J Korean Med Sci.  2010 May;25(5):691-697. 10.3346/jkms.2010.25.5.691.

Efficacy of Tandem High-Dose Chemotherapy and Autologous Stem Cell Rescue in Patients Over 1 Year of Age with Stage 4 Neuroblastoma: The Korean Society of Pediatric Hematology-Oncology Experience Over 6 Years (2000-2005)

Affiliations
  • 1Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
  • 2Department of Pediatrics, Cancer Research Institute, Seoul National University College of Medicine, Seoul, Korea.
  • 3Department of Pediatrics, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, Korea.
  • 4Department of Pediatrics, Kyung Hee University Medical Center, Kyung Hee University College of Medicine, Seoul, Korea.
  • 5Department of Pediatrics, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Hwasun, Korea.
  • 6Department of Pediatrics, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.
  • 7Division of Pediatric Hematology and Oncology, Department of Pediatrics, Severance Hospital, Yonsei University Health System, Seoul, Korea.
  • 8Department of Pediatrics8, Kyungpook National University Hospital, Kyungpook National University School of Medicine, Daegu, Korea. kslee@knu.ac.kr

Abstract

The efficacy of tandem high-dose chemotherapy and autologous stem cell rescue (HDCT/ASCR) was investigated in patients with high-risk neuroblastoma. Patients over 1 yr of age who were newly diagnosed with stage 4 neuroblastoma from January 2000 to December 2005 were enrolled in The Korean Society of Pediatric Hematology-Oncology registry. All patients who were assigned to receive HDCT/ASCR at diagnosis were retrospectively analyzed to investigate the efficacy of single or tandem HDCT/ASCR. Seventy and 71 patients were assigned to receive single or tandem HDCT/ASCR at diagnosis. Fifty-seven and 59 patients in the single or tandem HDCT group underwent single or tandem HDCT/ASCR as scheduled. Twenty-four and 38 patients in the single or tandem HDCT group remained event free with a median follow-up of 56 (24-88) months. When the survival rate was analyzed according to intent-to-treat at diagnosis, the probability of the 5-yr event-free survival+/-95% confidence intervals was higher in the tandem HDCT group than in the single HDCT group (51.2+/-12.4% vs. 31.3+/-11.5%, P=0.030). The results of the present study demonstrate that the tandem HDCT/ASCR strategy is significantly better than the single HDCT/ASCR strategy for improved survival in the treatment of high-risk neuroblastoma patients.

Keyword

Neuroblastoma; High-dose Chemotherapy; Transplantation, Autologous

MeSH Terms

Adolescent
Child
Child, Preschool
Combined Modality Therapy/mortality
Drug Therapy/*mortality
Female
Humans
Infant
Korea/epidemiology
Longitudinal Studies
Male
Neuroblastoma/*mortality/*therapy
Prevalence
Risk Assessment/methods
Risk Factors
Stem Cell Transplantation/*mortality
Survival Analysis
Survival Rate
Treatment Outcome

Figure

  • Fig. 1 Flow of patients. Fifty-seven (81.4%) out of 70 patients in the single group proceeded to the HDCT/ASCR as assigned at diagnosis. In the tandem group, 66 (93.0%) out of 71 patients proceeded to the first HDCT/ASCR and 59 (83.1%) patients received the second HDCT/ASCR as assigned at diagnosis.

  • Fig. 2 Results of the Kaplan-Meier analysis. (A) When the survival rate was analyzed according to intent-to-treat at diagnosis, the probability of the 5-yr EFS after diagnosis was higher in the tandem group than in the single group (51.2±12.4% vs. 31.3±11.5%, P=0.030). (B) When the analysis was confined to 123 patients who proceeded to HDCT/ASCR as assigned at diagnosis, the probability of 5-yr RFS after the first HDCT was higher in the tandem group than in the single group with borderline significance (59.7±13.5% vs. 41.6±14.5%, P=0.099). (C) However, the difference became significant when the analysis was confined to only patients who were not in CR prior to the first HDCT (55.7±17.0% vs. 0%, P=0.012).


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