Molecular profiling and targeted therapy in pediatric gliomas: review and consensus recommendations

Author:

Miklja Zachary1,Pasternak Amy2,Stallard Stefanie1,Nicolaides Theodore3,Kline-Nunnally Cassie4,Cole Bonnie5,Beroukhim Rameen6,Bandopadhayay Pratiti6,Chi Susan6,Ramkissoon Shakti H78,Mullan Brendan1,Bruzek Amy K1,Gauthier Angela9,Garcia Taylor1,Atchison Christie1,Marini Bernard2,Fouladi Maryam10,Parsons D William11,Leary Sarah5,Mueller Sabine4,Ligon Keith L12,Koschmann Carl1

Affiliation:

1. University of Michigan Medical School, Ann Arbor, Michigan

2. University of Michigan College of Pharmacy, Ann Arbor, Michigan

3. NYU School of Medicine, New York, New York

4. University of California San Francisco (UCSF) Benioff Children’s Hospital, San Francisco, California

5. Seattle Children’s Hospital/University of Washington (UW), Seattle, Washington

6. Dana-Farber Cancer Institute, Boston, Massachusetts

7. Foundation Medicine, Morrisville, North Carolina

8. Wake Forest School of Medicine, Winston-Salem, North Carolina

9. Yale Medical School, New Haven, Connecticut

10. Cincinnati Children’s Hospital, Cincinnati, Ohio

11. Texas Children’s Hospital, Houston, Texas

12. Brigham and Women’s Hospital/Harvard Medical School, Boston, Massachusetts

Abstract

Abstract As the field of neuro-oncology makes headway in uncovering the key oncogenic drivers in pediatric glioma, the role of precision diagnostics and therapies continues to rapidly evolve with important implications for the standard of care for clinical management of these patients. Four studies at major academic centers were published in the last year outlining the clinically integrated molecular profiling and targeting of pediatric brain tumors; all 4 demonstrated the feasibility and utility of incorporating sequencing into the care of children with brain tumors, in particular for children and young adults with glioma. Based on synthesis of the data from these studies and others, we provide consensus recommendations for the integration of precision diagnostics and therapeutics into the practice of pediatric neuro-oncology. Our primary consensus recommendation is that next-generation sequencing should be routinely included in the workup of most pediatric gliomas.

Funder

National Institutes of Health

National Institute of Neurological Disorders and Stroke

Publisher

Oxford University Press (OUP)

Subject

Cancer Research,Clinical Neurology,Oncology

Reference92 articles.

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3. The genomic landscape of diffuse intrinsic pontine glioma and pediatric non-brainstem high-grade glioma;Wu;Nat Genet,2014

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5. Targeted next-generation sequencing of pediatric neuro-oncology patients improves diagnosis, identifies pathogenic germline mutations, and directs targeted therapy;Kline;Neuro Oncol,2017

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