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.
authors.affiliation: Brain Tumor Center, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH, USA.
authors.forename: Joshua
authors.initials: J
authors.lastname: Baugh
authors.affiliation: Hospital for Sick Children, Toronto, ON, Canada.
authors.forename: Ute
authors.initials: U
authors.lastname: Bartels
authors.affiliation: Brain Tumor Center, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH, USA.
authors.forename: James
authors.initials: J
authors.lastname: Leach
authors.affiliation: Brain Tumor Center, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH, USA.
authors.forename: Blaise
authors.initials: B
authors.lastname: Jones
authors.affiliation: Brain Tumor Center, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH, USA.
authors.forename: Brooklyn
authors.initials: B
authors.lastname: Chaney
authors.affiliation: National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
authors.forename: Katherine E
authors.initials: KE
authors.lastname: Warren
authors.affiliation: Brain Tumor Center, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH, USA.
authors.forename: Jenavieve
authors.initials: J
authors.lastname: Kirkendall
authors.affiliation: Brain Tumor Center, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH, USA.
authors.forename: Renee
authors.initials: R
authors.lastname: Doughman
authors.affiliation: Hospital for Sick Children, Toronto, ON, Canada.
authors.forename: Cynthia
authors.initials: C
authors.lastname: Hawkins
authors.affiliation: Brain Tumor Center, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH, USA.
authors.forename: Lili
authors.initials: L
authors.lastname: Miles
authors.affiliation: Brain Tumor Center, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH, USA.
authors.forename: Christine
authors.initials: C
authors.lastname: Fuller
authors.affiliation: Lady Cilento Children's Hospital, Brisbane, Australia.
authors.forename: Tim
authors.initials: T
authors.lastname: Hassall
authors.affiliation: Hospital for Sick Children, Toronto, ON, Canada.
authors.forename: Eric
authors.initials: E
authors.lastname: Bouffet
authors.affiliation: Brain Tumor Center, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH, USA.
authors.forename: Adam
authors.initials: A
authors.lastname: Lane
authors.affiliation: Great Ormond Street Hospital for Children NHS Trust, London, UK.
authors.forename: Darren
authors.initials: D
authors.lastname: Hargrave
authors.affiliation: Gustave Roussy Institute, Villejuif, France.
authors.forename: Jacques
authors.initials: J
authors.lastname: Grill
authors.affiliation: Brain Tumor Center, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH, USA.
authors.forename: Lindsey M
authors.initials: LM
authors.lastname: Hoffman
authors.affiliation: Institute of Cancer Research, Sutton, UK.
authors.forename: Chris
authors.initials: C
authors.lastname: Jones
authors.affiliation: Brain Tumor Center, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH, USA.
authors.forename: Alex
authors.initials: A
authors.lastname: Towbin
authors.affiliation: National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.
authors.forename: Sharon A
authors.initials: SA
authors.lastname: Savage
authors.affiliation: Stanford University and Lucile Packard Children's Hospital, Palo Alto, CA, USA.
authors.forename: Michelle
authors.initials: M
authors.lastname: Monje
authors.affiliation: Texas Children's Hospital, Houston, TX, USA.
authors.forename: Xiao-Nan
authors.initials: XN
authors.lastname: Li
authors.affiliation: Sydney Children's Hospital, Randwick, NSW, Australia.|Children's Cancer Institute, University of New South Wales, Kensington, Australia.
authors.forename: David S
authors.initials: DS
authors.lastname: Ziegler
authors.affiliation: VU University Medical Center, Amsterdam, The Netherlands.
authors.forename: Sophie
authors.initials: S
authors.lastname: Veldhuijzen van Zanten
authors.affiliation: University of Göttingen, Göttingen, Germany.
authors.forename: Christof M
authors.initials: CM
authors.lastname: Kramm
authors.affiliation: VU University Medical Center, Amsterdam, The Netherlands.
authors.forename: Dannis G
authors.initials: DG
authors.lastname: van Vuurden
authors.affiliation: Brain Tumor Center, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH, USA. maryam.fouladi@cchmc.org.
authors.forename: Maryam
authors.initials: M
authors.lastname: Fouladi
mesh_terms: D000293:Adolescent; D000328:Adult; D001315:Australia; D020295:Brain Stem Neoplasms / Q000000981:diagnostic imaging / Q000453:epidemiology* / Q000473:pathology* / Q000628:therapy; D002170:Canada; D002648:Child; D002675:Child, Preschool; D005260:Female; D005910:Glioma / Q000000981:diagnostic imaging / Q000453:epidemiology* / Q000473:pathology* / Q000628:therapy; D006801:Humans; D007223:Infant; D007391:International Cooperation; D008279:Magnetic Resonance Imaging; D008297:Male; D009520:New Zealand; D011149:Pons / Q000000981:diagnostic imaging / Q000473:pathology*; D012042:Registries; D014481:United States; D055815:Young Adult
keywords: Brain tumor; Diffuse intrinsic pontine glioma; Registry