Craig Erker1, Adam Lane2, Brooklyn Chaney3, Sarah Leary4, Jane E Minturn5, Ute Bartels6, Roger J Packer7, Kathleen Dorris8, Nicholas G Gottardo9,10, Katherine E Warren11, Alberto Broniscer12,13, Mark W Kieran14, Xiaoting Zhu15,16,17, Peter White18, Phillip J Dexheimer18, Katie Black3, Anthony Asher3, Mariko DeWire3, Jordan R Hansford19, Sridharan Gururangan20, Javad Nazarian21,22, David S Ziegler23,24, Eric Sandler25, Allison Bartlett3, Stewart Goldman26, Chie-Schin Shih27, Tim Hassall28, Hetal Dholaria29, Pratiti Bandopadhayay11, Yvan Samson30, Michelle Monje31, Paul G Fisher31, Andrew Dodgshun32, Sarah Parkin32, Murali Chintagumpala33, Karen Tsui34, David Gass35, Valerie Larouche36, Emmett Broxson37, Mercedes Garcia Lombardi38, Stacie Shiqi Wang19, Jie Ma39, Cynthia Hawkins40, Dima Hamideh41, Lars Wagner42, Carl Koschmann43, Christine Fuller44, Rachid Drissi45,46, Blaise V Jones47, James Leach47, Maryam Fouladi46,48. 1. Department of Pediatrics, Dalhousie University and IWK Health Centre, Halifax, Nova Scotia, Canada. 2. Division of Bone Marrow Transplantation and Immune Deficiency, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA. 3. Division of Oncology, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA. 4. Division of Hematology/Oncology, Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA. 5. Division of Oncology, Children's Hospital of Philadelphia and Perelman School of Medicine, Philadelphia, Pennsylvania, USA. 6. Department of Pediatrics, Division of Oncology, University of Toronto and The Hospital for Sick Children, Toronto, Ontario, Canada. 7. Department of Neurology, Center for Neuroscience and Behavioral Medicine, Children's National Hospital, Washington, DC, USA. 8. Department of Pediatrics, University of Colorado School of Medicine, Aurora, Colorado, USA. 9. Department of Paediatric and Adolescent Oncology/Haematology, Perth Children's Hospital, Nedlands, Western Australia, Australia. 10. Brain Tumour Research Programme, Telethon Kids Institute, Nedlands, Western Australia, Australia. 11. Department of Pediatric Oncology, Dana-Farber Cancer Institute/Boston Children's Hospital, Boston, Massachusetts, USA. 12. Department of Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee, USA. 13. Department of Pediatrics, University of Tennessee Health Science Center, Memphis, Tennessee, USA. 14. Department of Pediatrics, Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Harvard Medical School, Boston, Massachusetts, USA. 15. Brain Tumor Center, Division of Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA. 16. Department of Electrical Engineering and Computer Science, University of Cincinnati College of Engineering and Applied Science, Cincinnati, Ohio, USA. 17. Department of Biomedical Informatics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA. 18. Department of Biomedical Informatics, Cincinnati Children's Hospital Medical Center and University of Cincinnati, Cincinnati, Ohio, USA. 19. Children's Cancer Centre, Royal Children's Hospital Melbourne Cancer, Cell Biology, Murdoch Children's Research Institute Department of Paediatrics, University of Melbourne, Melbourne, Victoria, Australia. 20. Preston A. Wells Center for Brain Tumor Therapy, UF Health Shands Hospital, Gainesville, Florida, USA. 21. Center for Genetic Medicine, Children's National Hospital, Washington, DC, USA. 22. Department of Oncology, University Children's Hospital, Zurich, Zurich, Switzerland. 23. Children's Cancer Institute Australia, Lowy Cancer Research Centre, UNSW and Kids Cancer Centre, Sydney's Children Hospital, Randwick, Sydney, New South Wales, Australia. 24. School of Women's and Children's Health, University of New South Wales, Sydney, New South Wales, Australia. 25. Department of Pediatrics, Wolfson Children's Hospital and Nemours Children's Specialty Care, Jacksonville, Florida, USA. 26. Division of Hematology/Oncology, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA. 27. Riley Children's Hospital, Indiana University, Indianapolis, Indiana, USA. 28. Lady Cilento Children's Hospital, Brisbane, Queensland, Australia. 29. Princess Margaret Hospital for Children and The University of Western Australia, Perth, Western Australia, Australia. 30. Départment de Pédiatrie, CHU Sainte-Justine and Université de Montréal, Montreal, Quebec, Canada. 31. Department of Neurology and Neurological Sciences, Stanford University and Lucile Packard Children's Hospital at Stanford, Stanford, California, USA. 32. Children's Haematology and Oncology Centre, Christchurch Hospital, Christchurch, New Zealand. 33. Department of Pediatrics, Section of Hematology/Oncology, Texas Children's Cancer Center, Houston, Texas, USA. 34. Starship Children's Hospital, Auckland, New Zealand. 35. Division Hematology and Oncology, Levine Children's Cancer & Blood Disorders, Charlotte, North Carolina, USA. 36. Department of Pediatrics, Centre Mère-enfant Soleil du CHU de Québec-Université Laval, Quebec City, Quebec, Canada. 37. Division of Hematology/Oncology, Wright State University and Dayton Children's Hospital, Dayton, Ohio, USA. 38. Department of Oncology, Hospital de Ninos Ricardo Gutierrez, Buenos Aires, Argentina. 39. Department of Pediatric Neurosurgery, Xin Hua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China. 40. Arthur and Sonia Labatt Brain Tumour Research Centre, The Hospital for Sick Children and the Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, Ontario, Canada. 41. Department of Pediatrics and Adolescent Medicine, American University of Beirut, Beirut, Lebanon. 42. Division of Pediatric Hematology/Oncology, Kentucky Children's Hospital, University of Kentucky, Lexington, Kentucky, USA. 43. Department of Pediatrics, C.S. Mott Children's Hospital and University of Michigan School of Medicine, Ann Arbor, Michigan, USA. 44. Department of Pathology, Upstate Medical University, Syracuse, New York, USA. 45. Center for Childhood Cancer & Blood Disorders, Nationwide Children's Hospital, Columbus, Ohio, USA. 46. The Ohio State University College of Medicine, Columbus, Ohio, USA. 47. Division of Radiology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA. 48. Pediatric Neuro-Oncology Program, Nationwide Children's Hospital, Columbus, Ohio, USA.
