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Neuro Oncol. 2018 Oct 9;20(11):1547-1555. doi: 10.1093/neuonc/noy070.

Prospective feasibility and safety assessment of surgical biopsy for patients with newly diagnosed diffuse intrinsic pontine glioma.

Author information

1
UCSF Benioff Children's Hospital & University of California San Francisco, San Francisco, California.
2
Dana-Farber Cancer Institute, Boston, Massachusetts.
3
Boston Children's Hospital, Boston, Massachusetts.
4
Ann & Robert H. Lurie Children's Hospital of Chicago & Northwestern University, Chicago, Illinois.
5
Washington University Medical Center & St. Louis Children's Hospital, St. Louis, Missouri.
6
Seattle Children's Hospital & University of Washington, Seattle, Washington.
7
Children's Hospital of Michigan & Wayne State University, Detroit, Michigan.
8
Children's Hospitals and Clinics of Minnesota, Minneapolis, Minnesota.
9
New York University, New York City, New York.
10
University of Louisville & Norton's Children's Hospital, Louisville, Kentucky.
11
University of Texas Southwestern Medical Center, Dallas, Texas.
12
Children's Healthcare of Atlanta & Emory University, Atlanta, Georgia.
13
Children's Hospital Los Angeles, Los Angeles, California.
14
Nemours Children's Clinic, Wolfson's Children's Hospital & University of Florida, Jacksonville, Florida.
15
Nicklaus Children's Hospital, Miami, Florida.
16
Medical University of South Carolina, South Carolina, Charleston, South Carolina.
17
Preston Robert Tisch Brain Tumor Center & Duke University Medical Center, Durham, North Carolina.
18
Johns Hopkins University, Baltimore, Maryland.
19
Children's Hospital of Colorado & University of Colorado School of Medicine, Denver, Colorado.
20
Penn State Health Children's Hospital, Hershey, Pennsylvania.
21
Oregon Health & Science University & Doernbecher Children's Hospital, Portland, Oregon.
22
Cook Children's Medical Center, Fort Worth, Texas.
23
Brigham and Women's Hospital, Boston, Massachusetts.
24
Broad Institute, Cambridge, Massachusetts.

Abstract

Background:

Diagnosis of diffuse intrinsic pontine glioma (DIPG) has relied on imaging studies, since the appearance is pathognomonic, and surgical risk was felt to be high and unlikely to affect therapy. The DIPG Biology and Treatment Study (DIPG-BATS) reported here incorporated a surgical biopsy at presentation and stratified subjects to receive FDA-approved agents chosen on the basis of specific biologic targets.

Methods:

Subjects were eligible for the trial if the clinical features and imaging appearance of a newly diagnosed tumor were consistent with a DIPG. Surgical biopsies were performed after enrollment and prior to definitive treatment. All subjects were treated with conventional external beam radiotherapy with bevacizumab, and then stratified to receive bevacizumab with erlotinib or temozolomide, both agents, or neither agent, based on O6-methylguanine-DNA methyltransferase status and epidermal growth factor receptor expression. Whole-genome sequencing and RNA sequencing were performed but not used for treatment assignment.

Results:

Fifty-three patients were enrolled at 23 institutions, and 50 underwent biopsy. The median age was 6.4 years, with 24 male and 29 female subjects. Surgical biopsies were performed with a specified technique and no deaths were attributed to the procedure. Two subjects experienced grade 3 toxicities during the procedure (apnea, n = 1; hypertension, n = 1). One subject experienced a neurologic deficit (left hemiparesis) that did not fully recover. Of the 50 tumors biopsied, 46 provided sufficient tissue to perform the study assays (92%, two-stage exact binomial 90% CI: 83%-97%).

Conclusions:

Surgical biopsy of DIPGs is technically feasible, associated with acceptable risks, and can provide biologic data that can inform treatment decisions.

PMID:
29741745
PMCID:
PMC6176802
[Available on 2019-10-09]
DOI:
10.1093/neuonc/noy070
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