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Outcome and prognostic factors in pediatric malignant peripheral nerve sheath tumors: An analysis of the European Pediatric Soft Tissue Sarcoma Group (EpSSG) NRSTS-2005 prospective study

van Noesel, MM; Orbach, D; Brennan, B; Kelsey, A; Zanetti, I; de Salvo, GL; Gaze, MN; ... Ferrari, A; + view all (2019) Outcome and prognostic factors in pediatric malignant peripheral nerve sheath tumors: An analysis of the European Pediatric Soft Tissue Sarcoma Group (EpSSG) NRSTS-2005 prospective study. Pediatric Blood and Cancer , 66 (10) , Article e27833. 10.1002/pbc.27833. Green open access

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Abstract

Background: Malignant peripheral nerve sheath tumors (MPNST) are rare tumors of childhood. The role of standard chemotherapy in unresectable MPNST is still unclear. We report the outcome and prognostic factors in the EpSSG risk-adapted prospective study for localized pediatric MPNST. Methods: Patients were stratified into four treatment groups defined by surgical resection, tumor size, and tumor grade (G): (a) surgery-only group—resected tumors G1; (b) adjuvant radiotherapy group—R0/R1, G2 tumors; (c) adjuvant chemotherapy group—R0/R1, G3 tumors; and (d) neoadjuvant chemotherapy group—R2 resected tumors and/or nodal involvement. Chemotherapy consisted of four courses of ifosfamide-doxorubicin and two courses of ifosfamide concomitant with radiotherapy (50.4-54 Gy). Results: Overall, the study included 51 patients. The 5-year event-free survival (EFS) and overall survival (OS) were 52.9% (95% confidence interval, 38.1-65.8) and 62.1% (46.7-74.3), respectively. The 5-year EFS was 92% (56.6-98.9) for treatment group 1 (N = 13), 33% (0.9-77.4) for treatment group 2 (N = 4), 29% (4.1-61.2) for treatment group 3 (N = 7), and 42% (23.1-60.1) for treatment group 4 (N = 27). Response rate to chemotherapy (partial response + complete response) in patients with measurable disease was 46%. The presence of neurofibromatosis type 1 (NF1; 51% of patients) was an independent poor prognostic factor for OS and EFS. Conclusion: The outcome for patients with resectable MPNST was excellent. Standard ifosfamide-doxorubicin for unresectable MPNST rendered the best reported outcome. Children with NF1 disease seem to have worse prognosis.

Type: Article
Title: Outcome and prognostic factors in pediatric malignant peripheral nerve sheath tumors: An analysis of the European Pediatric Soft Tissue Sarcoma Group (EpSSG) NRSTS-2005 prospective study
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1002/pbc.27833
Publisher version: https://doi.org/10.1002/pbc.27833
Language: English
Additional information: This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made. © 2019 The Authors. Pediatric Blood & Cancer Published by Wiley Periodicals, Inc.
Keywords: EpSSG study, MPNST outcome study, NF1, NRSTS, Phase 3 study, adjuvant chemothexrapy, outcomes research, sarcoma, soft tissue , Adolescent, Chemoradiotherapy, Adjuvant, Child, Child, Preschool, Disease-Free Survival, Europe, Female, Humans, Infant, Male, Neurofibrosarcoma, Prognosis, Prospective Studies, Treatment Outcome, Young Adult
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > Research Department of Oncology
URI: https://discovery.ucl.ac.uk/id/eprint/10182905
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