The clinical course and role of surgery in pediatric malignant peripheral nerve sheath tumors: a database study

OBJECTIVE Malignant peripheral nerve sheath tumors (MPNSTs) are rare tumors found throughout the body, with their clinical course in children still not completely understood. Correspondingly, this study aimed to determine survival outcomes and specific clinical predictors of survival in this population from a large national database. METHODS All patients with MPNSTs aged ≤ 18 years in the US National Cancer Database (NCDB) between 2005 and 2016 were retrospectively reviewed. Data were summarized, and overall survival was modeled using Kaplan-Meier and Cox regression analyses. RESULTS A total of 251 pediatric patients with MPNSTs (132 [53%] females and 119 [47%] males) were identified; the mean age at diagnosis was 13.1 years (range 1–18 years). There were 84 (33%) MPNSTs located in the extremities, 127 (51%) were smaller than 1 cm, and 22 (9%) had metastasis at the time of diagnosis. In terms of treatment, surgery was pursued in 187 patients (74%), chemotherapy in 116 patients (46%), and radiation therapy in 129 patients (61%). The 5-year overall survival rate was estimated at 52% (95% CI 45%–59%), with a median survival of 64 months (range 36–136 months). Multivariate regression revealed that older age (HR 1.10, p < 0.01), metastases at the time of diagnosis (HR 2.14, p = 0.01), and undergoing biopsy only (HR 2.98, p < 0.01) significantly and independently predicted a shorter overall survival. Chemotherapy and radiation therapy were not statistically significant. CONCLUSIONS In this study, the authors found that older patient age, tumor metastases at the time of diagnosis, and undergoing only biopsy significantly and independently predicted poorer outcomes. Only approximately half of patients survived to 5 years. These results have shown a clear survival benefit in pursuing maximal safe resection in pediatric patients with MPNSTs. As such, judicious workup with meticulous resection by an expert team should be considered the standard of care for these tumors in children.

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SURG-04. THE CLINICAL COURSE AND ROLE OF SURGERY IN PEDIATRIC MALIGNANT PERIPHERAL NERVE SHEATH TUMORS: A DATABASE STUDY

Neuro-Oncology ◽

10.1093/neuonc/noab196.774 ◽

2021 ◽

Vol 23 (Supplement_6) ◽

pp. vi195-vi195

Author(s):

Victor Lu ◽

Shelly Wang ◽

David Daniels ◽

Robert Spinner ◽

Allan Levi ◽

...

Keyword(s):

Overall Survival ◽

Radiation Therapy ◽

Peripheral Nerve ◽

Cox Regression ◽

Clinical Course ◽

The Body ◽

National Database ◽

Nerve Sheath Tumors ◽

Peripheral Nerve Sheath Tumors ◽

Nerve Sheath

Abstract BACKGROUND Malignant peripheral nerve sheath tumors (MPNST) are rare tumors found throughout the body, with their clinical course in children still to be completely understood. Correspondingly, this study aimed to determine survival outcomes and specific clinical predictors of survival in this demographic from a large national database. METHODS All MPNST patients aged ≤18 year in the U.S. National Cancer Database (NCDB) between 2005-2016 were retrospectively reviewed. Data were summarized and overall survival (OS) was modeled using Kaplan-Meier and Cox regression analyses. RESULTS A total of 251 pediatric MPNST cases were identified. Overall, mean age at diagnosis was 13.1 years (range, 1-18) with there being 132 (53%) females and 119 (47%) males. There were 84 (33%) MPNSTs located in extremities, 127 (51%) less than 1cm in size, and 22 (9%) have metastasis at diagnosis. In terms of treatment, surgery was pursued in 187 (74%) patients, chemotherapy in 116 (46%) patients, and radiation therapy in 129 (61%) patients. Five-year overall survival was estimated to be 52% (95% CI, 45-59%), and with median survival of 64 months (range, 36-136). Multivariate regression revealed older age (HR 1.10, P< 0.01), with metastases at time of diagnosis (HR 2.14, P=0.01), and biopsy only (HR 2.98, P< 0.01) all significantly and independently predicted shorter overall survival. Chemotherapy and radiation therapy were not statistically significant. CONCLUSIONS Pediatric MPNSTs are rare malignant entities, and only approximately half of patients will survive to five years. There remains a clear survival benefit in pursuing maximal safe resection in these patients. As such, judicious workup with meticulous surgical resection by an expert team should then be considered the standard of care for these tumors in children.

