Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2025 Jan 1;88(1):85-91.
doi: 10.1097/JCMA.0000000000001179. Epub 2024 Oct 11.

High-dose chemotherapy with autologous stem cell rescue in children and young adults with high-risk Ewing sarcoma

Chih-Ying Lee  1   2 , Ming-Hsin Hou  1   2 , Giun-Yi Hung  1   2 , Cheng-Yin Ho  1   2 , Ting-Yen Yu  3 , Po-Kuei Wu  2   4 , Chao-Ming Chen  2   4 , Chueh-Chuan Yen  2   5 , Cheng-Ying Shiau  2   6 , Paul Chih-Hsueh Chen  2   7 , Hung-Ta Hondar Wu  2   8 , Ching-Lan Wu  2   8 , Hsiu-Ju Yen  1   2 , Wei-Ming Chen  2   4
Affiliations

High-dose chemotherapy with autologous stem cell rescue in children and young adults with high-risk Ewing sarcoma

Chih-Ying Lee et al. J Chin Med Assoc. .

Abstract

Background: A combination treatment of surgery, chemotherapy, and radiotherapy can improve the survivals of pediatric patients with Ewing sarcoma (ES). However, prognosis remains poor for patients with metastatic disease at diagnosis or recurrence. Other high-risk (HR) features include large tumor burden, tumors of the axial skeleton, and poor histologic response. Several studies have documented high-dose chemotherapy with autologous stem cell rescue (HDC-ASCR) to be effective in such patients. In this retrospective study, we present the results of HDC-ASCR for high-risk ES (HRES) in children and young adults in a single institute.

Methods: From March 2004 to March 2021, patients with ES, Ewing-like sarcoma, or round cell sarcoma received HDC-ASCR as part of treatment were included. The patients' characteristics, disease status, stem cell dose, engraftment status, post-transplant complications, and outcomes were analyzed.

Results: Twenty patients receiving HDC-ASCR at complete response (n = 6), partial response (n = 13), and stable disease (n = 1) were enrolled. The male-to-female ratio was 11:9. Median age at diagnosis and transplant was 15.6 years old (range: 3.3-28.9) and 16.2 (range: 4.2-29.9), respectively. The conditioning regimens included ifosfamide-based in two and melphalan-based in 19. All patients achieved successful engraftment without transplant-related mortality. The 5-year progression-free and overall survival (OS) rate were 35% and 54.5%, respectively. The causes of death (n = 8) were all contributed to disease progression. Patients in the complete response group or with localized HRES exhibited a higher 5-year OS ( p = 0.047 and 0.05, respectively). Compared with the historical cohort without HDC-ASCR as part of primary treatment, the current cohort had a significantly better 5-year OS ( p = 0.018).

Conclusion: HDC-ASCR seems promising as an alternative treatment for HRES in improving OS in this retrospective study with a limited case number.

PubMed Disclaimer

Conflict of interest statement

Conflicts of interest: Dr. Po-Kuei Wu and Dr. Wei-Ming Chen, editorial board member at the Journal of the Chinese Medical Association , had no role in the peer review process or decision to publish this article. The other authors declare that they have no conflicts of interest related to the subject matter or materials discussed in this article.

References

    1. Hung GY, Horng JL, Lee YS, Yen HJ, Chen CC, Lee CY. Cancer incidence patterns among children and adolescents in Taiwan from 1995 to 2009: a population-based study. Cancer 2014;120:3545–53.
    1. Siegel DA, King J, Tai E, Buchanan N, Ajani UA, Li J. Cancer incidence rates and trends among children and adolescents in the United States, 2001-2009. Pediatrics 2014;134:e945–55.
    1. Lee CY, Yen CC, Yen HJ, Shiau CY, Chao TC, Wu PK, et al. Outcomes of 50 patients with Ewing sarcoma family of tumors treated at a single institution in Taiwan. Medicine (Baltimore) 2016;95:e3830.
    1. Cotterill SJ, Ahrens S, Paulussen M, Jurgens HF, Voute PA, Gadner H, et al. Prognostic factors in Ewing’s tumor of bone: analysis of 975 patients from the European Intergroup Cooperative Ewing’s Sarcoma Study Group. J Clin Oncol 2000;18:3108–14.
    1. Marina N, Granowetter L, Grier HE, Womer RB, Randall RL, Marcus KJ, et al. Age, tumor characteristics, and treatment regimen as event predictors in Ewing: a children’s Oncology Group Report. Sarcoma 2015;2015:927123.