title: Sarcomas in children with neurofibromatosis type 1-poor prognosis despite aggressive combined therapy in four patients treated in a single oncological institution.
abstract: OBJECTIVE
  Patients with neurofibromatosis type 1 (NF1) are predisposed to developing soft tissue sarcomas (STS).
  
  
  METHODS
  We report on four cases of STS diagnosed in locally advanced, unresectable stages in children with NF1 (three girls, one boy; age = 8 months-14 years). All patients received protocols for STS: Cooperative Weichteilsarkomstudie 91, 96 and 2002. One patient with limb rhabdomyosarcoma entered complete remission but developed late metastatic relapse and died of progression despite complete excision and autologous bone marrow transplantation. The other patient with bladder rhabdomyosarcoma died of neutropenia-related sepsis without remission. Patients with malignant peripheral nerve sheet tumour and malignant triton tumour located in the pelvis did not respond to therapy. One of them died of disease progression, while the other is disease-free 6 years post-therapy after mutilating tumour resection.
  
  
  CONCLUSIONS
  STS in NF1 seem to display poor prognosis in spite of combined therapy; thus, children with NF1 should remain under detailed control of the oncologist.
authors.affiliation: Department of Paediatrics, Haematology, Oncology and Endocrinology, Medical University, 7 Debinki Street, 80-211 Gdansk, Poland. ebien@amg.gda.pl
authors.forename: Ewa
authors.initials: E
authors.lastname: Bien
authors.forename: Teresa
authors.initials: T
authors.lastname: Stachowicz-Stencel
authors.forename: Danuta
authors.initials: D
authors.lastname: Sierota
authors.forename: Katarzyna
authors.initials: K
authors.lastname: Polczynska
authors.forename: Anna
authors.initials: A
authors.lastname: Szolkiewicz
authors.forename: Joanna
authors.initials: J
authors.lastname: Stefanowicz
authors.forename: Elzbieta
authors.initials: E
authors.lastname: Adamkiewicz-Drozynska
authors.forename: Piotr
authors.initials: P
authors.lastname: Czauderna
authors.forename: Wojciech
authors.initials: W
authors.lastname: Kosiak
authors.forename: Miroslawa
authors.initials: M
authors.lastname: Dubaniewicz-Wybieralska
authors.forename: Ewa
authors.initials: E
authors.lastname: Izycka-Swieszewska
authors.forename: Anna
authors.initials: A
authors.lastname: Balcerska
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use; D016026:Bone Marrow Transplantation; D002675:Child, Preschool; D003131:Combined Modality Therapy; D018450:Disease Progression; D017809:Fatal Outcome; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D019042:Muscle Neoplasms / Q000150:complications; D009362:Neoplasm Metastasis; D009456:Neurofibromatosis 1 / Q000150:complications*; D011379:Prognosis; D011878:Radiotherapy; D012186:Retroperitoneal Neoplasms / Q000150:complications; D012208:Rhabdomyosarcoma / Q000150:complications; D012509:Sarcoma / Q000150:complications* / Q000628:therapy*; D013125:Spinal Neoplasms / Q000150:complications; D014057:Tomography, X-Ray Computed; D001749:Urinary Bladder Neoplasms / Q000150:complications* / Q000628:therapy*
chemical_list: D000970:Antineoplastic Agents