SEATTLE – A research team led by Scott Ramsey, M.D., Ph.D., director of the Hutchinson Institute for Cancer Outcomes Research, or HICOR, has been approved for a $7.75 million, four-year funding award by the Patient-Centered Outcomes Research Institute, or PCORI, an independent, nonprofit organization based in Washington, D.C. The award will be used to conduct a pragmatic clinical trial evaluating the use of colony stimulating factor to reduce the risk of serious infection in patients undergoing chemotherapy for breast, colorectal or lung cancer.
This pragmatic trial, developed in partnership with the national clinical trials group SWOG, the SWOG Statistical Center at Fred Hutch, the nonprofit organization Cancer Research and Biostatistics, Columbia University and the University of Washington School of Pharmacy, will investigate whether a health-systems-based intervention can improve adherence to evidence-based guidelines for the use of supportive-care drugs known as colony-stimulating factors.
For patients whose chemotherapy carries a 20 percent or greater risk of febrile neutropenia, an infection caused by a loss of white blood cells, clinical practice guidelines recommend the use of primary prophylactic colony-stimulating factor, or PP-CSF, during the first cycle of chemo to reduce the risk of infection. PP-CSF use in patients at intermediate (10 to 20 percent) risk is suggested only when other risk factors are present, while it is not recommended for patients receiving low-risk chemotherapy. As a result, studies show that colony-stimulating factors are both under- and overused among chemotherapy patients.
“Underuse of colony-stimulating factor use can result in serious adverse health outcomes for chemotherapy patients that range from life-threatening infections to interruption of chemotherapy to reduced chemotherapy dose intensity,” said Ramsey, a health economist and internist in the Public Health Sciences Division at Fred Hutchinson Cancer Research Center, where HICOR is based. “Overuse and underuse of PP-CSF can harm patients and also can result in wasted resources.”
To evaluate and improve the use of colony-stimulating factor in high-risk cancer patients, Ramsey and colleagues will conduct a pragmatic trial that evaluates a guidelines-based, standing-order entry system for PP-CSF use versus usual care among breast, colorectal and lung cancer patients receiving chemotherapy. They’ll also conduct a randomized study of PP-CSF versus no PP-CSF for patients receiving intermediate-risk chemotherapy. The trial will involve 2,880 patients from more than 30 clinics nationwide.
Patient outcomes to be evaluated will include rates of febrile neutropenia and neutropenia-related emergency room visits/hospitalizations, health-related quality of life and mortality, and adherence to treatment guidelines, as well as patient out-of-pocket costs and knowledge of the risks, benefits and costs of colony-stimulating factor treatment.
The study was designed in consultation with patients, patient advocates, an ethicist, oncologists, insurance plan leaders, policy makers and leaders of the community clinics where the study will be implemented.
“Our goal is to improve adherence to clinical practice guidelines in the low- and high-risk chemotherapy settings, where the evidence for PP-CSF use is well-established,” Ramsey said. “At the same time, we will generate new evidence on the benefits and risks of PP-CSF for commonly prescribed, intermediate-risk chemotherapy regimens where more evidence is needed for patients and providers to make informed decisions.”
The Fred Hutch study is one of five awards totaling more than $64 million approved by PCORI that aim to provide patients with information that will help them make better-informed decisions about their care. It is also one of the first studies selected for funding through PCORI’s Pragmatic Clinical Studies Initiative, an effort to produce results that are broadly applicable to a greater variety of patients and care situations and can be more quickly taken up in routine clinical practice.
“In the end, this is about the patient and improving the delivery of optimal and more personalized supportive care in order to reduce the risk of serious complications while improving the quality, efficiency and overall value of cancer treatment,” said Gary Lyman, M.D., M.P.H., co-director of HICOR and a medical oncologist who is co-investigator on the grant.
At Fred Hutchinson Cancer Research Center, home to three Nobel laureates, interdisciplinary teams of world-renowned scientists seek new and innovative ways to prevent, diagnose and treat cancer, HIV/AIDS and other life-threatening diseases. Fred Hutch’s pioneering work in bone marrow transplantation led to the development of immunotherapy, which harnesses the power of the immune system to treat cancer. An independent, nonprofit research institute based in Seattle, Fred Hutch houses the nation’s first National Cancer Institute-funded cancer prevention research program, as well as the clinical coordinating center of the Women’s Health Initiative and the international headquarters of the HIV Vaccine Trials Network.