title: Preliminary results of a phase II trial of proton radiotherapy for pediatric rhabdomyosarcoma.
abstract: OBJECTIVE
  This prospective phase II study was designed to assess disease control and to describe acute and late adverse effects of treatment with proton radiotherapy in children with rhabdomyosarcoma (RMS).
  
  
  METHODS
  Fifty-seven patients with localized RMS (age 21 years or younger) or metastatic embryonal RMS (age 2 to 10 years) were enrolled between February 2005 and August 2012. All patients were treated with chemotherapy based on either vincristine, actinomycin, and cyclophosphamide or vincristine, actinomycin, and ifosfamide-based chemotherapy and proton radiation. Surgical resection was based on tumor site and accessibility. Common Terminology Criteria for Adverse Events, Version 3.0, was used to assess and grade adverse effects of treatment. Concurrent enrollment onto Children's Oncology Group or European Pediatric Sarcoma Study Group protocols was allowed. All pathology and imaging were reviewed at the treating institution.
  
  
  RESULTS
  Median follow-up was 47 months (range, 14 to 102 months) for survivors. Five-year event-free survival (EFS), overall survival (OS), and local control (LC) were 69%, 78%, and 81%, respectively, for the entire cohort. The 5-year LC by risk group was 93% for low-risk and 77% for intermediate-risk disease. There were 13 patients with grade 3 acute toxicity and three patients with grade 3 late toxicity. There were no acute or late toxicities higher than grade 3.
  