Abstract
BACKGROUND: Diffuse intrinsic pontine gliomas (DIPG) generally occur in young school-age children, although can occur in adolescents and young adults. The purpose of this study was to describe clinical, radiological, pathologic, and molecular characteristics in patients ≥10 years of age with DIPG enrolled in the International DIPG Registry (IDIPGR). METHODS: Patients ≥10 years of age at diagnosis enrolled in the IDIPGR with imaging confirmed DIPG diagnosis were included. The primary outcome was overall survival (OS) categorized as long-term survivors (LTS) (≥24 months) or short-term survivors (STS) (<24 months). RESULTS: Among 1010 patients, 208 (21%) were ≥10 years of age at diagnosis; 152 were eligible with a median age of 12 years (range 10-26.8). Median OS was 13 (2-82) months. The 1-, 3-, and 5-year OS was 59.2%, 5.3%, and 3.3%, respectively. The 18/152 (11.8%) LTS were more likely to be older (P < .01) and present with longer symptom duration (P < .01). Biopsy and/or autopsy were performed in 50 (33%) patients; 77%, 61%, 33%, and 6% of patients tested had H3K27M (H3F3A or HIST1H3B), TP53, ATRX, and ACVR1 mutations/genome alterations, respectively. Two of 18 patients with IDH1 testing were IDH1-mutant and 1 was a LTS. The presence or absence of H3 alterations did not affect survival. CONCLUSION: Patients ≥10 years old with DIPG have a median survival of 13 months. LTS present with longer symptom duration and are likely to be older at presentation compared to STS. ATRX mutation rates were higher in this population than the general DIPG population.
BACKGROUND: Diffuse intrinsic pontine gliomas (DIPG) generally occur in young school-age children, although can occur in adolescents and young adults. The purpose of this study was to describe clinical, radiological, pathologic, and molecular characteristics in patients ≥10 years of age with DIPG enrolled in the International DIPG Registry (IDIPGR). METHODS: Patients ≥10 years of age at diagnosis enrolled in the IDIPGR with imaging confirmed DIPG diagnosis were included. The primary outcome was overall survival (OS) categorized as long-term survivors (LTS) (≥24 months) or short-term survivors (STS) (<24 months). RESULTS: Among 1010 patients, 208 (21%) were ≥10 years of age at diagnosis; 152 were eligible with a median age of 12 years (range 10-26.8). Median OS was 13 (2-82) months. The 1-, 3-, and 5-year OS was 59.2%, 5.3%, and 3.3%, respectively. The 18/152 (11.8%) LTS were more likely to be older (P < .01) and present with longer symptom duration (P < .01). Biopsy and/or autopsy were performed in 50 (33%) patients; 77%, 61%, 33%, and 6% of patients tested had H3K27M (H3F3A or HIST1H3B), TP53, ATRX, and ACVR1 mutations/genome alterations, respectively. Two of 18 patients with IDH1 testing were IDH1-mutant and 1 was a LTS. The presence or absence of H3 alterations did not affect survival. CONCLUSION: Patients ≥10 years old with DIPG have a median survival of 13 months. LTS present with longer symptom duration and are likely to be older at presentation compared to STS. ATRX mutation rates were higher in this population than the general DIPG population.
Authors: S Bailey; A Howman; K Wheatley; D Wherton; N Boota; B Pizer; D Fisher; P Kearns; S Picton; F Saran; M Gibson; A Glaser; D J A Connolly; D Hargrave Journal: Eur J Cancer Date: 2013-09-04 Impact factor: 9.162
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Authors: Sophie E M Veldhuijzen van Zanten; Adam Lane; Martijn W Heymans; Joshua Baugh; Brooklyn Chaney; Lindsey M Hoffman; Renee Doughman; Marc H A Jansen; Esther Sanchez; William P Vandertop; Gertjan J L Kaspers; Dannis G van Vuurden; Maryam Fouladi; Blaise V Jones; James Leach Journal: J Neurooncol Date: 2017-05-30 Impact factor: 4.130
Authors: Daniele De Brasi; Francesca Orlando; Valeria Gaeta; Maria De Liso; Fabio Acquaviva; Luigi Martemucci; Augusto Mastrominico; Maja Di Rocco Journal: Genes (Basel) Date: 2021-07-30 Impact factor: 4.096
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