Neurofibromatosis
With Jill Knepprath
Educational content from recorded physician discussions — not medical advice. Always talk to your child's care team about your child's situation.
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Evidence & Research
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Malignant peripheral nerve sheath tumors: a report from Children’s Oncology Group study ARST0332
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Jacquelyn N Crane, Wei Xue, Amira Qumseya, Donald A Barkauskas, Khang Chau, Serena Y Tan, Susan Hiniker, Roshni Dasgupta, Rajkumar Venkatramani, Sheri L Spunt, Aaron R Weiss, Theodore W LaetschBackground: The cornerstone of the treatment of
video1:11 · Mar 2026
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A 2025 Children's Oncology Group prospective trial in *JNCI* stratified malignant peripheral nerve sheath tumor (MPNST) patients into observation, adjuvant radiation, adjuvant chemoradiation, and neoadjuvant chemoradiation arms. Resection remains the dominant survival determinant, outweighing neoadjuvant response. Female sex, absence of metastases, and low-risk classification independently predicted better outcomes. Among neoadjuvant recipients, 23% responded, 45% stabilized, and 32% progressed; critically, post-resection survival did not differ by neoadjuvant response category. This dissociation between radiographic response and surgical outcome argues for maximal resection even after apparent chemoradiation response, challenging response-adapted de-escalation strategies. The trial underscores surgery as non-negotiable when technically feasible, regardless of neoadjuvant effect.
- Complete resection is the primary survival driver in MPNST, superseding neoadjuvant response as a prognostic factor.[e11726-c3][e11726-c10][e11726-c11]
- Female sex, non-metastatic disease, and low-risk features independently improve MPNST outcomes.[e11726-c4][e11726-c5][e11726-c6]
- Neoadjuvant chemoradiation yields response in 23%, stability in 45%, and progression in 32% of patients.[e11726-c7][e11726-c8][e11726-c9]
- Post-resection survival is equivalent across neoadjuvant response categories, negating response-adapted surgical decisions.[e11726-c10]
- Resection should proceed after neoadjuvant therapy even in radiographic responders, per COG trial data.[e11726-c11]
For patients & families
Neurofibromatosis can sometimes lead to a rare cancer called malignant peripheral nerve sheath tumor. Doctors recently completed a large study looking at different ways to treat this cancer, including surgery, radiation, and chemotherapy given before or after surgery. The research showed that removing the tumor through surgery has the biggest impact on how well patients do. Doctors found that women, patients without cancer spread, and those considered lower risk tended to have better outcomes. When chemotherapy and radiation were given before surgery, about one in four patients saw their tumors shrink, nearly half stayed stable, and about one in three got worse. Importantly, even when tumors responded well to treatment before surgery, removing the tumor was still necessary for the best chance of survival. This means surgery remains the most important part of treatment whenever it can be safely done.
Neurofibromatosis can sometimes lead to a rare cancer called malignant peripheral nerve sheath tumor. Doctors recently completed a large study looking at different ways to treat this cancer, including surgery, radiation, and chemotherapy given before or after surgery. The research showed that removing the tumor through surgery has the biggest impact on how well patients do. Doctors found that women, patients without cancer spread, and those considered lower risk tended to have better outcomes. When chemotherapy and radiation were given before surgery, about one in four patients saw their tumors shrink, nearly half stayed stable, and about one in three got worse. Importantly, even when tumors responded well to treatment before surgery, removing the tumor was still necessary for the best chance of survival. This means surgery remains the most important part of treatment whenever it can be safely done.
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Malignant peripheral nerve sheath tumors: a report from Children’s Oncology Group study ARST0332
The Children's Oncology Group published a prospective trial on malignant peripheral nerve sheath tumors in the Journal of the National Cancer Institute in 2025.
clinicalJill Knepprath0:11 ↗
Patients were assigned to four treatment groups: observation, adjuvant radiation, adjuvant chemoradiation, and neoadjuvant chemoradiation.
clinicalJill Knepprath0:24 ↗
Resection has a major influence on survival for patients with malignant peripheral nerve sheath tumors.
clinicalJill Knepprath0:35 ↗
Female patients with malignant peripheral nerve sheath tumors had improved outcomes.
epidemiologicalJill Knepprath0:40 ↗
Patients without metastatic disease had improved outcomes.
clinicalJill Knepprath0:40 ↗
Low-risk patients had improved outcomes.
clinicalJill Knepprath0:40 ↗
In patients receiving neoadjuvant therapy, 23% showed a response.
clinicalJill Knepprath0:46 ↗
In patients receiving neoadjuvant therapy, 45% were stable.
clinicalJill Knepprath0:46 ↗
In patients receiving neoadjuvant therapy, 32% had progressive disease.
clinicalJill Knepprath0:46 ↗
There was no difference in survival based on the response to neoadjuvant therapy among patients who went on to have resection.
clinicalJill Knepprath0:59 ↗
Resection should be done for patients with malignant peripheral nerve sheath tumors whenever possible, even if they show a good response to chemoradiation.
guidelineJill Knepprath1:03 ↗
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