Oral Presentation Advances in Neuroblastoma Research Congress 2016

Revisions to the International Neuroblastoma Response Criteria: A consensus statement from the NCI-Clinical Trials Planning Meeting (#61)

Julie R. Park 1 , Rochelle Bagatell 2 , Susan L Cohn 3 , Andy Pearson 4 , Julia G Villablanca 5 , Frank Berthold 6 , Sue Burchill 7 , Ariane Boubaker 8 , Kieran McHugh 9 , Jed G Nuchtern 10 , Wendy B London 11 , Nita L Seibel 12 , O Wolf ]Lindwasser 13 , John M Maris 2 , Penelope Brock 9 , Gudrun Schleiermacher 14 , Ruth Ladenstein 15 , Katherine K Matthay 16 , Dominique Valteau-Couanet 17
  1. University of Washington, Seattle Children's Hospital, Seattle, WA, United States
  2. Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA
  3. University of Chicago, Chicago, Illinois, USA
  4. Divisions of Cancer Therapeutics and Clinical Studies, Institute of Cancer Research and Children and Young People’s Unit, The Royal Marsden NHS Foundation Trust, Sutton, Surrey , UK
  5. Children’s Hospital Los Angeles and Department of Pediatrics, USC Keck School of Medicine, Los Angeles, California, USA
  6. Children’s Hospital and University of Cologne, Koeln, Germany
  7. Leeds Institute of Cancer and Pathology, University of Leeds, Leeds, United Kingdom
  8. Nuclear Medicine Service, Central Hospital University Vaudois, Lausanne, CH
  9. Great Ormond Street Hospital for Children NHS Trust, London, United Kingdom
  10. Departments of Surgery and Pediatrics, Texas Children's Hospital and Baylor College of Medicine, Houston, Texas, USA
  11. Dana-Farber/Boston Children’s Cancer and Blood Disorder Center, Harvard Medical School, Boston, MA, USA
  12. Clinical Investigations Branch, National Cancer Institute, Bethesda, Maryland, USA
  13. Coordinating Center for Clinical Trials, National Cancer Institute, Bethesda, Maryland, USA
  14. Department of Pediatric Oncology, Institut Curie, Paris, France
  15. Children’s Cancer Research Institute, St Anna Children’s Hospital, VIenna, Austria
  16. Department of Pediatrics, , UCSF School of Medicine, SanFrancisco, California, USA
  17. Department of Pediatric Oncology, Gustave Roussy, Paris, France

Purpose: More than two decades ago, an international working group established the International Neuroblastoma Response Criteria (INRC) to assess treatment response in children with neuroblastoma. However, this system required modification to incorporate modern imaging techniques and new methods for quantifying bone marrow disease that were not previously widely available. The National Cancer Institute sponsored a Clinical Trials Planning Meeting (CTPM) in 2012 to update and refine response criteria for patients with neuroblastoma.  

Methods: Multi-disciplinary investigators from 13 countries reviewed data from published trials performed through cooperative groups, consortia and single institutions. Data from both prospective and retrospective trials were used to refine the INRC. Monthly international conference calls were held from 2011 – 2015 and consensus was reached through review by working group leadership and the NCI CTPM leadership council.

Results: Overall response in the revised INRC will integrate response with respect to 3 components: primary tumor, soft tissue and bone metastases, and bone marrow metastases. Primary and metastatic soft tissue sites will be assessed using RECIST and 123I-metaiodobenzylguanidine (123I-MIBG) or 18F-fluorodeoxyglucose (FDG)-Positive emission tomography (PET) scans if MIBG non-avid disease. 123I-MIBG or FDG-PET scans (if MIBG non-avid) replace 99Technetium (TC) bone scans to assess bone metastases. Bone marrow (BM) will be assessed by histology/immunohistology and immunocytology. BM with ≤5% tumor involvement will be classified as minimal BM disease. Urinary catecholamines will not be included in response assessment. Overall response categories will be defined as complete response, partial response, minor response, stable disease and progressive disease.

Conclusions: These revised criteria permit uniform application of consensus definitions of disease response, improve the interpretability of clinical trial results and facilitate collaborative trial designs.