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Updated: Oct 20, 2025

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
Outcomes after targeted treatment based on somatic tumor genetic testing for women with gynecologic cancers
Sahana Somasegar1, Claire Hoppenot1, Kristine Kuchta2
1Department of Obstetrics and Gynecology, University of Chicago Medicine, Chicago, IL, United States of America.
Objective:
Molecular tumor profiling and next-generation sequencing are being increasingly utilized, but there are limited data on the therapeutic implications and potential benefits of targeted treatments. We aim to characterize gynecologic oncology patients referred for somatic tumor genetic mutation testing and assess survival outcomes, efficacy, and toxicities of those receiving targeted therapy.
Methods:
We conducted a retrospective chart review of gynecologic oncology patients referred for somatic tumor testing by next generation sequencing between 1/1/2012-8/23/2019. The primary objective was to compare overall and progression free survival between those treated with targeted therapy (group 1) versus traditional treatment (group 2).
Results:
Most patients (70%) had additional treatment options available based on actionable mutations. The median number of somatic mutations identified was 5 (range 0-53). Patients in group 1 had more actionable somatic mutations (median 2 versus 0, p < 0.001). There was no difference in OS (median 64 versus 76 months, p = 0.97) or PFS (median 2 versus 8 months, p = 0.05) between the groups. While fewer patients in group 1 experienced neuropathy (0 versus 5, p = 0.02), grade I/II thrombocytopenia (7 versus 13, p = 0.03), grade III/IV thrombocytopenia (0 versus 4, p = 0.02), and grade III/IV neutropenia (1 versus 9, p = 0.002), all other non-hematologic toxicities were similar in the two groups.
Conclusions:
Most gynecologic cancer patients have actionable mutations and may benefit from a personalized targeted therapy treatment plan. Next generation sequencing can be used to identify clinically actionable mutations in gynecologic cancers and guide the selection of treatments, thereby expanding treatment options without worsening survival or toxicity.
Insights
Next-generation sequencing identifies actionable mutations in gynecologic cancers, enabling targeted therapies. These treatments expand options without negatively impacting survival or increasing toxicity.
Area of Science:
- Gynecologic Oncology
- Molecular Oncology
- Genomics
Background:
- Molecular tumor profiling and next-generation sequencing (NGS) are increasingly used.
- Limited data exist on the therapeutic implications of targeted treatments in gynecologic oncology.
Purpose of the Study:
- To characterize gynecologic oncology patients referred for somatic tumor genetic mutation testing.
- To assess survival outcomes, efficacy, and toxicities of patients receiving targeted therapy based on NGS results.
Main Methods:
- Retrospective chart review of gynecologic oncology patients undergoing NGS for somatic tumor testing (1/1/2012-8/23/2019).
- Comparison of overall survival (OS) and progression-free survival (PFS) between patients receiving targeted therapy versus traditional treatment.
Main Results:
- 70% of patients had actionable mutations, identifying additional treatment options.
- No significant difference in OS or PFS was observed between targeted therapy and traditional treatment groups.
- Targeted therapy was associated with reduced rates of neuropathy and certain hematologic toxicities.
Conclusions:
- Most gynecologic cancer patients possess actionable mutations, potentially benefiting from personalized targeted therapy.
- NGS effectively identifies actionable mutations in gynecologic cancers, guiding treatment selection.
- Targeted therapies can expand treatment options without worsening survival or toxicity.
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