title: Local therapy is critical in localised pelvic rhabdomyosarcoma: experience of the International Society of Pediatric Oncology Malignant Mesenchymal Tumor (SIOP-MMT) committee.
abstract: BACKGROUND
  Localised pelvic rhabdomyosarcomas (pRMS) are rare tumours with a poorer prognosis than the majority of RMS. This study analysed patient outcome according to the type of local therapy delivered and the effect of disease-related factors on prognosis.
  
  
  METHODS
  97 children with localised pRMS were enrolled in the SIOP-MMT84, 89 and 95 studies. After primary surgery or biopsy, all children received ifosfamide/actinomycin/vincristine-based chemotherapy. Radiotherapy and surgery were planned in patients failing to achieve complete remission.
  
  
  RESULTS
  Median age at diagnosis was 52 months [5 months-18 years]. IRS staging was I for five patients, II for 15 and III for 77. Patients had embryonal RMS (N = 41), alveolar RMS (N = 29), botryoid RMS (N = 3), or not otherwise specified RMS (N = 24).
  
  
  RESULTS
  87 patients achieved local control (90%), 37 relapsed (43%), mainly locally (84%). With a median follow-up of more than 10 years [4-22 years], 5-year OS was 66% (95% CI: 56-75%) and EFS was 52% (95% CI: 42-61%). Among the 18 IRS-I/II patients treated without radiotherapy, 15 survived. Seven out of the 20 IRS-III patients treated without local therapy died. In multivariate analysis, IRS staging, age greater than 10 years and lymph node involvement had a negative impact on OS. Perineal/perianal locations had a trend towards a worse prognosis.
  
  
  CONCLUSIONS
  pRMS still have a relatively poor prognosis. Radiotherapy or brachytherapy is necessary for all IRS-III patients including those with radiological complete remission after neoadjuvant chemotherapy with or without surgery. Radiotherapy may be withheld in IRS-I patients and children under 3 years with IRS-II pRMS.
authors.affiliation: Pediatric Haematology Oncology Department, Centre Hospitalo-Universitaire, 49000 Angers, France.
authors.forename: Yves
authors.initials: Y
authors.lastname: Réguerre
authors.forename: Hélène
authors.initials: H
authors.lastname: Martelli
authors.forename: Annie
authors.initials: A
authors.lastname: Rey
authors.forename: Timothy
authors.initials: T
authors.lastname: Rogers
authors.forename: Mark
authors.initials: M
authors.lastname: Gaze
authors.forename: Myriam Weyl
authors.initials: MW
authors.lastname: Ben Arush
authors.forename: Christine
authors.initials: C
authors.lastname: Devalck
authors.forename: Odile
authors.initials: O
authors.lastname: Oberlin
authors.forename: Michael
authors.initials: M
authors.lastname: Stevens
authors.forename: Daniel
authors.initials: D
authors.lastname: Orbach
mesh_terms: D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy / Q000532:radiotherapy; D010386:Pelvic Neoplasms / Q000188:drug therapy* / Q000532:radiotherapy*; D011379:Prognosis; D012074:Remission Induction; D012208:Rhabdomyosarcoma / Q000188:drug therapy* / Q000532:radiotherapy*; D016896:Treatment Outcome