyroid cancer: the M. D. Anderson Cancer Center experience.

Academic Article


  • INTRODUCTION: Thyroid cancer is the most common endocrine malignancy. The outcomes of patients with relapsed thyroid cancer treated on early-phase clinical trials have not been systematically analyzed. PATIENTS AND METHODS: We reviewed the records of consecutive patients with metastatic thyroid cancer referred to the Phase I Clinical Trials Program from March 2006 to April 2008. Best response was assessed by Response Evaluation Criteria in Solid Tumors. RESULTS: Fifty-six patients were identified. The median age was 55 yr (range 35-79 yr). Of 49 patients evaluable for response, nine (18.4%) had a partial response, and 16 (32.7%) had stable disease for 6 months or longer. The median progression-free survival was 1.12 yr. With a median follow-up of 15.6 months, the 1-yr survival rate was 81%. In univariate analysis, factors predicting shorter survival were anaplastic histology (P = 0.0002) and albumin levels less than 3.5 g/dl (P = 0.05). Among 26 patients with tumor decreases, none died (median follow-up 1.3 yr), whereas 52% of patients with any tumor increase died by 1 yr (P = 0.0001). The median time to failure in our phase I clinical trials was 11.5 months vs. 4.1 months for the previous treatment (P = 0.04). CONCLUSION: Patients with advanced thyroid cancer treated on phase I clinical trials had high rates of partial response and prolonged stable disease. Time to failure was significantly longer on the first phase I trial compared with the prior conventional treatment. Patients with any tumor decrease had significantly longer survival than those with any tumor increase.
  • Keywords

  • Aged, Analysis of Variance, Clinical Trials, Phase I as Topic, Creatinine, Disease-Free Survival, Female, Follow-Up Studies, Humans, Male, Middle Aged, Retrospective Studies, Survival Rate, Survivors, Thyroid Neoplasms
  • Digital Object Identifier (doi)

    Author List

  • Tsimberidou AM; Vaklavas C; Wen S; Hong D; Wheler J; Ng C; Naing A; Tse S; Busaidy N; Markman M
  • Start Page

  • 4423
  • End Page

  • 4432
  • Volume

  • 94
  • Issue

  • 11