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Pediatric Transplants

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

Bone Marrow Transplantation volume 48, pages 947–952 (2013)Cite this article

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Abstract

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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Acknowledgements

Supported in part by the WW Smith Charitable Trust, and the Foerderer-Murray, Weinberg and Sanford Funds of the Children’s Hospital of Philadelphia (S.A. Grupp); the Robert A. Good/ASBMT New Investigator Award (A.E. Seif); NIH grants U10 CA98413 (COG SDC grant) and U10 CA98543 (COG Chair’s grant). We would also like to thank Danniel Gaidula, MFA, for assistance in preparing Figure 1.

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Authors and Affiliations

  1. Department of Pediatrics, Division of Oncology, The Children’s Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia, PA, USA

    A E Seif, N J Bunin, J M Maris & S A Grupp

  2. Department of Biostatistics, Colleges of Medicine and Public Health & Health Professions, University of Florida, Gainesville, FL, USA

    A Naranjo & P W McGrady

  3. Children’s Oncology Group Statistics and Data Center, Arcadia, CA, USA

    A Naranjo, P W McGrady & W B London

  4. Department of Haematology and Oncology, Princess Margaret Hospital for Children, Perth, WA, Australia

    D L Baker

  5. Department of Pediatrics, Division of Hematology, Oncology and Stem Cell Transplant, Children’s Memorial Hospital and Northwestern University Feinberg School of Medicine, Chicago, IL, USA

    M Kletzel

  6. Brookline, MA, USA

    C S Kretschmar

  7. Department of Surgery, Division of General and Thoracic Surgery, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA

    D von Allmen

  8. Department of Pediatrics, Division of Hematology/Oncology, Comer Children’s Hospital, Chicago, IL, USA

    S L Cohn

  9. Department of Pediatrics, Division of Hematology/Oncology, Children’s Hospital Boston/Dana-Farber Cancer Institute, Boston, MA, USA

    W B London & L R Diller

  10. Department of Pediatrics, Division of Oncology, Seattle Children’s Hospital and University of Washington School of Medicine, Seattle, WA, USA

    J R Park

  11. Department of Pathology, The Children’s Hospital of Philadelphia and Perelman School of Medicine of the University of Pennsylvania, Philadelphia, PA, USA

    S A Grupp

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Correspondence to S A Grupp.

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Seif, A., Naranjo, A., Baker, D. et al. A pilot study of tandem high-dose chemotherapy with stem cell rescue as consolidation for high-risk neuroblastoma: Children’s Oncology Group study ANBL00P1. Bone Marrow Transplant 48, 947–952 (2013). https://doi.org/10.1038/bmt.2012.276

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