title: Tumour control and Quality of Life in children with rhabdomyosarcoma treated with pencil beam scanning proton therapy.
abstract: To assess clinical outcomes in children with rhabdomyosarcoma (RMS) treated with pencil beam scanning (PBS) proton therapy (PT).
  
  
  
  Eighty-three RMS (embryonal, n=74; 89%) patients treated between January 2000 and December 2014 were included. The median age was 4.5years (range, 0.8-15.5). All patients received systemic chemotherapy according to prospective protocols. Patients had low-, intermediate-, and high-risk disease in 24%, 63%, and 13% of cases, respectively. The median total dose delivered was 54Gy(RBE) (range, 41.4-64.8).
  
  
  
  After a median follow-up time of 55.5 months (range, 0.9-126.3), local failure occurred in 16 patients. The 5-year local-control survival rate was 78.5% [95% confidence interval (CI), 69.5-88.5%]. Significant predictors for local failure were group/stage, tumour location, and size. Fourteen patients (16%) died, all from tumour progression. The 5-year overall survival was 80.6% (95%CI, 71.8-90.0%). The 5-year incidence of grade 3 non-ocular late toxicity was 3.6% (95%CI, 1-12%). No grade 4-5 late toxicities were observed. One radiation-induced malignancy was observed (1.2%). The Quality of Life (QoL) scores increased significantly after PT compared to baseline values.
  
  
  
  PBS PT led to excellent outcome in children with RMS. Late non-ocular toxicity was minimal and QoL good.
authors.affiliation: Department of Radiation Oncology, Inselspital, Bern University Hospital, and University of Bern, Switzerland; Center for Proton Therapy, Paul Scherrer Institute, Switzerland.
authors.forename: Dominic
authors.initials: D
authors.lastname: Leiser
authors.affiliation: Department of Pediatric Hematology and Oncology, University Hospital Münster, Germany.
authors.forename: Gabriele
authors.initials: G
authors.lastname: Calaminus
authors.affiliation: Center for Proton Therapy, Paul Scherrer Institute, Switzerland.
authors.forename: Robert
authors.initials: R
authors.lastname: Malyapa
authors.affiliation: Department of Radiation Oncology, Inselspital, Bern University Hospital, and University of Bern, Switzerland; Center for Proton Therapy, Paul Scherrer Institute, Switzerland.
authors.forename: Beat
authors.initials: B
authors.lastname: Bojaxhiu
authors.affiliation: Center for Proton Therapy, Paul Scherrer Institute, Switzerland.
authors.forename: Francesca
authors.initials: F
authors.lastname: Albertini
authors.affiliation: Center for Proton Therapy, Paul Scherrer Institute, Switzerland.
authors.forename: Ulrike
authors.initials: U
authors.lastname: Kliebsch
authors.affiliation: Center for Proton Therapy, Paul Scherrer Institute, Switzerland.
authors.forename: Lorentzos
authors.initials: L
authors.lastname: Mikroutsikos
authors.affiliation: Center for Proton Therapy, Paul Scherrer Institute, Switzerland.
authors.forename: Petra
authors.initials: P
authors.lastname: Morach
authors.affiliation: Center for Proton Therapy, Paul Scherrer Institute, Switzerland.
authors.forename: Alessandra
authors.initials: A
authors.lastname: Bolsi
authors.affiliation: Center for Proton Therapy, Paul Scherrer Institute, Switzerland.
authors.forename: Marc
authors.initials: M
authors.lastname: Walser
authors.affiliation: Clinic for Particle Therapy, University Hospital Essen, West German Proton Therapy Center Essen (WPE), Germany.
authors.forename: Beate
authors.initials: B
authors.lastname: Timmermann
authors.affiliation: Department of Pediatric Hematology and Oncology, University Hospital Münster, Germany; ETH Zürich, Switzerland.
authors.forename: Tony
authors.initials: T
authors.lastname: Lomax
authors.affiliation: Center for Proton Therapy, Paul Scherrer Institute, Switzerland.
authors.forename: Ralf
authors.initials: R
authors.lastname: Schneider
authors.affiliation: Department of Radiation Oncology, Inselspital, Bern University Hospital, and University of Bern, Switzerland; Center for Proton Therapy, Paul Scherrer Institute, Switzerland; University of Bern, Switzerland; University of Zürich, Switzerland. Electronic address: damien.weber@psi.ch.
authors.forename: Damien C
authors.initials: DC
authors.lastname: Weber
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D061766:Proton Therapy / Q000379:methods*; D011788:Quality of Life; D012208:Rhabdomyosarcoma / Q000401:mortality / Q000523:psychology / Q000532:radiotherapy*
keywords: Children; Late toxicity; Pencil beam scanning proton therapy; Quality of Life; Radiation-induced tumours; Rhabdomyosarcoma