Related Experiment Video
Updated: Apr 27, 2026
Automated Multiplex Immunofluorescence Panel for Immuno-oncology Studies on Formalin-fixed Carcinoma Tissue Specimens
Published on: January 21, 2019
Personalized medicine for patients with advanced cancer in the phase I program at MD Anderson: validation and
Apostolia-Maria Tsimberidou1, Sijin Wen2, David S Hong3
1Department of Investigational Cancer Therapeutics, Phase I Clinical Trials Program, The University of Texas MD Anderson Cancer Center, Houston, Texas. atsimber@mdanderson.org.
Purpose:
The purpose of this study was to confirm our previous results that targeted agents matched with tumor molecular alterations were associated with improved outcomes compared with nonmatched therapy in patients with advanced cancer.
Experimental Design:
Outcomes of patients who were referred for treatment on phase I clinical trials at The University of Texas MD Anderson Cancer Center (Houston, TX) from March 2011 to January 2012 were compared between those who had received targeted therapy and those for whom no targeted therapy was available. Two-month landmark analyses for overall and progression-free survival (PFS) combining previously published and validation cohort patient data were performed.
Results:
In patients with one alteration, matched therapy (n = 143) compared with treatment without matching (n = 236) was associated with a higher objective response rate (12% vs. 5%; P < 0.0001), longer PFS (median, 3.9 vs. 2.2 months; P = 0.001), and longer survival (median, 11.4 vs. 8.6 months; P = 0.04). In multivariate analysis, matched therapy was an independent factor predicting response (P < 0.015) and PFS (P < 0.004). Two-month landmark analyses in the matched therapy group demonstrated that the median survival of responders was 30.5 months compared with 11.3 months for nonresponders (P = 0.01); and the median PFS was 38.7 months compared with 5.9 months, respectively (P < 0.0001). The respective values in the nonmatched therapy group were 9.8 and 9.4 months (P = 0.46) and 8.5 and 4.2 months (P = 0.18).
Conclusion:
This validation analysis confirms our previous observations. In the matched therapy group, 2-month landmark analyses demonstrated that responders have longer survival and PFS than nonresponders.
Insights
Targeted therapy matched with tumor molecular alterations significantly improves outcomes for advanced cancer patients. This validation study confirms that matched therapy leads to better response rates, progression-free survival (PFS), and overall survival compared to nonmatched treatments.
Area of Science:
- Oncology
- Molecular Biology
- Clinical Trials
Background:
- Targeted therapies offer personalized treatment strategies for cancer.
- Previous research suggested improved outcomes when targeted agents align with tumor molecular alterations.
Purpose of the Study:
- To validate previous findings on the association between matched targeted therapy and improved patient outcomes.
- To compare outcomes of advanced cancer patients receiving matched versus nonmatched targeted therapy.
Main Methods:
- Retrospective analysis of patients on phase I clinical trials (March 2011-January 2012).
- Comparison of outcomes between patients receiving targeted therapy and those without available targeted therapy.
- Two-month landmark analyses for overall survival and progression-free survival (PFS) were conducted.
Main Results:
- Matched therapy (n=143) showed higher objective response rates (12% vs 5%), longer PFS (median 3.9 vs 2.2 months), and longer survival (median 11.4 vs 8.6 months) compared to nonmatched therapy (n=236).
- Matched therapy was an independent predictor of response and PFS in multivariate analysis.
- In the matched group, responders had significantly longer survival (30.5 vs 11.3 months) and PFS (38.7 vs 5.9 months) than nonresponders at 2-month landmark analysis.
Conclusions:
- This validation analysis confirms that targeted agents matched with tumor molecular alterations improve outcomes in advanced cancer.
- Matched therapy is associated with superior response rates, PFS, and overall survival.
- Early responders to matched therapy demonstrate substantially prolonged survival and PFS.
