Abstract
BackgroundThe prognosis of patients with metastatic or advanced sarcomas is poor and there are few options for treatment. Several studies have shown that gemcitabine and docetaxel (GD) combination chemotherapy has antitumor activity against various subtypes of sarcoma. Recently, some studies have shown a favourable outcome for GD combination chemotherapy for relapsed high-grade osteosarcoma and spindle cell sarcoma of bone. If the effectiveness of GD is proven, this will result in new treatment options for advanced bone and soft tissue sarcomas (STS). The aim of this prospective Phase 2 study is to evaluate the efficacy and toxicity of the GD combination in patients with advanced bone sarcomas and STS.MethodsThis is a Phase 2, single-arm, open-label study to investigate the efficacy and safety of combination chemotherapy with GD for advanced bone sarcomas and STS and will enrol 20 patients. The patients will receive gemcitabine 900 mg/m2 on Days 1 and 8, and docetaxel 70 mg/m2 on Day 8 in 3-week cycles until disease progression or other evidence of treatment failure. The primary aim of this study is to analyse GD’s effect on progression-free survival (PFS). The secondary objectives are to analyse treatment efficacy and safety in terms of response rate, tumour control rate, overall survival, and adverse event rate. The length of follow-up will be 5 years.DiscussionThis study will evaluate the efficacy and safety of combination therapy with gemcitabine and docetaxel for bone sarcomas and STS. If this combination proves to be acceptable, it could be used for as second, third, or later line therapy for patients with sarcomas (especially bone sarcomas). In the future, the role of various treatments, including GD therapy, will be clarified for specific subtypes of sarcoma.Trial registrationThis study was registered as UMIN000031004 (University Hospital Medical Information Network-Clinical Trial Registry: UMIN-CTR) on 1 March 1 2018 and with the Japan Registry of Clinical Trials (jRCT) as jRCTs051180042 on 30 January 2019. The posted information will be updated as needed to reflect protocol amendments and study progress.
Highlights
The prognosis of patients with metastatic or advanced sarcomas is poor and there are few options for treatment
Small round-cell soft tissue sarcomas (STS) such as rhabdomyosarcoma and Ewing sarcoma, which usually occur in children and young adults, are sensitive to chemotherapy
The target sample size is based on expected response rate; the primary endpoint is defined as progression-free survival (PFS) because prolonging time to disease progression is important even if the response rate is less than expected
Summary
The prognosis of patients with metastatic or advanced sarcomas is poor and there are few options for treatment. If the effectiveness of GD is proven, this will result in new treatment options for advanced bone and soft tissue sarcomas (STS). The aim of this prospective Phase 2 study is to evaluate the efficacy and toxicity of the GD combination in patients with advanced bone sarcomas and STS. Small round-cell soft tissue sarcomas (STS) such as rhabdomyosarcoma and Ewing sarcoma, which usually occur in children and young adults, are sensitive to chemotherapy. Osteosarcoma and small round-cell sarcomas are treated with chemotherapy in combination with surgery or radiation therapy. The therapy for high-risk patients with high-grade large non-small round-cell STS, which usually occurs in older adults, is usually surgery and chemotherapy. There is no established chemotherapy for bone sarcomas other than osteosarcoma and Ewing sarcoma
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