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The international diffuse intrinsic pontine glioma registry: an infrastructure to accelerate collaborative research for an orphan disease

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Abstract

Diffuse intrinsic pontine glioma (DIPG), a rare, often fatal childhood brain tumor, remains a major therapeutic challenge. In 2012, investigators, funded by the DIPG Collaborative (a philanthropic partnership among 29 private foundations), launched the International DIPG Registry (IDIPGR) to advance understanding of DIPG. Comprised of comprehensive deidentified but linked clinical, imaging, histopathological, and genomic repositories, the IDIPGR uses standardized case report forms for uniform data collection; serial imaging and histopathology are centrally reviewed by IDIPGR neuro-radiologists and neuro-pathologists, respectively. Tissue and genomic data, and cell cultures derived from autopsies coordinated by the IDIPGR are available to investigators for studies approved by the Scientific Advisory Committee. From April 2012 to December 2016, 670 patients diagnosed with DIPG have been enrolled from 55 participating institutions in the US, Canada, Australia and New Zealand. The radiology repository contains 3558 studies from 448 patients. The pathology repository contains tissue on 81 patients with another 98 samples available for submission. Fresh DIPG tissue from seven autopsies has been sent to investigators to develop primary cell cultures. The bioinformatics repository contains next-generation sequencing data on 66 tumors. Nine projects using data/tissue from the IDIPGR by 13 principle investigators from around the world are now underway. The IDIPGR, a successful alliance among philanthropic agencies and investigators, has developed and maintained a highly collaborative, hypothesis-driven research infrastructure for interdisciplinary and translational projects in DIPG to improve diagnosis, response assessment, treatment and outcome for patients.

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Acknowledgements

We gratefully acknowledge all patients and their families who have supported and contributed to the International DIPG Registry. Supporting foundations including: The Cure Starts Now Foundation, Hope for Caroline Foundation, Julian Boivin Courage for Cures Foundation, Abbie’s Army, Michael Mosier Defeat DIPG Foundation, Brooke Healey Foundation, Reflections of Grace Foundation, The Cure Starts Now Australia, Soar with Grace Foundation, Jeffrey Thomas Hayden Foundation, Cure Brain Cancer Foundation, The Jones Family Foundation, Musella Foundation for Brain Tumor Research and Information, The Pray, Hope Believe Foundation, Benny’s World, Smiles for Sophie Forever, Aiden’s Avengers, Love, Chloe Foundation, A Cure from Caleb Society, Operation Grace White Foundation, Ryan’s Hope, American Childhood Cancer Organization, Juliana Rose Donnelly Trust, Joshua’s Wish, The Ellie Kavalieros DIPG Research Fund, Voices Against Brain Cancer, Wayland’s Warriors, Shelia Jones & Friends and The DIPG Collaborative.

Funding

This research was financially supported by the DIPG Collaborative.

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Authors and Affiliations

  1. Brain Tumor Center, Cancer and Blood Diseases Institute, Cincinnati Children’s Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH, USA

    Joshua Baugh, James Leach, Blaise Jones, Brooklyn Chaney, Jenavieve Kirkendall, Renee Doughman, Lili Miles, Christine Fuller, Adam Lane, Lindsey M. Hoffman, Alex Towbin & Maryam Fouladi

  2. Hospital for Sick Children, Toronto, ON, Canada

    Ute Bartels, Cynthia Hawkins & Eric Bouffet

  3. National Cancer Institute, National Institutes of Health, Bethesda, MD, USA

    Katherine E. Warren & Sharon A. Savage

  4. Lady Cilento Children’s Hospital, Brisbane, Australia

    Tim Hassall

  5. Great Ormond Street Hospital for Children NHS Trust, London, UK

    Darren Hargrave

  6. Gustave Roussy Institute, Villejuif, France

    Jacques Grill

  7. Institute of Cancer Research, Sutton, UK

    Chris Jones

  8. Stanford University and Lucile Packard Children’s Hospital, Palo Alto, CA, USA

    Michelle Monje

  9. Texas Children’s Hospital, Houston, TX, USA

    Xiao-Nan Li

  10. Sydney Children’s Hospital, Randwick, NSW, Australia

    David S. Ziegler

  11. Children’s Cancer Institute, University of New South Wales, Kensington, Australia

    David S. Ziegler

  12. VU University Medical Center, Amsterdam, The Netherlands

    Sophie Veldhuijzen van Zanten & Dannis G. van Vuurden

  13. University of Göttingen, Göttingen, Germany

    Christof M. Kramm

Authors
  1. Joshua Baugh
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  2. Ute Bartels
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  3. James Leach
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  4. Blaise Jones
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  6. Katherine E. Warren
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  7. Jenavieve Kirkendall
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  8. Renee Doughman
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  9. Cynthia Hawkins
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  11. Christine Fuller
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  12. Tim Hassall
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  13. Eric Bouffet
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  14. Adam Lane
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  15. Darren Hargrave
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  16. Jacques Grill
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  17. Lindsey M. Hoffman
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  18. Chris Jones
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  19. Alex Towbin
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  20. Sharon A. Savage
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  21. Michelle Monje
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  22. Xiao-Nan Li
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  23. David S. Ziegler
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  24. Sophie Veldhuijzen van Zanten
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  26. Dannis G. van Vuurden
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  27. Maryam Fouladi
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Corresponding author

Correspondence to Maryam Fouladi.

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Baugh, J., Bartels, U., Leach, J. et al. The international diffuse intrinsic pontine glioma registry: an infrastructure to accelerate collaborative research for an orphan disease. J Neurooncol 132, 323–331 (2017). https://doi.org/10.1007/s11060-017-2372-5

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  • DOI: https://doi.org/10.1007/s11060-017-2372-5

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