Pediatric neurofibrosarcoma

Background/Purpose: Neurofibrosarcoma is rare in children, and the natural history and prognostic factors are not well described. The authors present a 57-year review of their experience. Methods: The charts of children with neurofibrosarcoma were reviewed retrospectively. Statistical analysis was performed using the X² and unpaired t tests. Results: From 1944 to 2001, 38 patients under the age of 21 were diagnosed with neurofibrosarcoma. Twenty-two were boys. The average age at diagnosis was 13.8 years (range, 3 to 19.9 years). Nineteen patients (50%) had neurofibromatosis. The tumor site was as follows: extremity, 19 patients; trunk, 9 patients; head and neck, 7 patients; and retroperitoneum, 3 patients. The average tumor size was 10 cm. The margins after resection were as follows: grossly positive, 9; microscopically positive, 5; negative, 21; and unknown, 3. Patients with positive margins had a 22% survival rate, whereas those with negative or unknown margins had a 34% survival rate. Thirty-two patients achieved a complete response, 2 a partial response, and 4 progressed while on therapy. Twenty-six patients relapsed after a complete response (11 local, 10 distant, 5 both). Of the 15 patients with a distant relapse, 73% (11) relapsed in the lung. Twelve (32%) patients survived with an average follow-up of 14 years (range, 0.3 to 28 years). Nine patients were treated with chemotherapy, 9 with radiation, and 9 with both chemotherapy and radiation. Outcome was not significantly affected by gender, presence of neurofibromatosis, site, margin, or use of adjuvant therapy. Conclusion: Neurofibrosarcoma remains a rare disease in children with insufficient contemporary numbers to assess efficacy of therapy. Prognosis remains poor with a high incidence of relapse, particularly in the lungs, suggesting that more aggressive therapies to control both local and distant relapses are needed.