RT Journal Article T1 Activity of Pazopanib and Trabectedin in Advanced Alveolar Soft Part Sarcoma. A1 Stacchiotti, Silvia A1 Mir, Olivier A1 Le Cesne, Axel A1 Vincenzi, Bruno A1 Fedenko, Alexander A1 Maki, Robert G A1 Somaiah, Neeta A1 Patel, Shreyaskumar A1 Brahmi, Mehedi A1 Blay, Jean Y A1 Boye, Kjetil A1 Sundby-Hall, Kirsten A1 Gelderblom, Hans A1 Hindi, Nadia A1 Martin-Broto, Javier A1 Kosela, Hanna A1 Rutkowski, Piotr A1 Italiano, Antoine A1 Duffaud, Florence A1 Kobayashi, Eisuke A1 Casali, Paolo G A1 Provenzano, Salvatore A1 Kawai, Akira K1 Alveolar soft part sarcoma K1 Chemotherapy K1 Pazopanib K1 Sarcoma K1 Trabectedin AB Alveolar soft part sarcoma (ASPS) is an exceedingly rare and orphan disease, without active drugs approved in the front line. Pazopanib and trabectedin are licensed for sarcoma treatment from second-line, but very little and contradictory data are available on their activity in ASPS. Lacking ongoing and/or planned clinical trials, we conducted a multi-institutional study involving the reference sites for sarcoma in Europe, U.S., and Japan, within the World Sarcoma Network, to investigate the efficacy of pazopanib and trabectedin. From May 2007, 14 of the 27 centers that were asked to retrospectively review their databases had identified 44 advanced ASPS patients treated with pazopanib and/or trabectedin. Response was evaluated by Response Evaluation Criteria in Solid Tumors 1.1. Progression-free survival (PFS) and overall survival (OS) were computed by Kaplan-Meier method. Among 30 patients who received pazopanib, 18 were pretreated (13 with other antiangiogenics). Response was evaluable in 29/30 patients. Best responses were 1 complete response, 7 partial response (PR), 17 stable disease (SD), and 4 progressions. At a 19-month median follow-up, median PFS was 13.6 months (range: 1.6-32.2+), with 59% of patients progression-free at 1 year. Median OS was not reached.Among 23 patients treated with trabectedin, all were pretreated and evaluable for response. Best responses were 1 PR, 13 SD, and 9 progressions. At a 27-month median follow-up, median PFS was 3.7 months (range: 0.7-109), with 13% of patients progression-free at 1 year. Median OS was 9.1 months. The value of pazopanib in advanced ASPS is confirmed, with durable responses, whereas the value of trabectedin appears limited. These results are relevant to defining the best approach to advanced ASPS. This retrospective study, conducted among the world reference centers for treatment of sarcoma, confirms the value of pazopanib in patients with advanced alveolar soft part sarcoma (ASPS), with dimensional and durable responses, whereas trabectedin shows a limited activity. Alveolar soft part sarcoma is resistant to conventional cytotoxic chemotherapy. Pazopanib and trabectedin are licensed for treatment of sarcoma from second line; in the lack of prospective clinical trials, these results are relevant to defining ASPS best management and strongly support initiatives aimed at obtaining the approval of pazopanib in the front line of the disease. PB Oxford University Press YR 2017 FD 2017-07-28 LK http://hdl.handle.net/10668/11453 UL http://hdl.handle.net/10668/11453 LA en NO Stacchiotti S, Mir O, Le Cesne A, Vincenzi B, Fedenko A, Maki RG, et al. Activity of Pazopanib and Trabectedin in Advanced Alveolar Soft Part Sarcoma. Oncologist. 2018 Jan;23(1):62-70. DS RISalud RD Apr 11, 2025