title: Chemotherapy for the treatment of malignant peripheral nerve sheath tumors in neurofibromatosis 1: a 10-year institutional review.
abstract: BACKGROUND
  Neurofibromatosis 1 (NF1) is the most common autosomal dominant disorder, with an incidence of 1 in 2,500-3,300 live births. NF1 is associated with significant morbidity and mortality because of complications, especially malignant peripheral nerve sheath tumors (MPNSTs), which mainly develop during adulthood. We evaluated our experience with management of NF1 with MPNSTs by standard chemotherapy with anthracycline and/or ifosfamide in terms of time to treatment failure and overall survival.
  
  
  METHODS
  We performed a retrospective review of consecutive patients with NF1 and a diagnosis of MPNSTs between 1993 and 2003 in our referral center for NF1. Prognostic factors were evaluated by univariate analysis.
  
  
  RESULTS
  We evaluated data for 21 patients with grade 1 (n=1), grade 2 (n=8) and grade 3 (n=12) MPNST; 16 presented localized disease and underwent surgery: margins for 6 were tumor-free (including 3 patients with amputation), 2 showed microscopic residual disease and 8 showed macroscopic residual disease. All patients received chemotherapy and 9 radiotherapy. Median time to treatment failure and overall survival were 7.8 and 17 months, respectively. Two patients were still alive at 138 and 167 months. We found no significant relationship between type of chemotherapy and time to treatment failure or overall survival.
  
  
  CONCLUSIONS
  MPNSTs are highly aggressive in NF1. Conventional chemotherapy does not seem to reduce mortality, and its role must be questioned. Recent advances in the molecular biology of MPNSTs may provide new prognostic factors and targeted therapies.
authors: Department of Dermatology, Referral center for Neurofibromatosis, Henri Mondor Hospital, UPEC, Créteil, France. laurence.allanore@hmn.aphp.fr.
authors: Ouidad
authors: O
authors: Zehou
authors: Elizabeth
authors: E
authors: Fabre
authors: Laurent
authors: L
authors: Zelek
authors: Emilie
authors: E
authors: Sbidian
authors: Nicolas
authors: N
authors: Ortonne
authors: Eugeniu
authors: E
authors: Banu
authors: Pierre
authors: P
authors: Wolkenstein
authors: Laurence
authors: L
authors: Valeyrie-Allanore
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D018317:Nerve Sheath Neoplasms / Q000188:drug therapy* / Q000209:etiology* / Q000401:mortality / Q000601:surgery; D009456:Neurofibromatosis 1 / Q000150:complications* / Q000188:drug therapy / Q000401:mortality / Q000601:surgery; D012189:Retrospective Studies; D055815:Young Adult
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