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Malignant Peripheral Nerve Sheath Tumors in Children with Neurofibromatosis Type 1

Case Reports in Oncological Medicine ◽

10.1155/2014/843749 ◽

2014 ◽

Vol 2014 ◽

pp. 1-6 ◽

Cited By ~ 4

Author(s):

Apostolos Pourtsidis ◽

Dimitrios Doganis ◽

Margarita Baka ◽

Despina Bouhoutsou ◽

Maria Varvoutsi ◽

...

Keyword(s):

Radiation Therapy ◽

Peripheral Nerve ◽

Neurofibromatosis Type 1 ◽

Soft Tissue Sarcomas ◽

Neurofibromatosis Type ◽

Nerve Sheath Tumors ◽

Peripheral Nerve Sheath Tumors ◽

Nerve Sheath ◽

Increased Risk

Purpose. Malignant peripheral nerve sheath tumors (MPNSTs) are rare in children and account for approximately 5–10% of all soft tissue sarcomas in adults. MPNSTs may occur independently but individuals with neurofibromatosis type 1 (NF1) have a significantly increased risk. Our aim is to present patients with MPNST treated in our department.Cases and Results. In this report we present 4 cases of MPNSTs (3 females: 13, 12, and 13 years old and 1 male: 10 years old) arising in patients with NF1. All of them presented with an enlarging mass and pain at diagnosis. Tumor was located in the buttock, the spinal cord, the trunk, and the left leg proximal to the heel. Wide excision of the tumor and radiotherapy were applied to all and adjuvant chemotherapy was given to three of them after the disease was progressed. All four died 32, 18, 10, and 22 months after diagnosis with progressive disease locally and pulmonary metastases in two of them.Conclusions. In conclusion, MPNSTs arising in patients with NF1 are high grade sarcomas with short survival. Individuals with NF1 should be followed closely in order to identify early the development of MPNSTs. Aggressive surgery and complete excision significantly improves disease-free survival. The usefulness of radiation therapy in MPNSTs is not determined although all patients will receive radiation therapy at some stage of the disease. The role of chemotherapy is unclear.

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Postoperative Radiation Therapy Improves Progression-Free Survival in Malignant Peripheral Nerve Sheath Tumors

International Journal of Radiation Oncology*Biology*Physics ◽

10.1016/j.ijrobp.2019.06.2494 ◽

2019 ◽

Vol 105 (1) ◽

pp. E801-E802

Author(s):

H. Vasudevan ◽

C.H. Lucas ◽

S.J. Liu ◽

A. Choudhury ◽

S. Magill ◽

...

Keyword(s):

Radiation Therapy ◽

Peripheral Nerve ◽

Progression Free Survival ◽

Postoperative Radiation ◽

Postoperative Radiation Therapy ◽

Free Survival ◽

Nerve Sheath Tumors ◽

Peripheral Nerve Sheath Tumors ◽

Nerve Sheath

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Long-Term Outcomes of Radiation Therapy (RT) in the Management of Malignant Peripheral Nerve Sheath Tumors (MPNST) in Patients with Neurofibromatosis Type 1 (NF1)

International Journal of Radiation Oncology*Biology*Physics ◽

10.1016/j.ijrobp.2018.07.1357 ◽

2018 ◽

Vol 102 (3) ◽

pp. e474-e475 ◽

Cited By ~ 1

Author(s):

L. Sloan ◽

S.A. Terezakis ◽

J.O. Blakeley ◽

B. Slobogean ◽

L.R. Kleinberg

Keyword(s):

Radiation Therapy ◽

Peripheral Nerve ◽

Neurofibromatosis Type 1 ◽

Neurofibromatosis Type ◽

Long Term Outcomes ◽

Nerve Sheath Tumors ◽

Peripheral Nerve Sheath Tumors ◽

Nerve Sheath

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Role of resection of malignant peripheral nerve sheath tumors in patients with neurofibromatosis Type 1

Journal of Neurosurgery ◽

10.3171/2012.9.jns101610 ◽

2013 ◽

Vol 118 (1) ◽

pp. 142-148 ◽

Cited By ~ 32

Author(s):

Gavin P. Dunn ◽

Konstantinos Spiliopoulos ◽

Scott R. Plotkin ◽

Francis J. Hornicek ◽

David C. Harmon ◽

...

