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The ganglioside, GD2, as a circulating tumor biomarker for neuroblastoma

Citation

Balis, Frank et al. (2019), The ganglioside, GD2, as a circulating tumor biomarker for neuroblastoma, Dryad, Dataset, https://doi.org/10.5061/dryad.ksn02v706

Abstract

Background: GD2 is a ganglioside that is ubiquitously expressed in the plasma membrane of neuroblastoma

and is shed into the circulation.

Procedure: GD2 was measured with a high-pressure liquid chromatography/tandem mass spectrometry

assay in serum or plasma from 40 children without cancer (controls) and in biobanked

samples from 128 (73 high-risk) children with neuroblastic tumors at diagnosis, 56 children with

relapsed neuroblastoma, 14 children with high-risk neuroblastoma after treatment, and 8 to 12

children each with 10 other common childhood cancers at diagnosis.

Results: The C18 (18 carbon fatty acid) lipoform was the predominant circulating form of

GD2 in controls and in patients with neuroblastoma. The median concentration of GD2 in children

with high-risk neuroblastoma at diagnosis was 167 nM (range, 16.1-1060 nM), which was

30-fold higher than the median concentration (5.6 nM) in controls. GD2 was not elevated in

serum from children with the differentiated neuroblastic tumors, ganglioneuroma (n = 10) and

ganglioneuroblastoma-intermixed subtype (n = 12), and in children with 10 other childhood cancers.

GD2 concentrations were significantly higher in serum from children with MYCN-amplified

tumors (P = 0.0088), high-risk tumors (P < 0.00001), International Neuroblastoma Staging System

(INSS) stage 4 tumors (P < 0.00001), and in children who died (P = 0.034).

Conclusions: Circulating GD2 appears to be a specific and sensitive tumor biomarker for highrisk/

high-stage neuroblastoma and may prove to be clinically useful as a diagnostic or prognostic

circulating tumor biomarker. GD2 will be measured prospectively and longitudinally in children

enrolled on a high-risk neuroblastoma treatment trial to assess its ability to measure response to

treatment and predict survival.

Methods

This was a retrospective analysis. Serum samples were obtained from several biorepositories. The C18 and C20 lipoforms of GD2 were measured in the serum samples using a validated HPLC/MS/MS assay

Funding

Childhood Brain Tumor Tissue Consortium

Alex's Lemonade Stand Foundation for Childhood Cancer

National University Cancer Institute, Singapore

National Cancer Institute, Award: 1U10CA180884-01

National Cancer Institute, Award: U10CA180886

National Cancer Institute, Award: U10CA180899

National Cancer Institute, Award: U24CA114766