Clinical Outcomes of Patients with Desmoplastic Small Round Cell Tumor of the Peritoneum Undergoing Autologous HCT: a CIBMTR Retrospective Analysis

摘要

Desmoplastic small round cell tumor of the peritoneum (DSRCTP) is a rare, frequently fatal tumor. This retrospective study, based on CIBMTR registry data, describes the largest reported cohort of DSRCTP patients who have undergone autologous stem cell transplant (ASCT). The probabilities of disease-free survival (DFS) at one year for patients in complete remission (CR) and not in CR were 75% (95% confidence interval: 48–94%) and 35% (15–59%), respectively. The probability of overall survival (OS) at three years was 57% (29–83%) and 28% (9–51%) for patients in CR and not in CR respectively. Median survival for the entire cohort was 31 months (36 months and 21 months for those in CR and not in CR respectively). Engraftment at 42 days was 97% (88–100%). Treatment-related mortality was low, with only one death in the first 100 days. ASCT is a tolerable approach in patients with DSRCTP, with the greatest benefit seen in those patients who obtain CR. For those not in CR, the median OS in this series is greater than previously reported (21 months versus 17 months), suggesting ASCT is useful in prolonging DFS and OS, even in patients with residual or persistent disease pre transplant.