Randomized phase IIB trial of proton beam therapy versus intensity-modulated radiation therapy for locally advanced esophageal cancer

Steven H. Lin, Brian P. Hobbs, Vivek Verma, Rebecca S. Tidwell, Grace L. Smith, Xiudong Lei, Erin M. Corsini, Isabel Mok, Xiong Wei, Luyang Yao, Xin Wang, Ritsuko U. Komaki, Joe Y. Chang, Stephen G. Chun, Melenda D. Jeter, Stephen G. Swisher, Jaffer A. Ajani, Mariela Blum-Murphy, Ara A. Vaporciyan, Reza J. MehranAlbert C. Koong, Saumil J. Gandhi, Wayne L. Hofstetter, Theodore S. Hong, Thomas F. Delaney, Zhongxing Liao, Radhe Mohan

Research output: Contribution to journalArticlepeer-review

195 Scopus citations

Abstract

PURPOSE Whether dosimetric advantages of proton beam therapy (PBT) translate to improved clinical outcomes compared with intensity-modulated radiation therapy (IMRT) remains unclear. This randomized trial compared total toxicity burden (TTB) and progression-free survival (PFS) between these modalities for esophageal cancer. METHODS This phase IIB trial randomly assigned patients to PBT or IMRT (50.4 Gy), stratified for histology, resectability, induction chemotherapy, and stage. The prespecified coprimary end points were TTB and PFS. TTB, a composite score of 11 distinct adverse events (AEs), including common toxicities as well as postoperative complications (POCs) in operated patients, quantified the extent of AE severity experienced over the duration of 1 year following treatment. The trial was conducted using Bayesian group sequential design with three planned interim analyses at 33%, 50%, and 67% of expected accrual (adjusted for follow-up). RESULTS This trial (commenced April 2012) was approved for closure and analysis upon activation of NRGGI006 in March 2019, which occurred immediately prior to the planned 67% interim analysis. Altogether, 145 patients were randomly assigned (72 IMRT, 73 PBT), and 107 patients (61 IMRT, 46 PBT) were evaluable. Median follow-up was 44.1 months. Fifty-one patients (30 IMRT, 21 PBT) underwent esophagectomy; 80% of PBT was passive scattering. The posterior mean TTB was 2.3 times higher for IMRT (39.9; 95% highest posterior density interval, 26.2-54.9) than PBT (17.4; 10.5-25.0). The mean POC score was 7.6 times higher for IMRT (19.1; 7.3-32.3) versus PBT (2.5; 0.3-5.2). The posterior probability that mean TTB was lower for PBT compared with IMRT was 0.9989, which exceeded the trial’s stopping boundary of 0.9942 at the 67% interim analysis. The 3-year PFS rate (50.8% v 51.2%) and 3-year overall survival rates (44.5% v 44.5%) were similar. CONCLUSION For locally advanced esophageal cancer, PBT reduced the risk and severity of AEs compared with IMRT while maintaining similar PFS.

Original languageEnglish (US)
Pages (from-to)1569-1578
Number of pages10
JournalJournal of Clinical Oncology
Volume38
Issue number14
DOIs
StatePublished - 2020

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

MD Anderson CCSG core facilities

  • Biostatistics Resource Group
  • Clinical Trials Office

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