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

Sep 29, 2026

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Background: Multimodal surgery, chemotherapy, and radiotherapy improve pediatric Ewing sarcoma outcomes, yet prognosis remains poor in metastatic disease, recurrence, and other high-risk (HR) features such as large tumor burden, axial skeleton involvement, and poor histologic response. This retrospective single-institute study evaluates high-dose chemotherapy with autologous stem cell rescue (HDC-ASCR) for HR Ewing sarcoma (HRES). Methods: March 2004–March 2021 patients receiving HDC-ASCR were analyzed for characteristics, stem cell dose, engraftment, complications, and outcomes. Results: 5-year progression-free and overall survival were 35% and 54.5%, with deaths due to disease progression and better OS in complete response/localized disease; results also improved versus a historical cohort.

LanguageEnglish

CategoryHealthcare

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Pages7

Reading Time18 min

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Introduction

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J Chin MedAssoc Original article High-dose chemotherapy with autologous stem cell rescue in children and young adults with high-risk Ewing sarcoma Chih-Ying Leea,b, Ming-Hsin Houa,b, Giun-Yi Hunga,b, Cheng-Yin Hoa,b, Ting-Yen Yuc, Po-Kuei Wub,d, Chao-Ming Chenb,d, Chueh-Chuan Yenb,e, Cheng-Ying Shiaub,f, Paul Chih-Hsueh Chenb,g, Hung-Ta Hondar Wub,h, Ching-Lan Wub,h, Hsiu-Ju Yena,b,*, Wei-Ming Chenb,d aDivision of Pediatric Hematology and Oncology, Department of Pediatrics, Taipei Veterans General Hospital, Taipei, Taiwan, ROC; bFaculty of Medicine, School of Medicine, National Yang Ming Chiao Tung University, Taipei, Taiwan, ROC; cDepartment of Pediatrics, Far Eastern Memorial Hospital, New Taipei City, Taiwan, ROC; dDepartment of Orthopedics, Taipei Veterans General Hospital, Taipei, Taiwan, ROC; eDepartment of Oncology, Taipei Veterans General Hospital, Taipei, Taiwan, ROC; fDepartment of Heavy Particles and Radiation Oncology, Taipei Veterans General Hospital, Taipei, Taiwan, ROC; gDepartment of Pathology, Taipei Veterans General Hospital, Taipei, Taiwan, ROC; hDepartment of Radiology, Taipei Veterans General Hospital, Taipei, Taiwan, ROC 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. Keywords: Autologous stem cell rescue; Autologous stem cell transplant; Ewing sarcoma; High-dose chemotherapy; Pediatric 1. INTRODUCTION The Ewing sarcoma family of tumors (ESFT) are rare malignant diseases that can originate from bone or soft tissue. ESFT mostly *Address correspondence. Dr. Hsiu-Ju Yen, Department of Pediatrics, Taipei Veterans General Hospital, 201, Section 2, Shi-Pai Road, Taipei 112, Taiwan, [ROC. E-mail address: hjyen2@vghtpe.gov.tw](ROC. E-mail address: hjyen2@vghtpe.gov.tw) (H.-J. Yen). 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 t

FAQs

What patient groups were included in the HDC-ASCR study?

Patients with Ewing sarcoma, Ewing-like sarcoma, or round cell sarcoma who received HDC-ASCR as part of treatment were included from March 2004 to March 2021.

What outcomes were reported for progression-free and overall survival?

The 5-year progression-free survival was 35% and the 5-year overall survival was 54.5%, with all reported causes of death attributed to disease progression.

How did response status or disease localization affect overall survival?

Patients with complete response or with localized high-risk Ewing sarcoma showed higher 5-year overall survival compared with others, with reported p-values indicating statistical significance.

Sep 29, 2026

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