Feasibility, safety, and indications for surgical biopsy of intrinsic brainstem tumors in children

Cage, Tene A.; Samagh, Sonia P.; Mueller, Sabine; Nicolaides, Theodore; Haas‐Kogan, Daphne A.; Prados, Michael D.; Banerjee, Anu; Auguste, Kurtis I.; Gupta, Nalin

doi:10.1007/s00381-013-2101-0

Cited by 73 publications

(72 citation statements)

References 41 publications

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“…The treatment options for brain stem gliomas (BSG) are even more limited than those for GB. These precariously located intrinsic and infiltrative tumors are not amenable to resection although stereotactic biopsy may be feasible [6].…”

Section: Introductionmentioning

confidence: 99%

Pembrolizumab: first experience with recurrent primary central nervous system (CNS) tumors

et al. 2016

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Patients with progressive primary brain tumors (PBT) are attracted to promising new treatments, even prior to convincing data. Anti-PD1 immunotherapies have been in the spotlight since publication of groundbreaking results for metastatic melanoma with pembrolizumab (PBL). Our objective was to report on the response and toxicity of PBL in patients with advanced PBT. We retrospectively reviewed the charts of 22 patients (17 adults and 5 children) with recurrent central nervous system tumors treated with PBL. We analyzed prior antineoplastic therapies, steroid usage, and outcomes. Patients received a median of two neoplastic therapies prior to PBL, and a median of three infusions of PBL in adults and four in children. Twelve patients (9 adults and 3 children) started PBL on steroids (median dose in adults 4 mg; range 2-8, and in children 1.5 mg, range 0.5-4) and five patients received steroids later during PBL treatment. Twelve patients (10 adults and 2 children) received concomitant bevacizumab with PBL. Side effects were minimal. All patients showed progressive tumor growth during therapy. Median OS from the start of PBL was 2.6 months in adults and 3.2 months in children. Two GB patients underwent tumor resection following treatment with PBL. Tumor-lymphocytic response in these cases was unremarkable, and PD-L1 immuno-staining was negative. In this series of 22 patients with recurrent primary brain tumors, PBL showed no clinical or histologic efficacy. We do not recommend further use of PBL for recurrent PBT unless convincing prospective clinical trial data are published.

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Section: Introductionmentioning

confidence: 99%

Pembrolizumab: first experience with recurrent primary central nervous system (CNS) tumors

et al. 2016

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“…Unfortunately until reliable and minimally invasive methods are consistently available to detect target inhibition, most phase 1 central nervous system brain tumor trials will continue to use established clinical trial designs with dose-limiting toxicity as a primary determination for dose changes. 70,71 Biomarkers Biomarkers are characteristics that can be objectively measured and evaluated as an indicator of pathologic processes or pharmacologic responses to a therapeutic intervention. 72 Since phase 1 trials often recruit a small heterogeneous population of patients with different tumor types, biomarkers are primarily used to provide evidence that the agent reaches and modulates its target at the recommended dose for phase 2 trials.…”

Section: Clinical Trial Designmentioning

confidence: 99%

Experimental Therapeutic Trial Design for Pediatric Brain Tumors

Bornhorst

Hwang

2016

J Child Neurol

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Pediatric brain tumors are the leading cause of cancer-related death during childhood. Since the first pediatric brain tumor clinical trials, the field has seen improved outcomes in some, but not all tumor types. In the past few decades, a number of promising new therapeutic agents have emerged, yet only a few of these agents have been incorporated into clinical trials for pediatric brain tumors. In this review, the authors discuss the process of and challenges in pediatric clinical trial design; this will allow for highly efficient and effective clinical trials with appropriate endpoints to ensure rapid and safe investigation of novel therapeutics for children with brain tumors.

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“…2,11 In the series published to date, the majority of complications were transient cranial neuropathies, although more serious ones have been reported. Although the potential for surgical morbidity cannot be ignored or minimized, there is a path forward in terms of the molecular and biological characterization of DIPGs that can be used to further refine therapeutic decisions 10 : specifically, biopsy-directed clinical trials that use tissue to assign patients to rational therapeutic arms are under way.…”

Section: 6mentioning

confidence: 99%