Korean J Pediatr.  2013 Sep;56(9):401-406. 10.3345/kjp.2013.56.9.401.

High-dose chemotherapy and autologous peripheral blood stem cell transplantation in the treatment of children and adolescents with Ewing sarcoma family of tumors

• Seo J
• Kim DH
• Lim JS
• Koh JS
• Yoo JY
• Kong CB
• Song WS
• Cho WH
• Jeon DG
• Lee SY
• Lee JA

• KMID: 1493122
• DOI: http://doi.org/10.3345/kjp.2013.56.9.401

Abstract

PURPOSE
We performed a pilot study to determine the benefit of high-dose chemotherapy and autologous peripheral blood stem cell transplantation (HDCT/autoPBSCT) for patients with Ewing sarcoma family of tumors.
METHODS
We retrospectively analyzed the data of patients who received HDCT/autoPBSCT at Korea Cancer Center Hospital. Patients with relapsed, metastatic, or centrally located tumors were eligible for the study.
RESULTS
A total of 9 patients (3 male, 6 female), with a median age at HDCT/autoPBSCT of 13.4 years (range, 7.1 to 28.2 years), were included in this study. Patients underwent conventional chemotherapy and local control either by surgery or radiation therapy, and had achieved complete response (CR, n=7), partial response (n=1), or stable disease (n=1) prior to HDCT/autoPBSCT. There was no transplant-related mortality. However, the median duration of overall survival and event-free survival after HDCT/autoPBSCT were 13.3 months (range, 5.3 to 44.5 months) and 6.2 months (range, 2.1 to 44.5 months), respectively. At present, 4 patients are alive and 5 patients who experienced adverse events (2 metastasis, 2 local recur, and 1 progressive disease) survived for a median time of 2.8 months (range, 0.1 to 10.7 months). The 2-year survival after HDCT/autoPBSCT was 44.4%+/-16.6% and disease status at the time of HDCT/autoPBSCT tended to influence survival (57.1%+/-18.7% of cases with CR vs. 0% of cases with non-CR, P=0.07).
CONCLUSION
Disease status at HDCT/autoPBSCT tended to influence survival. Further studies are necessary to define the role of HDCT/autoPBSCT and to identify subgroup of patients who might benefit from this investigational treatment.

Keyword

Ewing sarcoma; High-dose chemotherapy; Stem cell transplantation

MeSH Terms

Adolescent
Child
Disease-Free Survival
Humans
Korea
Male
Neoplasm Metastasis
Peripheral Blood Stem Cell Transplantation
Pilot Projects
Retrospective Studies
Sarcoma, Ewing
Stem Cell Transplantation
Therapies, Investigational