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

Joshua Baugh; Ute Bartels; James Leach; Blaise Jones; Brooklyn Chaney; Katherine E. Warren; Jenavieve Kirkendall; Renee Doughman; Cynthia Hawkins; Lili Miles; Christine Fuller; Tim Hassall; Eric Bouffet; Adam Lane; Darren Hargrave; Jacques Grill; Lindsey M. Hoffman; Chris Jones; Alex Towbin; Sharon A. Savage; Michelle Monje; Xiao Nan Li; David S. Ziegler; Sophie Veldhuijzen van Zanten; Christof M. Kramm; Dannis G. van Vuurden; Maryam Fouladi

doi:10.1007/s11060-017-2372-5

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

Joshua Baugh, Ute Bartels, James Leach, Blaise Jones, Brooklyn Chaney, Katherine E. Warren, Jenavieve Kirkendall, Renee Doughman, Cynthia Hawkins, Lili Miles, Christine Fuller, Tim Hassall, Eric Bouffet, Adam Lane, Darren Hargrave, Jacques Grill, Lindsey M. Hoffman, Chris Jones, Alex Towbin, Sharon A. SavageMichelle Monje, Xiao Nan Li, David S. Ziegler, Sophie Veldhuijzen van Zanten, Christof M. Kramm, Dannis G. van Vuurden, Maryam Fouladi^*

^*Corresponding author for this work

Pediatrics

Research output: Contribution to journal › Article › peer-review

31 Scopus citations

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.

Original language	English (US)
Pages (from-to)	323-331
Number of pages	9
Journal	Journal of Neuro-Oncology
Volume	132
Issue number	2
DOIs	https://doi.org/10.1007/s11060-017-2372-5
State	Published - Apr 1 2017

Funding

In 2011, physicians, scientists, and patient advocacy groups met at the first International DIPG Symposium, and advocated for the establishment of a focused international effort to develop uniform criteria for diagnosis, classification, disease assessment, and to study DIPG biology and therapeutic strategies through the development of in vitro and in vivo models. In 2012, with financial support from the DIPG Collaborative, a philanthropic partnership which now includes 29 private foundations, and international investigators banded together to establish the International DIPG Registry (IDIPGR) and a parallel European SIOPE Registry. The IDIPGR continues to expand and maintains a highly-collaborative, hypothesis-driven research infrastructure to support a wide spectrum of interdisciplinary and translational projects in DIPG. Here, we report the logistical challenges, pitfalls, and successes of developing this registry, which we hope will serve as a model for other orphan disease registries.

Keywords

Brain tumor
Diffuse intrinsic pontine glioma
Registry

ASJC Scopus subject areas

Oncology
Neurology
Clinical Neurology
Cancer Research

Access to Document

10.1007/s11060-017-2372-5

Cite this

Baugh, J., Bartels, U., Leach, J., Jones, B., Chaney, B., Warren, K. E., Kirkendall, J., Doughman, R., Hawkins, C., Miles, L., Fuller, C., Hassall, T., Bouffet, E., Lane, A., Hargrave, D., Grill, J., Hoffman, L. M., Jones, C., Towbin, A., ... Fouladi, M. (2017). The international diffuse intrinsic pontine glioma registry: an infrastructure to accelerate collaborative research for an orphan disease. Journal of Neuro-Oncology, 132(2), 323-331. https://doi.org/10.1007/s11060-017-2372-5

@article{02357e765a90473889822c2acfd37f2f,

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

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.",

keywords = "Brain tumor, Diffuse intrinsic pontine glioma, Registry",

author = "Joshua Baugh and Ute Bartels and James Leach and Blaise Jones and Brooklyn Chaney and Warren, {Katherine E.} and Jenavieve Kirkendall and Renee Doughman and Cynthia Hawkins and Lili Miles and Christine Fuller and Tim Hassall and Eric Bouffet and Adam Lane and Darren Hargrave and Jacques Grill and Hoffman, {Lindsey M.} and Chris Jones and Alex Towbin and Savage, {Sharon A.} and Michelle Monje and Li, {Xiao Nan} and Ziegler, {David S.} and {Veldhuijzen van Zanten}, Sophie and Kramm, {Christof M.} and {van Vuurden}, {Dannis G.} and Maryam Fouladi",

