To transplant, or not to transplant? That is the question. A patient advocate evaluation of autologous stem cell transplant in neuroblastoma

Pediatr Blood Cancer. 2022 Aug;69(8):e29663. doi: 10.1002/pbc.29663. Epub 2022 Apr 4.


High-dose chemotherapy with autologous stem cell transplant (ASCT) has been a mainstay of high-risk neuroblastoma treatment for several decades, demonstrating improvements in event-free survival but with risks of serious or even life-threatening acute toxicities, severe long-term adverse health effects for survivors, and ongoing contention regarding overall survival benefit. The merits of ASCT in the modern era of immunotherapy are a source of debate among parents, advocates, and some physicians. Here we examine evidence for and against ASCT, explore parent attitudes and their turmoil over decision-making, and strongly encourage international research consortia to develop a coordinated strategy to accelerate progress toward a future that avoids the routine use of ASCT in high-risk neuroblastoma.

Keywords: Neuroblastoma; advocacy; advocate; autologous; cell; stem; transplant.

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Disease-Free Survival
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Immunotherapy
  • Neuroblastoma*
  • Patient Advocacy
  • Transplantation, Autologous