Refractory Stage M Ganglioneuroblastoma With Bone Metastases and a Favorable, Chronic Course of Disease: Description of a Patient Cohort
Tas, Michelle L; Molenaar, Jan J; Peek, Annemarie M L; Lequin, Maarten H; Verdijk, Rob M; de Krijger, Ronald R; Tytgat, Godelieve A M; van Noesel, Max M
(2022) Journal of Pediatric Hematology/oncology, volume 44, issue 1, pp. e5 - e13
(Article)
Abstract
Refractory stage M neuroblastoma (NB) is associated with a poor prognosis and a progressive course of disease. Here, we describe a unique group of patients with a discrepant clinical course. Seven histologically confirmed ganglioneuroblastoma (GNB) (n=6) and differentiating NB (n=1) patients were identified who were diagnosed with stage M disease
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based on iodine-123-metaiodobenzylguanidine avid bone metastases. Six patients started on high-risk treatment, without tumor response (stable disease). Treatment was discontinued before the start of consolidation treatment because of refractory response in all patients. Unexpectedly, after cessation of treatment no progression of disease occurred. In 2 patients, the primary tumors expanded (>25%) very slowly during 1.5 and 3 years, and remained stable thereafter. Metabolically, a slow decrease of urinary homovanillic acid and vanillylmandelic acid levels and iodine-123-metaiodobenzylguanidine avidity was observed. All patients are alive with presence of metastatic disease after a median follow-up of 17 years (range: 6.7 to 27 y). Interestingly, at diagnosis, 6 patients were asymptomatic, 6 patients had GNB morphology, and 5 patients had meningeal metastases. These are all features seen in only a small minority of stage M patients. This GNB entity illustrates the clinical heterogeneity of neuroblastic tumors and can be used to further study the developmental origin of different NB subtypes.
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Keywords: Bone Neoplasms/drug therapy, Child, Preschool, Chronic Disease, Consolidation Chemotherapy, Female, Ganglioneuroblastoma/drug therapy, Humans, Infant, Male, Neoplasm Metastasis, Neoplasm Staging, Retrospective Studies, Meningeal, Prognosis, Metastasis, Neuroblastoma, Refractory, Ganglioneuroblastoma, Hematology, Oncology, Pediatrics, Perinatology, and Child Health, Research Support, Non-U.S. Gov't, Journal Article
ISSN: 1077-4114
Publisher: Lippincott Williams and Wilkins
Note: Funding Information: This project was funded by the Villa Joep Foundation. Publisher Copyright: Copyright © 2021 The Author(s). Published by Wolters Kluwer Health, Inc. This is an open access article distributed under the terms of the Creative Commons Attribution-Non Commercial-No Derivatives License 4.0 (CCBY-NC-ND)
(Peer reviewed)