Autologous Hematopoietic Cell Transplantation Can Prolong Life in High-Risk Neuroblastoma Patients in Partial Remission

Recent recommendations for children with neuroblastoma are to not proceed to autologous transplantation (aHCT) unless a complete remission (CR) or a very good partial remission (VGPR) has been achieved according to the International Neuroblastoma Staging System Response Evaluation Criteria.  Because our anecdotal experience suggested that patients in partial remission (PR) may benefit, we reviewed data from the University of North Carolina at Chapel Hill for outcomes following aHCT in PR. Between September 1988 and June 2012, 102 patients were newly diagnosed, and 29 patients (22 males) at a median age of  4.1 years (range, 0.7-32.7 years) underwent aHCT at our institution between May 1995 and September 2012.  At the time of aHCT, 9 (31%) were in CR, 4 (13.8%) in VGPR, 15 (51.7%) in PR, and 1 (3.4%) had a mixed response (MR) with then-current response criteria. All received preparative regimens including melphalan, carboplatin, and etoposide. Thirteen (44.8%) received fractionated TBI as part of the conditioning regimen on the CCG 3891protocol; 2 received tandem aHCT on the COG ANBL 0532 protocol.  Twenty-six (89.7%) received peripheral blood stem cell grafts.  Of  these 29 patients, 13 (44.8%) remain alive at a median of 852 days (range, 103-6339 days) including 5/9 (55.6%) transplanted in CR, 2/4 (50%) in VGPR, and 6/15 (40%) in PR. Of those in PR at aHCT, 6 remain alive at a median of 526 days (range, 103-2506 days) and 9 died at a median of  404 days (range, 251-1043 days).   Disease status of 14 evaluable PR recipients at day 100 included 4 who achieved CR, 8 remaining in PR, and 2 with progressive disease. Twelve of these 15 are known to have received further therapy including local radiation, chimeric antibody, retinoic acid, and chemotherapy.  The 1-year overall survival for those transplanted in PR was approximately 36%.  Thus, aHCT in PR may not only contribute to improving survival for these high risk children but may also provide a bridge to other therapies that continue to provide hope to families.