A pilot study of tandem high-dose chemotherapy with stem cell rescue as consolidation for high-risk neuroblastoma: Children's Oncology Group study ANBL00P1

Seif, A. E.; Naranjo, A.; Baker, D. L.; Bunin, N. J.; Kletzel, M.; Kretschmar, C. S.; Maris, J. M.; McGrady, P. W.; von Allmen, D.; Cohn, S. L.; London, W. B.; Park, J. R.; Diller, L. R.; Grupp, S. A.

Bone Marrow Transplant. 2013 Jan 22; 48(7):947-52


Increasing treatment intensity has improved outcomes for children with neuroblastoma. We performed a pilot study in the Children's Oncology Group to assess the feasibility and toxicity of a tandem myeloablative regimen without TBI supported by autologous CD34-selected peripheral blood stem cells. Forty-one patients with high-risk neuroblastoma were enrolled; eight patients did not receive any myeloablative consolidation procedure and seven received only one. Two patients out of 41 (4.9%) experienced transplant-related mortality. CD34 selection was discontinued after subjects were enrolled due to serious viral illness. From the time of study enrollment, the overall 3-year EFS and OS were 44.8+/-9.6% and 59.2+/-9.2% (N=41). These results demonstrate that tandem transplantation in the cooperative group setting is feasible and support a randomized comparison of single vs tandem myeloablative consolidation with PBSC support for high-risk neuroblastoma.

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