Results from expanded access programs: a review of academic literature 161 7♦ leiomyosarcomas patients was 16.2 months, whereas this was 8.4 months for patients with other histology’s. These leiomyosarcomas patients also had a higher objective response rate (6.9% vs. 4.0%). Comparison with other data When comparing the expanded access program with previous reports on trabectedin, the authors conclude that: ‘Results of this expanded access program are consistent with previous reports of trabectedin, demonstrating disease control despite a low incidence of objective responses in advanced STS patients after failure of standard chemotherapy.’'(..) Also the safety profile observed in this study is similar to that observed throughout the development program of trabectedin.’ The authors also conclude that the higher OS and objective response rate in leiomyosarcomas and liposarcomas were consistent with those data reported in prior clinical trials. Several limitations to the study are mentioned: ‘Firsty, this was an open-label expanded access program and was not designed as a randomized study with a direct comparison to another agent. Another limitation of this study is that the findings may not reflect a general population in respect to race, since the majority of the population enrolled were White and were of relatively good performance status (median ECOG score = 1). Lastly, we did not capture information on progression-free survival, which would be of particular interest for this agent that is not associated with a high ORR, but often results in stable disease.’ Conclusion The authors conclude that: ‘Results of this expanded access program are consistent with previous reports of trabectedin, demonstrating disease control despite a low incidence of objective responses in advanced STS patients after failure of standard chemotherapy’. They further write that: ‘In this expanded access population of incurable advanced STS, trabectedin therapy results in durable disease control rates of approximately 30%, along with higher-than-expected rates of overall survival despite low rates of objective response. These data further support the palliative benefits of trabectedin in patients with advanced STS after failure of standard therapies.’ The authors do not provide specific suggestions for future studies.
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