note = "Funding Information: In 2011, physicians, scientists, and patient advocacy groups met at the first International DIPG Symposium, and advocated for the establishment of a focused international effort to develop uniform criteria for diagnosis, classification, disease assessment, and to study DIPG biology and therapeutic strategies through the development of in vitro and in vivo models. In 2012, with financial support from the DIPG Collaborative, a philanthropic partnership which now includes 29 private foundations, and international investigators banded together to establish the International DIPG Registry (IDIPGR) and a parallel European SIOPE Registry. The IDIPGR continues to expand and maintains a highly-collaborative, hypothesis-driven research infrastructure to support a wide spectrum of interdisciplinary and translational projects in DIPG. Here, we report the logistical challenges, pitfalls, and successes of developing this registry, which we hope will serve as a model for other orphan disease registries. Publisher Copyright: {\textcopyright} 2017, Springer Science+Business Media New York.",

year = "2017",

month = apr,

day = "1",

doi = "10.1007/s11060-017-2372-5",

language = "English (US)",

volume = "132",

pages = "323--331",

journal = "Journal of Neuro-Oncology",

issn = "0167-594X",

publisher = "Springer",

number = "2",

}

Baugh, J, Bartels, U, Leach, J, Jones, B, Chaney, B, Warren, KE, Kirkendall, J, Doughman, R, Hawkins, C, Miles, L, Fuller, C, Hassall, T, Bouffet, E, Lane, A, Hargrave, D, Grill, J, Hoffman, LM, Jones, C, Towbin, A, Savage, SA, Monje, M, Li, XN, Ziegler, DS, Veldhuijzen van Zanten, S, Kramm, CM, van Vuurden, DG & Fouladi, M 2017, 'The international diffuse intrinsic pontine glioma registry: an infrastructure to accelerate collaborative research for an orphan disease', Journal of Neuro-Oncology, vol. 132, no. 2, pp. 323-331. https://doi.org/10.1007/s11060-017-2372-5

TY - JOUR

T1 - The international diffuse intrinsic pontine glioma registry

T2 - an infrastructure to accelerate collaborative research for an orphan disease

AU - Baugh, Joshua

AU - Bartels, Ute

AU - Leach, James

AU - Jones, Blaise

AU - Chaney, Brooklyn

AU - Warren, Katherine E.

AU - Kirkendall, Jenavieve

AU - Doughman, Renee

AU - Hawkins, Cynthia

AU - Miles, Lili

AU - Fuller, Christine

AU - Hassall, Tim

AU - Bouffet, Eric

AU - Lane, Adam

AU - Hargrave, Darren

AU - Grill, Jacques

AU - Hoffman, Lindsey M.

AU - Jones, Chris

AU - Towbin, Alex

AU - Savage, Sharon A.

AU - Monje, Michelle

AU - Li, Xiao Nan

AU - Ziegler, David S.

AU - Veldhuijzen van Zanten, Sophie

AU - Kramm, Christof M.

AU - van Vuurden, Dannis G.

AU - Fouladi, Maryam

N1 - Funding Information: In 2011, physicians, scientists, and patient advocacy groups met at the first International DIPG Symposium, and advocated for the establishment of a focused international effort to develop uniform criteria for diagnosis, classification, disease assessment, and to study DIPG biology and therapeutic strategies through the development of in vitro and in vivo models. In 2012, with financial support from the DIPG Collaborative, a philanthropic partnership which now includes 29 private foundations, and international investigators banded together to establish the International DIPG Registry (IDIPGR) and a parallel European SIOPE Registry. The IDIPGR continues to expand and maintains a highly-collaborative, hypothesis-driven research infrastructure to support a wide spectrum of interdisciplinary and translational projects in DIPG. Here, we report the logistical challenges, pitfalls, and successes of developing this registry, which we hope will serve as a model for other orphan disease registries. Publisher Copyright: © 2017, Springer Science+Business Media New York.

PY - 2017/4/1

Y1 - 2017/4/1

N2 - 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.

AB - 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.

KW - Brain tumor

KW - Diffuse intrinsic pontine glioma

KW - Registry

UR - http://www.scopus.com/inward/record.url?scp=85009469009&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85009469009&partnerID=8YFLogxK

U2 - 10.1007/s11060-017-2372-5

DO - 10.1007/s11060-017-2372-5

M3 - Article

C2 - 28093680

AN - SCOPUS:85009469009

SN - 0167-594X

VL - 132

SP - 323

EP - 331

JO - Journal of Neuro-Oncology

JF - Journal of Neuro-Oncology

IS - 2

ER -

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

Abstract

Funding

Keywords

ASJC Scopus subject areas

Access to Document

Other files and links

Fingerprint

Cite this