Treatment of poor-risk neuroblastoma patients with high-dose chemotherapy and autologous peripheral stem cell rescue (original) (raw)

1997, Bone Marrow Transplantation

than 20% have long-term survival. 1,2 Because chemotherapy dose-intensity has been shown to correlate strongly with response and progression-free survival in metastatic A single institutional pilot study was conducted in which 12 poor-risk neuroblastoma (NB) patients were uni-NB, 3 many centers are currently treating poor-risk patients with consolidation regimens that include myeloablative formly treated with multi-agent induction chemotherapy followed by myeloablative consolidation chemo-chemotherapy or chemoradiotherapy followed by allogeneic bone marrow transplant (BMT) or autologous BMT. therapy and unpurged peripheral blood stem cell (PBSC) rescue. In addition to using standard criteria While the efficacy of myeloablative vs nonmyeloablative consolidation therapy has not been determined, some of the for evaluating response to induction chemotherapy, tumor cell contamination of the peripheral blood and/or best long-term survival rates have been reported with highdose myeloablative chemotherapy with or without total-bone marrow was analyzed in seven patients by immunocytology using a panel of five anti-NB mono-body irradiation, and bone marrow rescue. 2,4-8 However, the ability to provide bone marrow stem cells for patient clonal antibodies. Seven patients had morphologic evidence of bone marrow disease at the time of diagnosis, rescue following intensive therapy can be limited by marrow contamination with tumor cells, and by the lack of and two additional patients had tumor cells detected in bone marrow samples by immunocytology prior to the adequate marrow reserves to harvest sufficient numbers of stem cells. second cycle of chemotherapy. After three cycles of chemotherapy, two of the 12 patients continued to have Rescue with peripheral blood stem cells (PBSCs) has several advantages over autologous bone marrow. The pro-evidence of bone marrow disease. Samples from 29 PBSC harvests collected from nine patients were also genitor cell collection can be performed on an outpatient basis without general anesthesia, and in general PBSCs can analyzed for the presence of contaminating tumor cells by immunocytology. In each case, the stem cells were be successfully harvested in patients who do not have adequate marrow reserves. 9 In addition, rescue with PBSCs found to be free of tumor. Eleven of the 12 patients underwent myeloablative therapy and PBSC rescue; results in a significantly reduced cytopenic period after myeloablative therapy. 9-11 Further, studies in adults have five patients remain alive without disease progression, 28+ to 53+ months from diagnosis, and six patients have indicated that the peripheral blood is enriched with stem cells during the recovery phase following chemotherapy, developed recurrent disease. We conclude that PBSCs can be successfully harvested from children with NB, and with proper timing and growth factor support, higher yields of stem cells can be obtained from peripheral stem and used for hematopoietic reconstitution following myeloablative chemotherapy. However, more effective cell harvests than from conventional bone marrow harvests. 9,12 Although the experience with PBSC harvest and therapy for poor-risk NB patients is still urgently needed.