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Clinical Trial
. 2014 Nov 20;32(33):3762-70.
doi: 10.1200/JCO.2014.56.1548. Epub 2014 Oct 20.

Preliminary results of a phase II trial of proton radiotherapy for pediatric rhabdomyosarcoma

Affiliations
Clinical Trial

Preliminary results of a phase II trial of proton radiotherapy for pediatric rhabdomyosarcoma

Matthew M Ladra et al. J Clin Oncol. .

Erratum in

  • J Clin Oncol. 2015 Jan 10;33(2):228

Abstract

Purpose: 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).

Patients and 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.

Conclusion: 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.

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Conflict of interest statement

Authors' disclosures of potential conflicts of interest and author contributions are found at the end of this article.

Figures

Fig 1.
Fig 1.
Proton treatment plans for patients with primaries at (A) orbital, (B) parameningeal, (C) trunk, and (D) prostate sites.
Fig 2.
Fig 2.
(A) Event-free, (B) overall, and (C) local control survival for the entire cohort and by risk group. RT, radiation therapy.

Comment in

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