A pilot study of tandem high-dose chemotherapy with stem cell rescue as consolidation for high-risk neuroblastoma: Children's Oncology Group study ANBL00P1

Bone Marrow Transplant. 2013 Jul;48(7):947-52. doi: 10.1038/bmt.2012.276. Epub 2013 Jan 21.


Increasing treatment intensity has improved outcomes for children with neuroblastoma. We performed a pilot study in the Children's Oncology Group to assess the feasibility and toxicity of a tandem myeloablative regimen without TBI supported by autologous CD34-selected peripheral blood stem cells. Forty-one patients with high-risk neuroblastoma were enrolled; eight patients did not receive any myeloablative consolidation procedure and seven received only one. Two patients out of 41 (4.9%) experienced transplant-related mortality. CD34 selection was discontinued after subjects were enrolled due to serious viral illness. From the time of study enrollment, the overall 3-year EFS and OS were 44.8 ± 9.6% and 59.2 ± 9.2% (N=41). These results demonstrate that tandem transplantation in the cooperative group setting is feasible and support a randomized comparison of single vs tandem myeloablative consolidation with PBSC support for high-risk neuroblastoma.

Publication types

  • Clinical Trial
  • Multicenter Study
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Autografts
  • Female
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Neuroblastoma / mortality*
  • Neuroblastoma / therapy*
  • Peripheral Blood Stem Cell Transplantation*
  • Pilot Projects
  • Transplantation Conditioning / adverse effects
  • Transplantation Conditioning / methods*
  • Virus Diseases / etiology
  • Virus Diseases / mortality