  
  CONCLUSIONS
  Five-year LC, EFS, and OS rates were similar to those observed in comparable trials that used photon radiation. Acute and late toxicity rates were favorable. Proton radiation appears to represent a safe and effective radiation modality for pediatric RMS.
authors.affiliation: Matthew M. Ladra, Jackie Szymonifka, Alison M. Friedmann, Beow Yong Yeap, Claire Goebel, Shannon M. MacDonald, Nancy J. Tarbell, Torunn I. Yock Massachusetts General Hospital, Harvard Medical School;, Carlos Rodriguez-Galindo, Karen J. Marcus, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA; Anita Mahajan and David Grosshans, The University of Texas MD Anderson Cancer Center, Houston, TX.
authors.forename: Matthew M
authors.initials: MM
authors.lastname: Ladra
authors.affiliation: Matthew M. Ladra, Jackie Szymonifka, Alison M. Friedmann, Beow Yong Yeap, Claire Goebel, Shannon M. MacDonald, Nancy J. Tarbell, Torunn I. Yock Massachusetts General Hospital, Harvard Medical School;, Carlos Rodriguez-Galindo, Karen J. Marcus, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA; Anita Mahajan and David Grosshans, The University of Texas MD Anderson Cancer Center, Houston, TX.
authors.forename: Jackie D
authors.initials: JD
authors.lastname: Szymonifka
authors.affiliation: Matthew M. Ladra, Jackie Szymonifka, Alison M. Friedmann, Beow Yong Yeap, Claire Goebel, Shannon M. MacDonald, Nancy J. Tarbell, Torunn I. Yock Massachusetts General Hospital, Harvard Medical School;, Carlos Rodriguez-Galindo, Karen J. Marcus, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA; Anita Mahajan and David Grosshans, The University of Texas MD Anderson Cancer Center, Houston, TX.
authors.forename: Anita
authors.initials: A
authors.lastname: Mahajan
authors.affiliation: Matthew M. Ladra, Jackie Szymonifka, Alison M. Friedmann, Beow Yong Yeap, Claire Goebel, Shannon M. MacDonald, Nancy J. Tarbell, Torunn I. Yock Massachusetts General Hospital, Harvard Medical School;, Carlos Rodriguez-Galindo, Karen J. Marcus, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA; Anita Mahajan and David Grosshans, The University of Texas MD Anderson Cancer Center, Houston, TX.
authors.forename: Alison M
authors.initials: AM
authors.lastname: Friedmann
authors.affiliation: Matthew M. Ladra, Jackie Szymonifka, Alison M. Friedmann, Beow Yong Yeap, Claire Goebel, Shannon M. MacDonald, Nancy J. Tarbell, Torunn I. Yock Massachusetts General Hospital, Harvard Medical School;, Carlos Rodriguez-Galindo, Karen J. Marcus, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA; Anita Mahajan and David Grosshans, The University of Texas MD Anderson Cancer Center, Houston, TX.
authors.forename: Beow
authors.initials: B
authors.lastname: Yong Yeap
authors.affiliation: Matthew M. Ladra, Jackie Szymonifka, Alison M. Friedmann, Beow Yong Yeap, Claire Goebel, Shannon M. MacDonald, Nancy J. Tarbell, Torunn I. Yock Massachusetts General Hospital, Harvard Medical School;, Carlos Rodriguez-Galindo, Karen J. Marcus, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA; Anita Mahajan and David Grosshans, The University of Texas MD Anderson Cancer Center, Houston, TX.
authors.forename: Claire P
authors.initials: CP
authors.lastname: Goebel
authors.affiliation: Matthew M. Ladra, Jackie Szymonifka, Alison M. Friedmann, Beow Yong Yeap, Claire Goebel, Shannon M. MacDonald, Nancy J. Tarbell, Torunn I. Yock Massachusetts General Hospital, Harvard Medical School;, Carlos Rodriguez-Galindo, Karen J. Marcus, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA; Anita Mahajan and David Grosshans, The University of Texas MD Anderson Cancer Center, Houston, TX.
authors.forename: Shannon M
authors.initials: SM
authors.lastname: MacDonald
authors.affiliation: Matthew M. Ladra, Jackie Szymonifka, Alison M. Friedmann, Beow Yong Yeap, Claire Goebel, Shannon M. MacDonald, Nancy J. Tarbell, Torunn I. Yock Massachusetts General Hospital, Harvard Medical School;, Carlos Rodriguez-Galindo, Karen J. Marcus, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA; Anita Mahajan and David Grosshans, The University of Texas MD Anderson Cancer Center, Houston, TX.
authors.forename: David R
authors.initials: DR
authors.lastname: Grosshans
authors.affiliation: Matthew M. Ladra, Jackie Szymonifka, Alison M. Friedmann, Beow Yong Yeap, Claire Goebel, Shannon M. MacDonald, Nancy J. Tarbell, Torunn I. Yock Massachusetts General Hospital, Harvard Medical School;, Carlos Rodriguez-Galindo, Karen J. Marcus, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA; Anita Mahajan and David Grosshans, The University of Texas MD Anderson Cancer Center, Houston, TX.
authors.forename: Carlos
authors.initials: C
authors.lastname: Rodriguez-Galindo
authors.affiliation: Matthew M. Ladra, Jackie Szymonifka, Alison M. Friedmann, Beow Yong Yeap, Claire Goebel, Shannon M. MacDonald, Nancy J. Tarbell, Torunn I. Yock Massachusetts General Hospital, Harvard Medical School;, Carlos Rodriguez-Galindo, Karen J. Marcus, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA; Anita Mahajan and David Grosshans, The University of Texas MD Anderson Cancer Center, Houston, TX.
authors.forename: Karen J
authors.initials: KJ
authors.lastname: Marcus
authors.affiliation: Matthew M. Ladra, Jackie Szymonifka, Alison M. Friedmann, Beow Yong Yeap, Claire Goebel, Shannon M. MacDonald, Nancy J. Tarbell, Torunn I. Yock Massachusetts General Hospital, Harvard Medical School;, Carlos Rodriguez-Galindo, Karen J. Marcus, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA; Anita Mahajan and David Grosshans, The University of Texas MD Anderson Cancer Center, Houston, TX.
authors.forename: Nancy J
authors.initials: NJ
authors.lastname: Tarbell
authors.affiliation: Matthew M. Ladra, Jackie Szymonifka, Alison M. Friedmann, Beow Yong Yeap, Claire Goebel, Shannon M. MacDonald, Nancy J. Tarbell, Torunn I. Yock Massachusetts General Hospital, Harvard Medical School;, Carlos Rodriguez-Galindo, Karen J. Marcus, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA; Anita Mahajan and David Grosshans, The University of Texas MD Anderson Cancer Center, Houston, TX. tyock@partners.org.
authors.forename: Torunn I
authors.initials: TI
authors.lastname: Yock
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D011446:Prospective Studies; D061766:Proton Therapy; D011522:Protons / Q000009:adverse effects; D012208:Rhabdomyosarcoma / Q000188:drug therapy / Q000401:mortality / Q000532:radiotherapy*
chemical_list: D011522:Protons