Max M van Noesel1, Daniel Orbach2, Bernadette Brennan3, Anna Kelsey4, Ilaria Zanetti5, Gian Luca de Salvo6, Mark N Gaze7, Ross J Craigie8, Kieran McHugh9, Nadine Francotte10, Paola Collini11, Gianni Bisogno5, Michela Casanova12, Andrea Ferrari12. 1. Department of Pediatric Solid Tumors, Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands. 2. Institut Curie, SIREDO Oncology Center (Care, Innovation and Research for Children and AYA with Cancer), PSL Research University, Paris, France. 3. Department of Paediatric Oncology, Royal Manchester Children's Hospital, Manchester, UK. 4. Department of Pathology, Royal Manchester Children's Hospital, Manchester, UK. 5. Paediatric Hematology and Oncology Division, Padova University, Padova, Italy. 6. Department of EpSSG Sarcoma, EpSSG Data Centre Istituto Oncologico Veneto IRCCS, Padova, Italy. 7. Department of Oncology, University College London Hospitals NHS Foundation Trust, London, UK. 8. Department of Paediatric Surgery, Royal Manchester Children's Hospital, Manchester, UK. 9. Radiology Department, Great Ormond Street Hospital for Children, London, UK. 10. Department of Paediatric, CHC-Clinique Esperance, Montegnée, Belgium. 11. Department of Pathology, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy. 12. Department of Paediatric Oncology, Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy.
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.
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.
Authors: Sumanth Nagabushan; Loretta M S Lau; Paulette Barahona; Marie Wong; Alexandra Sherstyuk; Glenn M Marshall; Vanessa Tyrrell; Eva A Wegner; Paul G Ekert; Mark J Cowley; Chelsea Mayoh; Toby N Trahair; Philip Crowe; Antoinette Anazodo; David S Ziegler Journal: NPJ Precis Oncol Date: 2021-02-12
Authors: Andrea Ferrari; Bernadette Brennan; Michela Casanova; Nadege Corradini; Pablo Berlanga; Reineke A Schoot; Gema L Ramirez-Villar; Akmal Safwat; Gabriela Guillen Burrieza; Patrizia Dall'Igna; Rita Alaggio; Lisa Lyngsie Hjalgrim; Susanne Andrea Gatz; Daniel Orbach; Max M van Noesel Journal: Cancer Manag Res Date: 2022-09-23 Impact factor: 3.602