Sequential intensified chemotherapy with stem cell rescue for children and adolescents with desmoplastic small round-cell tumor

Abstract

The RMS4.99 study was designed to explore the role of early sequential intensified chemotherapy (SICT) with PBSC rescue in patients with soft tissue sarcoma with a poor prognosis. Fourteen patients with desmoplastic small round-cell tumor (DSRCT) were included in this study. Initial chemotherapy was followed by a course of CY and etoposide with subsequent PBSC harvest, then three consecutive intensified chemotherapy combinations followed by PBSC rescue and G-CSF administration: first cycle thiotepa (150 mg/m(2) x 2 on day 1) and melphalan (60 mg/m(2) on day 2), second cycle CY (2 g/m(2) on days 1 and 2) and thiotepa (150 mg/m(2) x 2 on day 3), third cycle melphalan (80 mg/m(2) on day 1). The interval between cycles had to be kept as short as possible. Then patients underwent surgery or radiotherapy or both, after which six courses of vincristine, actinomycin D, CY were administered. Ten patients received SICT, which was well tolerated. With a median follow-up of 27 months only three patients are alive without evidence of disease. The 3-year event-free and overall survival rates were 15.5 and 38.9%, respectively. The prognosis for pediatric patients with DSRCT did not improve after administering intensified chemotherapy early in their treatment, so different strategies are needed.

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

“…Bertuzzi et al reported on a cohort of 10 adult patients prospectively treated with high-dose melphalan plus mitoxantrone or thiotepa and found no improvements in overall survival [ 22 ]. Alternative approaches using sequential and multiple ASCTs administered earlier in the course of therapy were associated with equally poor outcomes [ 23 ]. Additional evidence of lack of effectiveness of ASCT was the poor response to the regimens used in our study with only a minor response noted in one patient.…”

Desmoplastic Small Round Blue Cell Tumor: A Review of Treatment and Potential Therapeutic Genomic Alterations

“…Doxorubicin is a common thread in the treatment of patients who either achieved long-term survival or had response to a standard consolidative radiotherapy dose of 30 Gy when delivered by external beam to the whole abdomen and pelvis [ 57 , 58 ]. Myeloablative chemotherapy with thiotepa and carboplatin, etoposide followed by autologous bone marrow, or peripheral stem cell rescue has been employed with limited success [ 56 , 61 ]. Case report of [ 90 Y]yttrium microspheres radioembolotherapy leading to a dramatic sustained reduction in the hepatic metastatic load has also been reported [ 59 , 62 ].…”

Primary desmoplastic small round cell tumor of the duodenum

“…For these advanced-stage patients, symptomatic treatment is more practical as it alleviates acute discomfort; however, it still leads to a poor outcome. Aggressive multimodality therapy with immunotherapy or bone marrow ablation and dose-intensive chemotherapy with autologous peripheral blood stem cell support may have potential benefit for DSRCTs and may be a promising new approach [8]. Recently, continuous hyperthermic peritoneal perfusion at the time of complete tumor resection has been developed as an adjunct to treat DSRCTs; this may prolong disease-free survival in selected cases [9].…”