title: Paediatric and adolescent alveolar soft part sarcoma: A joint series from European cooperative groups.
abstract: BACKGROUND
  Alveolar soft part sarcomas (ASPS) are generally chemo- and radio-resistant mesenchymal tumours, with no standardized treatment guidelines. We describe the clinical behaviour of paediatric ASPS and compare these features to previously reported adult series.
  
  
  METHODS
  The clinical data of 51 children and adolescents with ASPS, prospectively enrolled in or treated according to seven European Paediatric trials were analysed.
  
  
  RESULTS
  Median age was 13 years [range: 2-21]. Primary sites included mostly limbs (63%). IRS post-surgical staging was: IRS-I (complete resection) 35%, II (microscopic residual disease) 20%, III (gross residual disease) 18% and IV (metastases) 27%. Only 3 of the 18 evaluable patients (17%) obtained a response to conventional chemotherapy. After a median follow-up of 126 months (range: 9-240), 14/18 patients with IRS-I tumour, 10/10 IRS-II, 7/9 IRS-III and 2/14 IRS-IV were alive in remission. Sunitinib treatment achieved two very good partial responses in four patients. Ten-year overall survival (OS) and event free survival (EFS) was 78.0 ± 7% and 62.8 ± 7% respectively. Stage IV, size >5 cm and T2 tumours had a poorer outcome, but only IRS staging was an independent prognostic factor.
  
  
  CONCLUSIONS
  ASPS is a very rare tumour frequently arising in adolescents and in the extremities, and chemo resistant. Local surgical control is critical. ASPS is a poorly chemo sensitive tumour. For IRS-III/IV tumours, delayed radical local therapies including surgery are essential. Metastatic patients had a poor prognosis but targeted therapies showed promising results.
authors.affiliation: Department of Paediatric Oncology, Institut Curie, Paris, France.
authors.forename: D
authors.initials: D
authors.lastname: Orbach
authors.forename: B
authors.initials: B
authors.lastname: Brennan
authors.forename: M
authors.initials: M
authors.lastname: Casanova
authors.forename: C
authors.initials: C
authors.lastname: Bergeron
authors.forename: V
authors.initials: V
authors.lastname: Mosseri
authors.forename: N
authors.initials: N
authors.lastname: Francotte
authors.forename: M
authors.initials: M
authors.lastname: Van Noesel
authors.forename: A
authors.initials: A
authors.lastname: Rey
authors.forename: G
authors.initials: G
authors.lastname: Bisogno
authors.forename: G
authors.initials: G
authors.lastname: Pierron
authors.forename: A
authors.initials: A
authors.lastname: Ferrari
mesh_terms: D000293:Adolescent; D000970:Antineoplastic Agents / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D018572:Disease-Free Survival; D005060:Europe; D005260:Female; D006801:Humans; D053208:Kaplan-Meier Estimate; D008297:Male; D016016:Proportional Hazards Models; D011878:Radiotherapy; D018234:Sarcoma, Alveolar Soft Part / Q000401:mortality / Q000473:pathology* / Q000628:therapy*; D012983:Soft Tissue Neoplasms / Q000401:mortality / Q000473:pathology* / Q000628:therapy*; D055815:Young Adult
chemical_list: D000970:Antineoplastic Agents
keywords: Alveolar soft part sarcoma; adolescence; chemotherapy; childhood; radiotherapy; target therapy