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Title: Establishing Evidence-Based Indications for Proton Therapy: An Overview of Current Clinical Trials

Abstract

Purpose: To review and assess ongoing proton beam therapy (PBT) clinical trials and to identify major gaps. Methods and Materials: Active PBT clinical trials were identified from (clinicaltrials.gov) and the World Health Organization International Clinical Trials Platform Registry. Data on clinical trial disease site, age group, projected patient enrollment, expected start and end dates, study type, and funding source were extracted. Results: A total of 122 active PBT clinical trials were identified, with target enrollment of >42,000 patients worldwide. Ninety-six trials (79%), with a median planned sample size of 68, were classified as interventional studies. Observational studies accounted for 21% of trials but 71% (n=29,852) of planned patient enrollment. The most common PBT clinical trials focus on gastrointestinal tract tumors (21%, n=26), tumors of the central nervous system (15%, n=18), and prostate cancer (12%, n=15). Five active studies (lung, esophagus, head and neck, prostate, breast) will randomize patients between protons and photons, and 3 will randomize patients between protons and carbon ion therapy. Conclusions: The PBT clinical trial portfolio is expanding rapidly. Although the majority of ongoing studies are interventional, the majority of patients will be accrued to observational studies. Future efforts should focus on strategies to encourage optimal patient enrollmentmore » and retention, with an emphasis on randomized, controlled trials, which will require support from third-party payers. Results of ongoing PBT studies should be evaluated in terms of comparative effectiveness, as well as incremental effectiveness and value offered by PBT in comparison with conventional radiation modalities.« less

Authors:
 [1];  [2];  [3];  [1];  [4];  [1]
  1. Department of Radiation Oncology, University of Maryland School of Medicine, Baltimore, Maryland (United States)
  2. Department of Internal Medicine, University of Maryland School of Medicine, Baltimore, Maryland (United States)
  3. Department of Epidemiology and Public Health, University of Maryland School of Medicine, Baltimore, Maryland (United States)
  4. Miami Cancer Institute at Baptist Health South Florida, Miami, Florida (United States)
Publication Date:
OSTI Identifier:
22645758
Resource Type:
Journal Article
Resource Relation:
Journal Name: International Journal of Radiation Oncology, Biology and Physics; Journal Volume: 97; Journal Issue: 2; Other Information: Copyright (c) 2016 Elsevier Science B.V., Amsterdam, The Netherlands, All rights reserved.; Country of input: International Atomic Energy Agency (IAEA)
Country of Publication:
United States
Language:
English
Subject:
62 RADIOLOGY AND NUCLEAR MEDICINE; AGE GROUPS; CARBON IONS; CENTRAL NERVOUS SYSTEM; CLINICAL TRIALS; GASTROINTESTINAL TRACT; ION BEAMS; MAMMARY GLANDS; NEOPLASMS; PATIENTS; PROTON BEAMS; RADIOTHERAPY

Citation Formats

Mishra, Mark V., E-mail: mmishra@umm.edu, Aggarwal, Sameer, Bentzen, Soren M., Knight, Nancy, Mehta, Minesh P., and Regine, William F.. Establishing Evidence-Based Indications for Proton Therapy: An Overview of Current Clinical Trials. United States: N. p., 2017. Web. doi:10.1016/J.IJROBP.2016.10.045.
Mishra, Mark V., E-mail: mmishra@umm.edu, Aggarwal, Sameer, Bentzen, Soren M., Knight, Nancy, Mehta, Minesh P., & Regine, William F.. Establishing Evidence-Based Indications for Proton Therapy: An Overview of Current Clinical Trials. United States. doi:10.1016/J.IJROBP.2016.10.045.
Mishra, Mark V., E-mail: mmishra@umm.edu, Aggarwal, Sameer, Bentzen, Soren M., Knight, Nancy, Mehta, Minesh P., and Regine, William F.. Wed . "Establishing Evidence-Based Indications for Proton Therapy: An Overview of Current Clinical Trials". United States. doi:10.1016/J.IJROBP.2016.10.045.
@article{osti_22645758,
title = {Establishing Evidence-Based Indications for Proton Therapy: An Overview of Current Clinical Trials},
author = {Mishra, Mark V., E-mail: mmishra@umm.edu and Aggarwal, Sameer and Bentzen, Soren M. and Knight, Nancy and Mehta, Minesh P. and Regine, William F.},
abstractNote = {Purpose: To review and assess ongoing proton beam therapy (PBT) clinical trials and to identify major gaps. Methods and Materials: Active PBT clinical trials were identified from (clinicaltrials.gov) and the World Health Organization International Clinical Trials Platform Registry. Data on clinical trial disease site, age group, projected patient enrollment, expected start and end dates, study type, and funding source were extracted. Results: A total of 122 active PBT clinical trials were identified, with target enrollment of >42,000 patients worldwide. Ninety-six trials (79%), with a median planned sample size of 68, were classified as interventional studies. Observational studies accounted for 21% of trials but 71% (n=29,852) of planned patient enrollment. The most common PBT clinical trials focus on gastrointestinal tract tumors (21%, n=26), tumors of the central nervous system (15%, n=18), and prostate cancer (12%, n=15). Five active studies (lung, esophagus, head and neck, prostate, breast) will randomize patients between protons and photons, and 3 will randomize patients between protons and carbon ion therapy. Conclusions: The PBT clinical trial portfolio is expanding rapidly. Although the majority of ongoing studies are interventional, the majority of patients will be accrued to observational studies. Future efforts should focus on strategies to encourage optimal patient enrollment and retention, with an emphasis on randomized, controlled trials, which will require support from third-party payers. Results of ongoing PBT studies should be evaluated in terms of comparative effectiveness, as well as incremental effectiveness and value offered by PBT in comparison with conventional radiation modalities.},
doi = {10.1016/J.IJROBP.2016.10.045},
journal = {International Journal of Radiation Oncology, Biology and Physics},
number = 2,
volume = 97,
place = {United States},
year = {Wed Feb 01 00:00:00 EST 2017},
month = {Wed Feb 01 00:00:00 EST 2017}
}