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Trabectedin for desmoplastic small round cell tumours: a possible treatment option?
Anna Maria Frezza1, Jeremy S Whelan2, Palma Dileo2
1The London Sarcoma Service, University College Hospital, 1st Floor Central, 250 Euston Road, London NW1 2PG, UK ; Medical Oncology, University Campus Bio-Medico, Rome, Italy.
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.
Insights
Trabectedin shows promise for treating advanced Desmoplastic small round cell tumours (DSRCT) in young males. Two patients achieved disease stabilization or partial response, indicating potential as a new systemic treatment option for this rare cancer.
Area of Science:
- Oncology
- Medical Research
Background:
- Desmoplastic small round cell tumour (DSRCT) is a rare, aggressive sarcoma with a poor prognosis, often diagnosed at advanced stages.
- Current treatments, including alkylators and anthracyclines, yield limited durable responses, highlighting an unmet need for effective systemic therapies.
- Trabectedin is an established chemotherapeutic agent used for soft tissue sarcomas.
Observation:
- This study retrospectively reviewed two pretreated male patients with progressive DSRCT.
- Both patients received Trabectedin at a standard dose and schedule.
Findings:
- One patient achieved stable disease, while the other experienced a partial response (RECIST 1.1) after Trabectedin treatment.
- Both patients had a progression-free survival of 4 months.
- The treatment was generally well-tolerated, with manageable neutropenia in one case and no other grade 3-4 toxicities observed.
Implications:
- Trabectedin demonstrates activity and a favorable safety profile in pretreated DSRCT patients.
- These findings suggest Trabectedin could be a valuable treatment option for DSRCT.
- Further prospective studies are warranted to confirm these results and establish Trabectedin's role in DSRCT management.
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