Clinical Trial Will Clarify If More Patients Could Benefit from Treatment
The National Heart, Lung, and Blood Institute (NHLBI), one of the National Institutes of Health (NIH), and the Centers for Medicare & Medicaid Services (CMS) are launching the largest randomized clinical trial of the effectiveness and safety of long-term, home oxygen therapy for COPD (chronic obstructive pulmonary disease). The six-year, $28 million project will study patients with moderate disease.
COPD, a lung disease that over time makes it hard to breathe, is the fourth leading cause of death in the United States. In the Long-term Oxygen Treatment Trial, researchers at 14 clinical centers across the United States will study approximately 3,500 patients with moderate COPD to determine whether supplemental oxygen will help them lead longer, more active, and better quality lives. The results will help Medicare decide whether to extend coverage for home oxygen treatment to patients with moderate disease. Currently, Medicare limits coverage of home oxygen therapy to beneficiaries with severe COPD (very low blood oxygen levels while resting).
NHLBI will administer and oversee the study, and Medicare will cover the costs of items and medical services that are generally available through that program to beneficiaries enrolled in the trial. NIH and CMS are agencies of the U.S. Department of Health and Human Services (HHS).
"COPD is a devastating, highly disabling disease. The prospect that home oxygen therapy could lessen the disability of COPD and perhaps even prolong life when given earlier during the course of the disease is enticing, but we need more information to determine the risks and benefits," said NHLBI Director Elizabeth G. Nabel, M.D. "This study will provide important information to help patients and their health providers decide whether home oxygen treatment is a good choice for them."
About 12 million adults in the United States have been diagnosed with COPD, and another 12 million are believed to be undiagnosed. Approximately one million COPD patients currently receive supplemental oxygen treatment. Although oxygen therapy has been shown to improve survival in patients with severe COPD, the effects of treatment have not been adequately studied in patients with less severe disease.
The decision to undertake the study evolved from a scientific working group convened in May 2004 by NHLBI in cooperation with CMS and HHS' Agency for Healthcare Research and Quality. The group called for more research on the safety and efficacy of long-term oxygen therapy in patients with COPD. In March, Medicare announced that it will extend coverage of home oxygen treatment to Medicare-eligible patients enrolled in the study, who would not otherwise be covered.
"Medicare is committed to ensure that beneficiaries have access to the most appropriate treatments based on the best available science," commented CMS Chief Clinical Officer Barry Straube, M.D. "The results of this study will improve our understanding of the effects of oxygen treatment in a broader group of COPD patients and will help guide our policy decisions."
Patient recruitment for the Long-term Oxygen Treatment Trial is expected to begin in late 2007. Participants will be randomly selected to receive or not to receive supplemental oxygen for approximately three years. All participants will be periodically monitored; those who are not initially selected to receive oxygen will be prescribed oxygen if their blood oxygen levels worsen during the trial.
The 14 field study sites awarded contracts are:
- Brigham & Women's Hospital (John Reilly, M.D., Principal Investigator)
- Cleveland Clinic Foundation (James Stoller, M.D., M.S., Principal Investigator)
- Denver Health Medical Center (Rick Albert, M.D., Principal Investigator)
- Duke University (Neil MacIntyre, M.D., Principal Investigator)
- Kaiser Permanente Northwest (Thomas Stibolt, M.D., Principal Investigator)
- Los Angeles Biomedical Research Institute (Richard Casaburi, M.D., Principal Investigator)
- Ohio State University (Philip Diaz, M.D., Principal Investigator)
- Temple University (Gerard Criner, M.D., Principal Investigator)
- University of Alabama at Birmingham (J. Allen Cooper, Jr., M.D., Principal Investigator)
- University of Michigan (Fernando J. Martinez, M.D., M.S., Principal Investigator)
- University of Pittsburgh (Frank Sciurba, M.D., Principal Investigator)
- University of Utah (Richard Kanner, M.D., Principal Investigator)
- University of Washington (David Au, M.D., M.S., Principal Investigator)
- Washington University (Roger Yusen, M.D., M.P.H., Principal Investigator)
NHLBI awarded the contract for the study's data coordinating center to Johns Hopkins University (Steven Piantadosi, MD, PhD, Principal Investigator).
