Recurrent desmoplastic small round cell tumor responding to an mTOR inhibitor containing regimen

Area of Science:

• Oncology
• Sarcoma Research

Background:

• Desmoplastic small round cell tumor (DSRCT) is a rare and aggressive sarcoma.
• DSRCT typically presents as multiple abdominal masses.
• Relapsed DSRCT has a poor prognosis with no established standard therapies.

Purpose of the Study:

• To evaluate the efficacy and toxicity of a vinorelbine, cyclophosphamide, and temsirolimus (VCT) regimen.
• To assess the outcomes for patients with relapsed DSRCT treated with VCT.

Main Methods:

• Retrospective case series of five patients with relapsed DSRCT.
• Treatment involved a combination of vinorelbine, cyclophosphamide, and temsirolimus (VCT).
• Efficacy was assessed by response rate and time to progression/relapse; toxicity was recorded.

Main Results:

• All five patients achieved a partial response to VCT therapy.
• The median number of VCT courses administered was 7 (range 4-14).
• The median time to progression or relapse was 8.5 months (range 7-16 months).
• Most common toxicities included neutropenia and mucositis (n=4 each).

Conclusions:

• The VCT regimen demonstrated activity in patients with relapsed DSRCT.
• VCT induced partial responses and provided a median progression-free survival of 8.5 months.
• VCT represents a potential therapeutic option for advanced or relapsed DSRCT, warranting further investigation.