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Author Spotlight: Genetically Engineered Mouse Models and Pathological Characterization of Neurofibromatosis Type 1 Associated Tumors
Published on: May 17, 2024
Malignant peripheral nerve-sheath tumors in an adolescent patient with mosaic localized NF1: A case report
Hiroki Hagizawa1, Shigenori Nagata2, Toru Wakamatsu1
1Department of Orthopedic Surgery, Osaka International Cancer Institute, Osaka 541-8567, Japan.
Abstract:
Malignant peripheral nerve-sheath tumors (MPNSTs) are rare malignancies that are often observed in patients with neurofibromatosis type 1 (NF1). However, the occurrence of MPNST associated with mosaic localized NF1 is extremely rare. Previous reports have revealed that MPNST was associated with mosaic localized NF1 in only three patients who were >40 years of age. The present report details a 16-year-old man who presented with pain and a 3 cm mass on the medial side of the right knee. Magnetic resonance imaging revealed a circumscribed soft tissue tumor located in the subcutaneous tissue. His previous doctor believed that it was benign and conducted a marginal resection. However, postoperative histology results demonstrated spindle cell sarcoma, following which the patient was referred to The Osaka International Cancer Institute. Localized café-au-lait spots were identified in the affected leg, which inferred that the patient had NF1-related MPNST. A wide resection was performed to completely resect the residual tumor; however, a definitive histological diagnosis was challenging due to the small residual tumor. Hence, the genomic mutations of NF1 in the regional café-au-lait spots were analyzed. The result revealed an NF1 microdeletion and a consistently limited expression of NF1 in the tumor sample. Finally, the patient was diagnosed with MPNST with mosaic localized NF1. Local recurrence and distant metastasis were not observed 1.5 years after surgery. In conclusion, the present report presented MPNST in an adolescent patient with mosaic localized NF1. The occurrence of MPNSTs correlated with mosaic localized NF1 is extremely rare. However, it is of high-grade malignancy and therefore, its clinical features should be considered by orthopedists and pathologists.
Insights
Malignant peripheral nerve-sheath tumors (MPNSTs) are rare in mosaic localized neurofibromatosis type 1 (NF1). This report details an adolescent diagnosed with MPNST and mosaic localized NF1, highlighting its rarity and high-grade malignancy.
Area of Science:
- Oncology
- Genetics
- Pathology
Background:
- Malignant peripheral nerve-sheath tumors (MPNSTs) are rare cancers, frequently associated with neurofibromatosis type 1 (NF1).
- MPNST occurrence in mosaic localized NF1 is exceptionally rare, with only three prior cases reported in patients over 40.
- This case presents an adolescent with MPNST and mosaic localized NF1, a demographic previously undocumented.
Purpose of the Study:
- To report an extremely rare case of MPNST in an adolescent with mosaic localized NF1.
- To highlight the diagnostic challenges and genetic underpinnings of MPNST in this rare presentation.
- To emphasize the clinical significance for orthopedists and pathologists managing such high-grade malignancies.
Main Methods:
- Clinical presentation of a 16-year-old male with a right knee mass.
- Initial marginal resection followed by wide resection upon histological confirmation of sarcoma.
- Genomic analysis of NF1 mutations in café-au-lait spots to confirm mosaic localized NF1 and limited NF1 expression in the tumor.
Main Results:
- A 16-year-old male was diagnosed with MPNST associated with mosaic localized NF1.
- Genomic analysis revealed an NF1 microdeletion and reduced NF1 expression in the tumor.
- No local recurrence or distant metastasis was observed 1.5 years post-surgery.
Conclusions:
- MPNST in adolescent patients with mosaic localized NF1 is exceedingly rare.
- Despite rarity, MPNST is a high-grade malignancy requiring prompt recognition.
- Orthopedists and pathologists must consider these clinical features for accurate diagnosis and management.

