Trabectedin for advanced sarcomas failing doxorubicin: Analysis of 189 unreported patients in a compassionate use program

10574 Background: Between 2005 and 2008, 387 patients (pts) with advanced sarcoma failing doxorubicin were treated in a compassionate use program (ATU) of trabectedin in France using the standard 1.5mg/m2/CI 24h q21d regimen. The purpose of this study was to assess the outcome of pts treated in this program. Methods: From 2005 to 2008, 87 centers (ctrs) included at least 1 pt in the ATU program. Inclusion criteria were those of the EORTC trial (J Clin Oncol, 2005;23:5276), with no restriction on the previous number of lines. A simple CRF with 22 items was used to collect pts characteristics and outcome. One hundred eighty-nine pts files were collected as of December 20, 2008. Univariate and multivariate analyses of prognostic factors was performed. Results: Two hundred thirty-five pts were included in the 17 ctrs in which >4 pts were treated. Forty-four ctrs treated only 1 pt. Fifty-two percent were female; major histological subgroups were leiomyosarcoma (29%) and liposarcoma (20%). All pts had been treated with doxorubicin and ifosfamide, 3 (1.5%) in adjuvant setting only. Trabectedin was given in 1st, 2nd, 3rd, or 4th line in metastatic phase in n=8, 69, 66, 42 pts respectively (median: 3rd line). The median number of courses were 3 (range 1–24). Best response reported were PR, n=15 (8%), SD, n=68 (36%) and PD, n=94 (50%), NE, n=11 (6%). With a median follow-up of 805 days (d), median PFS and OS were 91 d and 309 d respectively. 27/127 (20%) evaluable pts had to be hospitalized for treatment related side effects. PFS was superior in myxoid liposarcoma (MyxLPS) (median 192 d vs 69 d, p=0.003), retroperitoneal sarcomas (median 104 d vs 69 d, p=0.006), and grade 1 tumors (median 141 d vs 70 d, p=0.01). In multivariate analysis (Cox model), tumor site, grade 1, histotype were the only independent prognostic factors for PFS. For OS, favorable prognostic factors in univariate analysis were histotypes (MyxLPS, MFH), grade 1 lesions, retroperitoneal site, no hospitalisation for toxicity (p<0.01 all) while Cox model identified female gender, tumor site, histotype as the only independent prognostic factors for OS. Conclusions: In this compassionate use program for heavily pretreated pts with advanced sarcomas, trabectedin yielded PFS and OS close to those observed in phase II and III trial. PFS and OS are superior in myxLPS, retroperitoneal sarcomas, and grade 1 tumors. [Table: see text]