Clinical Outcomes of Patients With Gastrointestinal Malignancies Participating in Phase I Clinical Trials

Aaron Denson, Nancy Burke, Georgine Wapinsky, Barbara Bertels, Tzu Hua Juan, Jae Lee, Gregory M. Springett, Jonathan R. Strosberg, Richard D. Kim, Dan M. Sullivan, Amit Mahipal

Research output: Contribution to journalArticlepeer-review

3 Scopus citations


Objectives: Early-phase clinical trials play a pivotal role in drug development. However, limited data are available on outcomes of gastrointestinal (GI) cancer patients enrolled in phase I clinical trials. Here, we evaluated the characteristics associated with survival in GI cancer patients participating in phase I clinical trials and attempted to validate previously established prognostic models. Materials and Methods: All consecutive patients with advanced GI tumors who participated in phase I clinical trials at our institution from January 2007 to December 2013 and received at least 1 dose of the study drug were included. Cox regression models were used to estimate multivariable-adjusted hazard ratio (HR) and 95% confidence interval. Results: In 243 study patients (median age, 62 y [range, 26 to 82 y]; 55% male), treatment included chemotherapy only (14%), targeted therapy (41%), chemotherapy + targeted therapy (42%), and others (2%) for the following disease types: pancreatic (42%), colorectal (34%), gastroesophageal (10%), hepatobiliary (13%), and others (2%). Response rate was 4%, with 38% achieving stable disease and 42% having progressive disease. Median survival was 5.8 months (range, 0.2 to 52.4 mo). Our multivariable Cox regression analyses included the following as predictors of survival: Eastern Cooperative Oncology Group performance score ≥ (HR = 1.76), prior systemic therapies ≥ (HR = 1.63), lactate dehydrogenase >618 IU/L (HR = 1.85), sodium >135 mmol/L (HR = 0.46), and white blood count >6×109/L (HR = 1.5). Our data set was consistent with previous prognostic scores. Conclusions: This is the largest study to assess clinical outcomes in this patient population. Phase I trials provide clinical benefit to patients with advanced GI malignancies and should be recommended as a treatment option in appropriate patients.

Original languageEnglish (US)
Pages (from-to)133-139
Number of pages7
JournalAmerican Journal of Clinical Oncology: Cancer Clinical Trials
Issue number2
StatePublished - 2018


  • clinical trial
  • clinical trial
  • gastrointestinal neoplasms
  • phase I
  • survival
  • therapeutics

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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