title: Treatment and outcome of patients suffering from perineal/perianal rhabdomyosarcoma: results from the CWS trials--retrospective clinical study.
abstract: OBJECTIVE
  To analyze the clinical course, treatment, complications, outcome, and quality of life (QOL) in patients with perineal/perianal rhabdomyosarcoma (PRMS) treated within the CWS-86, -91, -96, and -2002P trials.
  
  
  BACKGROUND
  Although multiple international study trials exist for the treatment of rhabdomyosarcoma, only very limited information is given on treatment, outcome, and QOL in PRMS.
  
  
  METHODS
  A total of 35 patients suffering from PRMS were treated with neoadjuvant chemotherapy. Local therapy with radiation and/or surgery was performed, followed by adjuvant chemotherapy. Functional long-term follow-up was evaluated by a gastrointestinal/QOL survey.
  
  
  RESULTS
  Thirty-two patients were evaluated (exclusion n = 3). Eight patients had embryonal histology, and 24 patients had alveolar histology. The median age was 108 months (median follow-up: 5.8 years). The 5-year overall survival was 47% (95% confidence interval: 29-64). Sixteen IRS (Intergroup Rhabdomyosarcoma Study) III and IV patients had locoregional lymph node involvement at diagnosis. Seven patients were treated with chemotherapy/surgery alone [5-year event-free survival (EFS): 85.7%]. Eleven patients received only radiochemotherapy (5-year EFS: 27.3%). Combined radiochemotherapy/surgery was used in 12 patients (5-year EFS: 63.6%). Two patients were treated only with chemotherapy and they died. Patients with embryonal histology had a significantly better 5-year EFS (87.5%) than patients with alveolar histology (39.1%; P = 0.013). Some patients reported symptoms of fecal incontinence. The median Wexner fecal incontinence score was 9 (possible range: 0-20), and the median QOL score was 90.5 (applicable range: 0-144).
  
  
  CONCLUSIONS
  The outcome of these patients remains unsatisfactory. Prognostic factors for a favorable outcome are tumor size of smaller than 5 cm, negative locoregional lymph nodes, age less than 10 years, low IRS group, and embryonal histology. Fecal incontinence seems to be a problem.
authors.affiliation: *Department of Pediatric Surgery and Pediatric Urology, University Children's Hospital, Tuebingen, Germany †Department of Pediatric Hematology, Oncology and Immunology, Olgahospital, Stuttgart, Germany ‡Department of Clinical Epidemiology and Applied Biometry, University of Tuebingen, Tuebingen, Germany §Department for Pediatric Hematology and Oncology, University Hospital, Frankfurt/Main, Germany ¶Department of Paidopathology, University Hospital, Kiel, Germany ‖Department of Radiotherapy, University Hospital, Muenster, Germany **Department of Oncology, University Children's Hospital, Zurich, Switzerland.
authors.forename: Joerg
authors.initials: J
authors.lastname: Fuchs
authors.forename: Tobias M
authors.initials: TM
authors.lastname: Dantonello
authors.forename: Gunnar
authors.initials: G
authors.lastname: Blumenstock
authors.forename: Daniel
authors.initials: D
authors.lastname: Kosztyla
authors.forename: Thomas
authors.initials: T
authors.lastname: Klingebiel
authors.forename: Ivo
authors.initials: I
authors.lastname: Leuschner
authors.forename: Andreas
authors.initials: A
authors.lastname: Schuck
authors.forename: Felix K
authors.initials: FK
authors.lastname: Niggli
authors.forename: Ewa
authors.initials: E
authors.lastname: Koscielniak
authors.forename: Guido
authors.initials: G
authors.lastname: Seitz
mesh_terms: D001003:Anal Canal; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D005858:Germany / Q000453:epidemiology; D006801:Humans; D008297:Male; D010386:Pelvic Neoplasms / Q000401:mortality / Q000628:therapy*; D010502:Perineum; D011788:Quality of Life; D012189:Retrospective Studies; D012208:Rhabdomyosarcoma / Q000401:mortality / Q000628:therapy*; D011795:Surveys and Questionnaires; D015996:Survival Rate / Q000639:trends; D013557:Switzerland / Q000453:epidemiology; D013997:Time Factors; D016896:Treatment Outcome