Desmoplastic small round cell tumours (DSRCT) is a rare disease affecting predominantly children and young adults characterised by a specific translocation t(11:22)(p13;q12) which fuses the ESWR1 gene to the WT1 gene. DSRCT shares characteristics with other small-round blue cell cancers including Ewing's sarcoma. . The best induction chemotherapy (IC) is not yet defined. The aim of this study is to evaluate the role of Adriamycin and Ifosfamide (AI)-based regimen as IC in DSRCT.
All pts diagnosed in our tertiary care center with DSRCT treated with AI-based regimen as IC between 1991 and 2017 were included in this study. Demographic, clinical and long-term characteristics were obtained from the electronic medical records and retrospectively analyzed.
34 pts were identified. With a median age of 25 yrs (range 18-56), 25 pts were male (74%). All pts had PS 0-1. 28 pts had symptoms (82%) with pain being the most common (n = 25). 14 pts (41%) had extra-peritoneal metastases (EPM) at diagnosis (liver mets (n = 14) associated with lung mets in 4 pts). Out of 34 pts, 26 pts (77%) received AI as IC. The remaining 8 pts had AI in combination to platinum agents (n = 4) or Etoposide (n = 4). Median number of cycles was 6±1 (range 3-9). Objective response and disease control rates were 71% and 91% respectively. Only 3 pts had progressive disease (PD) (9%). Dose reduction were noted in 2 pts with no treatment discontinuation. 41% had grade 3-4 toxicities. 7 pts (21%) developed febrile neutropenia. 17 pts (50%) underwent surgery (including 2 with EPM). Post-operative chemotherapy was given in 41 pts and radiotherapy in 21 pts. In pts who had surgery, local recurrence free survival was 10 months (95% CI: 7-12) while EPM-free survival was 13 months (95% CI: 7-19). Overall survival (OS) for pts without PD was 25.7 months (95% CI: 20.1-31.3). In pts who had surgery, OS was 28.3 months (95% CI: 13.6-43.1) while those who did not have surgery had an OS of 18.9 months (95% CI: 11.6-26.1).
AI-based regimen is a well-tolerated and active option with good response rate as an IC in DSRCT. Comparative data with currently used Ewing-type regimen are eagerly needed in the future.
Clinical trial identification
Legal entity responsible for the study
Institut Gustave Roussy.
Has not received any funding.
All authors have declared no conflicts of interest.