Keyword(s):

Multivariate Analysis ◽

Radiation Therapy ◽

Peripheral Nerve ◽

Neurofibromatosis Type ◽

Total Resection ◽

Nerve Sheath Tumors ◽

Peripheral Nerve Sheath Tumors ◽

Nerve Sheath

Object Malignant peripheral nerve sheath tumors (MPNSTs) are aggressive sarcomas that often arise from major peripheral nerves. Approximately half of MPNSTs arise in patients with neurofibromatosis Type 1 (NF1) who, in comparison with patients without NF1, present at younger ages and with larger tumors that are commonly associated with extensive plexiform neurofibromas. These tumors therefore pose a particularly difficult treatment challenge because of the morbidity often associated with attempted gross-total resection (GTR). Here, the authors aim to examine what role the extent of resection and other covariates play in the long-term survival of patients with NF1 in the setting of MPNST. Methods The authors retrospectively reviewed the records of 23 adult patients with NF1 who underwent surgery for MPNSTs at their institution between 1991 and 2008. The primary end points of the study were mortality, local recurrence, and metastasis. Kaplan-Meier survival curves were evaluated for all patients. Differences for each of the primary end points were evaluated based on cause-specific covariates, which included tiered tumor size, tumor location, grade, resection margin status, postoperative weakness, and use of chemotherapy and radiation therapy. Multivariate analysis was performed using Cox proportional hazards models. Results Gross-total resection (p = 0.01) and surgical margin status (p = 0.034) had a statistically important role in prolonging overall survival in patients with NF1 by univariate analysis. When tumor size, location, grade, postoperative weakness, and radiation therapy were also taken into account using multivariate analysis, GTR continued to be a significant prognostic factor (p = 0.035). Conclusions These findings suggest that GTR offers significant long-term benefit on survival in patients with NF1. Benefit on survival occurred independently of all other covariates, suggesting that complete resection should be the principal goal of treatment in this patient population.

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The implications of intradural extension in paraspinal malignant peripheral nerve sheath tumors: effects on central nervous system metastases and overall survival

Journal of Neurosurgery Spine ◽

10.3171/2018.5.spine18445 ◽

2018 ◽

Vol 29 (6) ◽

pp. 725-728 ◽

Cited By ~ 2

Author(s):

Hannah E. Gilder ◽

Ross C. Puffer ◽

Mohamad Bydon ◽

Robert J. Spinner

Keyword(s):

Overall Survival ◽

Peripheral Nerve ◽

Surgical Pathology ◽

Cns Metastases ◽

Tumor Extension ◽

Poor Prognostic Factor ◽

Nerve Sheath Tumors ◽

Peripheral Nerve Sheath Tumors ◽

Nerve Sheath ◽

Intradural Tumor

OBJECTIVEIn this study, the authors sought to compare tumors with intradural extension to those remaining in the epidural or paraspinal space with the hypothesis that intradural extension may be a mechanism for seeding of the CSF with malignant cells, thereby resulting in higher rates of CNS metastases and shorter overall survival.METHODSThe authors searched the medical record for cases of malignant peripheral nerve sheath tumors (MPNSTs) identified from 1994 to 2017. The charts of the identified patients were then reviewed for tumor location to identify patients with paraspinal malignancy. All patients included in the study had tumor specimens that were reviewed in the surgical pathology department. Paraspinal tumors with intradural extension were identified in the lumbar, sacral, and spinal accessory nerves, and attempts were made to match this cohort to another cohort of patients who had paraspinal tumors of the cranial nerves and lumbar and sacral spinal regions without intradural extension. Further information was collected on all patients with and without intradural extension, including date of diagnosis by pathology specimen review; nerve or nerves of tumor origin; presence, location, and diagnostic date of any CNS metastases; and either the date of death or date of last follow-up.RESULTSThe authors identified 6 of 179 (3.4%) patients who had intradural tumor extension and compared these patients with 12 patients who harbored paraspinal tumors that did not have intradural extension. All tumors were diagnosed as high-grade MPNSTs according to the surgical pathology findings. Four of 6 (66.7%) patients with intradural extension had documented CNS metastases. The presence of CNS metastases was significantly higher in the intradural group than in the paraspinal group (intradural, 66.7% vs paraspinal, 0%; p < 0.01). Time from diagnosis until death was 11.2 months in the intradural group and approximately 72 months in the paraspinal, extradural cohort.CONCLUSIONSIn patients with intradural extension of paraspinal MPNSTs, significantly higher rates of CNS metastases are seen with a reduced interval of time from diagnosis to metastatic lesion detection. Intradural tumor extension is also a poor prognostic factor for survival, with these patients showing a reduced mean time from diagnosis to death.

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