Malignant Peripheral Nerve Sheath Tumor in Children and Adolescents: Local Treatment in a Retrospective Single-Center Experience.

Abstract

Background: Malignant peripheral nerve sheath tumor (MPNST) is a rare and aggressive sarcoma often associated with neurofibromatosis type 1, whose clinical management remains complex and challenging. Few publications exist on pediatric MPNST, and limited data are available on the best treatment approach, in particular regarding local therapy.

Methods: This retrospective analysis concerned 45 patients less than 18 years old with MPNST, treated at a referral center for pediatric sarcomas from 1983 to 2023. Patients were treated using a multimodal approach, based on the protocols adopted at the time of their diagnosis.

Results: For the series as a whole, the median event-free survival (EFS) and overall survival (OS) were 16 and 26 months, respectively, and 5-year EFS and OS were 28.8% and 40.1%. The first event was local failure in 18 cases, local failure plus metastases in nine cases, and metastases-only in four cases. At univariable analysis, survival was better for males and patients younger than 15 years, and was influenced by tumor invasiveness and tumor size. With regard to treatment modalities, survival rates were significantly better for patients who responded to chemotherapy; EFS, local relapse-free survival (LRFS), and OS were better for patients who had a surgical resection; EFS and LRFS were better for patients who received radiotherapy combined with surgery, while OS was better for patients who had R0 resection.

Conclusions: Our study confirmed the unsatisfactory outcome of MPNST pediatric patients. Our series would suggest that a combined local treatment that included both surgical resection and radiotherapy could improve local control.