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Background  Desmoplastic small round cell tumor (DSRCT) is a very rare mesenchymal tumor that mainly affects teenagers and young adults with a mean age at diagnosis around 20–25 years. Although initial management still needs standardization, many centers will use multimodal treatment including intensive chemotherapy, extensive surgical resection followed by radiotherapy. Despite this, prognosis remains very poor and the median overall survival is 25 months. Recurrent disease is mainly treated by chemotherapy. Recently, due to the unmet medical need for recurrent disease, targeted therapies were explored for DSRCT.    Methods  In this study, we assessed the response rate and progression free survival in nine cases of progressive DSRCT included in the OUTC’s registry and treated with antiangiogenics targeted agents (sunitinib, sorafenib and bevacizumab). OUTC’s, a French national registry, collects data about the use of off-label targeted therapy in sarcoma.    Results  Eight males and one woman were included, with median age at diagnosis of 27.3 years (range from 9 to 48 years). They received a mean 3 lines (2–5) of treatment before antiangiogenic agent initiation. Six patients received sunitinib, two received sorafenib and one bevacizumab. Median progression free survival was 3.1 months (range 2–5.5 months) and best response observed was 5.5 months stable disease. Most patients had manageable low-grade toxicities, mainly fatigue, abdominal pain and skin toxicity.    Conclusions  Despite very limited activity of antiangiogenics in our study, prospective collection of cases of these rare tumors together with molecular data should guide therapeutic decision and enhance outcome.    Electronic supplementary material  The online version of this article (doi:10.1186/s13569-017-0076-4) contains supplementary material, which is available to authorized users.

clinical sarcoma research

Antiangiogenic effects in patients with progressive desmoplastic small round cell tumor: data from the French national registry dedicated to the use of off-labeled targeted therapy in sarcoma (OUTC’s)