Trabectedin for desmoplastic small round cell tumours: a possible treatment option?

BACKGROUND: Desmoplastic small round cell tumour (DSRCT) is a rare sarcoma typically affecting young males and usually widely metastatic at presentation. Despite multimodal treatment approaches, the prognosis for DSRCT is extremely poor. Alkylator- and anthracyclines- based regimens are widely used as therapy and an initial response is common. Durable responses are exceptionally rare so further systemic treatment options for these patients represent an unmet medical need. We report two cases of metastatic, pretreated DSRCT patients achieving disease stabilisation with Trabectedin. METHODS: Retrospective review of 2 patients with progressive DSRCT, treated with Trabectedin. RESULTS: Two males aged 19 and 23 years treated with Trabectedin, 1.5 mg/m(2) over 24 hours 3 weekly for 6 and 5 cycles respectively. Best responses were stable disease in patient 1 and partial response (RECIST 1.1) in patient 2. Progression free survival was 4 months in both cases. Persistent neutropenia required 4 weekly administration in one patient but no other grade 3-4 toxicities occurred. CONCLUSIONS: This report supports Trabectedin to be active and safe in pre-treated DSRCT patients. Further prospective and collaborative efforts are desirable to better define its role in the management of this disease.