Myeloablative Chemotherapy with Autologous Stem Cell Transplant for Desmoplastic Small Round Cell Tumor

Abstract

Desmoplastic small round cell tumor (DSRCT), a rare, aggressive neoplasm, has a poor prognosis. In this prospective study, we evaluated the role of myeloablative chemotherapy, followed by autologous stem cell transplant in improving survival in DSRCT. After high-dose induction chemotherapy and surgery, 19 patients with chemoresponsive DSRCT underwent autologous stem cell transplant. Myeloablative chemotherapy consisted of carboplatin (400–700 mg/m2/day for 3 days) + thiotepa (300 mg/m2/day for 3 days) ± topotecan (2 mg/m2/day for 5 days). All patients were engrafted and there was no treatment-related mortality. Seventeen patients received radiotherapy to sites of prior or residual disease at a median of 12 weeks after transplant. Five-year event-free and overall survival were 11 ± 7% and 16 ± 8%, respectively. Two patients survive disease-free 16 and 19 years after transplant (both in complete remission before transplant). 14 patients had progression and died of disease at a median of 18 months following autologous transplant. These data do not justify the use of myeloablative chemotherapy with carboplatin plus thiotepa in patients with DSRCT. Alternative therapies should be considered for this aggressive neoplasm.

Clinical Characteristics and Outcomes of Pediatric Patients with Desmoplastic Small round Cell Tumor

“… 13 In 19 pediatric and adult patients, overall survival following autologous stem cell transplant was 11%, with median overall survival for patients with complete remission after autologous transplant of 30.1 months compared to those who had measurable disease of 18.7 months. 18 These results, along with our current findings, highlight the importance of local control in the management of this disease. While the SEER database allowed access to a rare disease entity, there were limitations with using a registry.…”

Management of desmoplastic small round cell tumor

“…Based on preclinical data indicating elevated expression of pro-angiogenic factors in DSRCT 9 , irinotecan and temozolomide in combination with bevacizumab-induced blockade of angiogenesis administered prior to Ewing’s type chemotherapy are also being studied in a pilot study in newly diagnosed patients (clinicaltrials.gov NCT01189643). Attempts to improve outcomes with myeloablative chemotherapy with carboplatin, thiotepa and topotecan after debulking surgery have been unsuccessful 10 . Experience with other chemotherapeutic agents has mostly been anecdotal.…”

Early experience with cytoreduction and hyperthermic intraperitoneal chemotherapy at a newly developed center for peritoneal malignancy

“…Seven of the first 50 CRS/HIPEC procedures performed in our early experience were for DSRCT. Because the risk of recurrence is high, many go onto additional therapy including adjuvant systemic chemotherapy, whole abdomen radiation, and autologous stem cell transplantation (33,34). At last follow up, two patients have passed away of disease ( Figure 3C).…”