Universität zu Köln

Berthold, Frank ORCID: 0000-0002-7613-1723, Rosswog, Carolina, Christiansen, Holger, Fruhwald, Michael, Hemstedt, Nadine, Klingebiel, Thomas, Frohlich, Birgit, Schilling, Freimut H., Schmid, Irene, Simon, Thorsten ORCID: 0000-0002-3425-8451, Hero, Barbara, Fischer, Matthias and Ernst, Angela ORCID: 0000-0003-2375-1889 (2021). Clinical and molecular characterization of patients with stage 4(M) neuroblastoma aged less than 18 months without MYCN amplification. Pediatr. Blood Cancer, 68 (8). HOBOKEN: WILEY. ISSN 1545-5017

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Abstract

Introduction The survival of children with stage 4(M) neuroblastoma without MYCN amplification and below the age of 18 months is considered better than the still dismal outcome of older high-risk neuroblastoma patients. This study analyzes the impact of clinical and molecular characteristics on the long-term outcome. Patients and methods Clinical presentation, survival, and recurrence patterns of patients enrolled onto trials NB90, NB97, and NB2004 were retrospectively analyzed. Gene expression signatures based on RNA microarrays (TH10) were investigated if tumor material was available. Results Between 1990 and 2015, 177 patients with stage 4(M) MYCN nonamplified neuroblastoma aged less than 18 months at diagnosis were eligible. After a median follow-up of 9.7 years (IQR 5.0, 13.4), the proportions of 10-year event-free survival (EFS) and overall survival (OS) were 73% (95% confidence interval [CI] 67-79%) and 86% (95% CI 80-92%), respectively. Of the 27 neuroblastoma recurrences, 44% occurred in more than one site. Four additional patients presented histologically mature ganglioneuroma at recurrence. Six patients developed a secondary malignancy. The secondary 5-year EFS and OS of the 27 patients with neuroblastoma recurrence were 44% and 59%, respectively. TH10 gene expression signature was not prognostically predictive in the investigated subcohort. Conclusion The outcome of patients with stage 4(M) neuroblastoma aged less than 18 months is favorable when treated with high-risk or otherwise intensive therapy. The development of secondary malignancies and the potential of maturation to ganglioneuroma call for a controlled stepwise reduction of treatment intensity.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Berthold, Frank UNSPECIFIED orcid.org/0000-0002-7613-1723 UNSPECIFIED Rosswog, Carolina UNSPECIFIED UNSPECIFIED UNSPECIFIED Christiansen, Holger UNSPECIFIED UNSPECIFIED UNSPECIFIED Fruhwald, Michael UNSPECIFIED UNSPECIFIED UNSPECIFIED Hemstedt, Nadine UNSPECIFIED UNSPECIFIED UNSPECIFIED Klingebiel, Thomas UNSPECIFIED UNSPECIFIED UNSPECIFIED Frohlich, Birgit UNSPECIFIED UNSPECIFIED UNSPECIFIED Schilling, Freimut H. UNSPECIFIED UNSPECIFIED UNSPECIFIED Schmid, Irene UNSPECIFIED UNSPECIFIED UNSPECIFIED Simon, Thorsten UNSPECIFIED orcid.org/0000-0002-3425-8451 UNSPECIFIED Hero, Barbara UNSPECIFIED UNSPECIFIED UNSPECIFIED Fischer, Matthias UNSPECIFIED UNSPECIFIED UNSPECIFIED Ernst, Angela UNSPECIFIED orcid.org/0000-0003-2375-1889 UNSPECIFIED
URN:	urn:nbn:de:hbz:38-596335
DOI:	10.1002/pbc.29038
Journal or Publication Title:	Pediatr. Blood Cancer
Volume:	68
Number:	8
Date:	2021
Publisher:	WILEY
Place of Publication:	HOBOKEN
ISSN:	1545-5017
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language Oncology; Hematology; Pediatrics Multiple languages
URI:	http://kups.ub.uni-koeln.de/id/eprint/59633