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.