Trabectedin-irinotecan, a potentially promising combination in relapsed desmoplastic small round cell tumor: report of two cases. (17th February 2023)

• Record Type: Journal Article
• Authors: Ferrari, Andrea; Chiaravalli, Stefano; Bergamaschi, Luca; Nigro, Olga; Livellara, Virginia; Sironi, Giovanna; Gasparini, Patrizia; Pasquali, Sandro; Zaffaroni, Nadia; Stacchiotti, Silvia; Morosi, Carlo; Massimino, Maura; Casanova, Michela
• Is Part Of: Journal of chemotherapy. Volume 35:Number 2(2023)
• Page Start: 163
• Page End: 167
• Publication Date: 2023-02-17
• Subjects: Desmoplastic small round cell tumor -- trabectedin -- irinotecan -- combination -- new drug -- efficacy -- treatment; Chemotherapy -- Periodicals; Drug Therapy -- Periodicals; 615.58
• DOI: 10.1080/1120009X.2022.2067706
• Languages: English
• ISSNs: 1120-009X

Abstract

Abstract: Effective new drugs are urgently needed for desmoplastic small round cell tumor (DSRCT), an extremely rare and aggressive disease with a generally poor prognosis. We describe two heavily-pretreated young patients with advanced-stage DSRCT given third-line treatment with a combination of trabectedin and irinotecan, based on our preclinical data demonstrating its effect on patient-derived xenografts. This trabectedin-irinotecan treatment showed a limited toxicity. One patient had a mixed response (overall stable disease), the other a complete tumor remission. This is the first report of preliminary findings to suggest that combining trabectedin and irinotecan is worth further investigating as a potentially valuable chemotherapy for DSRCT.