Treatment patterns and outcomes in advanced clear cell sarcoma and gastrointestinal neuroectodermal tumors
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Updated: Oct 13, 2026
A Mouse Model of Incompletely Resected Soft Tissue Sarcoma for Testing (Neo)adjuvant Therapies
Published on: July 28, 2020
Adel Shahnam1, Daniel Reinhorn1, Mrinal M Gounder1,2
1Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, United States.
Background:
Clear cell sarcoma and gastrointestinal neuroectodermal tumor are ultra-rare soft tissue sarcomas with no established standard of care for advanced disease. Data evaluating outcomes of systemic therapy remain limited. We aimed to evaluate systemic therapy in patients with metastatic clear cell sarcoma and gastrointestinal neuroectodermal tumor treated at a tertiary referral institution.
Methods:
This is a retrospective, single-institution study of patients with metastatic clear cell sarcoma and gastrointestinal neuroectodermal tumor diagnosed between January 2000 and May 2025. The primary objective was to describe patterns of systemic therapy use, and secondary endpoints included overall survival (OS) and real-world progression-free survival (PFS). Survival outcomes were estimated using the Kaplan-Meier method. Hazard ratios were estimated using Cox proportional hazards models. Exploratory analyses evaluated outcomes by treatment class.
Results:
A total of 66 patients were included; 55 received systemic therapy. Median follow-up was 192 months, and patients received a median of 2 treatment lines with heterogeneous sequencing. Median OS was 18 months (95% CI = 13.6 to 28.0). In first-line analyses, tyrosine kinase inhibitor-based therapy and immune checkpoint inhibitors were associated with improved OS compared with doxorubicin-based chemotherapy (tyrosine kinase inhibitor: hazard ratio [HR] = 0.33, 95% CI = 0.14 to 0.79; P = .01; immune checkpoint inhibitor: HR = 0.39, 95% CI = 0.10 to 0.94; P = .04). Median first-line real-world PFS was 3.8 months (95% CI = 2.9 to 6.3), with no statistically significant differences between treatment classes. Nondoxorubicin-based chemotherapy was associated with shorter real-world PFS compared with doxorubicin regimens (HR = 4.1, 95% CI = 1.71 to 9.83; P < .001).
Conclusions:
Metastatic clear cell sarcoma and gastrointestinal neuroectodermal tumor is highly aggressive with limited effective systemic options. Outcomes remain poor, highlighting the need for novel therapies and collaborative studies.
