title: High-Dose Chemotherapy and Autologous Stem Cell Transplantation in Children with High-Risk or Recurrent Bone and Soft Tissue Sarcomas.
abstract: Despite increasing evidence that high-dose chemotherapy and autologous stem cell transplantation (HDCT/auto-SCT) might improve the survival of patients with high-risk or recurrent solid tumors, therapy effectiveness for bone and soft tissue sarcoma treatment remains unclear. This study retrospectively investigated the feasibility and effectiveness of HDCT/auto-SCT for high-risk or recurrent bone and soft tissue sarcoma. A total of 28 patients (18 high-risk and 10 recurrent) underwent single or tandem HDCT/auto-SCT between October 2004 and September 2014. During follow-up of a median 15.3 months, 18 patients exhibited disease progression and 2 died of treatment-related toxicities (1 veno-occlusive disease and 1 sepsis). Overall, 8 patients remained alive and progression-free. The 3-year overall survival (OS) and event-free survival (EFS) rates for all 28 patients were 28.7% and 26.3%, respectively. In the subgroup analysis, OS and EFS rates were higher in patients with complete or partial remission prior to HDCT/auto-SCT than in those with worse responses (OS, 39.1% vs. 0.0%, P = 0.002; EFS, 36.8% vs. 0.0%, P < 0.001). Therefore, careful selection of patients who can benefit from HDCT/auto-SCT and maximal effort to reduce tumor burden prior to treatment will be important to achieve favorable outcomes in patients with high-risk or recurrent bone and soft tissue sarcomas.
authors.affiliation: Department of Pediatrics, Chung-Ang University Hospital, Seoul, Korea .
authors.forename: Young Bae
authors.identifier: 0000-0001-7016-8827
authors.initials: YB
authors.lastname: Choi
authors.affiliation: Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea .
authors.forename: Eun Sang
authors.identifier: 0000-0001-8214-2106
authors.initials: ES
authors.lastname: Yi
authors.affiliation: Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea .
authors.forename: Ji Won
authors.identifier: 0000-0003-0084-1304
authors.initials: JW
authors.lastname: Lee
authors.affiliation: Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea .
authors.forename: Keon Hee
authors.identifier: 0000-0002-5980-7912
authors.initials: KH
authors.lastname: Yoo
authors.affiliation: Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea .
authors.forename: Ki Woong
authors.identifier: 0000-0001-5989-4772
authors.initials: KW
authors.lastname: Sung
authors.affiliation: Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea .
authors.forename: Hong Hoe
authors.identifier: 0000-0001-8082-1412
authors.initials: HH
authors.lastname: Koo
mesh_terms: D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001859:Bone Neoplasms / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D008297:Male; D012189:Retrospective Studies; D012509:Sarcoma / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D012983:Soft Tissue Neoplasms / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D033581:Stem Cell Transplantation; D015996:Survival Rate; D014182:Transplantation, Autologous; D016896:Treatment Outcome; D055815:Young Adult
keywords: Autologous Stem Cell Transplantation; Bone and Soft Tissue Sarcoma; Children; High-Dose Chemotherapy