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Published on: December 9, 2016
Real-world outcomes of patients with locally advanced or metastatic epithelioid sarcoma
Mrinal M Gounder1, Priscilla Merriam2, Ravin Ratan3
1Sarcoma Medical Oncology, Memorial Sloan Kettering Cancer Center and Weill Cornell Medical College, New York, New York.
Background:
Limited data are available on the real-world effectiveness and safety of systemic therapies for advanced (surgically unresectable and/or metastatic) epithelioid sarcoma (ES).
Methods:
A retrospective medical records review was conducted in patients with advanced ES who were initiating first-line or ≥2 lines of systemic therapy (2000-2017) at 5 US cancer centers. The real-world overall response rate (rwORR), the duration of response (rwDOR), the disease control rate (rwDCR) (defined as stable disease for ≥32 weeks or any duration of response), and progression-free survival (rwPFS) were assessed by radiology reports. Overall survival (OS), rwDOR, and rwPFS were estimated from the time therapy was initiated using the Kaplan-Meier method. Serious adverse events were assessed.
Results:
Of 74 patients (median age at diagnosis, 33 years; range, 10.6-76.3 years), 72% were male, and 85% had metastatic disease. The median number of lines of therapy was 2 (range, 1-7 lines of therapy), and 46 patients (62%) received ≥2 lines of systemic therapy. First-line regimens were usually anthracycline-based (54%) or gemcitabine-based (24%). For patients receiving first-line systemic therapy, the rwORR was 15%, the rwDCR was 20%, the median rwDOR was 3.3 months (95% CI, 2.1-5.2 months), the median rwPFS was 2.5 months (95% CI, 1.7, 6.9 months), and the median OS was 15.2 months (95% CI, 11.4-21.7 months). For those who received ≥2 lines of systemic therapy, the rwORR was 9%, the rwDCR was 20%, the median rwDOR was 4.5 months (95% CI, 0.7-5.6 months), and the median rwPFS was 6.0 months (95% CI, 3.2-7.4 months). Over one-half of patients (51.4%) experienced an adverse event, most frequently febrile neutropenia (14%), pain (10%), anemia, dyspnea, fever, thrombocytopenia, or transaminitis (5% each).
Conclusions:
Systemic therapies demonstrate limited efficacy in patients with advanced ES and have associated toxicities.
Insights
Systemic therapies show limited effectiveness for advanced epithelioid sarcoma (ES), with modest response rates and short progression-free survival. Treatments are associated with significant toxicities, including febrile neutropenia and pain.
Area of Science:
- Medical Oncology
- Sarcoma Research
- Clinical Effectiveness Studies
Background:
- Limited real-world data exists on systemic therapies for advanced epithelioid sarcoma (ES).
- Advanced ES is defined as surgically unresectable and/or metastatic disease.
Purpose of the Study:
- To evaluate the real-world effectiveness and safety of systemic therapies in advanced epithelioid sarcoma.
- To assess outcomes such as overall response rate, duration of response, disease control rate, and progression-free survival.
Main Methods:
- Retrospective review of medical records from 5 US cancer centers (2000-2017).
- Included patients with advanced ES receiving first-line or subsequent lines of systemic therapy.
- Assessed real-world overall response rate (rwORR), duration of response (rwDOR), disease control rate (rwDCR), progression-free survival (rwPFS), and overall survival (OS) using Kaplan-Meier method.
Main Results:
- 74 patients with advanced ES were analyzed; 85% had metastatic disease.
- First-line therapy showed a 15% rwORR and median rwPFS of 2.5 months.
- Patients receiving ≥2 lines of therapy had a 9% rwORR and median rwPFS of 6.0 months.
- Over 50% of patients experienced adverse events, most commonly febrile neutropenia.
Conclusions:
- Systemic therapies demonstrate limited efficacy in advanced epithelioid sarcoma.
- Treatment is associated with notable toxicities, impacting patient outcomes.
