Abstract
Prognosis of patients with advanced sarcoma after progression from FDA approved therapies remains grim. In this study, clinical outcomes of 100 patients with advanced sarcoma who received treatment on novel targeted therapy trials were evaluated. Outcomes of interest included best response, clinical benefit rate, progression-free survival (PFS) and overall survival (OS). Median patient age was 48 years (range 14–80). Patients had received a median of 2 prior lines of systemic treatment. Phase I treatments were anti-VEGF–based (n = 45), mTOR inhibitor–based (n = 15), and anti-VEGF + mTOR inhibitor–based (n = 17) or involved other targets (n = 23). Best responses included partial response (n = 4) and stable disease (n = 57). Clinical benefit rate was 36% (95% confidence interval 27–46%). Median OS was 9.6 months (95% Confidence Interval 8.1–14.2); median PFS was 3.5 months (95% Confidence Interval 2.4–4.7). RMH prognostic score of 2 or 3 was associated with lower median OS (log-rank p-value < 0.0001) and PFS (log-rank p-value 0.0081). Receiving cytotoxic chemotherapy as part of phase I trial was also associated with shorter median OS (log-rank p-value 0.039). Patients with advanced sarcoma treated on phase I clinical trials had a clinical benefit rate of 36% and RMH score predicted survival.
Highlights
We report the presenting characteristics and the outcomes of patients with sarcoma who were enrolled in phase I trials, primarily involving inhibitors of angiogenesis and mammalian target of rapamycin, at The University of Texas MD Anderson Cancer Center (MDACC) and explore putative associations between patient characteristics and survival outcomes
The four patients with partial responses included one patient with alveolar soft part sarcoma treated with an anti-VEGF agent, one with malignant fibrous histiocytoma treated with a combination of inhibitors of VEGF and histone deacetylase (HDAC), one with chondrosarcoma treated with a TRAIL
This study summarizes the clinical outcomes of patients with refractory sarcomas in phase I clinical studies after progression from standard US FDA approved therapies
Summary
Vivek Subbiah[1,2], Kenneth R. Clinical outcomes of 100 patients with advanced sarcoma who received treatment on novel targeted therapy trials were evaluated. Patients with advanced sarcoma treated on phase I clinical trials had a clinical benefit rate of 36% and RMH score predicted survival. Successful targeting of activating mutations in the KIT receptor tyrosine kinase with imatinib mesylate for gastrointestinal stromal tumor (GIST) illustrates how this approach can potentially change outcomes even for notoriously chemotherapy-resistant sarcoma subtypes[8,9]. We report the presenting characteristics and the outcomes of patients with sarcoma who were enrolled in phase I trials, primarily involving inhibitors of angiogenesis and mammalian target of rapamycin (mTOR), at The University of Texas MD Anderson Cancer Center (MDACC) and explore putative associations between patient characteristics and survival outcomes. We sought to validate the Royal Marsden Hospital (RMH) prognostic score among sarcoma patients enrolled in phase I clinical trials, as this score can help in patient prognostication[23,24]
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