High-dose chemotherapy and autologous peripheral blood stem cell transplantation in adult patients with high-risk or advanced Ewing and soft tissue sarcoma

Abstract

Purpose: Despite the availability of combined-modality treatment for Ewing sarcoma (ES) and soft tissue sarcomas (STS), results from independent groups still indicate a poor prognosis for high-risk and metastasized patients. The benefit of high-dose chemotherapy (HDCT) with autologous peripheral blood stem cell transplantation (ASCT) as compared to standard treatment is not defined.

Methods: Here, we report of HDCT in 35 consecutive adult patients with poor-risk ES or rhabdomyosarcoma (n = 11) and STS (n = 24) undergoing ASCT between July 1992 and March 2003. At a median follow-up of 100.6 months after ASCT, 11 patients are alive, with nine in sustained complete remission (CR) and each one in partial remission (PR) and stable disease. Median overall survival (OS) from ASCT was 17.1 months. Response to pretreatment, Karnofsky index > 80%, R (0) resection and first-line ASCT were associated with long-term OS (p < 0.05).

Conclusion: These data indicate that (1) patients achieving a CR or PR following induction, with preserved performance status and R (0) resection may benefit from ASCT and (2) that this can be an useful therapeutic modality in a subset of patients, in some achieving remarkable responses.

Fig. 1

a Overall survival rates in all patients stratified in those achieving PR or CR (thick line) prior to PBSCT versus SD or PD (dashed line). The vertical lines show 95% confidence intervals (CI) for the probability of survival at selected time points (6, 12, and 24 months) for both groups. b OS rates in all patients stratified in those with a Karnofsky performance index above 80% (thick line) versus KI of 80% or below (dashed line). The vertical lines show 95% CI for the probability of survival at selected time points (6, 12, and 24 months). c OS rates in all patients stratified in those with first-line PBSCT (thick line) versus those patients receiving the PBSCT with relapse (dashed line). The vertical lines show 95% CI for the probability of survival at selected time points (6, 12, and 24 months)