Oral Presentation Advances in Neuroblastoma Research Congress 2016

The way towards an international mIBG skeletal score for high risk neuroblastoma: the statistical perspective (#93)

Ulrike Pötschger 1 , Arlene Naranjo 2 , Gregory Yanik 3 , Maria-Rita Castellani 4 , Ariane Boubaker 5 , Bieke Lambert 6 , Valerie Lewington 7 , Zvi Bar-Sever 8 , Aurore Oudoux 9 , Anna Kaminska 10 , Katarina Taborska 11 , Lorenzo Biassoni 12 , Marguerite T. Parisi 13 , Barry L. Shulkin 14 , Helen R Nadel 15 , Michael J Gelfand 16 , Julie R. Park 17 , Susan G Kreissman 18 , Dominique Valteau-Couanet 19 , Katherine K. Matthay 20 , Ruth Ladenstein 21
  1. St. Anna Kinderkrebsforschung e.V., Vienna, Austria
  2. Children's Oncology Group Statistics and Data Center, University of Florida, Gainesville, FL, USA
  3. University of Michigan Medical Center, Ann Arbor, MI, USA
  4. Nuclear Medicine Division, Istituto Nazionale Tumori di Milano, Milan, Italy
  5. Centre Hospitalier Universitaire Vaudois, Lausanne, Switzerland
  6. Radiology and Nuclear Medicine, Ghent University , Ghent, Belgium
  7. Guy's and St. Thomas' NHS Foundation Trust, London, UK
  8. Schneider Children's Medical Center of Israel, Petah-Tikva, Israel
  9. Centre Oscar-Lambret, Lille, France
  10. Children's Memorial Health Institute, Warsaw, Poland
  11. Motol University Hospital, Prague, Czech Republic
  12. Great Ormond Street Hospital for Childen NHS Foundation Trust, London, UK
  13. Seattle Children's Hospital Research Foundation, Seattle, WA, USA
  14. St. Jude Children's Research Hospital, Memphis, TN, USA
  15. Children's and Women's Health Centre of British Columbia, Vancouver, BC, Canada
  16. Cincinnati Children's Hospital, Cincinnati, Ohio, USA
  17. Seattle Children's Hospital, Seattle, WA, USA
  18. Duke University Medical Center, Durham, NC, USA
  19. Institut Gustav Roussy, Villejuif, France
  20. University of California, San Francisco, CA, USA
  21. St. Anna Children's Hospital & Pediatric Medical University, Vienna, Austria

PURPOSE:

A collaborative effort was undertaken to derive an internationally agreed semi-quantitative meta-iodobenzylguanidine (mIBG) scoring method in neuroblastoma by harmonising previously established scoring systems, which were each found to have prognostic value at the end of induction (Yanik JNM 2013). The aim of this analysis was to investigate the individual effect on event-free-survival (EFS) of the two components of the scoring system: 1) the number of involved anatomic regions and 2) the scoring value within each segment and to evaluate the prognostic value of the new proposed scoring system.

PATIENTS AND METHODS:

COG and SIOPEN merged data of children with stage 4, mIBG avid, neuroblastoma entered on the COG-A3973 (216pts) and the SIOPEN/HR-NBL1 trial (341 pts). Two independent nuclear medicine review teams scored mIBG scans pre- and post-induction according to Curie- and SIOPEN-methodologies. Here, the SIOPEN score evaluating the skeletal (mIBG) uptake on a 0-6 scale in 12 anatomical regions was chosen for the statistical analysis due to the greater range of values. The two study cohorts were investigated separately and a bootstrap-based internal validation was performed.

RESULTS

In 557 pts the cumulative SIOPEN-score post induction had a significant impact on EFS with 5-years EFS of 41%, 33% and 15% for total scores of 0, 1-3 and >3, respectively. However, no increasing hazards with increasing scores per segment were observed. In contrast, the number of positive segments alone had a highly significant impact on EFS with 5-year EFS of 41%, 32% and 14% for patients with 0,1-2 and >2 positive segments post induction.

CONCLUSION

The number of positive segments was the most important prognostic factor. Weighting the involvement within segments did not improve the prognostic value of the scoring system. These results suggest a possible simplification of MIBG scoring, facilitating future international collaborations.