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A Mouse Model of Incompletely Resected Soft Tissue Sarcoma for Testing (Neo)adjuvant Therapies
Published on: July 28, 2020
Other endpoints in screening studies for soft tissue sarcomas
1Erasmus University Medical Center, Rotterdam, The Netherlands. j.verweij@erasmusmc.nl
Abstract:
Despite extensive research efforts over the past two decades to identify effective agents for the treatment of soft tissue sarcomas, few agents are available, and with modest utility. There is a high unmet medical need to develop novel therapies for the treatment of patients with soft tissue sarcomas. Clinical trials for soft tissue sarcomas should be optimally designed, and it is crucial that they identify and define the desired clinical outcome. Survival is often the ultimate endpoint; however, physiological and biological markers are often used to predict the potential therapeutic benefit of a new agent. These endpoints can be easily measured, but can lead to false-positive results and do not take into account the complicated nature of soft tissue sarcomas. Alternative endpoints that are currently being evaluated include the progression-free survival rate, time to progression, tumor growth rate, and progression arrest rate. This article discusses some of the limitations of current endpoint criteria and potential endpoint criteria that could be used to evaluate treatment options for patients with soft tissue sarcomas.
Insights
Developing novel therapies for soft tissue sarcomas is critical due to limited treatment options. This review examines current clinical trial endpoints and proposes improved criteria for evaluating new soft tissue sarcoma treatments.
Area of Science:
- Oncology
- Clinical Trial Design
- Sarcoma Research
Background:
- Soft tissue sarcomas (STS) have limited effective treatment options, highlighting a significant unmet medical need.
- Current research has yielded few highly effective agents for STS treatment over the past two decades.
- Optimizing clinical trial design is crucial for identifying beneficial novel therapies for STS patients.
Purpose of the Study:
- To discuss the limitations of current clinical trial endpoint criteria used in soft tissue sarcoma research.
- To explore potential alternative endpoint criteria for evaluating new STS treatment options.
- To improve the accuracy and relevance of clinical outcome measures in STS trials.
Main Methods:
- Review of existing literature on clinical trial endpoints for soft tissue sarcomas.
- Analysis of the limitations associated with traditional survival endpoints.
- Evaluation of alternative endpoints such as progression-free survival and time to progression.
Main Results:
- Traditional endpoints like overall survival can be difficult to measure and may not fully capture therapeutic benefit in STS.
- Physiological and biological markers, while easily measured, can lead to false-positive results.
- Alternative endpoints like progression-free survival rate, time to progression, tumor growth rate, and progression arrest rate are being explored.
Conclusions:
- Current endpoint criteria for soft tissue sarcoma clinical trials have significant limitations.
- There is a need for more robust and accurate endpoints to better evaluate novel therapeutic agents.
- Refining endpoint selection is essential for advancing the development of effective treatments for soft tissue sarcomas.
