Clinical outcome and prognostic markers for patients with gynecologic malignancies in phase 1 clinical trials: A single institution experience from 1999 to 2010

June Y. Hou, Santiago Aparo, Mohammad Ghalib, Imran Chaudhary, Umang Shah, Umang Swami, Mark Einstein, Gary L. Goldberg, Sridhar Mani, Sanjay Goel

Research output: Contribution to journalArticle

2 Scopus citations


Objectives. There is a scarcity of outcome data regarding phase 1 trials for patientswith gynecologic malignancy. The objective of this study was to assess toxicity, clinical benefit and prognosticators in gynecologic oncology patients participating in phase 1 trials. Methods. All phase 1 oncology trials conducted at Albert Einstein Cancer Center from 1999 to 2010 were reviewed and extracted for relevant demographic and clinical data concerning patients with gynecologic malignancy. Cox-proportional and logistic regression modeling were used for multivariate analysis. Results. 120 distinct patients with gynecologic malignancy participated in 41 trials, constituting 30.6% of all phase 1 patients enrolled in the same time period. The median age is 59 years. Out of the 184 patients enrolled, 160 individual responses were evaluable. Seventeen DLT events (9.2%) occurred, including 1 (0.5%) treatmentrelatedmortality. There were 27.2% ≥ grade 3 hematologic and 24.4% non-hematologic toxicity. Eighty patients had stable disease (SD, 50%), including 21.9% with SD ≥ 4 months, 11 (6.3%) with partial response (PR), and 3 (1.9%) achieving complete response (CR). The clinical benefit rate (CBR = SD + CR + PR) was 58.1%. Albumin (Alb) ≤ 3.5 g/dL and abnormal ANC were independent negative prognosticators of survival. We also found a continuous correlation between changes in Albumin (p = 0.02) and LDH (p = 0.02) and odds of achieving CBR ≥ 4 month. Conclusions. Our clinical outcome and safety data suggested that phase 1 trials may be a reasonable option for patients with advanced and recurrent gynecologic cancer. The potential prognosticators identified should be further validated in larger trials.

Original languageEnglish (US)
Pages (from-to)163-168
Number of pages6
JournalGynecologic Oncology
Issue number1
StatePublished - Oct 2013



  • Clinical trials
  • Gynecologic cancer
  • Phase 1
  • Prognosticator
  • Survival
  • Toxicity

ASJC Scopus subject areas

  • Oncology
  • Obstetrics and Gynecology

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