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07:55
Establishment of a Primary Culture of Patient-derived Soft Tissue Sarcoma
Published on: April 11, 2018
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Transcriptome based individualized therapy of refractory pediatric sarcomas: feasibility, tolerability and efficacy.
Bushra Weidenbusch1, Günther H S Richter1,2, Marie Sophie Kesper1,2
1Department of Pediatrics and Children's Cancer Research Center, Kinderklinik München Schwabing, Klinikum rechts der Isar, Fakultät für Medizin, Technische Universität München, Munich, Germany.
Oncotarget
|May 15, 2018
Summary
Gene expression profiling identified actionable targets in pediatric sarcomas. Targeted therapy significantly improved overall survival and progression-free survival in refractory cases, showing promise for future treatment strategies.
Area of Science:
- Pediatric Oncology
- Molecular Diagnostics
- Cancer Therapeutics
Background:
- Pediatric sarcoma survival rates have plateaued, particularly for adolescents and young adults (AYAs).
- Refractory cases require novel treatment strategies beyond conventional therapies.
- Gene expression profiling offers a method to characterize tumor transcriptomes and guide personalized treatment.
Purpose of the Study:
- To assess the feasibility and efficacy of expression-based targeted therapy (TT) in pediatric sarcoma patients.
- To identify actionable molecular targets in refractory pediatric sarcomas using gene expression profiling.
- To compare outcomes, including survival and adverse events, between patients receiving TT and those not.
Main Methods:
- Array-based gene expression profiling was performed on tumor biopsies from 20 pediatric sarcoma patients (ages 8-35).
- Actionable targets and deregulated pathways were identified to guide individualized TT selection.
- Patients receiving TT were compared to a control group (non TT) regarding disease status, survival, adverse events (AEs), and quality of life (QOL).
Main Results:
- Actionable targets were identified in all analyzed pediatric sarcoma biopsies.
- Nine patients received TT, while eleven did not; no significant differences in risk factors were observed between groups.
- TT significantly improved overall survival (OS) and progression-free survival (PFS) compared to non TT (median OS: 8.83 vs. 4.93 months; median PFS: 6.17 vs. 1.6 months).
- Quality of life (QOL) did not differ between groups. TT patients experienced fewer grade 1 AEs, with no difference in grade 2-4 AEs.
Conclusions:
- Expression-based targeted therapy is a feasible approach for refractory pediatric sarcomas.
- Targeted therapy demonstrated significant survival benefits in this patient cohort.
- Further investigation is warranted to confirm the efficacy and benefits of targeted therapy in pediatric sarcoma treatment.
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