Allogeneic hematopoietic cell transplantation for neuroblastoma: the CIBMTR experience

While the role of auto-HCT is well established in neuroblastoma, the role of allo-HCT is controversial. The CIBMTR conducted a retrospective review of 143 allo-HCT for NBL reported in 1990-2007. Patients were categorized into two different groups: those who had not (Group 1) and had (Group 2) undergone a prior auto HCT (n=46 and 97, respectively). One-year and five-year overall survival (OS) were 59% and 29% for Group 1 and 50% and 7% for Group 2. Amongst donor types, disease free survival (DFS) and OS were significantly lower for unrelated transplants at 1 and 3 years but not 5 years post-HCT. Patients in complete response (CR) or very good partial response (VGPR) at transplant had lower relapse rates and better DFS and OS, compared to those not in CR or VGPR. Our analysis indicates that allo-HCT can cure some neuroblastoma patients, with lower relapse rates and improved survival in patients without a history of prior auto-HCT as compared to those patients who had previously undergone auto-HCT. Although the data do not address why either strategy was chosen for patients, allo-HCT after a prior auto-HCT appears to offer minimal benefit. Disease recurrence remains the most common cause of treatment failure.