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

J Clin Endocrinol Metab. 2009 Nov;94(11):4423-32. doi: 10.1210/jc.2009-0743. Epub 2009 Oct 9.


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.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Aged
  • Analysis of Variance
  • Clinical Trials, Phase I as Topic / statistics & numerical data*
  • Creatinine / blood
  • Disease-Free Survival
  • Female
  • Follow-Up Studies
  • Humans
  • Male
  • Middle Aged
  • Retrospective Studies
  • Survival Rate
  • Survivors
  • Thyroid Neoplasms / mortality
  • Thyroid Neoplasms / therapy*


  • Creatinine