COPD is usually caused by smoking or by long-term exposure to dust or fumes. Symptoms include regular coughing (sometimes called "smoker's cough"), shortness of breath, excess sputum production, feeling like you can't breathe, not being able to take a deep breath, and wheezing. Quitting smoking is the single most important step patients can take to reduce the risk of developing COPD and to slow the progress of the disease.
Other treatments can help manage patients' symptoms, lower the rate of serious complications, and improve quality of life. Such treatments include exercise rehabilitation, medications such as bronchodilators and inhaled steroids to help open airways, supportive and preventive measures such as flu shots and pneumonia vaccine, and prompt treatment of respiratory infections. A limited number of patients with severe COPD may also benefit from lung volume reduction surgery (LVRS) or other surgeries.
NHLBI and CMS previously collaborated on the landmark study that determined which patients with severe COPD are most likely to benefit from or suffer complications from LVRS. Findings from the National Emphysema Treatment Trial were reported in May 2003, and Medicare subsequently began coverage of the procedure based on the results.
"As the population ages, the number of individuals affected by COPD is on the rise," noted James Kiley, PhD., director of the NHLBI Division of Lung Diseases. "It is more imperative than ever that we find treatments that will improve the health and function of patients with chronic lung diseases such as COPD."
November is National COPD Awareness Month. In addition, NHLBI is developing a national educational campaign to increase awareness and understanding of COPD and its risk factors and to underscore the benefits of early detection and treatment in slowing the disease and improving the quality of life.
To interview Thomas Croxton, M.D., Ph.D., NHLBI project officer of the study, contact the NHLBI Communications Office at 301-496-4236. To interview Dr. Reilly, call Kevin Myron at 617-534-1605; to interview Dr. Stoller, call Erinne Dyer at 216-444-8168 or Dr. Stoller at 216-444-1960; to interview Dr. Albert, call Lynn Ercolani at 303-436-6606; to interview Dr. MacIntyre, call Marla Broadfoot at 919-660-1306; to interview Dr. Stibolt, call Terry Fitzpatrick at 503-335-6602; to interview Dr. Casaburi, call David Feuerherd at 310-215-0234; to interview Dr. Diaz, call Sherri Kirk at 614-293-3737; to interview Dr. Criner, call Eryn Jelesiewicz at 215-707-0730; to interview Dr. Cooper, call Bob Shepard at 205-934-8934; to interview Dr. Martinez, call Katie Gazella at 734-764-2220; to interview Dr. Sciurba, call Michele Baum at 412-647-3555; to interview Dr. Kanner, call Chris Nelson at 801-581-7387; to interview Dr. Au, call Clare Hagerty at 206-685-1323; to interview Dr. Yusen, call Joni Westerhouse at 314-286-0120; to interview Dr. Piantadosi, call David Marsh at 410-955-1534.
- News release, "Medicare Launches Efforts To Improve Care For Patients Using Oxygen": http://www.cms.hhs.gov/apps/media/press/release.asp?Counter=1815
- "Long-term Oxygen Treatment in COPD" Working Group Executive Summary, May 10-11, 2004: http://www.nhlbi.nih.gov/research/reports/2004-oxygen-treatment
- News release, "NHLBI Study Shows Smoking Cessation Programs Improve Survival": http://www.nhlbi.nih.gov/news/press-releases/2005/nhlbi-study-shows-smoking-cessation-programs-improve-survival
- News release, "Landmark Cooperative Federal Study Defines the Role of Lung Surgery in the Treatment of Severe Emphysema": http://www.nhlbi.nih.gov/news/press-releases/2003/landmark-cooperative-federal-study-defines-the-role-of-lung-surgery-in-the-treatment-of-severe-emphysema
- Medicare coverage (general): http://www.cms.hhs.gov/coverage/default.asp [Link is no longer available]. For questions about Medicare please call 1-800-MEDICARE or visit www.medicare.gov.
- Resources to help smokers quit are available at www.smokefree.gov.