TY - JOUR
T1 - Survival after resection of malignant peripheral nerve sheath tumors
T2 - Introducing and validating a novel type-specific prognostic model
AU - Acem, Ibtissam
AU - Steyerberg, Ewout W
AU - Spreafico, Marta
AU - Grünhagen, Dirk J
AU - Callegaro, Dario
AU - Spinner, Robert J
AU - Pendleton, Courtney
AU - Coert, J Henk
AU - Miceli, Rosalba
AU - Abruzzese, Giulia
AU - Flucke, Uta E
AU - Slooff, Willem-Bart M
AU - van Dalen, Thijs
AU - Been, Lukas B
AU - Bonenkamp, Han J
AU - Anten, Monique H M E
AU - Broen, Martinus P G
AU - Bemelmans, Marc H A
AU - Bramer, Jos A M
AU - Schaap, Gerard R
AU - Kievit, Arthur J
AU - van der Hage, Jos
AU - van Houdt, Winan J
AU - van de Sande, Michiel A J
AU - Gronchi, Alessandro
AU - Verhoef, Cornelis
AU - Martin, Enrico
N1 - Publisher Copyright:
© 2024 The Author(s). Published by Oxford University Press, the Society for Neuro-Oncology and the European Association of Neuro-Oncology.
PY - 2024/6/5
Y1 - 2024/6/5
N2 - BACKGROUND: This study aimed to assess the performance of currently available risk calculators in a cohort of patients with malignant peripheral nerve sheath tumors (MPNST) and to create an MPNST-specific prognostic model including type-specific predictors for overall survival (OS).METHODS: This is a retrospective multicenter cohort study of patients with MPNST from 11 secondary or tertiary centers in The Netherlands, Italy and the United States of America. All patients diagnosed with primary MPNST who underwent macroscopically complete surgical resection from 2000 to 2019 were included in this study. A multivariable Cox proportional hazard model for OS was estimated with prespecified predictors (age, grade, size, NF-1 status, triton status, depth, tumor location, and surgical margin). Model performance was assessed for the Sarculator and PERSARC calculators by examining discrimination (C-index) and calibration (calibration plots and observed-expected statistic; O/E-statistic). Internal-external cross-validation by different regions was performed to evaluate the generalizability of the model.RESULTS: A total of 507 patients with primary MPNSTs were included from 11 centers in 7 regions. During follow-up (median 8.7 years), 211 patients died. The C-index was 0.60 (95% CI 0.53-0.67) for both Sarculator and PERSARC. The MPNST-specific model had a pooled C-index of 0.69 (95%CI 0.65-0.73) at validation, with adequate discrimination and calibration across regions.CONCLUSIONS: The MPNST-specific MONACO model can be used to predict 3-, 5-, and 10-year OS in patients with primary MPNST who underwent macroscopically complete surgical resection. Further validation may refine the model to inform patients and physicians on prognosis and support them in shared decision-making.
AB - BACKGROUND: This study aimed to assess the performance of currently available risk calculators in a cohort of patients with malignant peripheral nerve sheath tumors (MPNST) and to create an MPNST-specific prognostic model including type-specific predictors for overall survival (OS).METHODS: This is a retrospective multicenter cohort study of patients with MPNST from 11 secondary or tertiary centers in The Netherlands, Italy and the United States of America. All patients diagnosed with primary MPNST who underwent macroscopically complete surgical resection from 2000 to 2019 were included in this study. A multivariable Cox proportional hazard model for OS was estimated with prespecified predictors (age, grade, size, NF-1 status, triton status, depth, tumor location, and surgical margin). Model performance was assessed for the Sarculator and PERSARC calculators by examining discrimination (C-index) and calibration (calibration plots and observed-expected statistic; O/E-statistic). Internal-external cross-validation by different regions was performed to evaluate the generalizability of the model.RESULTS: A total of 507 patients with primary MPNSTs were included from 11 centers in 7 regions. During follow-up (median 8.7 years), 211 patients died. The C-index was 0.60 (95% CI 0.53-0.67) for both Sarculator and PERSARC. The MPNST-specific model had a pooled C-index of 0.69 (95%CI 0.65-0.73) at validation, with adequate discrimination and calibration across regions.CONCLUSIONS: The MPNST-specific MONACO model can be used to predict 3-, 5-, and 10-year OS in patients with primary MPNST who underwent macroscopically complete surgical resection. Further validation may refine the model to inform patients and physicians on prognosis and support them in shared decision-making.
KW - internal-external validation
KW - malignant peripheral nerve sheath tumors
KW - model performance
KW - neurofibromatosis 1
KW - prognosis
UR - http://www.scopus.com/inward/record.url?scp=85197632105&partnerID=8YFLogxK
U2 - 10.1093/noajnl/vdae083
DO - 10.1093/noajnl/vdae083
M3 - Article
C2 - 38946881
SN - 2632-2498
VL - 6
JO - Neuro-oncology advances
JF - Neuro-oncology advances
IS - 1
M1 - vdae083
ER -