Complete remission following decitabine/dendritic cell vaccine for relapsed neuroblastoma

Pediatrics. 2013 Jan;131(1):e336-41. doi: 10.1542/peds.2012-0376. Epub 2012 Dec 24.


Patients with relapsed stage 4 neuroblastoma have an extremely poor long-term prognosis, making the investigation of new agents of interest. We report the outcome of the first patient treated in a phase 1 study for relapsed neuroblastoma, using the chemotherapy agent decitabine to upregulate cancer testis antigen expression, followed by a dendritic cell vaccine targeting the cancer testis antigens MAGE-A1, MAGE-A3, and NY-ESO-1. Our patient had persistent tumor in his bone marrow after completion of standard therapy for neuroblastoma, including multiagent chemotherapy, tumor resection, stem cell transplantation, radiation therapy, and anti-GD2 monoclonal antibodies. His marrow disease persisted despite chemotherapy, which was given while the vaccine was being produced. After 3 cycles of decitabine and vaccine, this patient achieved a complete remission and is now 1 year from his last treatment, with no evidence of tumor in his bone marrow or other sites. This patient was noted to have an increase in MAGE-A3-specific T cells. This is the first report combining demethylating chemotherapy to enhance tumor antigen expression followed by a cancer antigen vaccine.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Azacitidine / administration & dosage
  • Azacitidine / analogs & derivatives*
  • Cancer Vaccines / administration & dosage*
  • Cancer Vaccines / immunology
  • Child
  • Decitabine
  • Dendritic Cells / transplantation*
  • Hematopoietic Stem Cell Transplantation / trends
  • Humans
  • Male
  • Neuroblastoma / diagnosis*
  • Neuroblastoma / immunology
  • Neuroblastoma / prevention & control*
  • Recurrence
  • Remission Induction / methods


  • Cancer Vaccines
  • Decitabine
  • Azacitidine