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Nivolumab in the Treatment of Recurrent or Refractory Pediatric Brain Tumors

A Single Institutional Experience

Gorsi, Hamza S., MD, MPH*,†; Malicki, Denise M., MD, PhD†,‡; Barsan, Valentin, MD†,§; Tumblin, Mark, BS†,∥; Yeh-Nayre, Lanipua, CPNP†,∥; Milburn, Mehrzad, BSN†,∥; Elster, Jennifer D., MD†,∥; Crawford, John R., MD, MS*,†,∥

Journal of Pediatric Hematology/Oncology: May 2019 - Volume 41 - Issue 4 - p e235–e241
doi: 10.1097/MPH.0000000000001339
Online Articles: Original Articles

Successful use of immune checkpoint inhibitors in a variety of cancers has generated interest in using this approach in pediatric brain tumors. We performed a retrospective review of 10 consecutive children (6 boys, 4 girls; ages, 2 to 17 y), with recurrent or refractory pediatric brain tumors (5 high-grade glioma, 1 low-grade glioma, pineoblastoma, medulloblastoma, ependymoma, and CNS embryonal tumor, NOS) treated at Rady Children’s Hospital San Diego from 2015 to 2017 with the immune checkpoint inhibitor nivolumab (3 mg/kg every 2 wk). Eight of 10 patients received prior chemotherapy and 9 radiation therapy. Nine patients had radiographic disease progression (median, 2.5 doses). Median time to progression was 5.5 weeks (1.6 to 24 wk). Three patients (2 with high-grade glioma, 1 with CNS embryonal tumor NOS) showed a partial response to treatment at the primary tumor site and 2 of 3 had progression of metastatic disease. Grade 2 toxicities were observed without dose limiting side effects. Tumor mutation burden (TMB) was low to intermediate (median, 1.3; range, 0 to 6.3). Median survival for PD-L1 positive patients was 13.7 weeks versus 4.2 weeks for PD-L1 negative patients (ρ=0.08) nivolumab was well tolerated in our series of pediatric recurrent brain tumors with some transient partial responses in patients with positive PD-L1 expression and higher TMB. Our findings suggest that the use of immune checkpoint inhibitors in pediatric brain tumor patients should be limited to those with elevated PD-L1 expression and TMB.

Departments of *Neurosciences



Pediatrics, Division of Hematology Oncology, University of California San Diego, La Jolla

Rady Children’s Hospital, San Diego, CA

V.B. is a paid consultant for Illumina. The remaining authors declare that they have nothing to disclose.

Reprints: Hamza S. Gorsi, MD, MPH, ORCID 0000-0002-1136-8543, Rady Children’s Hospital, MC 5009, 3020 Children’s Way, San Diego, CA 92123 (e-mails:;

Received June 15, 2018

Accepted September 24, 2018

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